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

• Status: Recruiting
• Trial end date: 2022-04-30
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Global Blood Therapeutics

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