NCT07743671PHASE3Recruiting
A Trial to Assess the Efficacy and Safety of Engasertib in Participants With Moderate to Severe Hereditary Hemorrhagic Telangiectasia (HHT)
Vaderis Therapeutics AG
Start Date
9/1/2026
Completion Date
6/1/2028
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.
Eligibility Criteria
Age Range: 18 years to No maximum
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 screening, and, in the judgement of the Investigator, participants are expected to have regular epistaxis that typically lasts for several minutes. This criterion is assessed at screening only and does not require reconfirmation prior to randomization on Day 1.
4. Participants have anemia OR in the prior 6 months have received a parenteral infusion of at least 250 mg of iron OR in the prior 6 months have received a red cell or whole blood transfusion.
Exclusion Criteria:
1. History or current diagnosis of clinically significant electrocardiogram (ECG) abnormalities.
2. History of significant or uncontrolled skin disorders per Investigator's judgement.
3. Local ablative (eg, cauterization) or surgical procedures on nasal telangiectases \<6 weeks before the Screening Visit.
4. 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.
5. Use of oral tranexamic or epsilon-aminocaproic acid unless they are on a stable dose for at least 4 weeks before the Screening Visit, which will need to be continued during the entire duration of the double-blind Treatment Period.
Interventions
DRUG
Engasertib
DRUG
Placebo
Conditions
Hereditary Hemorrhagic Telangiectasia
Locations
The University of Chicago Medical Center (UCMC)
Chicago, Illinois 60637
United States
Innovative Hematology, Inc.,
Indianapolis, Indiana 46260
United States
Mayo Clinic
Rochester, Minnesota 55905
United States
Washington University School of Medicine
St Louis, Missouri 63110
United States