Who is sponsoring NCT01753804?

NCT01753804 is sponsored by BioMarin Pharmaceutical.

What drugs are being tested in NCT01753804?

Interventions in NCT01753804: Observational study.

What condition does NCT01753804 study?

NCT01753804 studies Duchenne Muscular Dystrophy.

Is NCT01753804 still recruiting?

NCT01753804 is currently terminated. Last updated 8 December 2017.

Last reviewed 2017-12-08 · How we verify

NCT01753804

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.

Terminated Last updated 8 December 2017

What this trial tests

trial testing Observational study in Duchenne Muscular Dystrophy in 269 participants. Terminated before completion.

Timeline

1 September 2012

Primary endpoint
1 October 2016

1 October 2016

Quick facts

Lead sponsor	BioMarin Pharmaceutical
Status	Terminated
Study type	OBSERVATIONAL
Enrollment	269
Start date	1 September 2012
Primary completion	1 October 2016
Estimated completion	1 October 2016
Sites	16 locations across France, Italy, Netherlands, Belgium, Sweden, Germany, Argentina, United States

Drugs / interventions tested

Observational study — full drug profile →

Conditions studied

Duchenne Muscular Dystrophy — all drugs for Duchenne Muscular Dystrophy →

Sponsor

BioMarin Pharmaceutical — full company profile →

Who can join

Adults 3 to 18, male only, with Duchenne Muscular Dystrophy. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

To characterize the natural history and progression of Duchenne Muscular Dystrophy (DMD) to help inform the design of future studies, to capture biomarkers of safety and disease progression and to provide comparative data for the development of rare exons for which formal controlled trials are not feasible.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

MRI vastus lateralis fat fraction predicts loss of ambulation in Duchenne muscular dystrophy.
Naarding KJ, Reyngoudt H, van Zwet EW, Hooijmans MT, et al · · 2020 · cited 68× · PMID 31937624 · DOI 10.1212/wnl.0000000000008939
<i>DMD</i> Genotypes and Motor Function in Duchenne Muscular Dystrophy: A Multi-institution Meta-analysis With Implications for Clinical Trials.
Muntoni F, Signorovitch J, Sajeev G, Lane H, et al · · 2023 · cited 31× · PMID 36725339 · DOI 10.1212/wnl.0000000000201626
Global versus individual muscle segmentation to assess quantitative MRI-based fat fraction changes in neuromuscular diseases.
Reyngoudt H, Marty B, Boisserie JM, Le Louër J, et al · · 2021 · cited 30× · PMID 33219846 · DOI 10.1007/s00330-020-07487-0
Suitability of external controls for drug evaluation in Duchenne muscular dystrophy.
Goemans N, Signorovitch J, Sajeev G, Yao Z, et al · · 2020 · cited 29× · PMID 32611643 · DOI 10.1212/wnl.0000000000010170
Functional and Clinical Outcomes Associated with Steroid Treatment among Non-ambulatory Patients with Duchenne Muscular Dystrophy1.
McDonald CM, Mayer OH, Hor KN, Miller D, et al · · 2023 · cited 11× · PMID 36565131 · DOI 10.3233/jnd-221575
Functional trajectories before and after loss of ambulation in Duchenne muscular dystrophy and implications for clinical trials.
McDonald CM, Signorovitch J, Mercuri E, Niks EH, et al · · 2024 · cited 4× · PMID 38829874 · DOI 10.1371/journal.pone.0304099
Association between exon-skipping therapy with eteplirsen and cardiac outcomes in Duchenne muscular dystrophy.
Iff J, Desguerre I, Liu Y, Sarkozy F, et al · · 2026 · cited 2× · PMID 40831143 · DOI 10.1177/22143602251366721
Characterization of patients with Duchenne muscular dystrophy across previously developed health states.
Muntoni F, Goemans N, Posner N, Signorovitch J, et al · · 2024 · cited 2× · PMID 39475941 · DOI 10.1371/journal.pone.0307118

Verify or expand the search:

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Trials testing the same drug.

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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 BioMarin Pharmaceutical trials

Trials by the same sponsor.

NCT07477691 — Immune Modulation During Palynziq® Treatment in Adults (IMPALA) · Phase 4 · not yet recruiting
NCT07126262 — A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months · Phase 2 · recruiting
NCT06309979 — A Study to Assess Growth in Children With Idiopathic Short Stature · recruiting
NCT06455059 — Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia · Phase 3 · active not recruiting
NCT06305234 — A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade) · recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT01753804 (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 BioMarin Pharmaceutical
Last refreshed: 8 December 2017

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/NCT01753804.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing