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

CompletedPhase 1

Sponsor: Pfizer

Conditions: Sickle Cell Disease

Interventions: GBT021601

Countries: United States

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.

Eligibility overview

Sex: ALL

Age: 18 Years to 60 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria

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

Locations (3)

Miami, Florida, United States
Atlanta, Georgia, United States
Atlanta, Georgia, United States