NCT04983264

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)

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: Pfizer · Industry
• Sex: All
• Age: 18 Years – 60 Years
• Healthy volunteers: Not accepted

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.

Detailed description

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

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	GBT021601	Tablets and capsules which contain GBT021601 drug substance

Timeline

• Start date: 2021-05-21
• Primary completion: 2022-12-06
• Completion: 2022-12-06
• First posted: 2021-07-30
• Last updated: 2024-06-10
• Results posted: 2024-06-10

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study