Clinical, Genetic and Epigenetic Characterization of Patients With FSHD Type 1 and FSHD Type 2

NCT ID: NCT01970735

Last Updated: 2023-11-14

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

103 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-10-30

Study Completion Date

2014-12-16

Brief Summary

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The aim of the study was to compare the severity of illness between groups of patients (Facio-Scapulo-Humeral Dystrophy = FHSD1, FSHD2 and patients both FSHD1 and FSHD2).

Despite advances in research on the subject, answers are still needed on these diseases.

We also aim to determine whether the chromosomal genetic abnormality is involved in other diseases and the frequency of this mutation in the population of patients FSHD.

This study will increase our knowledge of the two forms of FSHD who present a common pathophysiological mechanism and may occur together in the same family with a worsening of the clinical phenotype worsening . In addition, epigenetic differences between FSHD type 1 and type 2 seems to have clinical consequences requiring appropriate management

Detailed Description

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Conditions

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Muscular Dystrophy, Facioscapulohumeral

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

BASIC_SCIENCE

Blinding Strategy

NONE

Study Groups

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FSHD patient

Group Type EXPERIMENTAL

Blood test

Intervention Type BIOLOGICAL

Interventions

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Blood test

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

* age ≥ 18 years and \<75 years
* FSHD patients 1 or 2 with or without genetic confirmation

Exclusion Criteria

\- Patient with all conditions considered by the investigator interfering with the proper conduct of the study.
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Centre Hospitalier Universitaire de Nice

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Hôpital Archet 1

Nice, , France

Site Status

Countries

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France

References

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Sacconi S, Briand-Suleau A, Gros M, Baudoin C, Lemmers RJLF, Rondeau S, Lagha N, Nigumann P, Cambieri C, Puma A, Chapon F, Stojkovic T, Vial C, Bouhour F, Cao M, Pegoraro E, Petiot P, Behin A, Marc B, Eymard B, Echaniz-Laguna A, Laforet P, Salviati L, Jeanpierre M, Cristofari G, van der Maarel SM. FSHD1 and FSHD2 form a disease continuum. Neurology. 2019 May 7;92(19):e2273-e2285. doi: 10.1212/WNL.0000000000007456. Epub 2019 Apr 12.

Reference Type DERIVED
PMID: 30979860 (View on PubMed)

Other Identifiers

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13-AOI-01

Identifier Type: -

Identifier Source: org_study_id

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