NCT05406362

Assess Safety and Efficacy of VAD044 in HHT Patients

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)

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 75 (actual)
• Sponsor: Vaderis Therapeutics AG · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

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 36 months.

Conditions

• Hereditary Hemorrhagic Telangiectasia (HHT)

Interventions

Type	Name	Description
DRUG	VAD044 Part I	capsules to be taken once daily for 12 weeks
DRUG	VAD044 Part II	capsules to be taken once daily for 36 months

Timeline

• Start date: 2022-07-18
• Primary completion: 2027-01-01
• Completion: 2027-01-01
• First posted: 2022-06-06
• Last updated: 2026-03-17

Locations

7 sites across 6 countries: United States, Belgium, France, Italy, Netherlands, Spain

Regulatory

• FDA-regulated drug study