Status:

ACTIVE_NOT_RECRUITING

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

Lead Sponsor:

Pfizer

Conditions:

Sickle Cell Disease

Eligibility:

All Genders

6+ years

Phase:

PHASE2

PHASE3

Brief 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 osi...

Eligibility Criteria

Inclusion

  • Part A, Part B, and Part C:
  • 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.

Exclusion

  • Part A, Part B, and Part C:
  • Participants who had more than 10 VOC within 12 months of screening
  • Female participant who is breastfeeding or pregnant
  • Participants who receive RBC transfusion therapy regularly or received an RBC transfusion ---for any reason within 90 days of Day 1
  • Participants hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF

Key Trial Info

Start Date :

September 22 2022

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

December 30 2032

Estimated Enrollment :

429 Patients enrolled

Trial Details

Trial ID

NCT05431088

Start Date

September 22 2022

End Date

December 30 2032

Last Update

June 17 2025

Active Locations (24)

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Page 1 of 6 (24 locations)

1

Smilow Cancer Hospital

New Haven, Connecticut, United States, 06511

2

Children's National Hospital

Washington D.C., District of Columbia, United States, 20010

3

Edward Jenner Research Group Center LLC

Plantation, Florida, United States, 33317

4

Pediatric Hematology / Oncology a division of Kidz Medical services

West Palm Beach, Florida, United States, 33407