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Purpose

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

Condition

Eligibility

Eligible Ages
Between 6 Months and 65 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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

Study Design

Phase
Phase 2/Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description
Part B only

Arm Groups

ArmDescriptionAssigned Intervention
Active Comparator
Part A 		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.
  • Drug: Osivelotor
    Tablets which contain drug substance
    Other names:
    • PF-07940367 or GBT021601
Placebo Comparator
Part B 		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
  • Drug: Osivelotor
    Tablets which contain drug substance
    Other names:
    • PF-07940367 or GBT021601
Experimental
Part C 		100 mg dose in cohort C1, dose level for cohorts C2 to C4 to be determined based on emerging data
  • Drug: Osivelotor
    Tablets which contain drug substance
    Other names:
    • PF-07940367 or GBT021601

Recruiting Locations

McGovern Medical School/Health Science Center Houston
Houston, Texas 77030

Memorial Hermann - TMC Investigational Drugs, IDS Pharmacy
Houston, Texas 77030

Memorial Hermann Hospital, Texas Medical Center - Clinical Research Unit (CRU)
Houston, Texas 77030

More Details

Status
Recruiting
Sponsor
Pfizer

Study Contact

Pfizer Pfizer CT.gov Call Center
1-800-718-1021
ClinicalTrials.gov_Inquiries@pfizer.com

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. 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.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.