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

Completed · Phase 1

Conditions studied: Sickle Cell Disease

In brief

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.

Key facts

Study ID

NCT04983264

Run by

Pfizer

People needed

6

Starts

2021-05-21

Expected to finish

2022-12-06

Last updated by the study team

2024-06-10

Who can join

Age: 18 and older, up to 60. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Advanced Pharma CR, LLC — Miami, Florida, United States
• Visionaries Clinical Research LLC — Atlanta, Georgia, United States
• Children's Healthcare of Atlanta AFLAC Center — Atlanta, Georgia, United States

Full record on ClinicalTrials.gov

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