Assess Safety and Efficacy of VAD044 in HHT Patients

NCT05406362 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: VAD044C002
• Study Type: interventional
• Target: 75
• Locations: 6 countries
• Sites: 7

Brief Summary

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.

Conditions

Hereditary Hemorrhagic Telangiectasia (HHT)

Keywords

Osler-Weber-Rendu disease; Arteriovenous Malformations

Outcome Measures

Primary Outcomes (2)

• Part I: Safety and Tolerability

  Type and severity of Adverse Events (AEs)

  12 weeks

• Part II: Safety and Tolerability

  Type and severity of Adverse Events (AEs)

  36 months

Secondary Outcomes (27)

• Part I: Change in Epistaxis episodes

  12 weeks

• Part I: Change in Epistaxis duration

  12 weeks

• Part I: Change in Epistaxis intensity

  12 weeks

• Part I: Change in Epistaxis Severity Score (ESS)

  12 weeks

• Part I: Change in Haemoglobin

  12 weeks

Study Arms (3)

30 mg

EXPERIMENTAL

30 mg VAD044

Drug: VAD044 Part I; Drug: VAD044 Part II

40 mg

EXPERIMENTAL

40 mg VAD044

Drug: VAD044 Part I; Drug: VAD044 Part II

Placebo

PLACEBO COMPARATOR

Placebo

Drug: VAD044 Part I

Interventions

VAD044 Part I DRUG

capsules to be taken once daily for 12 weeks

Also known as: VAD044 L-Tartrate gelatin capsules

30 mg; 40 mg; Placebo

VAD044 Part II DRUG

capsules to be taken once daily for 36 months

Also known as: VAD044 L-Tartrate gelatin capsules

30 mg; 40 mg

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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:
• 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
• \- Same as in Part I.

Sponsors & Collaborators

• Vaderis Therapeutics AG: lead

Study Sites (7)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Universitair Ziekenhuis Gent

Ghent, Belgium

Location

Hospices Civils de Lyon

Lyon, France

Location

Ospedale Maggiore di Crema

Crema, Italy

Location

St. Antonius Hospital

Nieuwegein, Netherlands

Location

Hospital Universiati De Bellvitge

Barcelona, Spain

Location

Hospital Universitario Ramón y Cajal

Madrid, Spain

Location

Related Publications (1)

• Al-Samkari H, Hessels J, Riera-Mestre A, Dupuis-Girod S, Van Zele T, Gomez Del Olmo V, Hodges PG, Torres-Iglesias R, Berte R, Saint-Mezard P, Lazar H, Benedict N, Barker D, Bernasconi C, Picard D, Buscarini E, Mager HJ. Engasertib versus Placebo for Bleeding in Hereditary Hemorrhagic Telangiectasia. N Engl J Med. 2025 Nov 27;393(21):2131-2141. doi: 10.1056/NEJMoa2504411.

  PMID: 41297007 RESULT

MeSH Terms

Conditions

Telangiectasia, Hereditary Hemorrhagic; Arteriovenous Malformations

Condition Hierarchy (Ancestors)

Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Telangiectasis; Hemorrhagic Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Vascular Malformations; Cardiovascular Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Damien Picard

  Vaderis Therapeutics AG

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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

Study Record Dates

• First Submitted: May 10, 2022
• First Posted: June 6, 2022
• Study Start: July 18, 2022
• Primary Completion (Estimated): January 1, 2027
• Study Completion (Estimated): January 1, 2027
• Last Updated: March 17, 2026

Record last verified: 2026-03

Locations

ES (2); FR (1); IT (1); NL (1); BE (1); US (1)