Assess Safety and Efficacy of VAD044 in HHT Patients

Vaderis Therapeutics

Status and phase: Enrolling; Phase 1

Conditions: Hereditary Hemorrhagic Telangiectasia (HHT)

Treatments: Drug: VAD044

Study type: Interventional

Funder types: Industry

Identifiers

NCT05406362

VAD044C002

Details and patient eligibility

About

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

Full 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)

Enrollment: 80 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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80 participants in 3 patient groups, including a placebo group

30 mg

Experimental group

Description:

30 mg VAD044

Treatment:

Drug: VAD044

40 mg

Experimental group

Description:

40 mg VAD044

Treatment:

Drug: VAD044

Placebo

Placebo Comparator group

Description:

Placebo

Treatment:

Drug: VAD044