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

April 5, 2024 updated by: Pfizer

A Phase 2/3 Randomized, Multicenter Study of GBT021601 Administered Orally to Participants With Sickle Cell Disease and an Open-Label Pharmacokinetics Study in Pediatric Participants With Sickle Cell Disease

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

517

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Kenya
      • Nairobi, Kenya, 00100
        • Not yet recruiting
        • Gertrude's Children's Hospital
  • Lebanon
      • Tripoli, Lebanon
        • Not yet recruiting
        • Nini Hospital s.a:l
      • Tripoli, Lebanon, 1300
        • Not yet recruiting
        • Nini Hospital s.a:l
  • Nigeria
      • Kano, Nigeria, 700233
        • Recruiting
        • Aminu Kano Teaching Hospital
      • Lagos, Nigeria, 100254
        • Recruiting
        • Lagos University Teaching Hospital
    • Oyo/ibadan North
      • Ibadan, Oyo/ibadan North, Nigeria, 200212
        • Recruiting
        • University College Hospital Ibadan
  • United States
    • Arkansas
      • Little Rock, Arkansas, United States, 72204
        • Active, not recruiting
        • Lynn Institute of the Ozarks
    • California
      • Torrance, California, United States, 90502
        • Not yet recruiting
        • Lundquist Institute for Biomedical Innovation at Harbor- UCLA Medical Center
    • Connecticut
      • New Haven, Connecticut, United States, 06510
        • Not yet recruiting
        • Smilow Cancer Center at Yale New Haven Hospital
      • New Haven, Connecticut, United States, 06511
        • Not yet recruiting
        • Smilow Cancer Center at Yale New Haven Hospital
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
        • Not yet recruiting
        • Children's National Health System
      • Washington, District of Columbia, United States, 20010
        • Not yet recruiting
        • Children's National Hospital
    • Florida
      • Miami, Florida, United States, 33317
        • Recruiting
        • Edward Jenner Research Group LLC.
      • West Palm Beach, Florida, United States, 33407
        • Not yet recruiting
        • Kidz Hematology / Oncology a division of Kidz Medical
      • West Palm Beach, Florida, United States, 33407
        • Not yet recruiting
        • St. Mary's Medical Center
    • Georgia
      • Dunwoody, Georgia, United States, 30350
        • Recruiting
        • Alpha Clinical Research Georgia
    • Illinois
      • Chicago, Illinois, United States, 60612-4333
        • Not yet recruiting
        • University of Illinois Hospital and Health Sciences System - Hospital Pharmacy Services
      • Chicago, Illinois, United States, 60612-7232
        • Not yet recruiting
        • University of Illinois Hospital and Health Sciences System
      • Chicago, Illinois, United States, 60612
        • Not yet recruiting
        • University of Illinois at Chicago Clinical Research Center
      • Chicago, Illinois, United States, 60612
        • Not yet recruiting
        • University of Illinois Hospital and Health Sciences System - Investigational Drug Services (IDS)
    • Louisiana
      • Baton Rouge, Louisiana, United States, 70805
        • Recruiting
        • LSU Health Baton Rouge-North Clinic
      • Baton Rouge, Louisiana, United States, 70805
        • Not yet recruiting
        • Our lady of the Lake Hospital
      • Baton Rouge, Louisiana, United States, 70808
        • Not yet recruiting
        • Our Lady of the Lake Hopistal, Inc
      • Baton Rouge, Louisiana, United States, 70808
        • Recruiting
        • Our Lady of the Lake Hopistal, Inc
      • Baton Rouge, Louisiana, United States, 70808
        • Recruiting
        • Our Lady of the Lake Hospital, Inc In-Patient Pharmacy
      • Baton Rouge, Louisiana, United States, 70808
        • Recruiting
        • Our Lady of the Lake Physicians Group, Medical Oncology
      • New Orleans, Louisiana, United States, 70112
        • Active, not recruiting
        • University Medical Center New Orleans
    • Mississippi
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27514
        • Not yet recruiting
        • UNC Hospitals, The University of North Carolina at Chapel Hill
      • Chapel Hill, North Carolina, United States, 27599-7600
        • Not yet recruiting
        • UNC Hospitals, The University of North Carolina at Chapel Hill
    • Texas
      • Houston, Texas, United States, 77004
        • Recruiting
        • UT Physicians Comprehensive Sickle Cell Clinic
      • Houston, Texas, United States, 77030
        • Recruiting
        • McGovern Medical School/Health Science Center Houston
      • Houston, Texas, United States, 77030
        • Recruiting
        • Memorial Hermann - TMC Investigational Drugs, IDS Pharmacy
      • Houston, Texas, United States, 77030
        • Recruiting
        • Memorial Hermann Hospital, Texas Medical Center - Clinical Research Unit (CRU)
      • Houston, Texas, United States, 77030
        • Recruiting
        • UT Physicians Comprehensive Sickle Cell Clinic
    • Virginia
      • Fairfax, Virginia, United States, 22031
        • Recruiting
        • Inova Schar Cancer Institute
      • Fairfax, Virginia, United States, 22031
        • Recruiting
        • Inova Schar Cancer Institute Infusion Pharmacy
      • Richmond, Virginia, United States, 23298
        • Not yet recruiting
        • Virginia Commonwealth University
      • Richmond, Virginia, United States, 23298
        • Not yet recruiting
        • Clinical Research Services Unit - Virginia Commonwealth University Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 months to 65 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Part A
Time Frame: Through week 12
Number of adult participants with change from baseline in hemoglobin (Hb) through week 12 as measured by change in osivelotor concentrations from baseline or percentage change from baseline of clinical measures of anemia Hb and hemolysis (including indirect bilirubin, reticulocytes and lactate dehydrogenase).
Through week 12
Part B
Time Frame: Through week 48

Co-primary endpoints: Hb response (increase from baseline of >1 g/dL) at Week 48 (based on average of Hb levels at Week 40 and Week 48) and the Annualized rate of VOC through end of Week 48.

A VOC is defined as an acute episode of pain that:

  • Has no medically determined cause other than a vaso-occlusive event, and
  • Results in a visit to a medical facility (hospitalization, emergency department, urgent care center, outpatient clinic, or infusion center), and
  • Requires parenteral narcotic agents, parenteral nonsteroidal anti-inflammatory drugs (NSAIDs), or an increase in treatment with oral narcotics.

Complicated VOCs of acute chest syndrome (ACS), hepatic sequestration, splenic sequestration, priapism, and dactylitis that meet the requirements listed above will be included in this co-primary endpoint.
Through week 48
Part C
Time Frame: Through Week 6

Assess the PK AUC0-24 after multiple dose administration of osivelotor in whole blood and plasma.

  • Cmax and Cmin
  • Accumulation ratios based on Cmax and AUC0-24. -% Hb occupancy
Through Week 6
Part C
Time Frame: Through Week 2
Assess the PK AUC0-last, AUC0-inf, and Cmax after single dose of osivelotor in whole blood and plasma.
Through Week 2

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Pfizer

Collaborators

Pfizer

Investigators

  • Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 22, 2022

Primary Completion (Estimated)

October 2, 2026

Study Completion (Estimated)

October 2, 2026

Study Registration Dates

First Submitted

June 21, 2022

First Submitted That Met QC Criteria

June 21, 2022

First Posted (Actual)

June 24, 2022

Study Record Updates

Last Update Posted (Actual)

April 8, 2024

Last Update Submitted That Met QC Criteria

April 5, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GBT021601-021
  • C5351004 (Other Identifier: Alias Study Number)
  • 2023-508766-14-00 (Registry Identifier: CTIS (EU))

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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