Assess Safety and Efficacy of VAD044 in HHT Patients

Phase 2Active Not RecruitingNCT05406362

Vaderis Therapeutics AG75 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Hereditary Hemorrhagic Telangiectasia (HHT)

Interventions

VAD044 Part IDRUG

capsules to be taken once daily for 12 weeks

VAD044 Part IIDRUG

capsules to be taken once daily for 12 months

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (7)

Massachusetts General Hospital

Boston, Massachusetts, United States

Universitair Ziekenhuis Gent

Ghent, Belgium

Hospices Civils de Lyon

Lyon, France

Ospedale Maggiore di Crema

Crema, Italy

Outcomes

Primary Outcomes

Part I: Safety and Tolerability

Type and severity of Adverse Events (AEs)

Time frame: 12 weeks

Part II: Safety and Tolerability

Type and severity of Adverse Events (AEs)

Time frame: 12 months

Secondary Outcomes

Part I: Change in Epistaxis episodes

The number of Epistaxis episodes

Time frame: 12 weeks

Part I: Change in Epistaxis duration

Epistaxis duration

Time frame: 12 weeks

Part I: Change in Epistaxis intensity

Epistaxis flow intensity

Time frame: 12 weeks

Part I: Change in Epistaxis Severity Score (ESS)

ESS used to evaluate the current severity of HHT patient nosebleeds (typically in the last three months) and can help health care providers to evaluate how a patient is responding to treatment. This score ranges from 0-10 and is automatically calculated after answering six simple questions. The higher the score the more severe.

Time frame: 12 weeks

Part I: Change in Haemoglobin

Haemoglobin

Time frame: 12 weeks

Part I: Change Ferritin

Ferritin

Time frame: 12 weeks

Part I: Change in blood Transferrin saturation level

Data from ClinicalTrials.gov

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