Engasertib: Engasertib will be administered as oral capsules.
Placebo: Placebo will be administered as oral capsules.
Study 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
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
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.
Primary outcome measure(s)
Total Number of Epistaxis Events Through Week 28 — Day 1 through Week 28
Trial sites (4)
Facility
City
Region
Status
The University of Chicago Medical Center (UCMC)
Chicago
Illinois
Recruiting
Innovative Hematology, Inc.,
Indianapolis
Indiana
Recruiting
Mayo Clinic
Rochester
Minnesota
Recruiting
Washington University School of Medicine
St Louis
Missouri
Recruiting
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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