A Phase 2/3 Study in Adult and Pediatric Participants With Sickle Cell Disease (SCD)

• Status: Not yet recruiting
• Trial end date: 2027-04-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of GBT021601.

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

Details

• Lead Sponsor: Global Blood Therapeutics

Criteria

Inclusion Criteria:

Part A, Part B, and Part C:

• Male or female with SCD

• Participants with stable hemoglobin value

• Participants on HU should be on stable dose for at least 90 days prior to signing ICF

Part B:

• Participants with SCD ages 12 to 65 years, inclusive

• Participants with more than or equal to 2 and less than or equal to 10 VOCs inclusive, within 12 months of Screening

Exclusion Criteria:

Part A, Part B, and Part C:

• Participants who had more than 10 VOC within 12 months of screening

• Female participant who is breastfeeding or pregnant

• Participants who receive RBC transfusion therapy regularly or received an RBC transfusion ---for any reason within 90 days of Day 1

• Participants hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF