NCT07743671 · Vaderis Therapeutics AG
A Trial to Assess the Efficacy and Safety of Engasertib in Participants With Moderate to Severe Hereditary Hemorrhagic Telangiectasia (HHT)
What this study is about
The primary objective of this trial is to assess the effectiveness of engasertib 40 mg once daily (QD) in reducing the frequency of epistaxis compared to placebo QD during 28 weeks of where neither patients nor doctors know which treatment is given treatment in participants with moderate to severe HHT.
View original scientific description
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.
Interventions
DRUG
Engasertib
Engasertib will be administered as oral capsules.
DRUG
Placebo
Placebo will be administered as oral capsules.
Primary outcome measures
Total Number of Epistaxis Events Through Week 28
Time frame: Day 1 through Week 28
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Participants are ≥18 years of age at the Screening Visit.
- 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:
- 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.
- 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.
- 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
- 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.
- 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
- Chicago, Illinois
- Indianapolis, Indiana
- Rochester, Minnesota
- St Louis, Missouri
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Sep 17, 2026 · Source of record for eligibility and locations