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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05431088
Other study ID # GBT021601-021
Secondary ID C53510042023-508
Status Recruiting
Phase Phase 2/Phase 3
First received
Last updated
Start date September 22, 2022
Est. completion date October 2, 2026

Study information

Verified date May 2024
Source Pfizer
Contact Pfizer Pfizer CT.gov Call Center
Phone 1-800-718-1021
Email ClinicalTrials.gov_Inquiries@pfizer.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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. 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.


Recruitment information / eligibility

Status Recruiting
Enrollment 517
Est. completion date October 2, 2026
Est. primary completion date October 2, 2026
Accepts healthy volunteers No
Gender All
Age group 6 Months to 65 Years
Eligibility 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


Related Conditions & MeSH terms


Intervention

Drug:
Osivelotor
Tablets which contain drug substance

Locations

Country Name City State
Kenya Gertrude's Children's Hospital Nairobi
Lebanon Nini Hospital s.a:l Tripoli
Lebanon Nini Hospital s.a:l Tripoli
Nigeria University College Hospital Ibadan Ibadan Oyo/ibadan North
Nigeria Aminu Kano Teaching Hospital Kano
Nigeria Lagos University Teaching Hospital Lagos
United States LSU Health Baton Rouge-North Clinic Baton Rouge Louisiana
United States Our Lady of the Lake Hopistal, Inc Baton Rouge Louisiana
United States Our Lady of the Lake Hopistal, Inc Baton Rouge Louisiana
United States Our lady of the Lake Hospital Baton Rouge Louisiana
United States Our Lady of the Lake Hospital, Inc In-Patient Pharmacy Baton Rouge Louisiana
United States Our Lady of the Lake Physicians Group, Medical Oncology Baton Rouge Louisiana
United States UNC Hospitals, The University of North Carolina at Chapel Hill Chapel Hill North Carolina
United States UNC Hospitals, The University of North Carolina at Chapel Hill Chapel Hill North Carolina
United States University of Illinois at Chicago Clinical Research Center Chicago Illinois
United States University of Illinois Hospital and Health Sciences System Chicago Illinois
United States University of Illinois Hospital and Health Sciences System - Hospital Pharmacy Services Chicago Illinois
United States University of Illinois Hospital and Health Sciences System - Investigational Drug Services (IDS) Chicago Illinois
United States Alpha Clinical Research Georgia Dunwoody Georgia
United States Inova Schar Cancer Institute Fairfax Virginia
United States Inova Schar Cancer Institute Infusion Pharmacy Fairfax Virginia
United States McGovern Medical School/Health Science Center Houston Houston Texas
United States Memorial Hermann - TMC Investigational Drugs, IDS Pharmacy Houston Texas
United States Memorial Hermann Hospital, Texas Medical Center - Clinical Research Unit (CRU) Houston Texas
United States UT Physicians Comprehensive Sickle Cell Clinic Houston Texas
United States UT Physicians Comprehensive Sickle Cell Clinic Houston Texas
United States Lynn Institute of the Ozarks Little Rock Arkansas
United States Mississippi Center for Advanced Medicine Madison Mississippi
United States Edward Jenner Research Group LLC. Miami Florida
United States Smilow Cancer Center at Yale New Haven Hospital New Haven Connecticut
United States Smilow Cancer Center at Yale New Haven Hospital New Haven Connecticut
United States University Medical Center New Orleans New Orleans Louisiana
United States Clinical Research Services Unit - Virginia Commonwealth University Medical Center Richmond Virginia
United States Virginia Commonwealth University Richmond Virginia
United States Lundquist Institute for Biomedical Innovation at Harbor- UCLA Medical Center Torrance California
United States Children's National Health System Washington District of Columbia
United States Children's National Hospital Washington District of Columbia
United States Kidz Hematology / Oncology a division of Kidz Medical West Palm Beach Florida
United States St. Mary's Medical Center West Palm Beach Florida

Sponsors (1)

Lead Sponsor Collaborator
Pfizer

Countries where clinical trial is conducted

United States,  Kenya,  Lebanon,  Nigeria, 

Outcome

Type Measure Description Time frame Safety issue
Primary Part A 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
Primary Part B 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
Primary Part C 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
Primary Part C Assess the PK AUC0-last, AUC0-inf, and Cmax after single dose of osivelotor in whole blood and plasma. Through Week 2
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