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

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ClinicalTrials.gov Identifier: NCT03695874
Recruitment Status : Not yet recruiting
First Posted : October 4, 2018
Last Update Posted : October 4, 2018
Sponsor:
Information provided by (Responsible Party):
Hospices Civils de Lyon

Brief Summary:

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.


Condition or disease Intervention/treatment
Hereditary Hemorrhagic Telangiectasia Other: Phase 1 : fill out 1 scale Other: Phase 2 : fill out 1 scale and 4 questionnaires

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Study Type : Observational
Estimated Enrollment : 600 participants
Observational Model: Other
Time Perspective: Prospective
Official Title: Development of a Quality of Life Measurement Scale in Hereditary Haemorrhagic Telangiectasia (HHT) Disease.
Estimated Study Start Date : October 2018
Estimated Primary Completion Date : June 2021
Estimated Study Completion Date : September 2021


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.





Primary Outcome Measures :
  1. 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.

  2. 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.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with an Hereditary Haemorrhagic Telangiectasia disease.
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03695874


Contacts
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Contact: Sylvie FOURDRINOY 04 27 85 65 26 ext +33 sylvie.fourdrinoy@chu-lyon.fr
Contact: Sophie DUPUIS-GIROD, PhD 04 27 85 65 25 ext +33 sophie.dupuis-girod@chu-lyon.fr

Locations
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France
Centre de Référence pour la Maladie de Rendu-Osler - Service de Génétique Clinique - HOSPICES CIVILS DE LYON - Groupement Hospitalier Est Not yet recruiting
Bron, France, 69677
Contact: Sylvie FOURDRINOY    04 27 85 65 26 ext +33    sylvie.fourdrinoy@chu-lyon.fr   
Contact: Sophie DUPUIS-GIROD, PhD    04 27 85 65 25 ext +33    sophie.dupuis-girod@chu-lyon.fr   
Sponsors and Collaborators
Hospices Civils de Lyon

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Responsible Party: Hospices Civils de Lyon
ClinicalTrials.gov Identifier: NCT03695874     History of Changes
Other Study ID Numbers: 69HCL18_0543
First Posted: October 4, 2018    Key Record Dates
Last Update Posted: October 4, 2018
Last Verified: September 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Telangiectasis
Telangiectasia, Hereditary Hemorrhagic
Vascular Diseases
Cardiovascular Diseases
Hemostatic Disorders
Hemorrhagic Disorders
Hematologic Diseases
Vascular Malformations
Cardiovascular Abnormalities
Congenital Abnormalities