A Phase 2/3 Study of Osivelotor in Adult and Adolescent Participants With SCD

NCT05431088 · tracked via the Priya Life Science USA tracker

Sponsor

Pfizer

Phase

Phase 2/3

Started

2022-09-22

Last updated

2026-08-18

Condition(s) studied

Sickle Cell Disease

Investigational drug(s) / intervention(s)

Osivelotor

Osivelotor: Tablets which contain drug substance

Study summary

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

Eligibility

Sex

ALL

Min age

12 Years

Max age

—

Healthy volunteers

Inclusion Criteria: Part A and Part B: * Male or female with SCD, HbSS and HbSB-zero * Participants with Hemoglobin ≥ 5.5 and ≤ 10.5 g/dL during Screening and considered stable 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 years and older, inclusive at screening. * Participants with more than or equal to 2 and ≤ 10 VOCs within 12 months of Screening. OLE: \- Participants who have completed the Part B successfully will be eligible. Exclusion Criteria: Part A and Part B: * 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 or anytime during the screening period. * Participants in Sub-Saharan Africa or who have relocated from Sub-Saharan Africa within 6 months. OLE: * Have any unresolved clinically significant adverse event, laboratory abnormality, or safety finding from Part B that, in the opinion of the Investigator, increases the risk of study drug administration. * Met permanent treatment discontinuation criteria during Part B. * Have withdrawn consent or are unable to comply with study procedure

Primary outcome measure(s)

Part A — 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).
Part B — 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.
OLE — Approximately 24 months after last patient enrolled
Incidence of Treatment Emergent Adverse Events: * Incidence of SAEs * Incidence of AEs leading to discontinuation * Change from baseline in laboratory parameters.

Trial sites (80)

Facility	City	Region	Status
AMR Clinical	Mobile	Alabama	Recruiting
Connecticut Children's Medical Center	Hartford	Connecticut	Not Yet Recruiting
Smilow Cancer Hospital	New Haven	Connecticut	Not Yet Recruiting
Edward Jenner Research Group LLC.	Plantation	Florida	Recruiting
Pediatric Hematology / Oncology a division of Kidz Medical services	West Palm Beach	Florida	Recruiting
St. Mary's Medical Center	West Palm Beach	Florida	Recruiting
Children's Center for Advanced Pediatrics	Atlanta	Georgia	Recruiting
Alpha Clinical Research Georgia	Dunwoody	Georgia	Active Not Recruiting
Sonar Clinical Research	Riverdale	Georgia	Active Not Recruiting
University of Illinois at Chicago Clinical Research Center	Chicago	Illinois	Recruiting
University of Illinois Hospital and Health Sciences System - Investigational Drug Services (IDS)	Chicago	Illinois	Recruiting
University of Illinois Hospital and Health Sciences System	Chicago	Illinois	Recruiting
LSU Health Baton Rouge-North Clinic	Baton Rouge	Louisiana	Active Not Recruiting
Our Lady of the Lake Hospital, Inc.	Baton Rouge	Louisiana	Active Not Recruiting
Our lady of the Lake Hospital	Baton Rouge	Louisiana	Active Not Recruiting
University Medical Center New Orleans	New Orleans	Louisiana	Active Not Recruiting
Children's Hospital of Michigan	Detroit	Michigan	Not Yet Recruiting
Great Lakes Research Institute, Flourish Detroit	Southfield	Michigan	Not Yet Recruiting
Southern Specialty Research	Flowood	Mississippi	Recruiting
Mississippi Center for Advanced Medicine	Madison	Mississippi	Active Not Recruiting
University Health	Kansas City	Missouri	Recruiting
Jack & Sheryl Morris Cancer Center	New Brunswick	New Jersey	Recruiting
Robert Wood Johnson University Hospital	New Brunswick	New Jersey	Recruiting
Rutgers Cancer Institute of New Jersey	New Brunswick	New Jersey	Recruiting
Icahn School of Medicine at Mount Sinai	New York	New York	Recruiting
Jacobi Medical Center	The Bronx	New York	Recruiting
Clinical & Translational Research Center (CTRC)	Chapel Hill	North Carolina	Not Yet Recruiting
UNC Health	Chapel Hill	North Carolina	Not Yet Recruiting
UNC Eastowne Medical Office Building - Consent Only	Chapel Hill	North Carolina	Not Yet Recruiting
Duke University IDS Pharmacy	Durham	North Carolina	Recruiting
Duke University Medical Center	Durham	North Carolina	Recruiting
ECU Physicians, Brody Outpatient Clinic	Greenville	North Carolina	Recruiting
UNC IDS	Morrisville	North Carolina	Not Yet Recruiting
Prisma Health Laurens County Hospital	Clinton	South Carolina	Recruiting
Prisma Health - Upstate	Greenville	South Carolina	Recruiting
Prisma Health Greenville Memorial Hospital	Greenville	South Carolina	Recruiting
Prisma Health Outpatient Radiology - Greenville	Greenville	South Carolina	Recruiting
Prisma Health Patewood Hospital	Greenville	South Carolina	Recruiting
Prisma Health Greer Memorial Hospital	Greer	South Carolina	Recruiting
Prisma Health Hillcrest Hospital	Simpsonville	South Carolina	Recruiting

+ 40 more sites — see the full list on the official registry below.

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05431088 on ClinicalTrials.gov ↗ ← All trials in the USA