Study Results
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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COMPLETED
32 participants
OBSERVATIONAL
2015-11-30
2019-09-10
Brief Summary
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Symptoms of classical FSHD start in adulthood. However, a small subgroup of FSHD patients have an early, childhood onset. This early onset is associated with faster progression and other symptoms like hearing loss and epilepsy.
The symptoms, natural history and clinical impact of FSHD in children are largely unknown.
The results of this study will be vital for adequate symptomatic management and trial-readiness.
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Detailed Description
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Therefore this study will focus on the total spectrum of FSHD in children.
In addition, an extensive genetic screening will be conducted, searching for (epi)genetic disease modifiers and severity predictors.
Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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iFSHD group
First recruitment group
No interventions assigned to this group
Eligibility Criteria
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Inclusion Criteria
* symptoms of facial, scapulohumeral or peroneal weakness
* genetically proven FSHD1 or FSHD2
* living in the Netherlands
Exclusion Criteria
17 Years
ALL
No
Sponsors
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Leiden University Medical Center
OTHER
Princess Beatrix Muscle Foundation
OTHER
University Medical Center Nijmegen
OTHER
Responsible Party
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RJM Goselink
Prof. dr. Baziel van Engelen
Principal Investigators
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Baziel van Engelen, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
Nijmegen University Medical Center
Locations
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Radboud University Medical Center
Nijmegen, Gelderland, Netherlands
Countries
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References
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Goselink RJ, Schreuder TH, Mul K, Voermans NC, Pelsma M, de Groot IJ, van Alfen N, Franck B, Theelen T, Lemmers RJ, Mah JK, van der Maarel SM, van Engelen BG, Erasmus CE. Facioscapulohumeral dystrophy in children: design of a prospective, observational study on natural history, predictors and clinical impact (iFocus FSHD). BMC Neurol. 2016 Aug 17;16:138. doi: 10.1186/s12883-016-0664-6.
Other Identifiers
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NL53213.091.15
Identifier Type: -
Identifier Source: org_study_id
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