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A Trial to Assess the Efficacy and Safety of Engasertib in Participants With Moderate to Severe Hereditary Hemorrhagic Telangiectasia (HHT)
Sponsor: Vaderis Therapeutics AG
Summary
The primary objective of this trial is to assess the efficacy of engasertib 40 mg once daily (QD) in reducing the frequency of epistaxis compared to placebo QD during 28 weeks of double-blind treatment in participants with moderate to severe HHT.
Official title: A Phase 3, Randomized, Double-blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Engasertib in Subjects With Moderate to Severe Hereditary Hemorrhagic Telangiectasia (HHT)
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
240
Start Date
2026-09
Completion Date
2028-06
Last Updated
2026-09-17
Healthy Volunteers
No
Conditions
Interventions
Engasertib
Engasertib will be administered as oral capsules.
Placebo
Placebo will be administered as oral capsules.
Locations (4)
The University of Chicago Medical Center (UCMC)
Chicago, Illinois, United States
Innovative Hematology, Inc.,
Indianapolis, Indiana, United States
Mayo Clinic
Rochester, Minnesota, United States
Washington University School of Medicine
St Louis, Missouri, United States