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Hereditary Hemorrhagic Telangiectasia (HHT)Phase 1/2NCT05406362

Assess Safety and Efficacy of VAD044 in HHT Patients

A Phase 1/2 study testing VAD044 Part I and VAD044 Part II in people with Hereditary Hemorrhagic Telangiectasia (HHT), run by Vaderis Therapeutics AG at 7 sites in 6 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

Part I: The purpose of this Phase 1b proof of concept study, randomised, placebo controlled, double blind, multicentre study is to asssess safety and efficacy of 2 doses of VAD044 in adult HHT patients. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
VAD044 Part I and VAD044 Part II
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
75 participants
Where
7 sitesin 6 countries
Study dates
18 Jul 2022to Jan 2027
Phase
Phase 1/2
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

  • Diagnosis of HHT by the Curaçao criteria
  • Several epistaxis/week
  • Anaemia
  • COVID-19 vaccination or positive COVID-19 antibody test
  • Patient has given written informed consent to participate in Part I
  • Completion of Part I of the study
  • All adverse events or serious adverse events occuring during Part I of the study have resolved
  • Patient has given written informed consent to participate in Part II

Who cannot join

  • Type 1 diabetes or uncontrolled type II diabetes (insulin or non-insulin dependent)
  • Active COVID-19 infection
  • active uncontrolled infection or known to be serologically positive for HIV, Hep B, Hep C infection
  • Recent procedures on nasal telangiectases (<6 weeks)
  • Requiring therapeutic anticoagulation
  • Use of drugs with anti-angiogenic properties in the past 8 weeks
Show all 9Show fewer
  • laboratory abnormalities
  • Fort Part II of the study:
  • Same as in Part I.

Trial locations and contacts

7 sites in 6 countries.

7 sites in 6 countries

Spain2 sites

  • Hospital Universiati De Bellvitge

    Barcelona

  • Hospital Universitario Ramón y Cajal

    Madrid

Other trials in Spain

United States1 site

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

Other trials in United States

Belgium1 site

  • Universitair Ziekenhuis Gent

    Ghent

Other trials in Belgium

France1 site

  • Hospices Civils de Lyon

    Lyon

Other trials in France

Italy1 site

  • Ospedale Maggiore di Crema

    Crema

Other trials in Italy

Netherlands1 site

  • St. Antonius Hospital

    Nieuwegein

Other trials in Netherlands

About this study

Official title: A Randomised, Placebo Controlled, Double Blind, Multicentre Proof of Concept Study to Assess the Safety and Efficacy of Two Doses of VAD044 in Patients With Hereditary Hemorrhagic Telangiectasia (HHT)

Part I: The purpose of this Phase 1b proof of concept study, randomised, placebo controlled, double blind, multicentre study is to asssess safety and efficacy of 2 doses of VAD044 in adult HHT patients.

Part II: The purpose of this open-label extension following the completion of the randomised double blind treatment and follow-up period (Part I of the study) is to assess the long-term safetty, tolerability and efficacy of VAD044 in adult HHT patients.

Read the detailed description

Part I: After being informed about the study and the potential risks, all patients giving written informed consent will undergo a two months screening and observation period to determine eligibility for study entry. At Day 0, patients who meet the eligibility requirements will be randomized in a double-blind manner (participant and investigator) in a 1:1:1 ratio to 30mg VAD044 (once daily), 40 mg (once daily) or placebo (once daily).

Part II: Patients who have completed the study Part I can participate in the open-label extension study (Part II).The patients can roll over immediately after last visit of the Part I or at any time at their convenience and according to their availability, but within a timeframe no longer than 8 months after the last visit (visit 12) of the part I. All patients in Part II will receive 30 mg of VAD044 once daily for the first 4 weeks afterwards the daily dose can be increased to 40 mg daily for up to 36 months.

Being tested
VAD044 Part I, VAD044 Part II
Keywords
Osler-Weber-Rendu disease, Arteriovenous Malformations
Medical subject headings
Telangiectasia, Hereditary Hemorrhagic, Arteriovenous Malformations

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • VAD044 Part IDrug

    Also called VAD044 L-Tartrate gelatin capsules

    capsules to be taken once daily for 12 weeks

  • VAD044 Part IIDrug

    Also called VAD044 L-Tartrate gelatin capsules

    capsules to be taken once daily for 36 months

Study groups in NCT05406362
Study groupRoleWhat they receive
30 mgExperimental30 mg VAD044
Receives: Drug: VAD044 Part I; Drug: VAD044 Part II
40 mgExperimental40 mg VAD044
Receives: Drug: VAD044 Part I; Drug: VAD044 Part II
PlaceboPlacebo comparatorPlacebo
Receives: Drug: VAD044 Part I

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Part I: Safety and Tolerability

    Type and severity of Adverse Events (AEs)

    Measured: 12 weeks

  2. Part II: Safety and Tolerability

    Type and severity of Adverse Events (AEs)

    Measured: 36 months

Secondary outcomes (27)

  1. Part I: Change in Epistaxis episodes

    The number of Epistaxis episodes

    Measured: 12 weeks

  2. Part I: Change in Epistaxis duration

    Epistaxis duration

    Measured: 12 weeks

  3. Part I: Change in Epistaxis intensity

    Epistaxis flow intensity

    Measured: 12 weeks

  4. Part I: Change in Epistaxis Severity Score (ESS)

    ESS used to evaluate the current severity of HHT patient nosebleeds (typically in the last three months) and can help health care providers to evaluate how a patient is responding to treatment. This score ranges from 0-10 and is automatically calculated after answering six simple questions. The higher the score the more severe.

    Measured: 12 weeks

  5. Part I: Change in Haemoglobin

    Haemoglobin

    Measured: 12 weeks

  6. Part I: Change Ferritin

    Ferritin

    Measured: 12 weeks

  7. Part I: Change in blood Transferrin saturation level

    Transferrin saturation level

    Measured: 12 weeks

  8. Part I: Change in Iron supplementation needs

    Iron supplementation needs

    Measured: 12 weeks

  9. Part I: Change Blood tranfusions requirements

    Blood tranfusions requirements

    Measured: 12 weeks

  10. Part I: Change in the Nasal Outcome for Epistaxis in Hereditary Hemorrhagic Telangiectasia score

    The Nasal Outcome for Epistaxis in Hereditary Hemorrhagic Telangiectasia (NOSE HHT questionnaire) wil be used to measure physical, social and emotional impacts of epistaxis. It is a 29-items questionnaire using a Likert scale. A higher score indicates a worse outcome

    Measured: 12 weeks

  11. Part I: Quality of Life Scale SF-12

    The SF-12 Quality of Life Scale is a scale to evaluate quality of life using 12 questions. In the SF-12, physical (SF12-PCS) and mental (SF12-MCS) component summary scores are calculated as sub-dimensions. The total score of the physical and mental component summary of the scale varies between 0-100. An increase in the score indicates well-being, and a decrease indicates a state of disability.

    Measured: 12 weeks

  12. Part I: Plasma concentration of VAD044

    Plasma concentration of VAD044

    Measured: 12 weeks

  13. Part I: Maximum concentration (Cmax) of VAD04

    Maximum concentration (Cmax) of VAD044

    Measured: 12 weeks

  14. Part I: Time of maximum concentration (Tmax) of VAD044

    Time of maximum concentration (Tmax) of VAD044

    Measured: 12 weeks

  15. Part I: Area under the curve (exposure to drug) during 24 hours (AUC0-24h) of VAD044

    Area under the curve (exposure to drug) during 24 hours (AUC0-24h) of VAD044

    Measured: 12 weeks

  16. Part I: Trough concentration (Ctrough) of VAD044

    Trough concentration (Ctrough) of VAD044

    Measured: 12 weeks

  17. Part I: Pharmacodynamics (PD) of VAD044

    PRP assay to measure the levels of pAKT in the blood in a subset of patients

    Measured: 12 weeks

  18. Part II: Change in The number of Epistaxis episodes

    The number of Epistaxis episodes

    Measured: 24 months

  19. Part II: Change in Epistaxis duration

    Epistaxis duration

    Measured: 24 months

  20. Part II: Change in Epistaxis flow intensity

    Epistaxis flow intensity

    Measured: 24 months

  21. Part II: Change in Epistaxis Severity Score (ESS)

    ESS used to evaluate the current severity of HHT patient nosebleeds (typically in the last three months) and can help health care providers to evaluate how a patient is responding to treatment. This score ranges from 0-10 and is automatically calculated after answering six simple questions. The higher the score the more severe.

    Measured: 24 months

  22. Part II: Change in Haemoglobin

    Haemoglobin

    Measured: 24 months

  23. Part II: Change in Ferritin

    Ferritin

    Measured: 24 months

  24. Part II: Change in Transferrin saturation level

    Transferrin saturation level

    Measured: 24 months

  25. Part II: Change in Iron supplementation needs

    Iron supplementation needs

    Measured: 24 months

  26. Part II: Change in blood tranfusions requirements

    blood tranfusions requirements

    Measured: 24 months

  27. Part II: Quality of Life Scale SF-12

    The SF-12 Quality of Life Scale is a scale to evaluate quality of life using 12 questions. In the SF-12, physical (SF12-PCS) and mental (SF12-MCS) component summary scores are calculated as sub-dimensions. The total score of the physical and mental component summary of the scale varies between 0-100. An increase in the score indicates well-being, and a decrease indicates a state of disability.

    Measured: 24 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 Jun 2022
  2. Study start18 Jul 2022
  3. Primary completionJan 2027 (estimated)
  4. Study completionJan 2027 (estimated)

Resources

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 1/2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
The patients will be randomised into VAD044 30 mg or 40 mg or placebo group in the ratio 1:1:1, according to a centralised randomisation process
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
75 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05406362
Sponsor's protocol ID
VAD044C002
Status last verified
Mar 2026
First submitted
10 May 2022
Last updated
17 Mar 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Al-Samkari H, Hessels J, Riera-Mestre A, Dupuis-Girod S, Van Zele T, Gomez Del Olmo V, Hodges PG, Torres-Iglesias R, Berte R, Saint-Mezard P, Lazar H, Benedict N, Barker D, Bernasconi C, Picard D, Buscarini E, Mager HJ. Engasertib versus Placebo for Bleeding in Hereditary Hemorrhagic Telangiectasia. N Engl J Med. 2025 Nov 27;393(21):2131-2141. doi: 10.1056/NEJMoa2504411.

    PubMed 41297007

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing VAD044 Part I and VAD044 Part II in people with Hereditary Hemorrhagic Telangiectasia (HHT), run by Vaderis Therapeutics AG at 7 sites in 6 countries.

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 8 inclusion and 9 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: under way, not looking for participants, as of the sponsor's last update on 17 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 sites in Spain, United States, Belgium, France, Italy and 1 more country. 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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Part I: Safety and Tolerability (time frame: 12 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05406362, which also lists 1 linked publication.

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 Mar 2026.

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