A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD)
NCT ID: NCT06924970
Last Updated: 2025-12-26
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
56 participants
INTERVENTIONAL
2025-05-01
2027-05-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Tebapivat 2.5 milligrams (mg)
Participants will receive 2.5 mg tebapivat orally, once daily (QD) for 12-weeks in the double-blind (DB) period. Participants who complete the DB Period will be eligible to receive the same dose in the Open-Label Extension (OLE) period for up to 52 weeks.
Tebapivat
Oral tablets.
Tebapivat 5.0 mg
Participants will receive 5.0 mg tebapivat orally, QD, for 12-weeks in the DB period. Participants who complete the DB Period will be eligible to receive the same dose in the OLE period for up to 52 weeks.
Tebapivat
Oral tablets.
Tebapivat 7.5 mg
Participants will receive 7.5 mg tebapivat orally, QD, for 12-weeks in the DB period. Participants who complete the DB Period will be eligible to receive the same dose in the OLE period for up to 52 weeks.
Tebapivat
Oral tablets.
Tebapivat Matched Placebo
Participants will receive a matched placebo, orally, QD, for 12-weeks in the DB period. Participants who complete the DB Period will be randomized in 1:1:1 to receive tebapivat 2.5 mg QD, tebapivat 5.0 mg QD, or tebapivat 7.5 mg QD in the OLE period for up to 52 weeks
Tebapivat
Oral tablets.
Tebapivat Matched Placebo
Oral tablets.
Interventions
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Tebapivat
Oral tablets.
Tebapivat Matched Placebo
Oral tablets.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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.
Exclusion Criteria
* \>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.
16 Years
ALL
No
Sponsors
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Agios Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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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
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
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
Kings College Hospital NHS Foundation Trust
London, , United Kingdom
University College London
London, , United Kingdom
Countries
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Central Contacts
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Other Identifiers
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2024-519746-70-01
Identifier Type: CTIS
Identifier Source: secondary_id
AG946-C-003
Identifier Type: -
Identifier Source: org_study_id