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Assess Safety and Efficacy of VAD044 in HHT Patients

Sponsor
Vaderis Therapeutics AG (Industry)
Overall Status
Not yet recruiting
CT.gov ID
NCT05406362
Collaborator
(none)
80
Enrollment
6
Locations
3
Arms
30
Anticipated Duration (Months)
13.3
Patients Per Site
0.4
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease Intervention/Treatment Phase
Phase 1

Detailed Description

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)

Study Design

Study Type:
Interventional
Anticipated Enrollment :
80 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Intervention 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 processThe 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 (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose:
Treatment
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)
Anticipated Study Start Date :
Jun 1, 2022
Anticipated Primary Completion Date :
Sep 1, 2024
Anticipated Study Completion Date :
Dec 1, 2024

Arms and Interventions

Arm Intervention/Treatment
Experimental: 30 mg

30 mg VAD044

Drug: VAD044
capsules to be taken once daily for 12 weeks
Other Names:
  • VAD044 L-Tartrate gelatin capsules

    		•
    Experimental: 40 mg

    40 mg VAD044

    Drug: VAD044
    capsules to be taken once daily for 12 weeks
    Other Names:
  • VAD044 L-Tartrate gelatin capsules

    		•
    Placebo Comparator: Placebo

    Placebo

    Drug: VAD044
    capsules to be taken once daily for 12 weeks
    Other Names:
  • VAD044 L-Tartrate gelatin capsules

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Safety and Tolerability [12 weeks]

      Type and severity of Adverse Events (AEs)

    Secondary Outcome Measures

    1. Change in Epistaxis Frequency [12 weeks]

      The number of Epistaxis episodes over four weeks

    2. Pharmacokinetics (PK) of VAD044 [12 weeks]

      Plasma concentration of VAD044

    3. Change in the Nasal Outcome for Epistaxis in Hereditary Hemorrhagic Telangiectasia score [12 weeks]

      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

    4. Quality of Life Scale SF-12 [12 weeks]

      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.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    • diagnosis of HHT by the Curaçao criteria

    • several epistaxis/week

    • anaemia

    • COVID-19 vaccination

    Exclusion Criteria:

    • 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

    • laboratory abnormalities

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Massachusetts General Hospital Boston Massachusetts United States 02114
    2 Washington University School of Medicine Saint Louis Missouri United States 63110
    3 St Michaels Hospital Toronto Canada
    4 Ospedale Maggiore di Crema Crema Italy
    5 St. Antonius Hospital Nieuwegein Netherlands
    6 Hospital Universiati De Bellvitge Barcelona Spain

    Sponsors and Collaborators

    • Vaderis Therapeutics AG

    Investigators

    • Study Director: Damien Picard, Vaderis Therapeutics AG

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Vaderis Therapeutics AG
    ClinicalTrials.gov Identifier:
    NCT05406362
    Other Study ID Numbers:
    • VAD044C002
    First Posted:
    Jun 6, 2022
    Last Update Posted:
    Jun 6, 2022
    Last Verified:
    Jun 1, 2022
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Vaderis Therapeutics AG
    Osler-Weber-Rendu disease
    Arteriovenous Malformations
    Additional relevant MeSH terms:
    Telangiectasis
    Telangiectasia, Hereditary Hemorrhagic

    Study Results

    No Results Posted as of Jun 6, 2022