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Clinical Trials in the UK / NCT06924970
Active, not recruiting Phase 2

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

NCT06924970 · tracked via the Priya Life Science UK tracker
Sponsor
Agios Pharmaceuticals, Inc.
Phase
Phase 2
Started
2025-05-01
Last updated
2026-08-20

Condition(s) studied

Sickle Cell Disease

Investigational drug(s) / intervention(s)

TebapivatTebapivat Matched Placebo

Tebapivat: Oral tablets.

Tebapivat Matched Placebo: Oral tablets.

Study 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.

Eligibility

Sex
ALL
Min age
16 Years
Max age
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (20)

FacilityCityRegionStatus
UConn Health Farmington Connecticut
MedStar Washington Hospital Center Washington D.C. District of Columbia
Emory-Children's Center/ Children's Healthcare of Atlanta: Arthur M. Blank Hospital Atlanta Georgia
Henry Ford Health System Detroit Michigan
Children's Hospital of Michigan Detroit Michigan
Icahn School of Medicine at Mt. Sinai New York New York
University of Pittsburgh Medical Center Pittsburgh Pennsylvania
Prisma Health Cancer Institute - Farris Road Greenville South Carolina
University of Texas Health Science Center of Houston Houston Texas
Fred Hutchinson Cancer Center, University of Washington Seattle Washington
CHR de la Citadelle Liège Wallonne
CHU Montreal Montreal Quebec
Hôpital Edouard Herriot, CHU de Lyon Lyon Auvergne-Rhône-Alpes
Institut Universitaire du Cancer de Toulouse - Oncopole Toulouse Midi Pyrenees
CHU Hôpital Henri Mondor Créteil Île-de-France Region
St. James Hospital Dublin Leinster
Amsterdam Universitair Medisch Centrum, Locatie AMC Amsterdam North Holland
Erasmus MC Rotterdam South Holland
Universitair Medisch Centrum Utrecht Utrecht Netherlands
University College London London United Kingdom

More Agios Pharmaceuticals, Inc. trials in the UK

Other trials for the same condition

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 NCT06924970 on ClinicalTrials.gov ↗ ← All trials in the UK