Assess Safety and Efficacy of VAD044 in HHT Patients

Purpose

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

  • Hereditary Hemorrhagic Telangiectasia (HHT)

Eligibility

Eligible Ages
Over 18 Years
Eligible Genders
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

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

Study Design

Phase
Phase 1
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
    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

Recruiting Locations

Massachusetts General Hospital
Boston, Massachusetts 02114
Contact:
Hedvika Lazar
hlazar@vaderis.com

More Details

Status
Recruiting
Sponsor
Vaderis Therapeutics AG

Study Contact

Hedvika Lazar
+41763217280
hlazar@vaderis.com

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)