Assess Safety and Efficacy of VAD044 in HHT Patients

Hereditary Hemorrhagic Telangiectasia (HHT) Clinical Trial

Official title:

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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05406362
• Other study ID #: VAD044C002
• Status: Recruiting
• Phase: Phase 1
• Start date: July 18, 2022
• Est. completion date: May 2024

Study information

• Verified date: May 2023
• Source: Vaderis Therapeutics AG
• Contact: Hedvika Lazar
• Phone: +41763217280
• Email: hlazar[@]vaderis.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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)

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 80
• Est. completion date: May 2024
• Est. primary completion date: March 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Hereditary Hemorrhagic Telangiectasia (HHT)
• Telangiectasia, Hereditary Hemorrhagic
• Telangiectasis

Intervention

Drug:
• VAD044
capsules to be taken once daily for 12 weeks

Locations

Country	Name	City	State
Belgium	Universitair Ziekenhuis Gent	Gent
France	Hospices Civils de Lyon	Lyon
Italy	Ospedale Maggiore di Crema	Crema
Netherlands	St. Antonius Hospital	Nieuwegein
Spain	Hospital Universiati De Bellvitge	Barcelona
Spain	Hospital Universitario Ramón y Cajal	Madrid
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	Washington University School of Medicine	Saint Louis	Missouri

Sponsors (1)

Lead Sponsor	Collaborator
Vaderis Therapeutics AG

Countries where clinical trial is conducted

United States,  Belgium,  France,  Italy,  Netherlands,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety and Tolerability	Type and severity of Adverse Events (AEs)	12 weeks
Secondary	Change in Epistaxis Frequency	The number of Epistaxis episodes over four weeks	12 weeks
Secondary	Pharmacokinetics (PK) of VAD044	Plasma concentration of VAD044	12 weeks
Secondary	Change in the Nasal Outcome for Epistaxis in Hereditary Hemorrhagic Telangiectasia score	The Nasal Outcome for Epistaxis in Hereditary Hemorrhagic Telangiectasia (NOSE HHT questionnaire) wil be used to measure physical, social and emotional impacts of epistaxis. It is a 29-items questionnaire using a Likert scale. A higher score indicates a worse outcome	12 weeks
Secondary	Quality of Life Scale SF-12	The SF-12 Quality of Life Scale is a scale to evaluate quality of life using 12 questions. In the SF-12, physical (SF12-PCS) and mental (SF12-MCS) component summary scores are calculated as sub-dimensions. The total score of the physical and mental component summary of the scale varies between 0-100. An increase in the score indicates well-being, and a decrease indicates a state of disability.	12 weeks