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

Last updated: April 16, 2025
Sponsor: Pfizer
Overall Status: Terminated

Phase

2/3

Condition

Sickle Cell Disease

Red Blood Cell Disorders

Treatment

open-label GBT021601

Osivelotor

Clinical Study ID

NCT05632354
GBT021601-022
C5351005
  • Ages > 6
  • All Genders

Study Summary

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

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Male or female aged 6 months or older with SCD who participated and received studydrug or placebo in a previous osivelotor clinical study and completed the end oftreatment visit. Note: Participants who discontinued study drug in the originating study due to anTEAE, but who remained on study, may be eligible for treatment in this studyprovided the TEAE does not pose a risk for treatment with osivelotor.

  2. Females of childbearing potential are required to have a negative urine pregnancytest prior to dosing on Day 1. Note: Females who become of childbearing potential during the study must be willingto have negative urine pregnancy tests to remain in the study.

  3. If sexually active, females of childbearing potential must consistently use highlyeffective methods of contraception consistently throughout the study and for atleast 120 days after the last dose of study drug. If sexually active, maleparticipants must use barrier methods of contraception until 84 days after the lastdose of study drug. Male participants are eligible to participate if they agree tothe following requirements during the study intervention period and for 84 daysafter the last dose of study intervention:

  • Refrain from donating sperm PLUS either

  • Be abstinent from heterosexual intercourse as their preferred and usuallifestyle OR

  • Must agree to use a male condom when engaging in any activity that allows forpassage of ejaculate to another person

  1. Participant has provided written informed consent/assent. For underage participants,both the consent of the participant's legal representative or legal guardian and theparticipant's assent (where applicable) must be obtained based on localrequirements.

Exclusion

Exclusion Criteria:

  • Participant withdrew consent or was noncompliant from the originating osivelotorclinical study

  • Current or recent use of voxelotor. Recent use is defined as within 10 days prior toDay 1

  • Current or recent use of crizanlizumab. Recent use is defined as within 90 daysprior to Day 1

  • Participant has any medical, psychological, safety, or behavioral conditions that,in the opinion of the Investigator, may confound safety interpretation, interferewith compliance, or preclude informed consent

  • Has received an investigational drug (including investigational vaccines) within 5times the elimination half-life (if known) or within 30 days (if the eliminationhalf-life- is unknown) prior to study drug administration or is concurrentlyenrolled in any research judged not to be scientifically or medically compatiblewith this study

Study Design

Total Participants: 47
Treatment Group(s): 2
Primary Treatment: open-label GBT021601
Phase: 2/3
Study Start date:
January 05, 2023
Estimated Completion Date:
February 13, 2025

Study Description

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

Connect with a study center

  • College of Medicine, University of Ibadan

    Ibadan, OYO State 200212
    Nigeria

    Site Not Available

  • University College Hospital Ibadan

    Ibadan, Oyo/ibadan North 200212
    Nigeria

    Site Not Available

  • Aminu kano Teaching Hospital

    Kano, 700233
    Nigeria

    Site Not Available

  • Lagos University Teaching Hospital

    Lagos, 100254
    Nigeria

    Site Not Available

  • Edward Jenner Research Group LLC.

    Miami, Florida 33317
    United States

    Site Not Available

  • Our Lady of the Lake Hospital, Inc.

    Baton Rouge, Louisiana 70808
    United States

    Site Not Available

  • University Medical Center Inpatient Pharmacy

    New Orleans, Louisiana 70112
    United States

    Site Not Available

  • University Medical Center New Orleans

    New Orleans, Louisiana 70112
    United States

    Site Not Available

  • Mississippi Center for Advanced Medicine

    Madison, Mississippi 39110
    United States

    Site Not Available

  • University of Texas Health Science Center

    Houston, Texas 77030
    United States

    Site Not Available

  • Inova Schar Cancer Institute

    Fairfax, Virginia 22031
    United States

    Site Not Available

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