A Phase 2/3 Randomized, Multicenter Study of Osivelotor Administered Orally to Adult and Adolescent Participants With Sickle Cell Disease
Pfizer
Summary
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of osivelotor.
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.
Eligibility
- Age range
- 12+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: Part A, Part B, and OLE: * Male or female with SCD * Participants with stable Hb value as judged by the Investigator * For participants taking hydroxyurea and/or L-glutamine, the dose must be stable for at least 90 days prior to signing the ICF or assent and with no anticipated need for dose adjustments during the study in the opinion of the Investigator. Part B: * Participants with SCD ages 12 to 65 years, inclusive * Participants with more than or equal to 2 and ≤ 10 VOCs within 12 months of Screening. OLE: \- Participants who have completed the Part B will be eligi…
Interventions
- DrugOsivelotor
Tablets which contain drug substance
Locations (49)
- Smilow Cancer HospitalNew Haven, Connecticut
- Edward Jenner Research Group Center LLCPlantation, Florida
- Pediatric Hematology / Oncology a division of Kidz Medical servicesWest Palm Beach, Florida
- St. Mary's Medical CenterWest Palm Beach, Florida
- Alpha Clinical Research GeorgiaDunwoody, Georgia
- Sonar Clinical ResearchRiverdale, Georgia