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Assess Safety and Efficacy of VAD044 in HHT Patients

Primary Purpose

Hereditary Hemorrhagic Telangiectasia (HHT)

Status
Recruiting
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
VAD044
Sponsored by
Vaderis Therapeutics AG
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hereditary Hemorrhagic Telangiectasia (HHT) focused on measuring Osler-Weber-Rendu disease, Arteriovenous Malformations

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • diagnosis of HHT by the Curaçao criteria
  • several epistaxis/week
  • anaemia
  • COVID-19 vaccination

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

Sites / Locations

  • Massachusetts General HospitalRecruiting
  • Washington University School of MedicineRecruiting
  • Universitair Ziekenhuis GentRecruiting
  • Hospices Civils de LyonRecruiting
  • Ospedale Maggiore di CremaRecruiting
  • St. Antonius HospitalRecruiting
  • Hospital Universiati De BellvitgeRecruiting
  • Hospital Universitario Ramón y CajalRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Placebo Comparator

Arm Label

30 mg

40 mg

Placebo

Arm Description

30 mg VAD044

40 mg VAD044

Placebo

Outcomes

Primary Outcome Measures

Safety and Tolerability
Type and severity of Adverse Events (AEs)

Secondary Outcome Measures

Change in Epistaxis Frequency
The number of Epistaxis episodes over four weeks
Pharmacokinetics (PK) of VAD044
Plasma concentration of VAD044
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
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.

Full Information

First Posted
May 10, 2022
Last Updated
May 3, 2023
Sponsor
Vaderis Therapeutics AG
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1. Study Identification

Unique Protocol Identification Number
NCT05406362
Brief Title
Assess Safety and Efficacy of VAD044 in HHT Patients
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)
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Recruiting
Study Start Date
July 18, 2022 (Actual)
Primary Completion Date
March 2024 (Anticipated)
Study Completion Date
May 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vaderis Therapeutics AG

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief 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
Detailed 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)

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Hemorrhagic Telangiectasia (HHT)
Keywords
Osler-Weber-Rendu disease, Arteriovenous Malformations

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Model Description
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
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
80 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
30 mg
Arm Type
Experimental
Arm Description
30 mg VAD044
Arm Title
40 mg
Arm Type
Experimental
Arm Description
40 mg VAD044
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo
Intervention Type
Drug
Intervention Name(s)
VAD044
Other Intervention Name(s)
VAD044 L-Tartrate gelatin capsules
Intervention Description
capsules to be taken once daily for 12 weeks
Primary Outcome Measure Information:
Title
Safety and Tolerability
Description
Type and severity of Adverse Events (AEs)
Time Frame
12 weeks
Secondary Outcome Measure Information:
Title
Change in Epistaxis Frequency
Description
The number of Epistaxis episodes over four weeks
Time Frame
12 weeks
Title
Pharmacokinetics (PK) of VAD044
Description
Plasma concentration of VAD044
Time Frame
12 weeks
Title
Change in the Nasal Outcome for Epistaxis in Hereditary Hemorrhagic Telangiectasia score
Description
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
Time Frame
12 weeks
Title
Quality of Life Scale SF-12
Description
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.
Time Frame
12 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Hedvika Lazar
Phone
+41763217280
Email
hlazar@vaderis.com
First Name & Middle Initial & Last Name or Official Title & Degree
Damien Picard
Email
dpicard@vaderis.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Damien Picard
Organizational Affiliation
Vaderis Therapeutics AG
Official's Role
Study Director
Facility Information:
Facility Name
Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hedvika Lazar
Email
hlazar@vaderis.com
Facility Name
Washington University School of Medicine
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hedvika Lazar
Email
hlazar@vaderis.com
Facility Name
Universitair Ziekenhuis Gent
City
Gent
Country
Belgium
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hedvika Lazar
Email
hlazar@vaderis.com
Facility Name
Hospices Civils de Lyon
City
Lyon
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hedvika Lazar
Email
hlazar@vaderis.com
Facility Name
Ospedale Maggiore di Crema
City
Crema
Country
Italy
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hedvika Lazar
Email
hlazar@vaderis.com
Facility Name
St. Antonius Hospital
City
Nieuwegein
Country
Netherlands
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hedvika Lazar
Email
hlazar@vaderis.com
Facility Name
Hospital Universiati De Bellvitge
City
Barcelona
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hedvika Lazar
Email
hlazar@vaderis.com
Facility Name
Hospital Universitario Ramón y Cajal
City
Madrid
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hedvika Lazar
Phone
+41763217280
Email
hlazar@vaderis.com
First Name & Middle Initial & Last Name & Degree
Viteri

12. IPD Sharing Statement

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Assess Safety and Efficacy of VAD044 in HHT Patients

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