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| ID | Type | Description | Link |
|---|---|---|---|
| C5351005 | Other Identifier | Alias Study Number |
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Study terminated as data obtained from this non-placebo-controlled trial may not be informative for the interpretation of the benefits and risks of osivelotor
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An Open-label Extension Study of GBT021601 in Participants with Sickle Cell Disease
An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of GBT021601 Administered to Participants with Sickle Cell Disease Who Have Participated in a GBT021601 Clinical Trial
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment | Experimental | Osivelotor |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Osivelotor | Drug | Osivelotor |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | An adverse event (AE) was any untoward medical occurrence in a participant administered a pharmaceutical product during the course of a clinical investigation. Serious adverse events (SAEs) were defined as any untoward medical occurrence that, at any dose, met one or more of the criteria: death, life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, congenital anomaly or birth defect and other medically significant events. TEAEs are events between first dose of study drug and up to 56 days after last dose that were absent before treatment or that worsened relative to pretreatment state. Relatedness of any AE to treatment was based on investigator decision. AEs included both SAEs and all non-serious AEs. | From first dose of study drug up to 56 days after last dose of study drug (approximately up to 736 days) |
| Change From Baseline in Hematocrit at Week 12 | Change from baseline in hematocrit at week 12 were reported in this outcome measure. | Baseline, Week 12 |
| Change From Baseline in Hematocrit at Week 48 | Change from baseline in hematocrit at week 48 were reported in this outcome measure. | Baseline, Week 48 |
| Change From Baseline in Leukocytes at Week 12 | Change from baseline in leukocytes at week 12 were reported in this outcome measure. | Baseline, Week 12 |
| Change From Baseline in Leukocytes Week 48 | Change from baseline in leukocytes at week 48 were reported in this outcome measure. | Baseline, Week 48 |
| Measure | Description | Time Frame |
|---|---|---|
| Annualized Rate of Vaso-Occlusive Crisis (VOC) | A VOC was defined as an acute episode of pain that had no medically determined cause other than a vaso-occlusive event, and resulted in a visit to a medical facility (hospitalization, emergency department, urgent care center, outpatient clinic, or infusion center), and required parenteral narcotic agents, parenteral nonsteroidal anti-inflammatory drugs (NSAIDs), or an increase in treatment with oral narcotics. Annualized rate of VOC was reported in this outcome measure. |
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Inclusion Criteria:
Male or female aged 6 months or older with SCD who participated and received study drug or placebo in a previous osivelotor clinical study and completed the end of treatment visit.
Note: Participants who discontinued study drug in the originating study due to an TEAE, but who remained on study, may be eligible for treatment in this study provided the TEAE does not pose a risk for treatment with osivelotor.
Females of childbearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1.
Note: Females who become of childbearing potential during the study must be willing to have negative urine pregnancy tests to remain in the study.
If sexually active, females of childbearing potential must consistently use highly effective methods of contraception consistently throughout the study and for at least 120 days after the last dose of study drug. If sexually active, male participants must use barrier methods of contraception until 84 days after the last dose of study drug. Male participants are eligible to participate if they agree to the following requirements during the study intervention period and for 84 days after the last dose of study intervention:
Participant has provided written informed consent/assent. For underage participants, both the consent of the participant's legal representative or legal guardian and the participant's assent (where applicable) must be obtained based on local requirements.
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Our Lady of the Lake Hospital, Inc. | Baton Rouge | Louisiana | 70808 | United States | ||
| University Medical Center Inpatient Pharmacy |
Not provided
| 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 47 participants from only Part A of the parent study C5351004 [NCT05431088] were enrolled in the current study C5351005 [NCT05632354]. Study was terminated based on Sponsor's decision. There were no participants enrolled in 'Participants With Delayed Start: Osivelotor 200 mg' group.
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| ID | Title | Description |
|---|---|---|
| FG000 | Participants Without Delayed Start: Osivelotor 100 Milligram (mg) | Participants with sickle cell disease (SCD) who participated in a previous osivelotor clinical study and received osivelotor 100 mg tablet orally and completed the end of treatment visit in this study for up to 680 days. Participants without any delay in start of treatment were included. |
| FG001 | Participants Without Delayed Start: Osivelotor 150 mg | Participants with SCD who participated in a previous osivelotor clinical study and received osivelotor 150 mg tablet orally and completed the end of treatment visit in this study for up to 680 days. Participants without any delay in start of treatment were included. |
| FG002 | Participants Without Delayed Start: Osivelotor 200 mg | Participants with SCD who participated in a previous osivelotor clinical study and received osivelotor 200 mg tablet orally and completed the end of treatment visit in this study for up to 680 days. Participants without any delay in start of treatment were included. |
| FG003 | Participants With Delayed Start: Osivelotor 100 mg | Participants with SCD who participated in a previous osivelotor clinical study and received 100 mg tablet orally and completed the end of treatment visit in this study for up to 680 days. Participants with delayed start are those with a gap in dosing greater than 42 consecutive days from completing the originating study (C5351005 Date of First Dose - C5351004 End of Treatment Date + 1 greater than 42). |
| FG004 | Participants With Delayed Start: Osivelotor 150 mg | Participants with SCD who participated in a previous osivelotor clinical study and received 150 mg tablet orally and completed the end of treatment visit in this study for up to 680 days. Participants with delayed start are those with a gap in dosing greater than 42 consecutive days from completing the originating study (C5351005 Date of First Dose - C5351004 End of Treatment Date + 1 greater than 42). |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Safety analysis set included all enrolled participants who had received at least one dose of study drug in this open-label extension (OLE) study. Participants were analyzed according to the assigned daily maintenance dose on study day 1 in the OLE study.
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| ID | Title | Description |
|---|---|---|
| BG000 | Participants Without Delayed Start: Osivelotor 100 mg | Participants with SCD who participated in a previous osivelotor clinical study and received osivelotor 100 mg tablet orally and completed the end of treatment visit in this study for up to 680 days. Participants without any delay in start of treatment were included. |
| BG001 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| 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 | Number of Participants With Treatment Emergent Adverse Events (TEAEs) | An adverse event (AE) was any untoward medical occurrence in a participant administered a pharmaceutical product during the course of a clinical investigation. Serious adverse events (SAEs) were defined as any untoward medical occurrence that, at any dose, met one or more of the criteria: death, life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, congenital anomaly or birth defect and other medically significant events. TEAEs are events between first dose of study drug and up to 56 days after last dose that were absent before treatment or that worsened relative to pretreatment state. Relatedness of any AE to treatment was based on investigator decision. AEs included both SAEs and all non-serious AEs. | Safety analysis set included all enrolled participants who had received at least one dose of study drug in this OLE study. Participants were analyzed according to the assigned daily maintenance dose on study day 1 in the OLE study. | Posted | Count of Participants | Participants | From first dose of study drug up to 56 days after last dose of study drug (approximately up to 736 days) |
From first dose of study drug up to 56 days after last dose of study drug (approximately up to 736 days)
Same event may appear as both SAE and non-SAE but are distinct events. An event may be categorized as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. Safety analysis set included all enrolled participants who had received at least one dose of study drug in this OLE study. Participants were analyzed according to the assigned daily maintenance dose on study day 1 in the OLE study.
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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 | Participants Without Delayed Start: Osivelotor 100 mg | Participants with SCD who participated in a previous osivelotor clinical study and received osivelotor 100 mg tablet orally and completed the end of treatment visit in this study for up to 680 days. Participants without any delay in start of treatment were included. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 28.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA 28.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer Inc. | 1-800-718-1021 | ClinicalTrials.gov_Inquiries@pfizer.com |
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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 | Feb 8, 2024 | Feb 10, 2026 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jun 23, 2025 | Feb 10, 2026 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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| Change From Baseline in Supine Blood Pressure (SBP) at Week 12 |
Change from baseline in SBP at week 12 were reported in this outcome measure. |
| Baseline, Week 12 |
| Change From Baseline in SBP at Week 48 | Change from baseline in SBP at week 48 were reported in this outcome measure. | Baseline, Week 48 |
| Change From Baseline in Diastolic Blood Pressure (DBP) at Week 12 | Change from baseline in DBP at week 12 were reported in this outcome measure. | Baseline, Week 12 |
| Change From Baseline in DBP at Week 48 | Change from baseline in DBP at week 48 were reported in this outcome measure. | Baseline, Week 48 |
| From the first dose of study drug up to last dose of study drug (approximately up to 680 days) |
| Number of Participants With Sickle Cell Disease (SCD) Related Serious Adverse Events (SAEs) | SCD is an inherited disorder caused by a point mutation in the beta globin gene which leads to formation of sickle hemoglobin (HbS). SAEs were defined as any untoward medical occurrence that, at any dose, meets one or more of the criteria: death, life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, congenital anomaly or birth defect and other medically significant events. Participants with delayed start are those with a gap in dosing greater than 42 consecutive days from completing the originating study (C5351005 Date of First Dose - C5351004 End of Treatment Date + 1 greater than 42). | From first dose of study drug up to 56 days after last dose of study drug (approximately up to 736 days) |
| Change From Baseline in Hemoglobin at Weeks 12, 24, 36, 48, and 60 | Change from baseline in hemoglobin at weeks 12, 24, 36, 48 and 60 were reported in this outcome measure. | Baseline, Weeks 12, 24, 36, 48, and 60 |
| Change From Baseline in Reticulocytes at Weeks 12, 24, 36, 48, and 60 | Change from baseline in reticulocytes at weeks 12, 24, 36, 48 and 60 were reported in this outcome measure. | Baseline, Weeks 12, 24, 36, 48, and 60 |
| Change From Baseline in Lactate Dehydrogenase (LDH) at Weeks 12, 24, 36, 48, and 60 | Change from baseline in LDH at weeks 12, 24, 36, 48 and 60 were reported in this outcome measure. | Baseline, Weeks 12, 24, 36, 48, and 60 |
| Change From Baseline in Unconjugated Bilirubin at Weeks 12, 24, 36, 48, and 60 | Change from baseline in unconjugated bilirubin at weeks 12, 24, 36, 48 and 60 were reported in this outcome measure. | Baseline, Weeks 12, 24, 36, 48, and 60 |
| New Orleans |
| Louisiana |
| 70112 |
| United States |
| University Medical Center New Orleans | New Orleans | Louisiana | 70112 | United States |
| Mississippi Center for Advanced Medicine | Madison | Mississippi | 39110 | United States |
| University of Texas Health Science Center | Houston | Texas | 77030 | United States |
| Inova Schar Cancer Institute | Fairfax | Virginia | 22031 | United States |
| University College Hospital Ibadan | Ibadan | Oyo/ibadan North | 200212 | Nigeria |
| Aminu kano Teaching Hospital | Kano | 700233 | Nigeria |
| Lagos University Teaching Hospital | Lagos | 100254 | Nigeria |
| Lost to Follow-up |
|
| Pregnancy |
|
| Study terminated by sponsor |
|
| Withdrawal by Subject |
|
| Non-compliance with study treatment |
|
| Participants Without Delayed Start: Osivelotor 150 mg |
Participants with SCD who participated in a previous osivelotor clinical study and received osivelotor 150 mg tablet orally and completed the end of treatment visit in this study for up to 680 days. Participants without any delay in start of treatment were included. |
| BG002 | Participants Without Delayed Start: Osivelotor 200 mg | Participants with SCD who participated in a previous osivelotor clinical study and received osivelotor 200 mg tablet orally and completed the end of treatment visit in this study for up to 680 days. Participants without any delay in start of treatment were included. |
| BG003 | Participants With Delayed Start: Osivelotor 100 mg | Participants with SCD who participated in a previous osivelotor clinical study and received 100 mg tablet orally and completed the end of treatment visit in this study for up to 680 days. Participants with delayed start are those with a gap in dosing greater than 42 consecutive days from completing the originating study (C5351005 Date of First Dose - C5351004 End of Treatment Date + 1 greater than 42). |
| BG004 | Participants With Delayed Start: Osivelotor 150 mg | Participants with SCD who participated in a previous osivelotor clinical study and received 150 mg tablet orally and completed the end of treatment visit in this study for up to 680 days. Participants with delayed start are those with a gap in dosing greater than 42 consecutive days from completing the originating study (C5351005 Date of First Dose - C5351004 End of Treatment Date + 1 greater than 42). |
| BG005 | Total | Total of all reporting groups |
| Years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| ID | Title | Description |
|---|---|---|
| OG000 | Participants Without Delayed Start: Osivelotor 100 mg | Participants with SCD who participated in a previous osivelotor clinical study and received osivelotor 100 mg tablet orally and completed the end of treatment visit in this study for up to 680 days. Participants without any delay in start of treatment were included. |
| OG001 | Participants Without Delayed Start: Osivelotor 150 mg | Participants with SCD who participated in a previous osivelotor clinical study and received osivelotor 150 mg tablet orally and completed the end of treatment visit in this study for up to 680 days. Participants without any delay in start of treatment were included. |
| OG002 | Participants Without Delayed Start: Osivelotor 200 mg | Participants with SCD who participated in a previous osivelotor clinical study and received osivelotor 200 mg tablet orally and completed the end of treatment visit in this study for up to 680 days. Participants without any delay in start of treatment were included. |
| OG003 | Participants With Delayed Start: Osivelotor 100 mg | Participants with SCD who participated in a previous osivelotor clinical study and received 100 mg tablet orally and completed the end of treatment visit in this study for up to 680 days. Participants with delayed start are those with a gap in dosing greater than 42 consecutive days from completing the originating study (C5351005 Date of First Dose - C5351004 End of Treatment Date + 1 greater than 42). |
| OG004 | Participants With Delayed Start: Osivelotor 150 mg | Participants with SCD who participated in a previous osivelotor clinical study and received 150 mg tablet orally and completed the end of treatment visit in this study for up to 680 days. Participants with delayed start are those with a gap in dosing greater than 42 consecutive days from completing the originating study (C5351005 Date of First Dose - C5351004 End of Treatment Date + 1 greater than 42). |
|
|
| Primary | Change From Baseline in Hematocrit at Week 12 | Change from baseline in hematocrit at week 12 were reported in this outcome measure. | Safety analysis set included all enrolled participants who had received at least one dose of study drug in this OLE study. Participants were analyzed according to the assigned daily maintenance dose on study day 1 in the OLE study. Here, 'Overall Number of Participants Analyzed' (N) signifies participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Percent | Baseline, Week 12 |
|
|
|
| Primary | Change From Baseline in Hematocrit at Week 48 | Change from baseline in hematocrit at week 48 were reported in this outcome measure. | Safety analysis set included all enrolled participants who had received at least one dose of study drug in this OLE study. Participants were analyzed according to the assigned daily maintenance dose on study day 1 in the OLE study. Here, 'Overall Number of Participants Analyzed' signifies participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Percent | Baseline, Week 48 |
|
|
|
| Primary | Change From Baseline in Leukocytes at Week 12 | Change from baseline in leukocytes at week 12 were reported in this outcome measure. | Safety analysis set included all enrolled participants who had received at least one dose of study drug in this OLE study. Participants were analyzed according to the assigned daily maintenance dose on study day 1 in the OLE study. Here, 'Overall Number of Participants Analyzed' signifies participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | 10^9 cells per liter (10^9 cells/L) | Baseline, Week 12 |
|
|
|
| Primary | Change From Baseline in Leukocytes Week 48 | Change from baseline in leukocytes at week 48 were reported in this outcome measure. | Safety analysis set included all enrolled participants who had received at least one dose of study drug in this OLE study. Participants were analyzed according to the assigned daily maintenance dose on study day 1 in the OLE study. Here, 'Overall Number of Participants Analyzed' signifies participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | 10^9 cells/L | Baseline, Week 48 |
|
|
|
| Primary | Change From Baseline in Supine Blood Pressure (SBP) at Week 12 | Change from baseline in SBP at week 12 were reported in this outcome measure. | Safety analysis set included all enrolled participants who had received at least one dose of study drug in this OLE study. Participants were analyzed according to the assigned daily maintenance dose on study day 1 in the OLE study. | Posted | Mean | Standard Deviation | Millimeters of mercury (mmHg) | Baseline, Week 12 |
|
|
|
| Primary | Change From Baseline in SBP at Week 48 | Change from baseline in SBP at week 48 were reported in this outcome measure. | Safety analysis set included all enrolled participants who had received at least one dose of study drug in this OLE study. Participants were analyzed according to the assigned daily maintenance dose on study day 1 in the OLE study. Here, 'Overall Number of Participants Analyzed' signifies participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | mmHg | Baseline, Week 48 |
|
|
|
| Primary | Change From Baseline in Diastolic Blood Pressure (DBP) at Week 12 | Change from baseline in DBP at week 12 were reported in this outcome measure. | Safety analysis set included all enrolled participants who had received at least one dose of study drug in this OLE study. Participants were analyzed according to the assigned daily maintenance dose on study day 1 in the OLE study. | Posted | Mean | Standard Deviation | mmHg | Baseline, Week 12 |
|
|
|
| Primary | Change From Baseline in DBP at Week 48 | Change from baseline in DBP at week 48 were reported in this outcome measure. | Safety analysis set included all enrolled participants who had received at least one dose of study drug in this OLE study. Participants were analyzed according to the assigned daily maintenance dose on study day 1 in the OLE study. Here, 'Overall Number of Participants Analyzed' signifies participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | mmHg | Baseline, Week 48 |
|
|
|
| Secondary | Annualized Rate of Vaso-Occlusive Crisis (VOC) | A VOC was defined as an acute episode of pain that had no medically determined cause other than a vaso-occlusive event, and resulted in a visit to a medical facility (hospitalization, emergency department, urgent care center, outpatient clinic, or infusion center), and required parenteral narcotic agents, parenteral nonsteroidal anti-inflammatory drugs (NSAIDs), or an increase in treatment with oral narcotics. Annualized rate of VOC was reported in this outcome measure. | Safety analysis set included all enrolled participants who had received at least one dose of study drug in this OLE study. Participants were analyzed according to the assigned daily maintenance dose on study day 1 in the OLE study. The annualized rate and corresponding 95% CI for each dose arm and overall (100 mg + 150 mg) was reported in this outcome measure as pre-specified in statistical analysis plan | Posted | Number | 95% Confidence Interval | VOC events per year | From the first dose of study drug up to last dose of study drug (approximately up to 680 days) |
|
|
|
| Secondary | Number of Participants With Sickle Cell Disease (SCD) Related Serious Adverse Events (SAEs) | SCD is an inherited disorder caused by a point mutation in the beta globin gene which leads to formation of sickle hemoglobin (HbS). SAEs were defined as any untoward medical occurrence that, at any dose, meets one or more of the criteria: death, life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, congenital anomaly or birth defect and other medically significant events. Participants with delayed start are those with a gap in dosing greater than 42 consecutive days from completing the originating study (C5351005 Date of First Dose - C5351004 End of Treatment Date + 1 greater than 42). | Safety analysis set included all enrolled participants who had received at least one dose of study drug in this OLE study. Participants were analyzed according to the assigned daily maintenance dose on study day 1 in the OLE study. | Posted | Count of Participants | Participants | From first dose of study drug up to 56 days after last dose of study drug (approximately up to 736 days) |
|
|
|
| Secondary | Change From Baseline in Hemoglobin at Weeks 12, 24, 36, 48, and 60 | Change from baseline in hemoglobin at weeks 12, 24, 36, 48 and 60 were reported in this outcome measure. | Safety analysis set included all enrolled participants who had received at least one dose of study drug in this OLE study. Participants were analyzed according to the assigned daily maintenance dose on study day 1 in the OLE study. Here 'Number Analyzed' (n) signifies participants evaluable at specified timepoints. | Posted | Mean | Standard Deviation | Grams per deciliter (g/dL) | Baseline, Weeks 12, 24, 36, 48, and 60 |
|
|
|
| Secondary | Change From Baseline in Reticulocytes at Weeks 12, 24, 36, 48, and 60 | Change from baseline in reticulocytes at weeks 12, 24, 36, 48 and 60 were reported in this outcome measure. | Safety analysis set included all enrolled participants who had received at least one dose of study drug in this OLE study. Participants were analyzed according to the assigned daily maintenance dose on study day 1 in the OLE study. Here 'Number Analyzed' signifies participants evaluable at specified timepoints. | Posted | Mean | Standard Deviation | 10^9 cells/L | Baseline, Weeks 12, 24, 36, 48, and 60 |
|
|
|
| Secondary | Change From Baseline in Lactate Dehydrogenase (LDH) at Weeks 12, 24, 36, 48, and 60 | Change from baseline in LDH at weeks 12, 24, 36, 48 and 60 were reported in this outcome measure. | Safety analysis set included all enrolled participants who had received at least one dose of study drug in this OLE study. Participants were analyzed according to the assigned daily maintenance dose on study day 1 in the OLE study. Here 'Number Analyzed' signifies participants evaluable at specified timepoints. | Posted | Mean | Standard Deviation | International units per liter (IU/L) | Baseline, Weeks 12, 24, 36, 48, and 60 |
|
|
|
| Secondary | Change From Baseline in Unconjugated Bilirubin at Weeks 12, 24, 36, 48, and 60 | Change from baseline in unconjugated bilirubin at weeks 12, 24, 36, 48 and 60 were reported in this outcome measure. | Safety analysis set. Participants were analyzed according to assigned daily maintenance dose on study day 1 in OLE study. Here, 'N'= participants evaluable for this outcome measure and 'n'=participants evaluable at specified timepoints. 'N'=0 for 'Participants without delayed start: Osivelotor 200 mg' group since data for the participant was missing at baseline, so change from baseline could not be calculated for unconjugated bilirubin parameter. | Posted | Mean | Standard Deviation | Micromoles per liter (mcmol/L) | Baseline, Weeks 12, 24, 36, 48, and 60 |
|
|
|
| 0 |
| 18 |
| 5 |
| 18 |
| 14 |
| 18 |
| EG001 | Participants Without Delayed Start: Osivelotor 150 mg | Participants with SCD who participated in a previous osivelotor clinical study and received osivelotor 150 mg tablet orally and completed the end of treatment visit in this study for up to 680 days. Participants without any delay in start of treatment were included. | 0 | 17 | 4 | 17 | 13 | 17 |
| EG002 | Participants Without Delayed Start: Osivelotor 200 mg | Participants with SCD who participated in a previous osivelotor clinical study and received osivelotor 200 mg tablet orally and completed the end of treatment visit in this study for up to 680 days. Participants without any delay in start of treatment were included. | 0 | 1 | 1 | 1 | 1 | 1 |
| EG003 | Participants With Delayed Start: Osivelotor 100 mg | Participants with SCD who participated in a previous osivelotor clinical study and received 100 mg tablet orally and completed the end of treatment visit in this study for up to 680 days. Participants with delayed start are those with a gap in dosing greater than 42 consecutive days from completing the originating study (C5351005 Date of First Dose - C5351004 End of Treatment Date + 1 greater than 42). | 1 | 5 | 2 | 5 | 5 | 5 |
| EG004 | Participants With Delayed Start: Osivelotor 150 mg | Participants with SCD who participated in a previous osivelotor clinical study and received 150 mg tablet orally and completed the end of treatment visit in this study for up to 680 days. Participants with delayed start are those with a gap in dosing greater than 42 consecutive days from completing the originating study (C5351005 Date of First Dose - C5351004 End of Treatment Date + 1 greater than 42). | 0 | 6 | 2 | 6 | 5 | 6 |
| Haemolysis | Blood and lymphatic system disorders | MedDRA 28.0 | Systematic Assessment |
|
| Tachycardia paroxysmal | Cardiac disorders | MedDRA 28.0 | Systematic Assessment |
|
| Cholecystitis | Hepatobiliary disorders | MedDRA 28.0 | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Sepsis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Vestibular neuronitis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Thermal burn | Injury, poisoning and procedural complications | MedDRA 28.0 | Systematic Assessment |
|
| Osteonecrosis | Musculoskeletal and connective tissue disorders | MedDRA 28.0 | Systematic Assessment |
|
| Cerebral infarction | Nervous system disorders | MedDRA 28.0 | Systematic Assessment |
|
| Posterior reversible encephalopathy syndrome | Nervous system disorders | MedDRA 28.0 | Systematic Assessment |
|
| Skin ulcer | Skin and subcutaneous tissue disorders | MedDRA 28.0 | Systematic Assessment |
|
| Anaemia | Blood and lymphatic system disorders | MedDRA 28.0 | Systematic Assessment |
|
| Neutropenia | Blood and lymphatic system disorders | MedDRA 28.0 | Systematic Assessment |
|
| Sickle cell anaemia with crisis | Blood and lymphatic system disorders | MedDRA 28.0 | Systematic Assessment |
|
| Tachycardia | Cardiac disorders | MedDRA 28.0 | Systematic Assessment |
|
| Hypoacusis | Ear and labyrinth disorders | MedDRA 28.0 | Systematic Assessment |
|
| Dry eye | Eye disorders | MedDRA 28.0 | Systematic Assessment |
|
| Vision blurred | Eye disorders | MedDRA 28.0 | Systematic Assessment |
|
| Dyspepsia | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
|
| Peptic ulcer | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
|
| Stress ulcer | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
|
| Dental caries | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
|
| Gastritis | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
|
| Pain | General disorders | MedDRA 28.0 | Systematic Assessment |
|
| Oedema | General disorders | MedDRA 28.0 | Systematic Assessment |
|
| Chest pain | General disorders | MedDRA 28.0 | Systematic Assessment |
|
| Oedema peripheral | General disorders | MedDRA 28.0 | Systematic Assessment |
|
| Peripheral swelling | General disorders | MedDRA 28.0 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 28.0 | Systematic Assessment |
|
| Hyperbilirubinaemia | Hepatobiliary disorders | MedDRA 28.0 | Systematic Assessment |
|
| Jaundice | Hepatobiliary disorders | MedDRA 28.0 | Systematic Assessment |
|
| Malaria | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Urinary tract infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Asymptomatic bacteriuria | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Respiratory tract infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Bronchitis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| COVID-19 | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Osteomyelitis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Otitis media | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Pharyngotonsillitis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Bacterial vaginosis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Conjunctivitis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Cystitis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Onychomycosis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Osteomyelitis acute | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Osteomyelitis chronic | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Otitis media acute | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Pharyngitis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Pharyngitis streptococcal | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Tetanus | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Tonsillitis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Urinary tract infection bacterial | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Animal bite | Injury, poisoning and procedural complications | MedDRA 28.0 | Systematic Assessment |
|
| Mouth injury | Injury, poisoning and procedural complications | MedDRA 28.0 | Systematic Assessment |
|
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 28.0 | Systematic Assessment |
|
| Skin laceration | Injury, poisoning and procedural complications | MedDRA 28.0 | Systematic Assessment |
|
| Alanine aminotransferase increased | Investigations | MedDRA 28.0 | Systematic Assessment |
|
| Aspartate aminotransferase increased | Investigations | MedDRA 28.0 | Systematic Assessment |
|
| Blood alkaline phosphatase increased | Investigations | MedDRA 28.0 | Systematic Assessment |
|
| Blood bilirubin increased | Investigations | MedDRA 28.0 | Systematic Assessment |
|
| Platelet count increased | Investigations | MedDRA 28.0 | Systematic Assessment |
|
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA 28.0 | Systematic Assessment |
|
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA 28.0 | Systematic Assessment |
|
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA 28.0 | Systematic Assessment |
|
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 28.0 | Systematic Assessment |
|
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA 28.0 | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 28.0 | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 28.0 | Systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 28.0 | Systematic Assessment |
|
| Osteitis | Musculoskeletal and connective tissue disorders | MedDRA 28.0 | Systematic Assessment |
|
| Skin papilloma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 28.0 | Systematic Assessment |
|
| Burning sensation | Nervous system disorders | MedDRA 28.0 | Systematic Assessment |
|
| Cluster headache | Nervous system disorders | MedDRA 28.0 | Systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA 28.0 | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 28.0 | Systematic Assessment |
|
| Migraine | Nervous system disorders | MedDRA 28.0 | Systematic Assessment |
|
| Sciatica | Nervous system disorders | MedDRA 28.0 | Systematic Assessment |
|
| Pregnancy | Pregnancy, puerperium and perinatal conditions | MedDRA 28.0 | Systematic Assessment |
|
| Anxiety | Psychiatric disorders | MedDRA 28.0 | Systematic Assessment |
|
| Depression | Psychiatric disorders | MedDRA 28.0 | Systematic Assessment |
|
| Microalbuminuria | Renal and urinary disorders | MedDRA 28.0 | Systematic Assessment |
|
| Albuminuria | Renal and urinary disorders | MedDRA 28.0 | Systematic Assessment |
|
| Glycosuria | Renal and urinary disorders | MedDRA 28.0 | Systematic Assessment |
|
| Haematuria | Renal and urinary disorders | MedDRA 28.0 | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 28.0 | Systematic Assessment |
|
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 28.0 | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA 28.0 | Systematic Assessment |
|
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA 28.0 | Systematic Assessment |
|
| Skin discolouration | Skin and subcutaneous tissue disorders | MedDRA 28.0 | Systematic Assessment |
|
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 28.0 | Systematic Assessment |
|
| Pallor | Vascular disorders | MedDRA 28.0 | Systematic Assessment |
|
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA 28.0 | 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.
| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| Change at week 24 |
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| Change at week 48 |
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| Change at week 36 |
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| Change at week 48 |
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| Change at week 60 |
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| Change at week 24 |
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| Change at week 36 |
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| Change at week 48 |
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| Change at week 60 |
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| Change at week 24 |
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| Change at week 36 |
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| Change at week 48 |
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| Change at week 60 |
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