Hereditary Hemorrhagic TelangiectasiaPhase 3NCT07743671
A Trial to Assess the Efficacy and Safety of Engasertib in Participants With Moderate to Severe Hereditary Hemorrhagic Telangiectasia (HHT)
A Phase 3 study testing Engasertib in people with Hereditary Hemorrhagic Telangiectasia, run by Vaderis Therapeutics AG at 4 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hereditary Hemorrhagic Telangiectasia
- What is being tested?
- Engasertib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 240 participants
- Where
- 4 sitesUnited States
- Study dates
- Sep 2026to Jun 2028
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States4 sites, 4 recruiting
- Recruiting
The University of Chicago Medical Center (UCMC)
Chicago, Illinois, 60637
Kenneth Cohen, MD, 773-702-4043, kcohen1@uchicagomedicine.org
- Recruiting
Innovative Hematology, Inc.,
Indianapolis, Indiana, 46260
Magdalena Lewandowska, MD, 317-871-0011 ext. 1236, mlewandowska@inheme.org
- Recruiting
Mayo Clinic
Rochester, Minnesota, 55905
Vivek N. Iyer, MD, MPH, 507-284-2158, iyer.vivek@mayo.edu
- Recruiting
Washington University School of Medicine
St Louis, Missouri, 63110
Murali Mohan Chakinala, MD, 314-454-8764, chakinalam@wustl.edu
About this study
Official title: A Phase 3, Randomized, Double-blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Engasertib in Subjects With Moderate to Severe Hereditary Hemorrhagic Telangiectasia (HHT)
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.
- Conditions
- Hereditary Hemorrhagic Telangiectasia
- Being tested
- Engasertib, Placebo
- Keywords
- Hereditary Hemorrhagic Telangiectasia, HHT, Engasertib, VAD044
- Medical subject headings
- Telangiectasia, Hereditary Hemorrhagic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EngasertibDrug
Also called VAD044
Engasertib will be administered as oral capsules.
PlaceboDrug
Placebo will be administered as oral capsules.
| Study group | Role | What they receive |
|---|---|---|
| Engasertib 40 mg | Experimental | Participants will receive engasertib as an oral capsule QD for 28 weeks. Receives: Drug: Engasertib |
| Placebo | Placebo comparator | Participants will receive matching placebo as an oral capsule QD for 28 weeks. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Total Number of Epistaxis Events Through Week 28
Measured: Day 1 through Week 28
Secondary outcomes (7)
Absolute Change from Baseline in Total Duration of Epistaxis at Week 28
Measured: Baseline and Week 28
Absolute Change from Baseline in Epistaxis Severity Score (ESS) at Week 28
Measured: Baseline and Week 28
Absolute Change from Baseline in the Nasal Outcome Score for Epistaxis in HHT (NOSE HHT) Score at Week 28
Measured: Baseline and Week 28
Absolute Change in Red Blood Cell (RBC) Unit Equivalents (RUEs) Received at Week 28
Measured: Baseline and Week 28
Patient Global Impression of Change (PGIC) Nosebleeds Sub-score at Week 28
Measured: Week 28
HHT-specific Quality of Life (HHT-QoL) Score at Week 28
Measured: Week 28
Absolute Change from Baseline in the Intensity-adjusted Duration of Epistaxis per Month at Week 28
Measured: Baseline and Week 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Aug 2026
- Study startSep 2026 (estimated)
- Primary completionJun 2028 (estimated)
- Study completionJun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Hemorrhagic TelangiectasiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 240 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Vaderis Therapeutics AG
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07743671
- Sponsor's protocol ID
- HHT-301
- Other IDs
- 2025-524490-17-00, 2025
- Status last verified
- Sep 2026
- First submitted
- 29 Jul 2026
- Last updated
- 17 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Randomized, Double-blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Engasertib in Subjects With Moderate to Severe Hereditary Hemorrhagic Telangiectasia (HHT)
- ClinicalTrials.gov ID
- NCT07743671
- Acronym / sponsor ID
- HHT-301
- Conditions
- Hereditary Hemorrhagic Telangiectasia
- Keywords
- Hereditary Hemorrhagic Telangiectasia, HHT, Engasertib, VAD044
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 240 participants (estimated)
- Lead sponsor
- Vaderis Therapeutics AGIndustry (pharma, biotech, devices)
- Study start
- Sep 2026 (estimated)
- Primary completion
- Jun 2028 (estimated)
- Study completion
- Jun 2028 (estimated)
- First posted
- 4 Aug 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Engasertib 40 mg | Experimental | Drug: EngasertibParticipants will receive engasertib as an oral capsule QD for 28 weeks. |
| Placebo | Placebo comparator | Drug: PlaceboParticipants will receive matching placebo as an oral capsule QD for 28 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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:
- 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.
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EngasertibAlso: VAD044Engasertib will be administered as oral capsules. | Drug | Engasertib 40 mg |
| PlaceboPlacebo will be administered as oral capsules. | Drug | Placebo |
Primary outcome
Total Number of Epistaxis Events Through Week 28
Time frame: Day 1 through Week 28
7 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Engasertib in people with Hereditary Hemorrhagic Telangiectasia, run by Vaderis Therapeutics AG at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 17 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Vaderis Therapeutics AG (844-823-3747 ext. +1, info@vaderis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Total Number of Epistaxis Events Through Week 28 (time frame: Day 1 through Week 28). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Sep 2026.