Assess Safety and Efficacy of VAD044 in HHT Patients

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

  • Hereditary Hemorrhagic Telangiectasia (HHT)

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.