Assess Safety and Efficacy of VAD044 in HHT Patients

Last updated: February 27, 2025
Sponsor: Vaderis Therapeutics AG
Overall Status: Active - Not Recruiting

Phase

1/2

Condition

N/A

Treatment

VAD044 Part I

VAD044 Part II

VAD044

Clinical Study ID

NCT05406362
VAD044C002
  • Ages > 18
  • All Genders

Study 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.

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.

Eligibility Criteria

Inclusion

For Part I of the study:

Inclusion Criteria:

  • 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

Exclusion

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

Fort Part II of the study:

Inclusion Criteria:

  • Completion of Part I of the study

  • All adverse events or serious adverse events occuring during Part I of the studyhave resolved

  • Patient has given written informed consent to participate in Part II

Exclusion Criteria:

  • Same as in Part I.

Study Design

Total Participants: 75
Treatment Group(s): 3
Primary Treatment: VAD044 Part I
Phase: 1/2
Study Start date:
July 18, 2022
Estimated Completion Date:
December 31, 2025

Study 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 12 months.

Connect with a study center

  • Universitair Ziekenhuis Gent

    Gent,
    Belgium

    Site Not Available

  • St Michaels Hospital

    Toronto,
    Canada

    Site Not Available

  • Hospices Civils de Lyon

    Lyon,
    France

    Site Not Available

  • Ospedale Maggiore di Crema

    Crema,
    Italy

    Site Not Available

  • St. Antonius Hospital

    Nieuwegein,
    Netherlands

    Site Not Available

  • Hospital Universiati De Bellvitge

    Barcelona,
    Spain

    Site Not Available

  • Hospital Universitario Ramón y Cajal

    Madrid,
    Spain

    Site Not Available

  • Massachusetts General Hospital

    Boston, Massachusetts 02114
    United States

    Site Not Available

  • Washington University School of Medicine

    Saint Louis, Missouri 63110
    United States

    Site Not Available

Map preview placeholder

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.