GBT021601-022: A Study of GBT021601 in Participants With Sickle Cell Disease (SCD)

NCT05632354 | Terminated Phase 2 Results FDA Drug

• 2 other identifiers: GBT021601-022C5351005
• Study Type: interventional
• Target: 47
• Locations: 2 countries
• Sites: 9

Brief Summary

An Open-label Extension Study of GBT021601 in Participants with Sickle Cell Disease

Conditions

Sickle Cell Disease

Outcome Measures

Primary Outcomes (9)

• 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

• 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

Secondary Outcomes (6)

• Annualized Rate of Vaso-Occlusive Crisis (VOC)

  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)

  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

  Baseline, Weeks 12, 24, 36, 48, and 60

• Change From Baseline in Reticulocytes at Weeks 12, 24, 36, 48, and 60

  Baseline, Weeks 12, 24, 36, 48, and 60

• Change From Baseline in Lactate Dehydrogenase (LDH) at Weeks 12, 24, 36, 48, and 60

  Baseline, Weeks 12, 24, 36, 48, and 60

Study Arms (1)

Treatment

EXPERIMENTAL

Osivelotor

Drug: Osivelotor

Interventions

Osivelotor DRUG

Osivelotor

Treatment

Eligibility Criteria

• Age: 6 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• Refrain from donating sperm PLUS either
• Be abstinent from heterosexual intercourse as their preferred and usual lifestyle OR
• Must agree to use a male condom when engaging in any activity that allows for passage of ejaculate to another person
• 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.

You may not qualify if:

• Participant withdrew consent or was noncompliant from the originating osivelotor clinical study
• Current or recent use of voxelotor. Recent use is defined as within 10 days prior to Day 1
• Current or recent use of crizanlizumab. Recent use is defined as within 90 days prior to Day 1
• Participant has any medical, psychological, safety, or behavioral conditions that, in the opinion of the Investigator, may confound safety interpretation, interfere with compliance, or preclude informed consent
• Has received an investigational drug (including investigational vaccines) within 5 times the elimination half-life (if known) or within 30 days (if the elimination half-life- is unknown) prior to study drug administration or is concurrently enrolled in any research judged not to be scientifically or medically compatible with this study

Sponsors & Collaborators

• Pfizer: lead

Study Sites (9)

Our Lady of the Lake Hospital, Inc.

Baton Rouge, Louisiana, 70808, United States

Location

University Medical Center Inpatient Pharmacy

New Orleans, Louisiana, 70112, United States

Location

University Medical Center New Orleans

New Orleans, Louisiana, 70112, United States

Location

Mississippi Center for Advanced Medicine

Madison, Mississippi, 39110, United States

Location

University of Texas Health Science Center

Houston, Texas, 77030, United States

Location

Inova Schar Cancer Institute

Fairfax, Virginia, 22031, United States

Location

University College Hospital Ibadan

Ibadan, Oyo/ibadan North, 200212, Nigeria

Location

Aminu kano Teaching Hospital

Kano, 700233, Nigeria

Location

Lagos University Teaching Hospital

Lagos, 100254, Nigeria

Location

Related Links

• To obtain contact information for a study center near you, click here.

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 1, 2022
• First Posted: November 30, 2022
• Study Start: January 5, 2023
• Primary Completion: February 13, 2025
• Study Completion: February 13, 2025
• Last Updated: March 19, 2026
• Results First Posted: March 19, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share

Locations

US (6); NG (3)