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RecruitingNCT07743671Updated Sep 17, 2026

A Trial to Assess the Efficacy and Safety of Engasertib in Participants With Moderate to Severe Hereditary Hemorrhagic Telangiectasia (HHT)

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

Phase
Phase 3
Study type
Interventional
Enrollment
240
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

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.

02

Conditions studied

  • Hereditary Hemorrhagic Telangiectasia

Keywords

  • Hereditary Hemorrhagic Telangiectasia
  • HHT
  • Engasertib
  • VAD044
03

In context

Telangiectasia, Hereditary Hemorrhagic

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 →

Lead sponsor

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.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

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.

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

Exclusion Criteria:

  1. History or current diagnosis of (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.
Terms in these criteria, in plain language(2)

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.

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.

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.

05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Double (Participant, Investigator)
Enrollment
240 participants (estimated)

Study arms

  • Experimental
    Engasertib 40 mg

    Participants will receive engasertib as an oral capsule QD for 28 weeks.

    Drug: Engasertib

  • Placebo comparator
    Placebo

    Participants will receive matching placebo as an oral capsule QD for 28 weeks.

    Drug: Placebo

Interventions

  • Engasertib will be administered as oral capsules.

    Also known as: VAD044

  • DrugPlacebo

    Placebo will be administered as oral capsules.

06

What researchers measure

Primary outcomes

  1. Total Number of Epistaxis Events Through Week 28

    Time frame: Day 1 through Week 28

Secondary outcomes

  1. Absolute Change from Baseline in Total Duration of Epistaxis at Week 28

    Time frame: Baseline and Week 28

  2. Absolute Change from Baseline in Epistaxis Severity Score (ESS) at Week 28

    Time frame: Baseline and Week 28

  3. 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

  4. Absolute Change in Red Blood Cell (RBC) Unit Equivalents (RUEs) Received at Week 28

    Time frame: Baseline and Week 28

  5. Patient Global Impression of Change (PGIC) Nosebleeds Sub-score at Week 28

    Time frame: Week 28

  6. HHT-specific Quality of Life (HHT-QoL) Score at Week 28

    Time frame: Week 28

  7. Absolute Change from Baseline in the Intensity-adjusted Duration of Epistaxis per Month at Week 28

    Time frame: Baseline and Week 28

07

Study locations

4 of 4 sites recruiting
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Sep 17, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT07743671
Lead sponsor
Vaderis Therapeutics AG
Responsible party
Sponsor
First posted
Aug 4, 2026
Start date
Sep 2026 (estimated)
Primary completion
Jun 2028 (estimated)
Completion
Jun 2028 (estimated)
Last update
Sep 17, 2026

Study contacts

Vaderis Therapeutics AG
Contact
info@vaderis.com
844-823-3747 ext. +1

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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Studies of the drug tested here: Engasertib

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