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

Sickle Cell Disease Clinical Trial

Official title:

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

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
• Start date: September 22, 2022
• Est. completion date: October 2, 2026

Study information

• Verified date: April 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
• 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

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

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

External Links

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