Assess Safety and Efficacy of VAD044 in HHT Patients

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

75 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-07-18

Study Completion Date

2025-12-31

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Hereditary Hemorrhagic Telangiectasia (HHT)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

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 Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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30 mg

30 mg VAD044

Group Type EXPERIMENTAL

VAD044 Part I

Intervention Type DRUG

capsules to be taken once daily for 12 weeks

VAD044 Part II

Intervention Type DRUG

capsules to be taken once daily for 12 months

40 mg

40 mg VAD044

Group Type EXPERIMENTAL

VAD044 Part I

Intervention Type DRUG

capsules to be taken once daily for 12 weeks

VAD044 Part II

Intervention Type DRUG

capsules to be taken once daily for 12 months

Placebo

Group Type PLACEBO_COMPARATOR

VAD044 Part I

Intervention Type DRUG

capsules to be taken once daily for 12 weeks

Interventions

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VAD044 Part I

capsules to be taken once daily for 12 weeks

Intervention Type DRUG

VAD044 Part II

capsules to be taken once daily for 12 months

Intervention Type DRUG

Other Intervention Names

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VAD044 L-Tartrate gelatin capsules VAD044 L-Tartrate gelatin capsules

Eligibility Criteria

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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

* 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

* 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:

\- Same as in Part I.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No