A Phase 2, Double-blind, Randomized, Placebo-Controlled, Multicenter, Dose- Finding, Efficacy, and Safety Study of Tebapivat in Participants With Sickle Cell Disease
Status: Recruiting
Location: See all (24) 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
RECRUITING
Aurora
Connecticut
UConn Health
RECRUITING
Farmington
Washington, D.c.
MedStar Washington Hospital Center
RECRUITING
Washington D.c.
Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITING
Chicago
Massachusetts
Boston Medical Center & Boston University School of Medicine
RECRUITING
Boston
Michigan
Children's Hospital of Michigan
RECRUITING
Detroit
New York
Icahn School of Medicine at Mt. Sinai
RECRUITING
New York
Montefiore Medical Center
RECRUITING
The Bronx
Pennsylvania
University of Pittsburgh Medical Center
RECRUITING
Pittsburgh
Rhode Island
Lifespan at Rhode Island Hospital
RECRUITING
Providence
South Carolina
Prisma Health Cancer Institute - Farris Road
RECRUITING
Greenville
Texas
University of Texas Health Science Center of Houston
RECRUITING
Houston
Other Locations
Belgium
CHR de la Citadelle
RECRUITING
Liège
Clinique CHC MontLégia
RECRUITING
Liège
Canada
CHU Montreal
RECRUITING
Montreal
McGill University Health Center
RECRUITING
Montreal
Scarborough Health Network - Centenary Hospital
RECRUITING
Scarborough Village
France
CHU Hôpital Henri Mondor
RECRUITING
Créteil
Hôpital Edouard Herriot, CHU de Lyon
RECRUITING
Lyon
Institut Universitaire du Cancer de Toulouse - Oncopole
RECRUITING
Toulouse
Ireland
St. James Hospital
RECRUITING
Dublin
Netherlands
Amsterdam Universitair Medisch Centrum, Locatie AMC
RECRUITING
Amsterdam
Universitair Medisch Centrum Utrecht
RECRUITING
Utrecht
United Kingdom
University College London
RECRUITING
London
Contact Information
Primary
Agios Medical Affairs
medinfo@agios.com
833-228-8474
Time Frame
Start Date: 2025-05-01
Estimated Completion Date: 2027-05
Participants
Target number of participants: 56
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.