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| ID | Type | Description | Link |
|---|---|---|---|
| C5341020 | Other Identifier | Alias Study Number |
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Emerging clinical data evaluated by Pfizer and shared with regulatory authorities indicates that the risk profile of voxelotor in people with SCD exceeds the benefits observed in previously generated global research and requires further assessment.
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This study consists of four parts, Parts A, B, C, and D.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Voxelotor | Experimental | Subjects to receive daily oral dosing of voxelotor according to which Part (A, B, C, or D), the subject is participating in:
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Voxelotor | Drug |
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| Measure | Description | Time Frame |
|---|---|---|
| Part A: Maximum Concentration (Cmax) of Voxelotor in Whole Blood | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose | |
| Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Voxelotor in Whole Blood | AUC0-last was calculated using the linear/log trapezoid rule. | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
| Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 Extrapolated to Infinity (AUC0-inf) of Voxelotor in Whole Blood | AUC0-inf was calculated as AUCt + Ct/lambdaz, where AUCt=area under the concentration-time curve at designated time, Ct is the last quantifiable concentration for whole blood and lambdaz=elimination rate constant. | pre-dose, 2, 8, 24, 48, 96,168 and 336 hours post-dose |
| Part B: Change From Baseline to Week 24 in Hemoglobin Level | Baseline, Week 24 | |
| Part C: Change From Baseline to Week 48 in Cerebral Blood Flow | Cerebral blood flow was measured using transcranial Doppler (TCD) sonography. Change from baseline in cerebral blood flow as measured by the time-averaged mean of the maximum (TAMM) TCD velocity is reported. | Baseline, Week 48 |
| Part D: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | An adverse event (AE) was defined as any untoward medical occurrence in a participant administered a pharmaceutical product during the course of a clinical investigation. TEAE was defined as an AE that emerged on or after initiation of study drug (having been absent pre-treatment), or an AE that existed pre-treatment and worsened on treatment (relative to the pre-treatment state) through 28 days after study drug discontinuation. An SAE was any AE that resulted in any of the following outcomes: death, life threatening adverse drug experience, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, congenital anomaly or birth defect, other important medical events. AEs were classified as SCD-related and non-SCD related. |
| Measure | Description | Time Frame |
|---|---|---|
| Part A: Maximum Concentration (Cmax) of Voxelotor in Plasma | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose | |
| Part A: Maximum Concentration (Cmax) of Voxelotor in Red Blood Cells (RBC) | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
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Inclusion Criteria:
Male or female participants with homozygous hemoglobin SS (HbSS) or hemoglobin S beta0 thalassemia (HbS β0thal)
Age:
Hydroxyurea (HU) therapy:
Hemoglobin (HB):
For Part C only: Participants 12 to 17 years of age must have a TCD velocity of ≥ 140 cm/sec measured anytime during screening.
Exclusion Criteria:
Any one of the following requiring medical attention within 14 days of signing the Informed Consent Form (ICF):
Requires chronic transfusion therapy
History of stroke or meeting criteria for primary stroke prophylaxis (history of two TCD measurements ≥ 200 cm/sec by non-imaging TCD or ≥185 cm/sec by TCDi).
Transfusion within 30 days prior to signing the ICF
Exclusion Criteria for Part D Only:
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Brentwood Clinic UCSF Benioff Children's Hospital Oakland | Brentwood | California | 94513 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 35451176 | Derived | Estepp JH, Kalpatthi R, Woods G, Trompeter S, Liem RI, Sims K, Inati A, Inusa BPD, Campbell A, Piccone C, Abboud MR, Smith-Whitley K, Dixon S, Tonda M, Washington C, Griffin NM, Brown C. Safety and efficacy of voxelotor in pediatric patients with sickle cell disease aged 4 to 11 years. Pediatr Blood Cancer. 2022 Aug;69(8):e29716. doi: 10.1002/pbc.29716. Epub 2022 Apr 21. |
| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
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Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.
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A total of 147 pediatric participants with SCD (13 in Part A, 40 in Part B, 62 in Part C and 32 in Part D) were enrolled in the study.
This study consisted of four parts-Part A, B, C and D. The study was terminated as the emerging clinical data indicated that the risk profile of voxelotor in people with sickle cell disease (SCD) exceeded the benefits observed in previously generated global research and required further assessment.
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| ID | Title | Description |
|---|---|---|
| FG000 | Part A: Participants Aged 12 to 17 Years: Voxelotor 600 mg | Participants aged 12 to 17 years received a single oral dose of voxelotor 600 milligrams (mg) on Day 1. |
| FG001 | Part A: Participants Aged 6 to 11 Years: Voxelotor 600 mg |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Part A (Up to 15 Days) |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 28, 2021 | May 16, 2025 |
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| From start of study treatment up to 28 days after study treatment discontinuation (Up to 52 weeks) |
| Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Voxelotor in Plasma | AUC0-last was calculated using the linear/log trapezoid rule. | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
| Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Voxelotor in RBC | AUC0-last was calculated using the linear/log trapezoid rule. | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
| Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 Extrapolated to Infinity (AUC0-inf) of Voxelotor in Plasma | AUC0-inf was calculated as AUCt + Ct/lambdaz, where AUCt=area under the concentration-time curve at designated time, Ct is the last quantifiable concentration for plasma and lambdaz is the elimination rate constant. | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
| Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 Extrapolated to Infinity (AUC0-inf) of Voxelotor in RBC | AUC0-inf was calculated as AUCt + Ct/lambdaz, where AUCt=area under the concentration-time curve at designated time, Ct is the last quantifiable concentration and lambdaz is the elimination rate constant. | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
| Part A: Time at Which Cmax Was Observed (Tmax) for Voxelotor in Whole Blood | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
| Part A: Time at Which Cmax Was Observed (Tmax) for Voxelotor in Plasma | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
| Part A: Time at Which Cmax Was Observed (Tmax) for Voxelotor in RBC | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
| Part A: Terminal Elimination Half-Life (T1/2) for Voxelotor in Whole Blood | T1/2 was the time measured for the drug concentration to decrease by one half. | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
| Part A: Terminal Elimination Half-Life (T1/2) for Voxelotor in Plasma | T1/2 was the time measured for the drug concentration to decrease by one half. | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
| Part A: Terminal Elimination Half-Life (T1/2) for Voxelotor in RBC | T1/2 was the time measured for the drug concentration to decrease by one half. | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
| Part A: Percentage Hemoglobin (Hb) Occupancy | Percentage hemoglobin occupancy (% Hb Occupancy) refers to the percentage of hemoglobin molecules within red blood cells that were bound to study drug. Concentration of voxelotor in whole blood and plasma was used to calculate %Hb occupancy. Percentage Hb occupancy within RBCs was estimated using the formula: ([Concentration of voxelotor in whole blood- {1-hematocrit}]*[Concentration of voxelotor in plasma/hematocrit])/5000*100. | 15 days |
| Part B: Percentage of Days With SCD Symptom Exacerbation During the First 24 Weeks of Treatment | SCD symptoms were measured using the Patient Reported Outcome (PRO), Sickle Cell Disease Severity Measure (SCDSM) which was a self-administered 9-item questionnaire of SCD core symptoms including pain severity, frequency, and type, as well as fatigue and mental acuity, on a 4-point response scale that was completed daily using a handheld electronic device by the participants. | From Day 1 up to 24 weeks |
| Part B: Change From Baseline to Week 21 to 24 in the Sickle Cell Disease Severity Measure (SCDSM) Total Symptom Score (TSS) | The SCDSM was a self-administered 9-item questionnaire of SCD core symptoms including pain severity, frequency, and type, as well as fatigue and mental acuity, on a 4-point response scale with a range of 0 (strongly disagree) to 4 (strongly agree) that was completed daily using a handheld electronic device by the participants. TSS was calculated as the sum of the 9-item questionnaire scores scaled to a 100-point scale with a range of 0 (no symptoms) to 100 (most severe symptoms). Baseline TSS was the average of the non-missing score during the Screening period. The average of change from baseline in SCDSM TSS score for the 4-week period (Week 21 to 24) is reported. | Baseline, Weeks 21 to 24 |
| Part B: Percent Change From Baseline to Weeks 12 and 24 in Lactate Dehydrogenase (LDH) | Baseline, Weeks 12 and 24 |
| Part B: Percent Change From Baseline to Weeks 12 and 24 in Indirect Bilirubin | Baseline, Weeks 12 and 24 |
| Part B: Percent Change From Baseline to Weeks 12 and 24 in Percentage Reticulocytes | Baseline, Weeks 12 and 24 |
| Part B: Cmax of Voxelotor in Whole Blood and Plasma | Day 1 (pre-dose, 2, 8, 24 hours post-dose), pre-dose on Weeks 2, 4, 8, 12, 16, 20 and 24 |
| Part B: Area Under the Concentration-Time Curve (AUC) From Time 0 Extrapolated to Infinity (AUC0-inf) of Voxelotor for Whole Blood and Plasma | AUC0-inf was calculated as AUCt + Ct/lambdaz, where AUCt=area under the concentration-time curve at designated time, Ct is the last quantifiable concentration and lambdaz is the elimination rate constant. | Day 1 (pre-dose, 2, 8, 24 hours post-dose), pre-dose on Weeks 2, 4, 8, 12, 16, 20 and 24 |
| Part B: Terminal Elimination Half-life of Voxelotor for Plasma and Whole Blood | T1/2 was the time measured for the drug concentration to decrease by one half. | Day 1 (pre-dose, 2, 8, 24 hours post-dose), pre-dose on Weeks 2, 4, 8, 12, 16, 20 and 24 |
| Part B: Accumulation Ratio (Rac) of Voxelotor for Plasma and Whole Blood | Accumulation ratio was calculated as ratio of area under the concentration-time curve from time 0 to 24 hours (AUC0-24) at steady-state (Day 28) to AUC0-24 on Day 1. | Day 1 (0 to 24 hours post-dose) and Day 28 (0 to 24 hours post-dose) |
| Part B: Percentage Hemoglobin Occupancy | Percentage hemoglobin occupancy (% Hb Occupancy) refers to the percentage of hemoglobin molecules within red blood cells that were bound to study drug. Concentration of voxelotor in whole blood and plasma was used to calculate %Hb occupancy. Percentage Hb occupancy within RBCs was estimated using the formula: ([Concentration of voxelotor in whole blood- {1-hematocrit}]*[Concentration of voxelotor in plasma/hematocrit])/5000*100. | Day 28 |
| Part B: Change From Baseline to Week 12 and 24 in Cerebral Blood Flow | Cerebral blood flow was measured using TCD sonography. Change from baseline in cerebral blood flow as measured by TAMM TCD velocity is reported. | Baseline, Weeks 12 and 24 |
| Part C: Change From Baseline to Weeks 24 and 48 in Hemoglobin Level | Baseline, Weeks 24 and 48 |
| Part C: Percent Change From Baseline to Weeks 24 and 48 in LDH | Baseline, Weeks 24 and 48 |
| Part C: Percent Change From Baseline to Weeks 24 and 48 in Indirect Bilirubin | Baseline, Weeks 24 and 48 |
| Part C: Percent Change From Baseline to Weeks 24 and 48 in Percentage Reticulocytes | Baseline, Weeks 24 and 48 |
| Part C: Change From Baseline to Week 24 in Cerebral Blood Flow | Cerebral blood flow was measured using TCD sonography. Change from baseline in cerebral blood flow as measured by TAMM TCD velocity is reported. | Baseline, Week 24 |
| Part C: Time to Initial Hemoglobin Response | Time to initial Hb response was defined as the time from first dose of study treatment to the first occurrence of a change from baseline in Hb > 1 gram per deciliter (g/dL). | From first dose of study treatment (Day 1) up to Week 48 |
| Part C: Cmax of Voxelotor for Plasma and Whole Blood | Day 1 (15 minutes to 2 hours post-dose), pre-dose on Weeks 4, 8, 12, 16, 20, 24, 36 and 48 |
| Part C: Area Under the Concentration-Time Curve (AUC) From Time 0 Extrapolated to Infinity (AUC0-inf) of Voxelotor for Whole Blood and Plasma | AUC0-inf was calculated as AUCt + Ct/lambdaz, where AUCt=area under the concentration-time curve at designated time, Ct is the last quantifiable concentration and lambdaz is the elimination rate constant. | Day 1 (15 minutes to 2 hours post-dose), pre-dose on Weeks 4, 8, 12, 16, 20, 24, 36 and 48 |
| Part C: Terminal Elimination Half-life (T1/2) of Voxelotor for Whole Blood and Plasma | T1/2 was the time measured for the drug concentration to decrease by one half. | Day 1 (15 minutes to 2 hours post-dose), pre-dose on Weeks 4, 8, 12, 16, 20, 24, 36 and 48 |
| Part C: Percentage Hemoglobin Occupancy of Voxelotor | Percentage hemoglobin occupancy (% Hb Occupancy) refers to the percentage of hemoglobin molecules within red blood cells that were bound to study drug. Concentration of voxelotor in whole blood and plasma was used to calculate %Hb occupancy. Percentage Hb occupancy within RBCs was estimated using the formula: ([Concentration of voxelotor in whole blood- {1-hematocrit}]*[Concentration of voxelotor in plasma/hematocrit])/5000*100. | Day 28 |
| Part C: Percentage of Participants With Normal Transcranial Doppler (TCD) Flow Velocity at Week 48 | Normal TCD flow velocity was considered as < 170 centimeter per second (cm/sec) by non-imagining TCD or < 155 cm/sec by imaging transcranial Doppler (TCDi). Percentage of participants with normal TCD flow velocity at Week 48 by Baseline TCD group (i.e. Baseline normal TCD [<170 cm/sec] and Baseline conditional TCD [>=170 cm/sec] is reported. | Week 48 |
| Part C: Annualized Incidence Rate of Vaso-occlusive Crisis (VOC) Events | VOC events included preferred terms of sickle cell anaemia with crisis, acute chest syndrome, pneumonia necrotising and pneumonia. Annualized incidence rate was calculated as total number of events divided by total person years. Total person years=sum of participants treatment period in years, which covered the time from first dose to last dose. | Up to Week 48 |
| Part C: Annualized Incidence Rate of Stroke Events | Annualized incidence rate was calculated as total number of events divided by total person years. Total person years=sum of participants treatment period in years, which covered the time from first dose to last dose. | Up to Week 48 |
| Part D: Cmax of Voxelotor for Plasma and Whole Blood | Day 1 (anytime between 15 minutes to 2 hours post-dose), pre-dose on Weeks 2, 8, 12, 16, 24, 36 and 48 |
| Part D: Area Under the Concentration-Time Curve (AUC) From Time 0 Extrapolated to Infinity (AUC0-inf) of Voxelotor for Plasma and Whole Blood | AUC0-inf was calculated as AUCt + Ct/lambdaz, where AUCt=area under the concentration-time curve at designated time, Ct is the last quantifiable concentration and lambdaz is the elimination rate constant. | Day 1 (anytime between 15 minutes to 2 hours post-dose), pre-dose on Weeks 2, 8, 12, 16, 24, 36 and 48 |
| Part D: T1/2 of Voxelotor for Plasma and Whole Blood | T1/2 was the time measured for the drug concentration to decrease by one half. | Day 1 (anytime between 15 minutes to 2 hours post-dose), pre-dose on Weeks 2, 8, 12, 16, 24, 36 and 48 |
| Part D: Percentage Hemoglobin Occupancy | Percentage hemoglobin occupancy (% Hb Occupancy) refers to the percentage of hemoglobin molecules within red blood cells that were bound to study drug. Concentration of voxelotor in whole blood and plasma was used to calculate %Hb occupancy. Percentage Hb occupancy within RBCs was estimated using the formula: ([Concentration of voxelotor in whole blood- {1-hematocrit}]*[Concentration of voxelotor in plasma/hematocrit])/5000*100. | Day 28 |
| Part D: Change From Baseline to Weeks 24 and 48 in Hemoglobin Level | Baseline, Weeks 24 and 48 |
| Part D: Percent Change From Baseline to Weeks 24 and 48 in LDH | Baseline, Weeks 24 and 48 |
| Part D: Percent Change From Baseline to Weeks 24 and 48 in Indirect Bilirubin | Baseline, Weeks 24 and 48 |
| Part D: Percent Change From Baseline to Weeks 24 and 48 in Reticulocytes Count | Baseline, Weeks 24 and 48 |
| Part D: Time to Initial Hemoglobin Response | Time to initial Hb response, defined as the time from first dose of study treatment to the first occurrence of a change from baseline in Hb > 1 g/dL. | From first dose of study treatment (Day 1) up to Week 48 |
| Part D: Annualized Incidence Rate of VOC Events | VOC events included preferred terms of 'Sickle cell anemia with crisis', 'Acute chest syndrome', 'Pneumonia necrotising,' and 'Pneumonia. Annualized incidence rate was calculated as total number of events divided by total person years. Total person years=sum of participants treatment period in years, which covered the time from first dose to last dose. | Up to Week 48 |
| Part D: Annualized Incidence Rate of Stroke Events | Annualized incidence rate was calculated as total number of events divided by total person years. Total person years=sum of participants treatment period in years, which covered the time from first dose to last dose. | Up to Week 48 |
| Children's National Medical Center |
| Washington D.C. |
| District of Columbia |
| 20010 |
| United States |
| Children's Healthcare of Atlanta Scottish Rite | Atlanta | Georgia | 30342 | United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| University of Illinois at Chicago Clinical Research Center | Chicago | Illinois | 60612 | United States |
| Our Lady of the Lake Children's Hospital (IP Address) | Baton Rouge | Louisiana | 70809 | United States |
| Children's Mercy Hospital | Kansas City | Missouri | 64108 | United States |
| Rutgers-Robert Wood Johnson Medical School | New Brunswick | New Jersey | 08903 | United States |
| Brody School of Medicine at East Carolina University | Greenville | North Carolina | 27834 | United States |
| University Hospitals Cleveland Medical Center, Rainbow Babies & Children's Hospital | Cleveland | Ohio | 44106 | United States |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | 15224 | United States |
| St. Jude Children's Research Hospital | Memphis | Tennessee | 38105 | United States |
| American University of Beirut - Medical Center | Beirut | Lebanon |
| Rafik Hariri University Hospital | Beirut | Lebanon |
| Nini Hospital | Tripoli | Lebanon |
| University College London Hospital, NHS Foundation Trust | London | Greater London | NW1 2PG | United Kingdom |
| Barts Health NHS Trust, The Royal London Hospital | London | E1 1BB | United Kingdom |
| Guy's and St Thoma's NHS Foundation Trust, Evelina London Children's Hospital | London | SE1 7EH | United Kingdom |
| Manchester University NHS Foundation Trust, Royal Manchester Children's Hospital | Manchester | M13 9WL | United Kingdom |
Participants aged 6 to 11 years received a single oral dose of voxelotor 600 mg on Day 1.
| FG002 | Part B: Voxelotor 900 mg QD | Participants aged 12 to 17 years received once daily (QD) oral dose of voxelotor 900 mg for 24 weeks. |
| FG003 | Part B: Voxelotor 1500 mg QD | Participants aged 12 to 17 years received once daily oral dose of voxelotor 1500 mg for 24 weeks. |
| FG004 | Part C: Participants Aged 4 to 11 Years: Voxelotor 1500 mg QD | Participants aged 4 to 11 years received once daily oral dose of voxelotor 1500 mg for 48 weeks. |
| FG005 | Part C: Participants Aged 12 to 17 Years: Voxelotor 1500 mg QD | Participants aged 12 to 17 years received once daily oral dose of voxelotor 1500 mg for 48 weeks. |
| FG006 | Part D: Participants Aged 6 Months to <2 Years: Voxelotor 1500 mg QD | Participants aged 6 months to <2 years received once daily oral dose of voxelotor 1500 mg for 48 weeks. |
| FG007 | Part D: Participants Aged 2 to <4 Years: Voxelotor 1500 mg QD | Participants aged 2 to <4 years received once daily oral dose of voxelotor 1500 mg for 48 weeks. |
| COMPLETED |
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| NOT COMPLETED |
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| Part B (Up to 28 Weeks) |
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| Part C (Up to 52 Weeks) |
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| Part D (Up to 52 Weeks) |
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Safety Population comprised of all participants who received at least one dose of study drug.
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| ID | Title | Description |
|---|---|---|
| BG000 | Part A: Participants Aged 12 to 17 Years: Voxelotor 600 mg | Participants aged 12 to 17 years received a single oral dose of voxelotor 600 milligrams (mg) on Day 1. |
| BG001 | Part A: Participants Aged 6 to 11 Years: Voxelotor 600 mg | Participants aged 6 to 11 years received a single oral dose of voxelotor 600 mg on Day 1. |
| BG002 | Part B: Voxelotor 900 mg QD | Participants aged 12 to 17 years received once daily (QD) oral dose of voxelotor 900 mg for 24 weeks. |
| BG003 | Part B: Voxelotor 1500 mg QD | Participants aged 12 to 17 years received once daily oral dose of voxelotor 1500 mg for 24 weeks. |
| BG004 | Part C: Participants Aged 4 to 11 Years: Voxelotor 1500 mg QD | Participants aged 4 to 11 years received once daily oral dose of voxelotor 1500 mg for 48 weeks. |
| BG005 | Part C: Participants Aged 12 to 17 Years: Voxelotor 1500 mg QD | Participants aged 12 to 17 years received once daily oral dose of voxelotor 1500 mg for 48 weeks. |
| BG006 | Part D: Participants Aged 6 Months to <2 Years: Voxelotor 1500 mg QD | Participants aged 6 months to <2 years received once daily oral dose of voxelotor 1500 mg for 48 weeks. |
| BG007 | Part D: Participants Aged 2 to <4 Years: Voxelotor 1500 mg QD | Participants aged 2 to <4 years received once daily oral dose of voxelotor 1500 mg for 48 weeks. |
| BG008 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||
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| Primary | Part A: Maximum Concentration (Cmax) of Voxelotor in Whole Blood | Pharmacokinetic (PK) population comprised of all participants who received any amount of study drug and who provided PK data from at least one post-dose sample in plasma. | Posted | Geometric Mean | Geometric Coefficient of Variation | Nanogram per milliliter | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
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| Primary | Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Voxelotor in Whole Blood | AUC0-last was calculated using the linear/log trapezoid rule. | PK population comprised of all participants who received any amount of study drug and who provided PK data from at least one post-dose sample in plasma. | Posted | Geometric Mean | Geometric Coefficient of Variation | Hours*nanogram per milliliter | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
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| Primary | Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 Extrapolated to Infinity (AUC0-inf) of Voxelotor in Whole Blood | AUC0-inf was calculated as AUCt + Ct/lambdaz, where AUCt=area under the concentration-time curve at designated time, Ct is the last quantifiable concentration for whole blood and lambdaz=elimination rate constant. | PK population comprised of all participants who received any amount of study drug and who provided PK data from at least one post-dose sample in plasma. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Geometric Mean | Geometric Coefficient of Variation | Hours*nanogram per milliliter | pre-dose, 2, 8, 24, 48, 96,168 and 336 hours post-dose |
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| Primary | Part B: Change From Baseline to Week 24 in Hemoglobin Level | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Gram per deciliter | Baseline, Week 24 |
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| Primary | Part C: Change From Baseline to Week 48 in Cerebral Blood Flow | Cerebral blood flow was measured using transcranial Doppler (TCD) sonography. Change from baseline in cerebral blood flow as measured by the time-averaged mean of the maximum (TAMM) TCD velocity is reported. | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Centimeter per second | Baseline, Week 48 |
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| Primary | Part D: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | An adverse event (AE) was defined as any untoward medical occurrence in a participant administered a pharmaceutical product during the course of a clinical investigation. TEAE was defined as an AE that emerged on or after initiation of study drug (having been absent pre-treatment), or an AE that existed pre-treatment and worsened on treatment (relative to the pre-treatment state) through 28 days after study drug discontinuation. An SAE was any AE that resulted in any of the following outcomes: death, life threatening adverse drug experience, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, congenital anomaly or birth defect, other important medical events. AEs were classified as SCD-related and non-SCD related. | Safety population comprised of all participants who received any amount of study drug. | Posted | Count of Participants | Participants | From start of study treatment up to 28 days after study treatment discontinuation (Up to 52 weeks) |
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| Secondary | Part A: Maximum Concentration (Cmax) of Voxelotor in Plasma | PK population comprised of all participants who received any amount of study drug and who provided PK data from at least one post-dose sample in plasma. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Geometric Mean | Geometric Coefficient of Variation | Nanogram per milliliter | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
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| Secondary | Part A: Maximum Concentration (Cmax) of Voxelotor in Red Blood Cells (RBC) | PK population comprised of all participants who received any amount of study drug and who provided PK data from at least one post-dose sample in plasma. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Geometric Mean | Geometric Coefficient of Variation | Nanogram per milliliter | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
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| Secondary | Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Voxelotor in Plasma | AUC0-last was calculated using the linear/log trapezoid rule. | PK population comprised of all participants who received any amount of study drug and who provided PK data from at least one post-dose sample in plasma. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Geometric Mean | Geometric Coefficient of Variation | Hours*nanogram per milliliter | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
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| Secondary | Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Voxelotor in RBC | AUC0-last was calculated using the linear/log trapezoid rule. | PK population comprised of all participants who received any amount of study drug and who provided PK data from at least one post-dose sample in plasma. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Geometric Mean | Geometric Coefficient of Variation | Hours*nanogram per milliliter | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
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| Secondary | Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 Extrapolated to Infinity (AUC0-inf) of Voxelotor in Plasma | AUC0-inf was calculated as AUCt + Ct/lambdaz, where AUCt=area under the concentration-time curve at designated time, Ct is the last quantifiable concentration for plasma and lambdaz is the elimination rate constant. | PK population comprised of all participants who received any amount of study drug and who provided PK data from at least one post-dose sample in plasma. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Geometric Mean | Geometric Coefficient of Variation | Hours*nanogram per milliliter | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
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| Secondary | Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 Extrapolated to Infinity (AUC0-inf) of Voxelotor in RBC | AUC0-inf was calculated as AUCt + Ct/lambdaz, where AUCt=area under the concentration-time curve at designated time, Ct is the last quantifiable concentration and lambdaz is the elimination rate constant. | PK population comprised of all participants who received any amount of study drug and who provided PK data from at least one post-dose sample in plasma. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Geometric Mean | Geometric Coefficient of Variation | Hours*nanogram per milliliter | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
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| Secondary | Part A: Time at Which Cmax Was Observed (Tmax) for Voxelotor in Whole Blood | PK population comprised of all participants who received any amount of study drug and who provided PK data from at least one post-dose sample in plasma. | Posted | Median | Full Range | Hours | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
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| Secondary | Part A: Time at Which Cmax Was Observed (Tmax) for Voxelotor in Plasma | PK population comprised of all participants who received any amount of study drug and who provided PK data from at least one post-dose sample in plasma. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Median | Full Range | Hours | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
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| Secondary | Part A: Time at Which Cmax Was Observed (Tmax) for Voxelotor in RBC | PK population comprised of all participants who received any amount of study drug and who provided PK data from at least one post-dose sample in plasma. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Median | Full Range | Hours | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
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| Secondary | Part A: Terminal Elimination Half-Life (T1/2) for Voxelotor in Whole Blood | T1/2 was the time measured for the drug concentration to decrease by one half. | PK population comprised of all participants who received any amount of study drug and who provided PK data from at least one post-dose sample in plasma. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Geometric Mean | Geometric Coefficient of Variation | Hours | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
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| Secondary | Part A: Terminal Elimination Half-Life (T1/2) for Voxelotor in Plasma | T1/2 was the time measured for the drug concentration to decrease by one half. | PK population comprised of all participants who received any amount of study drug and who provided PK data from at least one post-dose sample in plasma. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Geometric Mean | Geometric Coefficient of Variation | Hours | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
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| Secondary | Part A: Terminal Elimination Half-Life (T1/2) for Voxelotor in RBC | T1/2 was the time measured for the drug concentration to decrease by one half. | PK population comprised of all participants who received any amount of study drug and who provided PK data from at least one post-dose sample in plasma. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Geometric Mean | Geometric Coefficient of Variation | Hours | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose |
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| Secondary | Part A: Percentage Hemoglobin (Hb) Occupancy | Percentage hemoglobin occupancy (% Hb Occupancy) refers to the percentage of hemoglobin molecules within red blood cells that were bound to study drug. Concentration of voxelotor in whole blood and plasma was used to calculate %Hb occupancy. Percentage Hb occupancy within RBCs was estimated using the formula: ([Concentration of voxelotor in whole blood- {1-hematocrit}]*[Concentration of voxelotor in plasma/hematocrit])/5000*100. | Data was not collected as the model for Hb occupancy within RBCs was not developed for single dose administration (Part A). The model used to determine % Hb Occupancy was constructed via populational PK modelling and required data from multiple dose administration, thus cannot be applied to estimate the % Hb Occupancy for single doses. | Posted | 15 days |
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| Secondary | Part B: Percentage of Days With SCD Symptom Exacerbation During the First 24 Weeks of Treatment | SCD symptoms were measured using the Patient Reported Outcome (PRO), Sickle Cell Disease Severity Measure (SCDSM) which was a self-administered 9-item questionnaire of SCD core symptoms including pain severity, frequency, and type, as well as fatigue and mental acuity, on a 4-point response scale that was completed daily using a handheld electronic device by the participants. | The variable represents a derivation from a new instrument developed by the sponsor specifically for this clinical program. Due to challenges in the validation of "Percentage of Days With SCD Symptom Exacerbation" from the instrument, specifically with the impact of missing data, the construct of this variable was not possible. A decision not to analyze this variable was documented in the statistical analysis plan. | Posted | From Day 1 up to 24 weeks |
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| Secondary | Part B: Change From Baseline to Week 21 to 24 in the Sickle Cell Disease Severity Measure (SCDSM) Total Symptom Score (TSS) | The SCDSM was a self-administered 9-item questionnaire of SCD core symptoms including pain severity, frequency, and type, as well as fatigue and mental acuity, on a 4-point response scale with a range of 0 (strongly disagree) to 4 (strongly agree) that was completed daily using a handheld electronic device by the participants. TSS was calculated as the sum of the 9-item questionnaire scores scaled to a 100-point scale with a range of 0 (no symptoms) to 100 (most severe symptoms). Baseline TSS was the average of the non-missing score during the Screening period. The average of change from baseline in SCDSM TSS score for the 4-week period (Week 21 to 24) is reported. | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Units on a scale | Baseline, Weeks 21 to 24 |
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| Secondary | Part B: Percent Change From Baseline to Weeks 12 and 24 in Lactate Dehydrogenase (LDH) | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'Number Analyzed' signifies number of participants evaluable for the specified rows. | Posted | Mean | Standard Deviation | Percent change | Baseline, Weeks 12 and 24 |
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| Secondary | Part B: Percent Change From Baseline to Weeks 12 and 24 in Indirect Bilirubin | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'Number Analyzed' signifies number of participants evaluable for the specified rows. | Posted | Mean | Standard Deviation | Percent change | Baseline, Weeks 12 and 24 |
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| Secondary | Part B: Percent Change From Baseline to Weeks 12 and 24 in Percentage Reticulocytes | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'Number Analyzed' signifies number of participants evaluable for the specified rows. | Posted | Mean | Standard Deviation | Percent change | Baseline, Weeks 12 and 24 |
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| Secondary | Part B: Cmax of Voxelotor in Whole Blood and Plasma | PK population comprised of participants who received at least one dose of voxelotor and had at least 1 measurable voxelotor concentration observation in plasma or whole blood with associated sampling time and dosing information. | Posted | Geometric Mean | Geometric Coefficient of Variation | Microgram per milliliter | Day 1 (pre-dose, 2, 8, 24 hours post-dose), pre-dose on Weeks 2, 4, 8, 12, 16, 20 and 24 |
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| Secondary | Part B: Area Under the Concentration-Time Curve (AUC) From Time 0 Extrapolated to Infinity (AUC0-inf) of Voxelotor for Whole Blood and Plasma | AUC0-inf was calculated as AUCt + Ct/lambdaz, where AUCt=area under the concentration-time curve at designated time, Ct is the last quantifiable concentration and lambdaz is the elimination rate constant. | PK population comprised of participants who received at least one dose of voxelotor and had at least 1 measurable voxelotor concentration observation in plasma or whole blood with associated sampling time and dosing information. | Posted | Geometric Mean | Geometric Coefficient of Variation | Hours*microgram per milliliter | Day 1 (pre-dose, 2, 8, 24 hours post-dose), pre-dose on Weeks 2, 4, 8, 12, 16, 20 and 24 |
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| Secondary | Part B: Terminal Elimination Half-life of Voxelotor for Plasma and Whole Blood | T1/2 was the time measured for the drug concentration to decrease by one half. | PK population comprised of participants who received at least one dose of voxelotor and had at least 1 measurable voxelotor concentration observation in plasma or whole blood with associated sampling time and dosing information. | Posted | Geometric Mean | Geometric Coefficient of Variation | Hours | Day 1 (pre-dose, 2, 8, 24 hours post-dose), pre-dose on Weeks 2, 4, 8, 12, 16, 20 and 24 |
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| Secondary | Part B: Accumulation Ratio (Rac) of Voxelotor for Plasma and Whole Blood | Accumulation ratio was calculated as ratio of area under the concentration-time curve from time 0 to 24 hours (AUC0-24) at steady-state (Day 28) to AUC0-24 on Day 1. | PK population comprised of participants who received at least one dose of voxelotor and had at least 1 measurable voxelotor concentration observation in plasma or whole blood with associated sampling time and dosing information. | Posted | Geometric Mean | Geometric Coefficient of Variation | Ratio | Day 1 (0 to 24 hours post-dose) and Day 28 (0 to 24 hours post-dose) |
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| Secondary | Part B: Percentage Hemoglobin Occupancy | Percentage hemoglobin occupancy (% Hb Occupancy) refers to the percentage of hemoglobin molecules within red blood cells that were bound to study drug. Concentration of voxelotor in whole blood and plasma was used to calculate %Hb occupancy. Percentage Hb occupancy within RBCs was estimated using the formula: ([Concentration of voxelotor in whole blood- {1-hematocrit}]*[Concentration of voxelotor in plasma/hematocrit])/5000*100. | PK population comprised of participants who received at least one dose of voxelotor and had at least 1 measurable voxelotor concentration observation in plasma or whole blood with associated sampling time and dosing information. | Posted | Geometric Mean | Geometric Coefficient of Variation | Percentage of bound hemoglobin | Day 28 |
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| Secondary | Part B: Change From Baseline to Week 12 and 24 in Cerebral Blood Flow | Cerebral blood flow was measured using TCD sonography. Change from baseline in cerebral blood flow as measured by TAMM TCD velocity is reported. | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'Number Analyzed' signifies number of participants evaluable for the specified rows. | Posted | Mean | Standard Deviation | Centimeter per second | Baseline, Weeks 12 and 24 |
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| Secondary | Part C: Change From Baseline to Weeks 24 and 48 in Hemoglobin Level | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'Number Analyzed' signifies number of participants evaluable for the specified rows. | Posted | Mean | Standard Deviation | Grams per deciliter | Baseline, Weeks 24 and 48 |
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| Secondary | Part C: Percent Change From Baseline to Weeks 24 and 48 in LDH | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'Number Analyzed' signifies number of participants evaluable for the specified rows. | Posted | Mean | Standard Deviation | Percent change | Baseline, Weeks 24 and 48 |
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| Secondary | Part C: Percent Change From Baseline to Weeks 24 and 48 in Indirect Bilirubin | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'Number Analyzed' signifies number of participants evaluable for the specified rows. | Posted | Mean | Standard Deviation | Percent change | Baseline, Weeks 24 and 48 |
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| Secondary | Part C: Percent Change From Baseline to Weeks 24 and 48 in Percentage Reticulocytes | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'Number Analyzed' signifies number of participants evaluable for the specified rows. | Posted | Mean | Standard Deviation | Percent change | Baseline, Weeks 24 and 48 |
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| Secondary | Part C: Change From Baseline to Week 24 in Cerebral Blood Flow | Cerebral blood flow was measured using TCD sonography. Change from baseline in cerebral blood flow as measured by TAMM TCD velocity is reported. | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Centimeter per second | Baseline, Week 24 |
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| Secondary | Part C: Time to Initial Hemoglobin Response | Time to initial Hb response was defined as the time from first dose of study treatment to the first occurrence of a change from baseline in Hb > 1 gram per deciliter (g/dL). | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Weeks | From first dose of study treatment (Day 1) up to Week 48 |
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| Secondary | Part C: Cmax of Voxelotor for Plasma and Whole Blood | PK population comprised of participants who received at least one dose of voxelotor and had at least 1 measurable voxelotor concentration observation in plasma or whole blood with associated sampling time and dosing information. Here, 'Overall Number of Participants Analyzed' signifies participants evaluable for this outcome measure. | Posted | Geometric Mean | Geometric Coefficient of Variation | Microgram per milliliter | Day 1 (15 minutes to 2 hours post-dose), pre-dose on Weeks 4, 8, 12, 16, 20, 24, 36 and 48 |
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| Secondary | Part C: Area Under the Concentration-Time Curve (AUC) From Time 0 Extrapolated to Infinity (AUC0-inf) of Voxelotor for Whole Blood and Plasma | AUC0-inf was calculated as AUCt + Ct/lambdaz, where AUCt=area under the concentration-time curve at designated time, Ct is the last quantifiable concentration and lambdaz is the elimination rate constant. | PK population comprised of participants who received at least one dose of voxelotor and had at least 1 measurable voxelotor concentration observation in plasma or whole blood with associated sampling time and dosing information. Here, 'Overall Number of Participants Analyzed' signifies participants evaluable for this outcome measure. | Posted | Geometric Mean | Geometric Coefficient of Variation | Hours*microgram per milliliter | Day 1 (15 minutes to 2 hours post-dose), pre-dose on Weeks 4, 8, 12, 16, 20, 24, 36 and 48 |
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| Secondary | Part C: Terminal Elimination Half-life (T1/2) of Voxelotor for Whole Blood and Plasma | T1/2 was the time measured for the drug concentration to decrease by one half. | PK population comprised of participants who received at least one dose of voxelotor and had at least 1 measurable voxelotor concentration observation in plasma or whole blood with associated sampling time and dosing information. Here, 'Overall Number of Participants Analyzed' signifies participants evaluable for this outcome measure. | Posted | Geometric Mean | Geometric Coefficient of Variation | Hours | Day 1 (15 minutes to 2 hours post-dose), pre-dose on Weeks 4, 8, 12, 16, 20, 24, 36 and 48 |
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| Secondary | Part C: Percentage Hemoglobin Occupancy of Voxelotor | Percentage hemoglobin occupancy (% Hb Occupancy) refers to the percentage of hemoglobin molecules within red blood cells that were bound to study drug. Concentration of voxelotor in whole blood and plasma was used to calculate %Hb occupancy. Percentage Hb occupancy within RBCs was estimated using the formula: ([Concentration of voxelotor in whole blood- {1-hematocrit}]*[Concentration of voxelotor in plasma/hematocrit])/5000*100. | PK population comprised of participants who received at least one dose of voxelotor and had at least 1 measurable voxelotor concentration observation in plasma or whole blood with associated sampling time and dosing information. Here, 'Overall Number of Participants Analyzed' signifies participants evaluable for this outcome measure. | Posted | Geometric Mean | Geometric Coefficient of Variation | Percentage of bound hemoglobin | Day 28 |
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| Secondary | Part C: Percentage of Participants With Normal Transcranial Doppler (TCD) Flow Velocity at Week 48 | Normal TCD flow velocity was considered as < 170 centimeter per second (cm/sec) by non-imagining TCD or < 155 cm/sec by imaging transcranial Doppler (TCDi). Percentage of participants with normal TCD flow velocity at Week 48 by Baseline TCD group (i.e. Baseline normal TCD [<170 cm/sec] and Baseline conditional TCD [>=170 cm/sec] is reported. | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. All participants reported under, 'Overall Number of Participants Analyzed' contributed data to the table; however, may not have evaluable data for each row. Here, 'Number Analyzed ' signifies number of participants evaluable for each row. | Posted | Number | Percentage of participants | Week 48 |
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| Secondary | Part C: Annualized Incidence Rate of Vaso-occlusive Crisis (VOC) Events | VOC events included preferred terms of sickle cell anaemia with crisis, acute chest syndrome, pneumonia necrotising and pneumonia. Annualized incidence rate was calculated as total number of events divided by total person years. Total person years=sum of participants treatment period in years, which covered the time from first dose to last dose. | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. | Posted | Number | 95% Confidence Interval | VOC events per person year | Up to Week 48 |
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| Secondary | Part C: Annualized Incidence Rate of Stroke Events | Annualized incidence rate was calculated as total number of events divided by total person years. Total person years=sum of participants treatment period in years, which covered the time from first dose to last dose. | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. | Posted | Number | 95% Confidence Interval | Stroke events per person year | Up to Week 48 |
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| Secondary | Part D: Cmax of Voxelotor for Plasma and Whole Blood | PK population comprised of participants who received at least one dose of voxelotor and had at least 1 measurable voxelotor concentration observation in plasma or whole blood with associated sampling time and dosing information. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Geometric Mean | Geometric Coefficient of Variation | Microgram per milliliter | Day 1 (anytime between 15 minutes to 2 hours post-dose), pre-dose on Weeks 2, 8, 12, 16, 24, 36 and 48 |
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| Secondary | Part D: Area Under the Concentration-Time Curve (AUC) From Time 0 Extrapolated to Infinity (AUC0-inf) of Voxelotor for Plasma and Whole Blood | AUC0-inf was calculated as AUCt + Ct/lambdaz, where AUCt=area under the concentration-time curve at designated time, Ct is the last quantifiable concentration and lambdaz is the elimination rate constant. | PK population comprised of participants who received at least one dose of voxelotor and had at least 1 measurable voxelotor concentration observation in plasma or whole blood with associated sampling time and dosing information. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Geometric Mean | Geometric Coefficient of Variation | Hours*microgram per milliliter | Day 1 (anytime between 15 minutes to 2 hours post-dose), pre-dose on Weeks 2, 8, 12, 16, 24, 36 and 48 |
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| Secondary | Part D: T1/2 of Voxelotor for Plasma and Whole Blood | T1/2 was the time measured for the drug concentration to decrease by one half. | PK population comprised of participants who received at least one dose of voxelotor and had at least 1 measurable voxelotor concentration observation in plasma or whole blood with associated sampling time and dosing information. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Geometric Mean | Geometric Coefficient of Variation | Hours | Day 1 (anytime between 15 minutes to 2 hours post-dose), pre-dose on Weeks 2, 8, 12, 16, 24, 36 and 48 |
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| Secondary | Part D: Percentage Hemoglobin Occupancy | Percentage hemoglobin occupancy (% Hb Occupancy) refers to the percentage of hemoglobin molecules within red blood cells that were bound to study drug. Concentration of voxelotor in whole blood and plasma was used to calculate %Hb occupancy. Percentage Hb occupancy within RBCs was estimated using the formula: ([Concentration of voxelotor in whole blood- {1-hematocrit}]*[Concentration of voxelotor in plasma/hematocrit])/5000*100. | PK population comprised of participants who received at least one dose of voxelotor and had at least 1 measurable voxelotor concentration observation in plasma or whole blood with associated sampling time and dosing information. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Geometric Mean | Geometric Coefficient of Variation | Percentage of bound hemoglobin | Day 28 |
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| Secondary | Part D: Change From Baseline to Weeks 24 and 48 in Hemoglobin Level | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Grams per deciliter | Baseline, Weeks 24 and 48 |
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| Secondary | Part D: Percent Change From Baseline to Weeks 24 and 48 in LDH | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'Number Analyzed' signifies participants evaluable for the specified rows. | Posted | Mean | Standard Deviation | Percent change | Baseline, Weeks 24 and 48 |
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| Secondary | Part D: Percent Change From Baseline to Weeks 24 and 48 in Indirect Bilirubin | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'Number Analyzed' signifies participants evaluable for the specified rows. | Posted | Mean | Standard Deviation | Percent change | Baseline, Weeks 24 and 48 |
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| Secondary | Part D: Percent Change From Baseline to Weeks 24 and 48 in Reticulocytes Count | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure and 'Number Analyzed' signifies participants evaluable for the specified rows. | Posted | Mean | Standard Deviation | Percent change | Baseline, Weeks 24 and 48 |
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| Secondary | Part D: Time to Initial Hemoglobin Response | Time to initial Hb response, defined as the time from first dose of study treatment to the first occurrence of a change from baseline in Hb > 1 g/dL. | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Weeks | From first dose of study treatment (Day 1) up to Week 48 |
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| Secondary | Part D: Annualized Incidence Rate of VOC Events | VOC events included preferred terms of 'Sickle cell anemia with crisis', 'Acute chest syndrome', 'Pneumonia necrotising,' and 'Pneumonia. Annualized incidence rate was calculated as total number of events divided by total person years. Total person years=sum of participants treatment period in years, which covered the time from first dose to last dose. | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. | Posted | Number | 95% Confidence Interval | VOC events per person year | Up to Week 48 |
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| Secondary | Part D: Annualized Incidence Rate of Stroke Events | Annualized incidence rate was calculated as total number of events divided by total person years. Total person years=sum of participants treatment period in years, which covered the time from first dose to last dose. | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. | Posted | Number | 95% Confidence Interval | Stroke events per person year | Up to Week 48 |
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Part A: From start of treatment on Day 1 up to 2 weeks after last dose (up to Day 15); Part B, C and D: From start of treatment on Day 1 up to 4 weeks after last dose (up to Week 28 for Part B and up to Week 52 for Parts C and D).
An AE term may be reported as both serious and non-serious AE but are distinct events. An AE may be serious for 1 participant and non-serious for another participant, or a participant may have experienced both a serious and non-serious episode of the same event. Safety population comprised of all participants who received any amount of study drug.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Part A: Participants Aged 12 to 17 Years: Voxelotor 600 mg | Participants aged 12 to 17 years received a single oral dose of voxelotor 600 milligrams (mg) on Day 1. | 0 | 7 | 1 | 7 | 6 | 7 |
| EG001 | Part A: Participants Aged 6 to 11 Years: Voxelotor 600 mg | Participants aged 6 to 11 years received a single oral dose of voxelotor 600 mg on Day 1. | 0 | 6 | 0 | 6 | 3 | 6 |
| EG002 | Part B: Voxelotor 900 mg QD | Participants aged 12 to 17 years received once daily (QD) oral dose of voxelotor 900 mg for 24 weeks. | 0 | 25 | 12 | 25 | 22 | 25 |
| EG003 | Part B: Voxelotor 1500 mg QD | Participants aged 12 to 17 years received once daily oral dose of voxelotor 1500 mg for 24 weeks. | 0 | 15 | 7 | 15 | 15 | 15 |
| EG004 | Part C: Participants Aged 4 to 11 Years: Voxelotor 1500 mg QD | Participants aged 4 to 11 years received once daily oral dose of voxelotor 1500 mg for 48 weeks. | 0 | 51 | 23 | 51 | 46 | 51 |
| EG005 | Part C: Participants Aged 12 to 17 Years: Voxelotor 1500 mg QD | Participants aged 12 to 17 years received once daily oral dose of voxelotor 1500 mg for 48 weeks. | 0 | 11 | 8 | 11 | 11 | 11 |
| EG006 | Part D: Participants Aged 6 Months to <2 Years: Voxelotor 1500 mg QD | Participants aged 6 months to <2 years received once daily oral dose of voxelotor 1500 mg for 48 weeks. | 0 | 9 | 7 | 9 | 9 | 9 |
| EG007 | Part D: Participants Aged 2 to <4 Years: Voxelotor 1500 mg QD | Participants aged 2 to <4 years received once daily oral dose of voxelotor 1500 mg for 48 weeks. | 0 | 23 | 14 | 23 | 21 | 23 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Sickle cell anaemia with crisis | Blood and lymphatic system disorders | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Influenza | Infections and infestations | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Tonsillitis | Infections and infestations | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Viral infection | Infections and infestations | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Hemolytic anaemia | Blood and lymphatic system disorders | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Priapism | Reproductive system and breast disorders | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Acute chest syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Pneumonia | Infections and infestations | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Pyrexia | General disorders | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Hypersplenism | Blood and lymphatic system disorders | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Platelet disorder | Blood and lymphatic system disorders | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Salmonellosis | Infections and infestations | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Electrocardiogram T wave inversion | Investigations | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Haemoglobin decreased | Investigations | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Periorbital swelling | Eye disorders | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Cholecystitis acute | Hepatobiliary disorders | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Hypersensitivity | Immune system disorders | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Dehydration | Metabolism and nutrition disorders | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Pallor | Vascular disorders | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Anaemia | Blood and lymphatic system disorders | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Bacteraemia | Infections and infestations | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Bronchiolitis | Infections and infestations | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Bronchitis | Infections and infestations | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Malaria | Infections and infestations | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Rotavirus infection | Infections and infestations | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Sepsis | Infections and infestations | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Constipation | Gastrointestinal disorders | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Vomiting | Gastrointestinal disorders | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Ultrasound head abnormal | Investigations | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Splenectomy | Surgical and medical procedures | MedDRA19.1 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Pneumonia necrotising | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Sickle cell anaemia with crisis | Blood and lymphatic system disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Acute chest syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Osteonecrosis | Musculoskeletal and connective tissue disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Priapism | Reproductive system and breast disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Nausea | Gastrointestinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Vomiting | Gastrointestinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Constipation | Gastrointestinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Abdominal distension | Gastrointestinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Dental caries | Gastrointestinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Gastritis | Gastrointestinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Toothache | Gastrointestinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Flank pain | Musculoskeletal and connective tissue disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Viral infection | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Acute sinusitis | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Body tinea | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Cellulitis | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Conjunctivitis | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Gastroenteritis | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Pharyngitis streptococcal | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Rhinitis | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Sinusitis | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Headache | Nervous system disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Dizziness | Nervous system disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Lethargy | Nervous system disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Migraine | Nervous system disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Syncope | Nervous system disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Non-cardiac chest pain | General disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Pyrexia | General disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Pain | General disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Fatigue | General disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Chest discomfort | General disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Chest pain | General disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Peripheral swelling | General disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Upper-airway cough syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Rash generalised | Skin and subcutaneous tissue disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Rash papular | Skin and subcutaneous tissue disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Anaemia | Blood and lymphatic system disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Neutropenia | Blood and lymphatic system disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Reticulocytopenia | Blood and lymphatic system disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Thrombocytosis | Blood and lymphatic system disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Fall | Injury, poisoning and procedural complications | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Joint injury | Injury, poisoning and procedural complications | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Laceration | Injury, poisoning and procedural complications | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Transaminases increased | Investigations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Bacterial test positive | Investigations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Anxiety | Psychiatric disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Insomnia | Psychiatric disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Mental status changes | Psychiatric disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Stress | Psychiatric disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Cholelithiasis | Hepatobiliary disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Ocular icterus | Hepatobiliary disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Tachycardia | Cardiac disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Hypoacusis | Ear and labyrinth disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Tinnitus | Ear and labyrinth disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Retinopathy sickle cell | Eye disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Seasonal allergy | Immune system disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Micturition urgency | Renal and urinary disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| gastrointestinal disorder | Gastrointestinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Influenza | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Lower respiratory tract infection | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Nasopharyngitis | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Otitis media | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Urinary tract infection | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| COVID-19 | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Febrile infection | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Hepatitis A | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Impetigo | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Sepsis | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Tinea faciei | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Tooth abscess | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Viral upper respiratory tract infection | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Gait disturbance | General disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Pain in jaw | Musculoskeletal and connective tissue disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Obstructive sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Paranasal sinus hypersecretion | Respiratory, thoracic and mediastinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Tonsillar erythema | Respiratory, thoracic and mediastinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Tonsillar hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Depressed level of consciousness | Nervous system disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Hypersomnia | Nervous system disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Dermatitis allergic | Skin and subcutaneous tissue disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Alanine aminotransferase increased | Investigations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Aspartate aminotransferase increased | Investigations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Blood bilirubin increased | Investigations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Breath sounds abnormal | Investigations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Electrocardiogram QT prolonged | Investigations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Haemoglobin decreased | Investigations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Influenza A virus test positive | Investigations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Neutrophil count decreased | Investigations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Splenomegaly | Blood and lymphatic system disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Cardiomegaly | Cardiac disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Diastolic dysfunction | Cardiac disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Left atrial dilatation | Cardiac disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Ventricular hypertrophy | Cardiac disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Sports injury | Injury, poisoning and procedural complications | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Epiphyseal fracture | Injury, poisoning and procedural complications | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Foot fracture | Injury, poisoning and procedural complications | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Muscle strain | Injury, poisoning and procedural complications | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Eye pain | Eye disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Visual impairment | Eye disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Albuminuria | Renal and urinary disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Dysuria | Renal and urinary disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Proteinuria | Renal and urinary disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Allergic oedema | Immune system disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Decreased appetite | Metabolism and nutrition disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Dehydration | Metabolism and nutrition disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Encopresis | Psychiatric disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Enuresis | Psychiatric disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Post-traumatic stress disorder | Psychiatric disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Hypertransaminasaemia | Hepatobiliary disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Hypersplenism | Blood and lymphatic system disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Pneumonia | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Dactylitis | Musculoskeletal and connective tissue disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Respiratory tract infection viral | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Tonsillitis | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Gastroenteritis rotavirus | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Lip infection | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Oral herpes | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Respiratory syncytial virus infection | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Rotavirus infection | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Viral rash | Infections and infestations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Lip blister | Gastrointestinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Hypercoagulation | Blood and lymphatic system disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Rash pruritic | Skin and subcutaneous tissue disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Blood alkaline phosphatase increased | Investigations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Ultrasound head abnormal | Investigations | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Foreign body ingestion | Injury, poisoning and procedural complications | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Jaundice | Hepatobiliary disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Benign lymph node neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Hypertonic bladder | Renal and urinary disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Flatulence | Gastrointestinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
| Allergic respiratory symptom | Respiratory, thoracic and mediastinal disorders | MedDRA26.0 | Non-systematic Assessment | MedDRA v19.1 for Part A and Part B arms, MedDRA v25.1 for Part C arms and MedDRA v26.0 for Part D arms |
|
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer Inc. | 1-800-718-1021 | ClinicalTrials.gov_Inquiries@pfizer.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 3, 2023 | May 16, 2025 | SAP_001.pdf |
| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| ID | Term |
|---|---|
| C000628792 | voxelotor |
Not provided
Not provided
Not provided
| Withdrawal by Subject |
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| Lost to Follow-up |
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| Non compliance |
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| Withdrawal by Subject |
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| Physician Decision |
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| Other |
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| Withdrawal by Subject |
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| Physician Decision |
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| Other |
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| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Black or African American |
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| White |
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| Multi-racial |
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| Middle Eastern or North African |
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