Assess Safety and Efficacy of VAD044 in HHT Patients

Vaderis Therapeutics

Status and phase: Active, not recruiting; Phase 2; Phase 1

Conditions: Hereditary Hemorrhagic Telangiectasia (HHT)

Treatments: Drug: VAD044 Part II; Drug: VAD044 Part I

Study type: Interventional

Funder types: Industry

Identifiers

NCT05406362

VAD044C002

Details and patient eligibility

About

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.

Full 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.

Enrollment: 75 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

For Part I of the study:

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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75 participants in 3 patient groups, including a placebo group

30 mg

Experimental group

Description:

30 mg VAD044

Treatment:

Drug: VAD044 Part I

Drug: VAD044 Part II

40 mg

Experimental group

Description:

40 mg VAD044

Treatment:

Drug: VAD044 Part I

Drug: VAD044 Part II

Placebo

Placebo Comparator group

Description:

Placebo

Treatment:

Drug: VAD044 Part I