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Purpose

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.

Condition

Eligibility

Eligible Ages
Over 18 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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 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 study have resolved - Patient has given written informed consent to participate in Part II Exclusion Criteria: - Same as in Part I.

Study Design

Phase
Phase 1/Phase 2
Study Type
Interventional
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 process
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
30 mg 		30 mg VAD044
  • Drug: VAD044 Part I
    capsules to be taken once daily for 12 weeks
    Other names:
    • VAD044 L-Tartrate gelatin capsules
  • Drug: VAD044 Part II
    capsules to be taken once daily for 12 months
    Other names:
    • VAD044 L-Tartrate gelatin capsules
Experimental
40 mg 		40 mg VAD044
  • Drug: VAD044 Part I
    capsules to be taken once daily for 12 weeks
    Other names:
    • VAD044 L-Tartrate gelatin capsules
  • Drug: VAD044 Part II
    capsules to be taken once daily for 12 months
    Other names:
    • VAD044 L-Tartrate gelatin capsules
Placebo Comparator
Placebo 		Placebo
  • Drug: VAD044 Part I
    capsules to be taken once daily for 12 weeks
    Other names:
    • VAD044 L-Tartrate gelatin capsules

More Details

Status
Active, not recruiting
Sponsor
Vaderis Therapeutics AG

Study Contact

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 12 months.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.