Development of a Quality of Life Measurement Scale in Hereditary Haemorrhagic Telangiectasia (HHT) Disease. (ELECT-RO)

February 3, 2021 updated by: Hospices Civils de Lyon

Hereditary Haemorrhagic Telangiectasia (HHT) is a rare inherited genetic disease of autosomal dominant inheritance with a prevalence of 1/6000. It is manifested by haemorrhages, mucocutaneous telangiectasias and visceral arteriovenous malformations. These symptoms significantly affect the daily lives of patients, their social relationships and their working lives.

HAS (Haute Autorité de Santé) national recommendations focus on assessing and improving the quality of life (QOL) of patients. Many scales for measuring QOL exist but they are most often general and therefore have the disadvantage of not taking into account the particularities of pathologies and their symptoms and do not allow to have a precise vision of their impact on QOL.

It is important to be able to evaluate this impact, to determine its nature and to quantify it so that health professional can adapt their proposal for the management of HHT patients. And only the development of a specific and validated QOL measurement scale will allow them to access this information.

The aim of this study is to develop a scale of measurement of quality of life in HHT disease and to validate it, a scale specific to HHT, simple and fast to fill by the patients themselves.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Actual)

643

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Bron, France, 69677
        • Centre de Référence pour la Maladie de Rendu-Osler - Service de Génétique Clinique - HOSPICES CIVILS DE LYON - Groupement Hospitalier Est

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patients with an Hereditary Haemorrhagic Telangiectasia disease.

Description

Inclusion Criteria:

  • over 18 years
  • able to read French
  • with clinically confirmed Hereditary Haemorrhagic Telangiectasia disease (presence of at least 3 Curaçao criteria) and / or molecular biology
  • who received the information and did not object to participate in the study

Exclusion Criteria:

  • None

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Phase 1: statistical purification

400 Hereditary Haemorrhagic Telangiectasia patients:

  • over 18 years
  • able to read French
  • with clinically confirmed Hereditary Haemorrhagic Telangiectasia disease (presence of at least 3 Curaçao criteria) and / or molecular biology
  • who received the information and did not object to participate in the study
Other: Phase 1 : fill out 1 scale

Patients will be asked to complete questionnaires.

Group 1: the purpose is to simplify a 75 items scale by a statistical analysis of the data collected from 400 patients. The statistical purification will allow to finalize an abbreviated scale (20-25 items).

Doctors specialized in Hereditary Haemorrhagic Telangiectasia may suggest that patients enter the study. After submission of the information notice and collection of their non-opposition, patients will have the choice to fill out the paper version or the electronic version of the scale and questionnaires.
Phase 2: statistical validation

200 Hereditary Haemorrhagic Telangiectasia patients:

  • over 18 years
  • able to read French
  • with clinically confirmed Hereditary Haemorrhagic Telangiectasia disease (presence of at least 3 Curaçao criteria) and / or molecular biology
  • who received the information and did not object to participate in the study
Other: Phase 2 : fill out 1 scale and 4 questionnaires

Patients will be asked to complete questionnaires.

Group 2: the purpose is to validate the abbreviated scale by a statistical analysis of the data collected from 200 patients. The statistical validation will allow to check the consistency and correlation of the abbreviated scale with other scales: a general Quality Of Life scale (SF36), an anxiety and depression scale (HAD), a social support scale (SSQ6) and a scale of regulation emotional (CERQ) and to test and re-test to check fidelity of the abbreviated scale.

Doctors specialized in Hereditary Haemorrhagic Telangiectasia may suggest that patients enter the study. After submission of the information notice and collection of their non-opposition, patients will have the choice to fill out the paper version or the electronic version of the scale and questionnaires.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Validity of the factor structure of the developed measurement scale.
Time Frame: 1 hour
Saturation coefficients of the exploratory factor analysis observed on the sample of 400 participants, confirmatory factor analysis (CFA) adjustment indices on the sample of 200 participants, and factorial weights observed on the AFC will be combined to evaluate the structural validity of the measurement scale.
1 hour
Validity of the reliability of the developed measurement scale.
Time Frame: 1 hour
Cronbach alphas coefficients, composite reliability values (rho) and percentages of extracted variance will be combined to assess the Reliability of the measurement scale.
1 hour

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Hospices Civils de Lyon

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 28, 2019

Primary Completion (Actual)

December 28, 2020

Study Completion (Actual)

December 28, 2020

Study Registration Dates

First Submitted

September 30, 2018

First Submitted That Met QC Criteria

October 2, 2018

First Posted (Actual)

October 4, 2018

Study Record Updates

Last Update Posted (Actual)

February 8, 2021

Last Update Submitted That Met QC Criteria

February 3, 2021

Last Verified

February 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 69HCL18_0543

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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