RecruitingPhase 2Phase 3NCT05431088

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


Sponsor

Pfizer

Enrollment

389 participants

Start Date

Sep 22, 2022

Study Type

INTERVENTIONAL

Conditions

Summary

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


Eligibility

Min Age: 12 Years

Inclusion Criteria9

  • 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 Criteria5

  • 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.

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Interventions

DRUGOsivelotor

Tablets which contain drug substance


Locations(49)

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

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

Houston, Texas, United States

The University of Texas Health Science Center at Houston

Houston, Texas, United States

UT Physicians Comprehensive Sickle Cell Clinic

Houston, Texas, United States

Inova Schar Cancer Institute

Fairfax, Virginia, United States

Hospital Universitario Professor Edgar Santos

Salvador, Estado de Bahia, Brazil

Multihemo Servicos Medicos S/A

Recife, Pernambuco, Brazil

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

Ribeirão Preto, São Paulo, Brazil

Fundacao Faculdade Regional de Medicina de Sao Jose do Rio Preto

São José do Rio Preto, São Paulo, Brazil

BP A Beneficência Portuguesa de São Paulo

São Paulo, São Paulo, Brazil

BP - A Beneficência Portuguesa de São Paulo

São Paulo, São Paulo, Brazil

Casa de Saude Santa Marcelina

São Paulo, São Paulo, Brazil

Casa de Saude Santa Marcelina

São Paulo, São Paulo, Brazil

Esho Empresa de Servicos Hospitalares S A

São Paulo, SÃO Paulo- Brasil, Brazil

Instituto Estadual de Hematologia Arthur Siqueira Cavalcanti - HEMORIO

Rio de Janeiro, Brazil

Esho Empresa de Servicos Hospitalares S A

São Paulo, Brazil

Real e Benemerita Associacao Portuguesa de Sao Paulo

São Paulo, Brazil

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

São Paulo, Brazil

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

São Paulo, Brazil

Nirmal Hospital Pvt Ltd

Surat, Gujarat, India

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

Nashik, Maharashtra, India

KEMRI/CRDR, Siaya, KEMRI Clinical Research Annex

Kisumu, Siaya County, Kenya

Gertrude's Children's Hospital

Nairobi, Kenya

Kenya Medical Research Institute - Centre for Respiratory Disease Research

Nairobi, Kenya

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

Nairobi, Kenya

University College Hospital Ibadan

Ibadan, Oyo/ibadan North, Nigeria

Aminu Kano Teaching Hospital

Kano, Nigeria

Lagos University Teaching Hospital

Lagos, Nigeria

University Hospitals Bristol and Weston NHS Foundation Trust

Bristol, United Kingdom

Bristol Royal Infirmary

Bristol, United Kingdom

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NCT05431088


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