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

Phase 2

• Conditions: Haematological Disorders

• Registration Number: PACTR202208531640912
• Lead Sponsor: Global Blood Therapeutics Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-10-29
• Last Update Posted: 29 May 2023

Recruitment & Eligibility

• Status: Pending
• Sex: All
• Target Recruitment: 480

Inclusion 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

Study & Design

• Study Type: Interventional
• Study Design: Not specified