An Intrapatient Single Dose and Multiple Ascending Dose Study to Evaluate the Pharmacokinetics, Safety, Tolerability, and Pharmacodynamics of GBT021601, a Hemoglobin S Polymerization Inhibitor, in Participants With Sickle Cell Disease (SCD)

About the study

The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK), and pharmacodynamics (i.e., how the body absorbs, distributes, breaks down, and excretes) of GBT021601, a hemoglobin S (HbS) polymerization inhibitor, in participants with SCD, following single and multiple ascending doses.

Study point of contact

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


2 United States sites


18 to 60 Years


Phase 1

Study type



18 Years - 60 Years







participation requirements

Male or Female with SCD
Participants with SCD ages 18 to 60 years, inclusive.
Participant has provided documented informed consent.
Patients with stable and close to baseline hemoglobin value
Patients on HU should be on stable dose for at least 90 days prior to signing ICF

participation restrictions

Patients had more than 10 VOC within 12 months of screening
Patients who are pregnant or nursing
Patients who receive RBC transfusion therapy regularly or received an RBC transfusion for any reason within 60 days of signing the ICF
Hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF or within 24 days prior to Day 1 treatment


  • Miami, Florida, United States, Advanced Pharma CR, LLC, 33147 [Withdrawn]
  • Atlanta, Georgia, United States, Children's Healthcare of Atlanta, 30342 [Recruiting]
Last updated 2021-12-21