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.
Participants are ≥18 years of age at the Screening Visit.
Multiple telangiectases at characteristic sites: lips, oral cavity, fingers, or nose;
Visceral lesions: gastrointestinal telangiectasia and/or pulmonary, hepatic, cerebral, or spinal arteriovenous malformations (AVMs); or
A first degree relative with HHT according to these criteria.
History or current diagnosis of clinically significant electrocardiogram (ECG) abnormalities.
History of significant or uncontrolled skin disorders per Investigator's judgement.
Local ablative (eg, cauterization) or surgical procedures on nasal telangiectases <6 weeks before the Screening Visit.
Use of drugs with anti-angiogenic properties, including, but not limited to, bevacizumab, pazopanib, thalidomide, lenalidomide, pomalidomide, tacrolimus, sirolimus, or selective estrogen response modulators (tamoxifen, raloxifene, or bazedoxifene) < 6 weeks before the Screening Visit.
Parallel
Engasertib
DrugEngasertib will be administered as oral capsules.
Placebo
DrugPlacebo will be administered as oral capsules.
Total Number of Epistaxis Events Through Week 28
Absolute Change from Baseline in Total Duration of Epistaxis at Week 28
Absolute Change from Baseline in Epistaxis Severity Score (ESS) at Week 28
Absolute Change from Baseline in the Nasal Outcome Score for Epistaxis in HHT (NOSE HHT) Score at Week 28
Absolute Change in Red Blood Cell (RBC) Unit Equivalents (RUEs) Received at Week 28
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