NCT05431088

A Phase 2/3 Study in Adult and Adolescent Participants With SCD

A Phase 2/3 Randomized, Multicenter Study of Osivelotor Administered Orally to Adult and Adolescent Participants With Sickle Cell Disease

• Status: Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 389 (estimated)
• Sponsor: Pfizer · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of osivelotor.

Detailed description

This is a three-part, multicenter, Phase 2/3 study of orally administered osivelotor in participants with sickle cell disease (SCD). Part A will evaluate the safety, tolerability, and efficacy of osivelotor in adult participants with SCD to determine an optimal dose. Part B will evaluate the efficacy of osivelotor versus placebo in adult and adolescent participants with SCD for 48 weeks. Open Label Extension (OLE) will evaluate the long-term safety and hematologic responses of open-label osivelotor in adult and adolescent participants having completed Part B.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	Osivelotor	Tablets which contain drug substance

Timeline

• Start date: 2022-09-22
• Primary completion: 2030-12-30
• Completion: 2032-12-31
• First posted: 2022-06-24
• Last updated: 2026-03-20

Locations

49 sites across 6 countries: United States, Brazil, India, Kenya, Nigeria, United Kingdom

Regulatory

• FDA-regulated drug study