An Open-label Extension Study to Evaluate the Long-term Safety of GBT021601 Administered to Participants With Sickle Cell Disease Who Have Participated in a GBT021601 Clinical Trial

Official Summary

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

Eligibility Requirements

• Minimum Age: 6 Months

Study Design

• Study Type: INTERVENTIONAL
• Allocation: NA
• Model: SINGLE_GROUP
• Masking: NONE
• Enrollment: 47 participants

Study Arms

• Treatment (EXPERIMENTAL)
  Osivelotor

Interventions

• DRUG: Osivelotor — Osivelotor

Primary Outcomes

• Number of Participants With Treatment Emergent Adverse Events (TEAEs) (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 (Baseline, Week 12)
• Change From Baseline in Hematocrit at Week 48 (Baseline, Week 48)
• Change From Baseline in Leukocytes at Week 12 (Baseline, Week 12)
• Change From Baseline in Leukocytes Week 48 (Baseline, Week 48)

Secondary Outcomes

• 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)

Eligibility Criteria

Inclusion Criteria:

1. 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.
2. 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.
3. 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
4. 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:

* 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

Trial Locations

• Our Lady of the Lake Hospital, Inc., Baton Rouge, Louisiana, United States
• University Medical Center Inpatient Pharmacy, New Orleans, Louisiana, United States
• University Medical Center New Orleans, New Orleans, Louisiana, United States
• Mississippi Center for Advanced Medicine, Madison, Mississippi, United States
• University of Texas Health Science Center, Houston, Texas, United States
• Inova Schar Cancer Institute, Fairfax, Virginia, United States
• University College Hospital Ibadan, Ibadan, Oyo/ibadan North, Nigeria
• Aminu kano Teaching Hospital, Kano, Nigeria
• Lagos University Teaching Hospital, Lagos, Nigeria

Study Officials

• Pfizer CT.gov Call Center — STUDY_DIRECTOR
  Pfizer

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