Engasertib for moderate to severe HHT: see if daily 40 mg helps with nosebleeds

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a Phase 3, randomized, double-blind study evaluating the efficacy and safety of engasertib 40 mg once daily versus placebo in adults with moderate to severe hereditary hemorrhagic telangiectasia (HHT). Participants will be treated for 28 weeks to determine if engasertib can decrease epistaxis frequency. Eligibility requires confirmed HHT by Curaçao criteria, regular epistaxis, and anemia or recent iron therapy, among other criteria. Primary outcome focuses on reduction of nosebleed events; safety will also be monitored. The study is sponsored by Vaderis Therapeutics AG with sites at UCMC, Innovative Hematology, Mayo Clinic, and collaborating centers.

Why it matters

Who can join: the full rules

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.

Where it's running

• The University of Chicago Medical Center (UCMC)
  Chicago, Illinois, United States Recruiting
• Innovative Hematology, Inc.,
  Indianapolis, Indiana, United States Recruiting
• Mayo Clinic
  Rochester, Minnesota, United States Recruiting
• Washington University School of Medicine
  St Louis, Missouri, United States Recruiting

Study contacts

• Study coordinator: Vaderis Therapeutics AG
• Email: info@vaderis.com
• Phone: 844-823-3747

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Examining the role of hypoxia in hereditary hemorrhagic telangiectasia

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• Using nintedanib to treat nosebleeds in patients with hereditary hemorrhagic telangiectasia

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.