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.
| Condition | Sickle Cell Disease |
|---|---|
| Treatment | GBT021601 |
| Clinical Study Identifier | NCT04983264 |
| Sponsor | Global Blood Therapeutics |
| Last Modified on | 6 July 2022 |
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