A Phase 2/3 Randomized, Multicenter Study of GBT021601 Administered Orally to Participants With Sickle Cell Disease and an Open-Label Pharmacokinetics Study in Pediatric Participants With Sickle Cell Disease

About the study

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

Study point of contact

Eleanor Lisbon, MD, MPH
[email protected]
Jessica Guider
[email protected]


6 Months - 65 Years


Phase 2/Phase 3

Study type






participation requirements

Part A, Part B, and Part C:

Male or female with SCD
Participants with stable Hb value as judged by the Investigator
For participants taking hydroxyurea and/or L-glutamine, the dose must be stable for at least 90 days prior to signing the ICF or assent and with no anticipated need for dose adjustments during the study in the opinion of the Investigator.

Part B:

Participants with SCD ages 12 to 65 years, inclusive
Participants with more than or equal to 2 and ≤ 10 VOCs within 12 months of Screening.

participation restrictions

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

Last updated 2022-11-07