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RECRUITINGPhase 2 / Phase 3INTERVENTIONAL

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

A Phase 2/3 Randomized, Multicenter Study of Osivelotor Administered Orally to Adult and Adolescent Participants With Sickle Cell Disease

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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

Who May Be Eligible (Plain English)

Who May Qualify: Part A, Part B, and OLE: - 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. OLE: \- Participants who have completed the Part B will be eligible. Who Should NOT Join This Trial: Part A, Part B, and OLE: - 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. Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
Inclusion Criteria: Part A, Part B, and OLE: * 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. OLE: \- Participants who have completed the Part B will be eligible. Exclusion Criteria: Part A, Part B, and OLE: * 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.

Treatments Being Tested

DRUG

Osivelotor

Tablets which contain drug substance

Locations (20)

Smilow Cancer Hospital
New Haven, Connecticut, United States
Edward Jenner Research Group Center LLC
Plantation, Florida, United States
Pediatric Hematology / Oncology a division of Kidz Medical services
West Palm Beach, Florida, United States
St. Mary's Medical Center
West Palm Beach, Florida, United States
Alpha Clinical Research Georgia
Dunwoody, Georgia, United States
Sonar Clinical Research
Riverdale, Georgia, United States
University of Illinois at Chicago Clinical Research Center
Chicago, Illinois, United States
University of Illinois Hospital and Health Sciences System - Investigational Drug Services (IDS)
Chicago, Illinois, United States
University of Illinois Hospital and Health Sciences System
Chicago, Illinois, United States
LSU Health Baton Rouge-North Clinic
Baton Rouge, Louisiana, United States
Our Lady of the Lake Hospital, Inc.
Baton Rouge, Louisiana, United States
Our lady of the Lake Hospital
Baton Rouge, Louisiana, United States
University Medical Center New Orleans
New Orleans, Louisiana, United States
Mississippi Center for Advanced Medicine
Madison, Mississippi, United States
University Health
Kansas City, Missouri, United States
Clinical & Translational Research Center (CTRC)
Chapel Hill, North Carolina, United States
UNC Health
Chapel Hill, North Carolina, United States
UNC Eastowne Medical Office Building - Consent Only
Chapel Hill, North Carolina, United States
UNC IDS
Morrisville, North Carolina, United States
McGovern Medical School at UTHealth
Houston, Texas, United States