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 (8) 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
Connecticut
UConn Health
RECRUITING
Farmington
Washington, D.c.
MedStar Washington Hospital Center
RECRUITING
Washington D.c.
New York
Icahn School of Medicine at Mt. Sinai
RECRUITING
New York
South Carolina
Prisma Health Cancer Institute - Farris Road
RECRUITING
Greenville
Texas
University of Texas Health Science Center of Houston
RECRUITING
Houston
Other Locations
Belgium
Clinique CHC MontLégia
RECRUITING
Liège
Canada
CHU Montreal
RECRUITING
Montreal
Ireland
St. James Hospital
RECRUITING
Dublin
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