A Phase 3 interventional study of Engasertib and Placebo in Hereditary Hemorrhagic Telangiectasia, sponsored by Vaderis Therapeutics AG. Recruiting at 4 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-09-17.
Sponsored by Vaderis Therapeutics AG · Phase 3, Interventional, and Treatment
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.
106 studies on the registry are indexed under Telangiectasia, Hereditary Hemorrhagic; 24 are open to participants now.
This study's planned enrollment of 240 is above the median of 33 across 61 interventional studies indexed under Telangiectasia, Hereditary Hemorrhagic.
Browse Telangiectasia, Hereditary Hemorrhagic studies →Vaderis Therapeutics AG is the lead sponsor of 3 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
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:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants will receive engasertib as an oral capsule QD for 28 weeks.
Drug: Engasertib
Participants will receive matching placebo as an oral capsule QD for 28 weeks.
Drug: Placebo
Engasertib will be administered as oral capsules.
Also known as: VAD044
Placebo will be administered as oral capsules.
Total Number of Epistaxis Events Through Week 28
Time frame: Day 1 through Week 28
Absolute Change from Baseline in Total Duration of Epistaxis at Week 28
Time frame: Baseline and Week 28
Absolute Change from Baseline in Epistaxis Severity Score (ESS) at Week 28
Time frame: Baseline and Week 28
Absolute Change from Baseline in the Nasal Outcome Score for Epistaxis in HHT (NOSE HHT) Score at Week 28
Time frame: Baseline and Week 28
Absolute Change in Red Blood Cell (RBC) Unit Equivalents (RUEs) Received at Week 28
Time frame: Baseline and Week 28
Patient Global Impression of Change (PGIC) Nosebleeds Sub-score at Week 28
Time frame: Week 28
HHT-specific Quality of Life (HHT-QoL) Score at Week 28
Time frame: Week 28
Absolute Change from Baseline in the Intensity-adjusted Duration of Epistaxis per Month at Week 28
Time frame: Baseline and Week 28
Plan to share: No
No publications or documents are linked to this record.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Engasertib
Telangiectasia, Hereditary Hemorrhagic →