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NCT05431088PHASE2, PHASE3Recruiting

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

Pfizer

Start Date

9/22/2022

Completion Date

12/31/2032

Summary

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

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. (Complete) Part B will evaluate the efficacy of osivelotor versus placebo in adult and adolescent participants with SCD for 48 weeks. Open Label Extension (OLE) will evaluate the long-term safety and hematologic responses of open-label osivelotor in adult and adolescent participants having completed Part B.

Eligibility Criteria

Age Range: 12 years to No maximum

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

Interventions

DRUG

Osivelotor

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Conditions

Sickle Cell Disease

Locations

AMR Clinical

Mobile, Alabama 36608

United States

Connecticut Children's Medical Center

Hartford, Connecticut 06106

United States

Smilow Cancer Hospital

New Haven, Connecticut 06511

United States

Edward Jenner Research Group LLC.

Plantation, Florida 33317

United States

Pediatric Hematology / Oncology a division of Kidz Medical services

West Palm Beach, Florida 33407

United States

St. Mary's Medical Center

West Palm Beach, Florida 33407

United States

Children's Center for Advanced Pediatrics

Atlanta, Georgia 30329

United States

Alpha Clinical Research Georgia

Dunwoody, Georgia 30350

United States

Sonar Clinical Research

Riverdale, Georgia 30274

United States

University of Illinois at Chicago Clinical Research Center

Chicago, Illinois 60612

United States

University of Illinois Hospital and Health Sciences System - Investigational Drug Services (IDS)

Chicago, Illinois 60612

United States

University of Illinois Hospital and Health Sciences System

Chicago, Illinois 60612

United States

LSU Health Baton Rouge-North Clinic

Baton Rouge, Louisiana 70805

United States

Our Lady of the Lake Hospital, Inc.

Baton Rouge, Louisiana 70808

United States

Our lady of the Lake Hospital

Baton Rouge, Louisiana 70808

United States

University Medical Center New Orleans

New Orleans, Louisiana 70112

United States

Children's Hospital of Michigan

Detroit, Michigan 48201

United States

Great Lakes Research Institute, Flourish Detroit

Southfield, Michigan 48075

United States

Southern Specialty Research

Flowood, Mississippi 39232

United States

Mississippi Center for Advanced Medicine

Madison, Mississippi 39110

United States

University Health

Kansas City, Missouri 64108

United States

Jack & Sheryl Morris Cancer Center

New Brunswick, New Jersey 08901

United States

Robert Wood Johnson University Hospital

New Brunswick, New Jersey 08901

United States

Rutgers Cancer Institute of New Jersey

New Brunswick, New Jersey 08901

United States

Icahn School of Medicine at Mount Sinai

New York, New York 10029

United States

Jacobi Medical Center

The Bronx, New York 10461

United States

Clinical & Translational Research Center (CTRC)

Chapel Hill, North Carolina 27514

United States

UNC Health

Chapel Hill, North Carolina 27514

United States

UNC Eastowne Medical Office Building - Consent Only

Chapel Hill, North Carolina 27517

United States

Duke University IDS Pharmacy

Durham, North Carolina 27710

United States

Duke University Medical Center

Durham, North Carolina 27710

United States

ECU Physicians, Brody Outpatient Clinic

Greenville, North Carolina 27834

United States

UNC IDS

Morrisville, North Carolina 27560

United States

Prisma Health Laurens County Hospital

Clinton, South Carolina 29325

United States

Prisma Health - Upstate

Greenville, South Carolina 29605

United States

Prisma Health Greenville Memorial Hospital

Greenville, South Carolina 29605

United States

Prisma Health Outpatient Radiology - Greenville

Greenville, South Carolina 29605

United States

Prisma Health Patewood Hospital

Greenville, South Carolina 29615

United States

Prisma Health Greer Memorial Hospital

Greer, South Carolina 29650

United States

Prisma Health Hillcrest Hospital

Simpsonville, South Carolina 29681

United States

Prisma Health North Greenville Hospital

Travelers Rest, South Carolina 29690

United States

McGovern Medical School at UTHealth

Houston, Texas 77030

United States

Memorial Hermann Hospital, Texas Medical Center - Clinical Research Unit (CRU)

Houston, Texas 77030

United States

The University of Texas Health Science Center at Houston

Houston, Texas 77030

United States

UT Physicians Comprehensive Sickle Cell Clinic

Houston, Texas 77030

United States

Inova Schar Cancer Institute

Fairfax, Virginia 22031

United States

Multihemo Servicos Medicos S/A

Recife, Pernambuco 50070-460

Brazil

Hospital Das Clinicas da Faculdade de Medicina de Ribeirão Preto - USP

Ribeirão Preto, São Paulo 14051-140

Brazil

Fundacao Faculdade Regional de Medicina de Sao Jose do Rio Preto

São José do Rio Preto, São Paulo 15090-000

Brazil

BP A Beneficência Portuguesa de São Paulo

São Paulo, São Paulo 01321 001

Brazil

BP - A Beneficência Portuguesa de São Paulo

São Paulo, São Paulo 01323-900

Brazil

Casa de Saude Santa Marcelina

São Paulo, São Paulo 08270-070

Brazil

Casa de Saude Santa Marcelina

São Paulo, São Paulo 08270-120

Brazil

CPHosp Medicina, Ensino e Pesquisa Ltda

São, São Paulo 01228-000

Brazil

Esho Empresa de Servicos Hospitalares S A

São Paulo, SÃO Paulo- Brasil 01232-011

Brazil

Instituto Estadual de Hematologia Arthur Siqueira Cavalcanti - HEMORIO

Rio de Janeiro, 20211-030

Brazil

Esho Empresa de Servicos Hospitalares S A

São Paulo, 01232-010

Brazil

Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo

São Paulo, 05403-000

Brazil

Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo

São Paulo, 05403-010

Brazil

AIIMS Raipur

Raipur, Chhattisgarh 492099

India

Nirmal Hospital Pvt Ltd

Surat, Gujarat 395002

India

Haemato Oncology Care Centre

Vadodara, Gujarat 390020

India

Chopda Medicare & Research Centre Pvt. Ltd: Magnum Heart Institute

Nashik, Maharashtra 422005

India

AIIMS Delhi

New Delhi, National Capital Territory of Delhi 110029

India

AIIMS, Bhubaneswar

Bhubaneswar, Odisha 751019

India

KEMRI/CRDR, Siaya, KEMRI Clinical Research Annex

Kisumu, Siaya County 40600

Kenya

Gertrude's Children's Hospital

Nairobi, 00100

Kenya

Kenya Medical Research Institute - Centre for Respiratory Disease Research

Nairobi, 00100

Kenya

Center for Research In Therapeutic Sciences (CREATES), Strathmore University Medical Centre

Nairobi, 00200

Kenya

University College Hospital Ibadan

Ibadan, Oyo/ibadan North 200212

Nigeria

Aminu Kano Teaching Hospital

Kano, 700233

Nigeria

Lagos University Teaching Hospital

Lagos, 100254

Nigeria

King Khalid University Hospital

Riyadh, Riyadh Region 11472

Saudi Arabia

University Hospitals Bristol and Weston NHS Foundation Trust

Bristol, BS2 8ED

United Kingdom

Bristol Royal Infirmary

Bristol, BS2 8EX

United Kingdom

University College London Hospital

London, NW1 2PG

United Kingdom

Guy's & St Thomas' NHS Foundation Trust

London, SE1 9RT

United Kingdom

King's College Hospital NHS Foundation Trust

London, SE5 9RS

United Kingdom

Hammersmith Hospital, Imperial College Healthcare NHS Trust

London, W12 0HS

United Kingdom

Hammersmith Hospital

London, W12 0HS

United Kingdom