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NCT04668716
Brain Involvement in Dystrophinopathies Part 2
trial in Duchenne Muscular Dystrophy in 339 participants. Completed in 30 June 2024.
30 June 2024
Quick facts
| Lead sponsor | Great Ormond Street Hospital for Children NHS Foundation Trust |
|---|---|
| Status | Completed |
| Study type | OBSERVATIONAL |
| Enrollment | 339 |
| Start date | 11 October 2021 |
| Primary completion | 30 June 2024 |
| Estimated completion | 30 June 2024 |
| Sites | 7 locations across Denmark, France, Netherlands, Italy, United Kingdom, Spain |
Conditions studied
- Duchenne Muscular Dystrophy — all drugs for Duchenne Muscular Dystrophy →
- Becker Muscular Dystrophy — all drugs for Becker Muscular Dystrophy →
Sponsor
Great Ormond Street Hospital for Children NHS Foundation Trust
Who can join
Adults 5 to 50, male only, with Duchenne Muscular Dystrophy or Becker Muscular Dystrophy. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
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.
Publications & conference data
1 peer-reviewed publication reference this trial (live from Europe PMC):
-
Characterisation of a large, single-centre cohort of patients with Becker muscular dystrophy to inform standardised care guidelines.
Riguzzi P, Borland H, James MK, Bourke J, et al · · 2025 · cited 2× · PMID 40483375 · DOI 10.1007/s00415-025-13126-9
Verify or expand the search:
- PubMed search for NCT04668716
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other recruiting trials for Duchenne Muscular Dystrophy
Currently open trials in the same condition.
- NCT07287189 — Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients · Phase 2 · recruiting
- NCT06817382 — A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Ma · Phase 1 · recruiting
- NCT06402942 — Gamified Occupational Therapy for Adolescents With Duchenne Muscular Dystrophy · NA · recruiting
- NCT06450639 — A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD) · Phase 2 · active not recruiting
- NCT06692426 — Trial of Cell Based Therapy for DMD · Phase 1 · recruiting
Other Great Ormond Street Hospital for Children NHS Foundation Trust trials
Trials by the same sponsor.
- NCT07412886 — Advanced MRI in IVH · NA · not yet recruiting
- NCT07112794 — Calprotectin and Gut Disease Study · not yet recruiting
- NCT06770335 — The FLaME Cognitive Rehabilitation Study for Childhood Brain Tumour · NA · not yet recruiting
- NCT06899581 — Gut Health in Children With Cancer · recruiting
- NCT06481527 — Seen and Be Heard: Ensuring Fair Cancer Care for All Children · not yet recruiting
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT04668716 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Great Ormond Street Hospital for Children NHS Foundation Trust
- Last refreshed: 22 July 2024
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04668716.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing