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RECRUITING

NCT06924970

PHASE2

A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD)

Sponsor: Agios Pharmaceuticals, Inc.

View on ClinicalTrials.gov

Summary

The main purpose of this study is to compare the effect of tebapivat versus placebo on anemia and to detect a dose-response for hemoglobin (Hb) response in participants with SCD.

Official title: A Phase 2, Double-blind, Randomized, Placebo-Controlled, Multicenter, Dose- Finding, Efficacy, and Safety Study of Tebapivat in Participants With Sickle Cell Disease

Key Details

Gender

All

Age Range

16 Years - Any

Study Type

INTERVENTIONAL

Enrollment

Start Date

2025-05-01

Completion Date

2027-05

Last Updated

2026-03-30

Healthy Volunteers

Conditions

Sickle Cell Disease

Interventions

DRUG

Tebapivat

Oral tablets.

DRUG

Tebapivat Matched Placebo

Oral tablets.

Key Inclusion Criteria: * Documented diagnosis of SCD (HbSS, HbSC \[combined heterozygosity for hemoglobins S and C\], sickle hemoglobin \[HbS\]/β0-thalassemia, HbS/β+-thalassemia, or other sickle cell syndrome variants). * Hemoglobin ≥5.5 and ≤10.5 grams per decilitre (g/dL). Hemoglobin concentration must be based on an average of at least 2 Hb concentration measurements (separated by ≥7 days) collected during the screening period. * If taking hydroxyurea, the hydroxyurea dose must be stable for at least 90 days before randomization. Discontinuation of hydroxyurea requires a 90-day washout before providing informed consent. Key Exclusion Criteria: * Receiving regularly scheduled red blood cell (RBC) transfusion therapy (also termed chronic, prophylactic, or preventative transfusion); episodic transfusion in response to worsened anemia or vaso-occlusive crisis (VOC) is permitted. Additionally, a participant who requires episodic transfusion(s) may not have received a transfusion(s) within 60 days before providing informed consent or during the screening period. * \>10 sickle cell pain crisis (SCPCs) in the 12 months before providing informed consent. * Receiving anabolic steroids that have not been stopped for at least 4 weeks before randomization. Testosterone replacement therapy to treat hypogonadism is allowed; the testosterone dose and preparation must be stable for ≥10 weeks before randomization. * Hospitalized for an SCPC and/or other vaso-occlusive event within 14 days before providing informed consent or within 14 days before randomization. If an SCPC occurs during the screening period, the screening period may be extended with Medical Monitor approval. * Receiving treatment with voxelotor, crizanlizumab, or L-glutamine within 90 days before randomization. * Platelet count \<lower limit of normal (LLN) for the local laboratory or \<150×109/liter (L) (whichever is lower) during screening. Platelet transfusions received within 28 days before consent or during screening. * Receiving treatment with hematopoietic stimulating agents within 90 days before randomization. * Prior exposure to gene therapy or prior bone marrow or stem cell transplantation, including any conditioning regimen.

Locations (32)

UCHealth at University of Colorado Anschutz Medical Campus

Aurora, Colorado, United States

UConn Health

Farmington, Connecticut, United States

Children's National Hospital

Washington D.C., District of Columbia, United States

MedStar Washington Hospital Center

Washington D.C., District of Columbia, United States

Emory-Children's Center/ Children's Healthcare of Atlanta: Arthur M. Blank Hospital

Atlanta, Georgia, United States

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Boston Medical Center & Boston University School of Medicine

Boston, Massachusetts, United States

Henry Ford Health System

Detroit, Michigan, United States

Children's Hospital of Michigan

Detroit, Michigan, United States

Icahn School of Medicine at Mt. Sinai

New York, New York, United States

Montefiore Medical Center

The Bronx, New York, United States

Thomas Jefferson University Hospital

Philadelphia, Pennsylvania, United States

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, United States

Lifespan at Rhode Island Hospital

Providence, Rhode Island, United States

Prisma Health Cancer Institute - Farris Road

Greenville, South Carolina, United States

University of Texas Health Science Center of Houston

Houston, Texas, United States

Fred Hutchinson Cancer Center, University of Washington

Seattle, Washington, United States

CHR de la Citadelle

Liège, Wallonne, Belgium

Clinique CHC MontLégia

Liège, Wallonne, Belgium

Scarborough Health Network - Centenary Hospital

Scarborough Village, Ontario, Canada

CHU Montreal

Montreal, Quebec, Canada

McGill University Health Center

Montreal, Quebec, Canada

Hôpital Edouard Herriot, CHU de Lyon

Lyon, Auvergne-Rhône-Alpes, France

Institut Universitaire du Cancer de Toulouse - Oncopole

Toulouse, Midi Pyrenees, France

CHU Hôpital Henri Mondor

Créteil, Île-de-France Region, France

St. James Hospital

Dublin, Leinster, Ireland

Amsterdam Universitair Medisch Centrum, Locatie AMC

Amsterdam, North Holland, Netherlands

Erasmus MC

Rotterdam, South Holland, Netherlands

Universitair Medisch Centrum Utrecht

Utrecht, Netherlands

Evelina London Children's Hospital, Guy's and St. Thomas' NHS Foundation Trust

London, United Kingdom

Kings College Hospital NHS Foundation Trust

London, United Kingdom

University College London

London, United Kingdom

Clinical Research Directory

A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD)

Summary

Key Details

Conditions

Interventions

Eligibility Criteria

Locations (32)