Vaderis Therapeutics AG
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.
Inclusion Criteria: 1. Participants are ≥18 years of age at the Screening Visit. 2. Participants have a definite diagnosis of HHT by the Curaçao criteria, defined as spontaneous and recurrent epistaxis and having at least 2 of the following criteria: 1. Multiple telangiectases at characteristic sites: lips, oral cavity, fingers, or nose; 2. Visceral lesions: gastrointestinal telangiectasia and/or pulmonary, hepatic, cerebral, or spinal arteriovenous malformations (AVMs); or 3. A first degree relative with HHT according to these criteria. 3. Participants must have an ESS \>4 at scree…
Engasertib will be administered as oral capsules.
Placebo will be administered as oral capsules.
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