A Phase 2, Double-blind, Randomized, Placebo-Controlled, Multicenter, Dose- Finding, Efficacy, and Safety Study of Tebapivat in Participants With Sickle Cell Disease
Status: Active_not_recruiting
Location: See all (30) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 16
Healthy Volunteers: f
View:
• 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.
Locations
United States
Colorado
UCHealth at University of Colorado Anschutz Medical Campus
Aurora
Connecticut
UConn Health
Farmington
Washington, D.c.
Children's National Hospital
Washington D.c.
MedStar Washington Hospital Center
Washington D.c.
Georgia
Emory-Children's Center/ Children's Healthcare of Atlanta: Arthur M. Blank Hospital
Atlanta
Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago
Massachusetts
Boston Medical Center & Boston University School of Medicine
Boston
Michigan
Children's Hospital of Michigan
Detroit
Henry Ford Health System
Detroit
New York
Icahn School of Medicine at Mt. Sinai
New York
Montefiore Medical Center
The Bronx
Pennsylvania
Thomas Jefferson University Hospital
Philadelphia
University of Pittsburgh Medical Center
Pittsburgh
Rhode Island
Lifespan at Rhode Island Hospital
Providence
South Carolina
Prisma Health Cancer Institute - Farris Road
Greenville
Texas
University of Texas Health Science Center of Houston
Houston
Washington
Fred Hutchinson Cancer Center, University of Washington
Seattle
Other Locations
Belgium
CHR de la Citadelle
Liège
Clinique CHC MontLégia
Liège
Canada
CHU Montreal
Montreal
France
CHU Hôpital Henri Mondor
Créteil
Hôpital Edouard Herriot, CHU de Lyon
Lyon
Institut Universitaire du Cancer de Toulouse - Oncopole
Toulouse
Ireland
St. James Hospital
Dublin
Netherlands
Amsterdam Universitair Medisch Centrum, Locatie AMC
Amsterdam
Erasmus MC
Rotterdam
Universitair Medisch Centrum Utrecht
Utrecht
United Kingdom
Evelina London Children's Hospital, Guy's and St. Thomas' NHS Foundation Trust
London
Kings College Hospital NHS Foundation Trust
London
University College London
London
Time Frame
Start Date: 2025-05-01
Completion Date: 2027-05
Participants
Target number of participants: 59
Treatments
Experimental: 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.
Experimental: 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.
Experimental: 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.
Placebo_comparator: 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
Related Therapeutic Areas
Sponsors
Leads: Agios Pharmaceuticals, Inc.