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

• Status: Not yet recruiting
• Trial end date: 2028-03-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Global Blood Therapeutics

Criteria

Inclusion Criteria:

1. Male or female age 6 months or older with SCD who participated and received study drug or placebo in the previous GBT-Sponsored GBT021601 clinical study and remained on the previous study within 30 calendar days of the Day 1 visit for Study GBT021601-022.

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 GBT021601.

2. Female participants of childbearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1.

Note: Female participants 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, female participants 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 120 days after the last dose of study drug.

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 GBT021601 clinical study.

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