Duchenne Muscular Dystrophy Clinical Trial
Official title:
Brain INvolvement in Dystrophinopathies (BIND): Deep Functional Phenotyping of Duchenne Muscular Dystrophy and Becker Muscular Dystrophy Patients (WP5 and WP6) Part 2: a Neurobehavioural and MRI Study
NCT number | NCT04668716 |
Other study ID # | 20NM35 |
Secondary ID | |
Status | Recruiting |
Phase | |
First received | |
Last updated | |
Start date | October 11, 2021 |
Est. completion date | May 2024 |
The objective of this study is to understand the relationship between DMD and BMD brain comorbidities, and the location of the gene mutation which causes the disease.
Status | Recruiting |
Enrollment | 270 |
Est. completion date | May 2024 |
Est. primary completion date | May 2024 |
Accepts healthy volunteers | Accepts Healthy Volunteers |
Gender | Male |
Age group | 5 Years to 50 Years |
Eligibility | Inclusion Criteria: For DMD patients: - Male - age 5-17 years - genetically-proven diagnosis of DMD - genetic mutation that abrogates expression of Dp427 alone (assigned in DMD Group 1: Dp427-/Dp140+) or both Dp427 and Dp140 (assigned to DMD Group 2: Dp427-/Dp140-); or all isoforms (assigned to DMD group 3) For BMD patients: - age 5-50 years - genetically-proven diagnosis of BMD - genetic mutation that decreases expression of Dp427 alone (assigned to BMD Group 1), of both Dp427 and Dp140 (assigned to BMD Group 2), or of all the isoforms (assigned to BMD group 3). For MRI controls: - Male - age 5-50 years Exclusion Criteria: For DMD and BMD patients: - Lack of a molecular diagnosis of DMD or BMD - Mutation falls outside the regions of interest - A severe co-morbidity or planned surgical intervention within 6 months from the study which could interfere with the well-being of the participant For MRI controls: - any muscle disease - a brain disorder (such as severe brain concussion in past history, congenital brain anomalies, epilepsy) General exclusion criteria for MRI: - Claustrophobia - Pacemakers and defibrillators - Nerve stimulators - Intracranial clips - Intraorbital or intraocular metallic fragments - Cochlear implants - Ferromagnetic implants (e.g. thoracic implant for scoliosis) - Inability to lie supine during less than 45 minutes - not having a general practitioner - severe learning disability which will require a general anaesthetic |
Country | Name | City | State |
---|---|---|---|
Denmark | Copenhagen Neuromuscular Center | Copenhagen | |
France | Imagine Institut Des Maladies Genetiques Necker Enfants Malades Fondation | Paris | |
Italy | Universita Cattolica Del Sacro Cuore | Rome | |
Netherlands | STICHTING KEMPENHAEGHE/Leiden University Medical Center | Heeze | |
Spain | Universidad Complutense de Madrid | Madrid | |
United Kingdom | UCL/GOSH | London | |
United Kingdom | University of Newcastle Upon Tyne | Newcastle |
Lead Sponsor | Collaborator |
---|---|
Great Ormond Street Hospital for Children NHS Foundation Trust | Catholic University of the Sacred Heart, Institut Necker Enfants Malades, Leiden University Medical Center, Newcastle-upon-Tyne Hospitals NHS Trust, Region Hovedstadens Apotek, Stichting Kempenhaeghe, Universidad Complutense de Madrid, Università degli Studi di Ferrara |
Denmark, France, Italy, Netherlands, Spain, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | CNS Comorbidity Phenotyping | Correlate CNS comorbidity phenotypes with genotype in DMD and BMD patients | 170 minutes |
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