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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-000903-26 | EudraCT Number | ||
| C5341021 | 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 is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ≥ 2 to < 15 years old, with Sickle Cell Disease. The primary objective is to evaluate the effect of voxelotor on the TCD (Transcranial Doppler Ultrasound) measurements in SCD participants in this age range.
This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ≥ 2 to < 15 years old, with Sickle Cell Disease. The study will be conducted at approximately 50 international clinical sites, and will enroll approximately 224 participants. Participants will be randomized in a 1:1 ratio to receive voxelotor or placebo. All participants younger than 12 years of age and randomized to voxelotor will receive a dose based on their body weight, to provide exposure corresponding to the adult dose of 1500 mg/day.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Voxelotor | Experimental | Voxelotor 1500mg or equivalent daily as a tablet, dispersible tablet, or as powder for oral suspension. |
|
| Placebo | Placebo Comparator | Matching placebo. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Voxelotor | Drug | Participants are randomized 1:1 to receive voxelotor or placebo. |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Time-Averaged Maximum of Mean Velocity (TAMMV) Arterial Cerebral Blood Flow at Week 24 | TAMMV is an ultrasound measurement used in transcranial Doppler (TCD) to assess blood flow in cerebral arteries. TCD flow velocities were categorized as follows: (i) Normal: < 170 centimeter per second (cm/sec); (ii) Conditional: 170 to < 200 cm/sec - the eligible participant population for this study; (iii) Abnormal: >= 200 cm/sec. | Baseline (value at screening), Week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in TAMMV Arterial Cerebral Blood Flow at Week 48 | TAMMV is an ultrasound measurement used in TCD to assess blood flow in cerebral arteries. TCD flow velocities were categorized as follows: (i) Normal: < 170 cm/sec; (ii) Conditional: 170 to < 200 cm/sec - the eligible participant population for this study; (iii) Abnormal: >= 200 cm/sec. | Baseline (value at Screening), Weeks 48 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's National Medical Center | Washington D.C. | District of Columbia | 20010 | United States | ||
| University of Miami |
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| 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 236 participants were assigned to the study treatment. Study was terminated based on sponsor decision.
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| ID | Title | Description |
|---|---|---|
| FG000 | Voxelotor | Participants aged greater than or equal to 12 years and less than 15 years of age were to receive 1500 milligrams (mg) voxelotor tablet orally once daily for 96 weeks or study termination, whichever occurred first. Participants less than 12 years of age received voxelotor at a weight based (1500 mg-equivalent) dose. Participants were followed up to 4 weeks after the last dose of study drug. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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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 | Jul 19, 2023 | May 29, 2025 |
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Double-Blind, Placebo-Controlled
| Placebo |
| Drug |
Matching placebo. |
|
| Time to Conversion to Abnormal TCD Flow | Time to conversion was the number of weeks from the date of randomization to the date of first determined TCD assessment when an abnormal TCD flow velocity (>= 200 cm/sec) was determined. TCD flow velocities were categorized as follows: (i) Normal: < 170 cm/sec; (ii) Conditional: 170 to < 200 cm/sec - the eligible participant population for this study; (iii) Abnormal: >= 200 cm/sec. The stratified Log Rank Test was stratified by stratification factors: baseline HU use (yes; no), age group (2 to <= 8 years; >8 to <15 years), and baseline TAMMV value (170 cm/sec to < 185 cm/sec; 185 cm/sec to < 200 cm/sec). | Up to 96 weeks |
| Time to Reversion to Normal TCD Flow | TCD was used to assess cerebral artery blood flow velocity in children with sickle cell disease (SCD). Time to first normal TCD flow was the number of weeks from randomization to the date of first determined normal TCD flow. Normal is < 170 cm/sec. | Up to 96 weeks |
| Percentage of Participants With TAMMV Reduced by >=15 cm/Sec From Baseline at Weeks 24, 48 and 96 | In this outcome measure, percentage of participants whose TAMMV reduced by >=15 cm/sec from Baseline at Weeks 24, 48 and 96 is reported. TAMMV is an ultrasound measurement used in TCD to assess blood flow in cerebral arteries. TCD flow velocities are categorized as follows: (i) Normal: < 170 cm/sec; (ii) Conditional: 170 to < 200 cm/sec - the eligible participant population for this study; (iii) Abnormal: >= 200 cm/sec. | At Weeks 24, 48 and 96 |
| Change From Baseline in Hemoglobin (Hb) at Weeks 24, 48 and 96 | Change from baseline in hemoglobin at weeks 24, 48 and 96 were reported in this outcome measure. | Baseline (value at Screening), Weeks 24, 48 and 96 |
| Percent Change From Baseline in Unconjugated Bilirubin at Weeks 24, 48 and 96 | Percent change from baseline in unconjugated bilirubin at weeks 24, 48 and 96 was reported in this outcome measure. | Baseline (value at Screening), Weeks 24, 48 and 96 |
| Percent Change From Baseline in Reticulocyte at Weeks 24, 48 and 96 | Percent change from baseline in reticulocyte at weeks 24, 48 and 96 was reported in this outcome measure. | Baseline (value at Screening), Weeks 24, 48 and 96 |
| Percent Change From Baseline in Absolute Reticulocyte at Weeks 24, 48 and 96 | Percent change from baseline in absolute reticulocyte at weeks 24, 48 and 96 was reported in this outcome measure. | Baseline (value at Screening), Weeks 24, 48 and 96 |
| Percent Change From Baseline in Lactate Dehydrogenase (LDH) at Weeks 24, 48 and 96 | Percent change from baseline in LDH at weeks 24, 48 and 96 was reported in this outcome measure. | Baseline (value at Screening), Weeks 24, 48 and 96 |
| Annualized Incidence Rate of Vaso-Occlusive Crises (VOCs) | VOC was defined as a composite of acute painful crisis and/or acute chest syndrome (ACS). Annualized incidence rate was defined as total number of events per total person-years on treatment. Total person-years was the sum of participants treatment period in years, which included the time from randomization date to the earliest of (last dose date, post-randomization HU initiation for participants with no HU at baseline, end of study, or study termination). The 95% CI of rate displayed the exact Poisson confidence limits. | From randomization to the last dose date, post-randomization HU initiation with no HU at baseline, end of study or study termination, whichever occurred earlier (maximum up to 110 weeks) |
| Miami |
| Florida |
| 33136 |
| United States |
| Children's Healthcare of Atlanta: Hughes Spalding | Atlanta | Georgia | 30303 | United States |
| Boston Children's Hospital - Clinical Research Pharmacy | Boston | Massachusetts | 02115 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| University of Mississippi Medical Center | Jackson | Mississippi | 39216 | United States |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Medical University of South Carolina: Investigational Drug Services | Charleston | South Carolina | 29425 | United States |
| Medical University of South Carolina: Shawn Jenkins Women's and Children's Hospital | Charleston | South Carolina | 29425 | United States |
| Medical University of South Carolina | Charleston | South Carolina | 29425 | United States |
| Texas Children's Hospital - Investigational Pharmacy | Houston | Texas | 77030 | United States |
| Texas Children's Hospital- Wallace Tower | Houston | Texas | 77030 | United States |
| Texas Children's Hospital | Houston | Texas | 77030 | United States |
| Ain Shams University Hospital- Clinical Research Center (MASRI) | Cairo | Abassia | 11566 | Egypt |
| Zagazig University Hospital | Zagazig | Alsharkia | Egypt |
| Alexandria Clinical Research Center, Faculty of Medicine, Alexandria University | Alexandria | 21131 | Egypt |
| Abu El Rich Hospital, Cairo University Hospital | Cairo | 11562 | Egypt |
| Komfo Anokye Teaching Hospital | Kumasi | Ashanti Region | 00233 | Ghana |
| Department of Child Health, University of Ghana Medical School, College of Health Sciences, Korle-Bu | Accra | Greater Accra Region | GA-221-1570 | Ghana |
| Azienda Ospedaliera Universitaria Meyer "A.O.U. Meyer" - SOC "Oncologia, Ematologia e TCSE" | Florence | 50139 | Italy |
| Azienda Ospedaliera Universitaria (A.O.U.) "Luigi Vanvitelli" | Naples | 80138 | Italy |
| Azienda Ospedaliera Universita' (AOU ) Padova | Padova | 35128 | Italy |
| Azienda Ospedaliera Universita' (AOU) Padova | Padova | 35128 | Italy |
| Kemri/Crdr,Siaya,Kemri Clinical Research Annex | Kisumu | Siaya County | 40600 | Kenya |
| KEMRI CRDR Clinical Research Annex | Nairobi | 00100 | Kenya |
| Strathmore University Medical Centre | Nairobi | 00200 | Kenya |
| Gertrude's Children Hospital | Nairobi | 100 | Kenya |
| Lagos University Teaching Hospital | Surulere | Lagos | 100254 | Nigeria |
| College of Medicine, University of Ibadan | Ibadan | Oyo State | 200212 | Nigeria |
| University of Nigeria Teaching Hospital | Enugu | 460000 | Nigeria |
| Barau Dikko Teaching/Kaduna State University | Kaduna | 800212 | Nigeria |
| Aminu Kano Teaching Hospital | Kano | 700233 | Nigeria |
| Sultan ÙŽQaboos University Hospital | Muscat | Oman |
| King Abdullah International Medical Research Center (KAIMRC), Ministry of National Guard - Health | Riyadh | 11481 | Saudi Arabia |
| Barts Health NHS Trust | London | E1 1BB | United Kingdom |
| FG001 | Placebo | Participants were to receive voxelotor matched placebo orally once daily for 96 weeks or study termination, whichever occurred first. Participants were followed up to 4 weeks after the last dose of study drug. |
| COMPLETED |
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| NOT COMPLETED |
|
|
Intent-to-Treat (ITT) population included all randomized participants.
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| ID | Title | Description |
|---|---|---|
| BG000 | Voxelotor | Participants aged greater than or equal to 12 years and less than 15 years of age were to receive 1500 mg voxelotor tablet orally once daily for 96 weeks or study termination, whichever occurred first. Participants less than 12 years of age received voxelotor at a weight based (1500 mg-equivalent) dose. Participants were followed up to 4 weeks after the last dose of study drug. |
| BG001 | Placebo | Participants were to receive voxelotor matched placebo orally once daily for 96 weeks or study termination, whichever occurred first. Participants were followed up to 4 weeks after the last dose of study drug. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Count of Participants | Participants |
| ||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race/Ethnicity, Customized | Race was reported. | 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 | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change From Baseline in Time-Averaged Maximum of Mean Velocity (TAMMV) Arterial Cerebral Blood Flow at Week 24 | TAMMV is an ultrasound measurement used in transcranial Doppler (TCD) to assess blood flow in cerebral arteries. TCD flow velocities were categorized as follows: (i) Normal: < 170 centimeter per second (cm/sec); (ii) Conditional: 170 to < 200 cm/sec - the eligible participant population for this study; (iii) Abnormal: >= 200 cm/sec. | ITT analysis population included all randomized participants. Here, "Overall Number of Participants Analyzed'' signifies number of participants evaluable for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | cm/sec | Baseline (value at screening), Week 24 |
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| Secondary | Change From Baseline in TAMMV Arterial Cerebral Blood Flow at Week 48 | TAMMV is an ultrasound measurement used in TCD to assess blood flow in cerebral arteries. TCD flow velocities were categorized as follows: (i) Normal: < 170 cm/sec; (ii) Conditional: 170 to < 200 cm/sec - the eligible participant population for this study; (iii) Abnormal: >= 200 cm/sec. | ITT analysis population included all randomized participants. Here, "Overall Number of Participants Analyzed'' signifies number of participants evaluable for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | cm/sec | Baseline (value at Screening), Weeks 48 |
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| Secondary | Time to Conversion to Abnormal TCD Flow | Time to conversion was the number of weeks from the date of randomization to the date of first determined TCD assessment when an abnormal TCD flow velocity (>= 200 cm/sec) was determined. TCD flow velocities were categorized as follows: (i) Normal: < 170 cm/sec; (ii) Conditional: 170 to < 200 cm/sec - the eligible participant population for this study; (iii) Abnormal: >= 200 cm/sec. The stratified Log Rank Test was stratified by stratification factors: baseline HU use (yes; no), age group (2 to <= 8 years; >8 to <15 years), and baseline TAMMV value (170 cm/sec to < 185 cm/sec; 185 cm/sec to < 200 cm/sec). | ITT analysis population included all randomized participants. Participants in the analysis included all randomized participants (Intent-to Treat) population. Participants with an event after postbaseline HU initiation or without event are censored at the earliest date of last non-missing TAMMV assessment prior to start of HU or end of study date. | Posted | Median | 95% Confidence Interval | Weeks | Up to 96 weeks |
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| Secondary | Time to Reversion to Normal TCD Flow | TCD was used to assess cerebral artery blood flow velocity in children with sickle cell disease (SCD). Time to first normal TCD flow was the number of weeks from randomization to the date of first determined normal TCD flow. Normal is < 170 cm/sec. | ITT analysis population included all randomized participants. Here, "Overall Number of Participants Analyzed'' signifies number of participants evaluable for this outcome measure. | Posted | Median | Full Range | Weeks | Up to 96 weeks |
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| Secondary | Percentage of Participants With TAMMV Reduced by >=15 cm/Sec From Baseline at Weeks 24, 48 and 96 | In this outcome measure, percentage of participants whose TAMMV reduced by >=15 cm/sec from Baseline at Weeks 24, 48 and 96 is reported. TAMMV is an ultrasound measurement used in TCD to assess blood flow in cerebral arteries. TCD flow velocities are categorized as follows: (i) Normal: < 170 cm/sec; (ii) Conditional: 170 to < 200 cm/sec - the eligible participant population for this study; (iii) Abnormal: >= 200 cm/sec. | ITT analysis population=all randomized participants and at each timepoint ITT= total number of all randomized participants used in the analysis. Participants with any of the following scenarios were counted as non-responders:(i) TCD missing at assessment timepoint (including due to abnormal TCD discontinuation) (ii) post-randomization HU use prior to assessment timepoint for subjects with no HU use at baseline".For any missing values, data was imputed using last TAMMV value prior to transfusion. | Posted | Number | Percentage of participants | At Weeks 24, 48 and 96 |
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| Secondary | Change From Baseline in Hemoglobin (Hb) at Weeks 24, 48 and 96 | Change from baseline in hemoglobin at weeks 24, 48 and 96 were reported in this outcome measure. | ITT analysis population included all randomized participants. Here, "Overall Number of Participants Analyzed'' = number of participants evaluable for this outcome. All participants reported under "Overall Number of Participants Analyzed'' contributed data to table but may not have evaluable data for every row. "Number Analyzed" = participants evaluable for each row. | Posted | Least Squares Mean | 95% Confidence Interval | Grams per deciliter (g/dL) | Baseline (value at Screening), Weeks 24, 48 and 96 |
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| Secondary | Percent Change From Baseline in Unconjugated Bilirubin at Weeks 24, 48 and 96 | Percent change from baseline in unconjugated bilirubin at weeks 24, 48 and 96 was reported in this outcome measure. | ITT analysis population included all randomized participants. Here, "Overall Number of Participants Analyzed'' = number of participants evaluable for this outcome. All participants reported under "Overall Number of Participants Analyzed'' contributed data to table but may not have evaluable data for every row. "Number Analyzed" = participants evaluable for each row. | Posted | Least Squares Mean | 95% Confidence Interval | Percentage change | Baseline (value at Screening), Weeks 24, 48 and 96 |
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| Secondary | Percent Change From Baseline in Reticulocyte at Weeks 24, 48 and 96 | Percent change from baseline in reticulocyte at weeks 24, 48 and 96 was reported in this outcome measure. | ITT analysis population included all randomized participants. Here, "Overall Number of Participants Analyzed'' = number of participants evaluable for this outcome. All participants reported under this contributed data to table but may not have evaluable data for every row. 'Number Analyzed'=participants evaluable for each row. | Posted | Least Squares Mean | 95% Confidence Interval | Percent change | Baseline (value at Screening), Weeks 24, 48 and 96 |
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| Secondary | Percent Change From Baseline in Absolute Reticulocyte at Weeks 24, 48 and 96 | Percent change from baseline in absolute reticulocyte at weeks 24, 48 and 96 was reported in this outcome measure. | ITT analysis population included all randomized participants. Here, "Overall Number of Participants Analyzed'' = number of participants evaluable for this outcome. All participants reported under this contributed data to table but may not have evaluable data for every row. 'Number Analyzed'= participants evaluable for each row. | Posted | Least Squares Mean | 95% Confidence Interval | Percent change | Baseline (value at Screening), Weeks 24, 48 and 96 |
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| Secondary | Percent Change From Baseline in Lactate Dehydrogenase (LDH) at Weeks 24, 48 and 96 | Percent change from baseline in LDH at weeks 24, 48 and 96 was reported in this outcome measure. | ITT analysis population included all randomized participants. Here, "Overall Number of Participants Analyzed'' = number of participants evaluable for this outcome. All participants reported under this contributed data to table but may not have evaluable data for every row. 'Number Analyzed'= participants evaluable for each row. | Posted | Least Squares Mean | 95% Confidence Interval | Percent change | Baseline (value at Screening), Weeks 24, 48 and 96 |
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| Secondary | Annualized Incidence Rate of Vaso-Occlusive Crises (VOCs) | VOC was defined as a composite of acute painful crisis and/or acute chest syndrome (ACS). Annualized incidence rate was defined as total number of events per total person-years on treatment. Total person-years was the sum of participants treatment period in years, which included the time from randomization date to the earliest of (last dose date, post-randomization HU initiation for participants with no HU at baseline, end of study, or study termination). The 95% CI of rate displayed the exact Poisson confidence limits. | ITT analysis population included all randomized participants. | Posted | Number | 95% Confidence Interval | Events/ person-years | From randomization to the last dose date, post-randomization HU initiation with no HU at baseline, end of study or study termination, whichever occurred earlier (maximum up to 110 weeks) |
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From randomization to the last dose date, post-randomization HU initiation with no HU at baseline, end of study or study termination, whichever occurred earlier (maximum up to 110 weeks)
Same event may appear as both SAE and non-SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both a serious and non-serious event during the study. Analysis population included all randomized participants.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Voxelotor | Participants aged greater than or equal to 12 years and less than 15 years of age were to receive 1500 mg voxelotor tablet orally once daily for 96 weeks or study termination, whichever occurred first. Participants less than 12 years of age received voxelotor at a weight based (1500 mg-equivalent) dose. Participants were followed up to 4 weeks after the last dose of study drug. | 8 | 120 | 63 | 120 | 81 | 120 |
| EG001 | Placebo | Participants were to receive voxelotor matched placebo orally once daily for 96 weeks or study termination, whichever occurred first. Participants were followed up to 4 weeks after the last dose of study drug. | 2 | 116 | 43 | 116 | 83 | 116 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Sickle cell anaemia with crisis | Blood and lymphatic system disorders | MedDRA v27.1 | Non-systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Haemolysis | Blood and lymphatic system disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Leukocytosis | Blood and lymphatic system disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Thrombocytosis | Blood and lymphatic system disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Acute chest syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Pneumonia viral | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Malaria | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Arthritis bacterial | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Diphtheria | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Meningitis | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Osteomyelitis | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Osteomyelitis chronic | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
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| Pharyngitis | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
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| Septic shock | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
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| Tonsillitis | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
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| Osteonecrosis | Musculoskeletal and connective tissue disorders | MedDRA v27.1 | Systematic Assessment |
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| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA v27.1 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA v27.1 | Systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | MedDRA v27.1 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA v27.1 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA v27.1 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA v27.1 | Systematic Assessment |
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| Seizure | Nervous system disorders | MedDRA v27.1 | Systematic Assessment |
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| Cerebrovascular accident | Nervous system disorders | MedDRA v27.1 | Systematic Assessment |
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| Haemorrhagic stroke | Nervous system disorders | MedDRA v27.1 | Systematic Assessment |
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| Posterior reversible encephalopathy syndrome | Nervous system disorders | MedDRA v27.1 | Systematic Assessment |
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| Angina pectoris | Cardiac disorders | MedDRA v27.1 | Systematic Assessment |
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| Cardiac failure | Cardiac disorders | MedDRA v27.1 | Systematic Assessment |
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| Coronary artery thrombosis | Cardiac disorders | MedDRA v27.1 | Systematic Assessment |
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| Cholecystitis | Hepatobiliary disorders | MedDRA v27.1 | Systematic Assessment |
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| Hepatomegaly | Hepatobiliary disorders | MedDRA v27.1 | Systematic Assessment |
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| Hyperbilirubinaemia | Hepatobiliary disorders | MedDRA v27.1 | Systematic Assessment |
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| Eyelid ptosis congenital | Congenital, familial and genetic disorders | MedDRA v27.1 | Systematic Assessment |
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| Periorbital oedema | Eye disorders | MedDRA v27.1 | Systematic Assessment |
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| Liver function test abnormal | Investigations | MedDRA v27.1 | Systematic Assessment |
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| Glomerulonephritis acute | Renal and urinary disorders | MedDRA v27.1 | Systematic Assessment |
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| Hypertensive emergency | Vascular disorders | MedDRA v27.1 | Systematic Assessment |
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| Hypersplenism | Blood and lymphatic system disorders | MedDRAv27.1 | Systematic Assessment |
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| Abdominal distension | Gastrointestinal disorders | MedDRAv27.1 | Systematic Assessment |
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| Bacterial infection | Infections and infestations | MedDRAv27.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Sickle cell anaemia with crisis | Blood and lymphatic system disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Thrombocytosis | Blood and lymphatic system disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Leukocytosis | Blood and lymphatic system disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Neutrophilia | Blood and lymphatic system disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Splenomegaly | Blood and lymphatic system disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Haemolysis | Blood and lymphatic system disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Leukopenia | Blood and lymphatic system disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Malaria | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Pharyngotonsillitis | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Varicella | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Bacterial infection | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Body tinea | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Escherichia urinary tract infection | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Metapneumovirus infection | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Mumps | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Osteomyelitis acute | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Parotitis | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Rash pustular | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Carbuncle | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Respiratory syncytial virus infection | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA v27.1 | Systematic Assessment |
| |
| Osteonecrosis | Musculoskeletal and connective tissue disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Acute chest syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Tonsillar hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Dyspnoea exertional | Respiratory, thoracic and mediastinal disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Nasal turbinate hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Gait disturbance | General disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Mouth ulceration | Gastrointestinal disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA v27.1 | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA v27.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA v27.1 | Systematic Assessment |
| |
| Blood lactate dehydrogenase increased | Investigations | MedDRA v27.1 | Systematic Assessment |
| |
| Cardiac murmur | Investigations | MedDRA v27.1 | Systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA v27.1 | Systematic Assessment |
| |
| Urine albumin/creatinine ratio increased | Investigations | MedDRA v27.1 | Systematic Assessment |
| |
| Bilirubin conjugated increased | Investigations | MedDRA v27.1 | Systematic Assessment |
| |
| Blood albumin decreased | Investigations | MedDRA v27.1 | Systematic Assessment |
| |
| Heart rate irregular | Investigations | MedDRA v27.1 | Systematic Assessment |
| |
| Platelet count increased | Investigations | MedDRA v27.1 | Systematic Assessment |
| |
| Vitamin D decreased | Investigations | MedDRA v27.1 | Systematic Assessment |
| |
| Ocular icterus | Hepatobiliary disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Hepatomegaly | Hepatobiliary disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Hepatosplenomegaly | Hepatobiliary disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Hyperbilirubinaemia | Hepatobiliary disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Jaundice | Hepatobiliary disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Macule | Skin and subcutaneous tissue disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Rash macular | Skin and subcutaneous tissue disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Rash papular | Skin and subcutaneous tissue disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Bone fissure | Injury, poisoning and procedural complications | MedDRA v27.1 | Systematic Assessment |
| |
| Soft tissue injury | Injury, poisoning and procedural complications | MedDRA v27.1 | Systematic Assessment |
| |
| Wound | Injury, poisoning and procedural complications | MedDRA v27.1 | Systematic Assessment |
| |
| Nail injury | Injury, poisoning and procedural complications | MedDRA v27.1 | Systematic Assessment |
| |
| Overdose | Injury, poisoning and procedural complications | MedDRA v27.1 | Systematic Assessment |
| |
| Upper limb fracture | Injury, poisoning and procedural complications | MedDRA v27.1 | Systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Pallor | Vascular disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Night blindness | Eye disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Vernal keratoconjunctivitis | Eye disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Eye pruritus | Eye disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Ocular hyperaemia | Eye disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Cardiac failure congestive | Cardiac disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Right ventricular failure | Cardiac disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Motion sickness | Ear and labyrinth disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Pollakiuria | Renal and urinary disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Sickle cell nephropathy | Renal and urinary disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Dysmenorrhoea | Reproductive system and breast disorders | MedDRA v27.1 | Systematic Assessment |
| |
| Acrodermatitis | Infections and infestations | MedDRAv27.1 | Systematic Assessment |
| |
| Abdominal tenderness | Gastrointestinal disorders | MedDRAv27.1 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRAv27.1 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRAv27.1 | Systematic Assessment |
| |
| Dactylitis | Musculoskeletal and connective tissue disorders | MedDRAv27.1 | Systematic Assessment |
| |
| Sciatica | Nervous system disorders | MedDRAv27.1 | Systematic Assessment |
| |
| Catarrh | Respiratory, thoracic and mediastinal disorders | MedDRAv27.1 | Systematic Assessment |
| |
| Keratosis pilaris | Skin and subcutaneous tissue disorders | MedDRAv27.1 | Systematic Assessment |
| |
| Miliaria | Skin and subcutaneous tissue disorders | MedDRAv27.1 | Systematic Assessment |
|
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 | Jan 31, 2025 | Oct 23, 2025 | SAP_002.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
| 12 to < 15 Years |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Arab |
|
| Black or African American |
|
| White |
|
| Multi-Racial |
|
Mixed Model Repeated Measures analysis
| Units | Counts |
|---|---|
| Participants |
|
|
|
Participants were to receive voxelotor matched placebo orally once daily for 96 weeks or study termination, whichever occurred first. Participants were followed up to 4 weeks after the last dose of study drug.
|
|
|
| Participants |
|
|
Participants were to receive voxelotor matched placebo orally once daily for 96 weeks or study termination, whichever occurred first. Participants were followed up to 4 weeks after the last dose of study drug. |
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
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| Units | Counts |
|---|---|
| Participants |
|
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| Units | Counts |
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| Participants |
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|
| Units | Counts |
|---|---|
| Participants |
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| Units | Counts |
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| Participants |
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