NCT04583917Completed
Brain Involvement in Dystrophinopathies Part 1
Brain INvolvement in Dystrophinopathies (BIND): Deep Functional Phenotyping of Duchenne Muscular Dystrophy and Becker Muscular Dystrophy Patients (WP5) Part 1: a Multicentre Online Phenotyping and Neurobehavioural Data Collection Study
Source: ClinicalTrials.gov · Last updated 22 Jul 2024
Brief summary (registry, in English)
The objective of this study is to collect data from a large cohort of individuals with DMD and BMD focusing on the neurobehavioural aspects of these conditions and their correlation to the location of the DMD gene mutation.
- Status (registry)
- Completed
- Study type
- Observational
- Enrollment
- 377 (actual)
- First submitted
- 5 Oct 2020
- Start date
- Mar 2021
- Primary completion date
- Jun 2024
- Lead sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
- Diseases
- Duchenne muscular dystrophy
- NCT number
- NCT04583917
What you commit to
- Study type: Observational
- Participants enrolled: 377
- From Mar 2021 to the primary completion date, Jun 2024
- 7 sites in 6 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- A severe co-morbidity or planned surgical intervention within 6 months from the study which could interfere with the well-being of the participant
Other
- Lack of a molecular diagnosis of DMD or BMD
- Mutation falls outside the regions of interest
Who the study is looking for
- 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).
Age in the registry: 5 years to 50 years · Sex: Male · Does not accept healthy volunteers
Sites (7)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Copenhagen Neuromuscular Center Coordinator of this site? Confirm its status | Copenhagen | Denmark | Unknown status |
| Universidad Complutense de Madrid Coordinator of this site? Confirm its status | Madrid | Spain | Unknown status |
| Imagine Institut Des Maladies Genetiques Necker Enfants Malades Fondation Coordinator of this site? Confirm its status | Paris | France | Unknown status |
| GOSH/UCL Coordinator of this site? Confirm its status | London | United Kingdom | Unknown status |
| University of Newcastle Upon Tyne Coordinator of this site? Confirm its status | Newcastle | United Kingdom | Unknown status |
| Università Cattolica del Sacro Cuore Coordinator of this site? Confirm its status | Rome | Italy | Unknown status |
| Stichting Kempenhaeghe Coordinator of this site? Confirm its status | Heeze | Netherlands | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.