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The trial is taking place at:
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St. Mary's Medical Center | West Palm Beach - Neurology Department

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

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Pfizer

Status and phase

Enrolling
Phase 3
Phase 2

Conditions

Sickle Cell Disease

Treatments

Drug: Osivelotor

Study type

Interventional

Funder types

Industry

Identifiers

NCT05431088
2023-508766-14-00 (Registry Identifier)
C5351004 (Other Identifier)
GBT021601-021

Details and patient eligibility

About

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

Full 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.

Part C will evaluate the pharmacokinetics (PK) and safety of single and multiple doses (MD) of open-label single arm osivelotor administered to pediatric participants.

Enrollment

517 estimated patients

Sex

All

Ages

6 months to 65 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

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 criteria

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

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Parallel Assignment

Masking

Quadruple Blind

517 participants in 3 patient groups, including a placebo group

Part A
Active Comparator group
Description:
Initially, participants will be randomized 1:1 to 100 mg and 150 mg daily. Upon review of the 150 mg safety data from at least 6 participants, there will be 1:1:1 randomization: 100 mg, 150 mg, and up to 200 mg. Participants will then receive maintenance once daily doses through Week 12.
Treatment:
Drug: Osivelotor
Part B
Placebo Comparator group
Description:
Following the selection of the optimal safe and effective dose from Part A of the study, Part B of the study will assess the efficacy and safety of 48 weeks of the optimal dose, compared to placebo
Treatment:
Drug: Osivelotor
Part C
Experimental group
Description:
100 mg dose in cohort C1, dose level for cohorts C2 to C4 to be determined based on emerging data
Treatment:
Drug: Osivelotor

Trial contacts and locations

38

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Central trial contact

Pfizer Pfizer CT.gov Call Center

Data sourced from clinicaltrials.gov

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