A Study to Evaluate GBT021601-012 Single Dose and Multiple Dose in Participants With Sickle Cell Disease (SCD)

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)

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.

This is an open-label intrapatient single dose followed by a multiple dose escalation study in at least six (6) participants with SCD.

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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