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)

Condition studied

Hereditary Hemorrhagic Telangiectasia (HHT)

Investigational medicinal product(s)

Every trial of Engasertib across our registers →

Eligibility

Primary endpoint

Total number of epistaxis events through Week 28 as assessed by the HHT eDiary

Endpoint detail

Absolute change from baseline in total duration of epistaxis at Week 28 as assessed by the HHTeDiary, Absolute change from baseline in ESS at Week 28, based on symptoms as they have occurred over the last 4 weeks as assessed by the ESS questionnaire, Absolute change from baseline in the NOSE HHT score at Week 28, based on symptoms as they have occurred over the past 2 weeks as assessed by the NOSE HHT questionnaire, Absolute change in red blood cell (RBC) unit equivalents (RUEs) received at Week 28