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

NCT01753804 | Terminated

• 1 other identifier: PRO-DMD-01
• Study Type: observational
• Target: 269
• Locations: 10 countries
• Sites: 16

Brief Summary

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.

Conditions

Duchenne Muscular Dystrophy

Keywords

DMD; Myopathy; Natural History; Muscular dystrophy; Biomarkers; Muscle testing

Outcome Measures

Primary Outcomes (1)

• 6 minute walk distance

  Participants are asked to walk at their own preferred speed on a fixed distance for 6 minutes. Subjects are warned of the time and that they may stop earlier if they feel unable to continue. Total distance walked within 6 minutes (or until stopping) is recorded.

  Change from visit 1 walking distance

Study Arms (1)

Study participants

All participants will follow the same protocol, including muscle strength and function testing, and blood and urine collection, for a maximum of 7 visits over 3 years.

Other: Observational study

Interventions

Observational study OTHER

There is no medication or device tested in this study. This is an obversational study on the progression of the disease.

Study participants

Eligibility Criteria

• Age: 3 Years - 18 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Subjects diagnosed with DMD resulting from a mutation in the DMD gene which is confirmed by a state of the art DNA diagnostic technique covering all DMD gene exons

You may qualify if:

• Diagnosis of DMD resulting from a mutation in the DMD gene confirmed by a state of the art DNA diagnostic technique covering all DMD gene exons.
• Age 3 - 18 years
• Willing and able to comply with protocol requirements
• Life expectancy of at least 3 years
• Able to give informed assent and/or consent in writing signed by the subject and/or parent(s)/legal guardian (according to local regulations)

You may not qualify if:

• Current participation in a clinical study with an Investigational Medicinal Product (IMP)
• Participation within the previous 1 month in a clinical study with an IMP

Sponsors & Collaborators

• BioMarin Pharmaceutical: lead

Study Sites (16)

UC Davis Health System

Sacramento, California, 95817, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Hospital de Pediatria Prof Dr Juan P Garrahan

Buenos Aires, Argentina

Location

Universitair Ziekenhuis

Ghent, Belgium

Location

Universitair Ziekenhuis Leuven

Leuven, Belgium

Location

Hospital das Clinicas da Faculdade de Medicina da USP

São Paulo, Brazil

Location

CHU Hopital des enfants

Toulouse, France

Location

Universitaetsklinikum Essen

Essen, Germany

Location

Universitaetsklinikum Freiburg

Freiburg im Breisgau, Germany

Location

Azienda Ospedaliera Universitaria Policlinico G. Martino

Messina, Italy

Location

Policlinico Univsersitario Agostino Gemelli

Rome, Italy

Location

Leids Universitair Medisch Centrum

Leiden, Netherlands

Location

UMC St. Radboud

Nijmegen, Netherlands

Location

Drottning Silvias Barn- ochungdomssjukhus

Gothenburg, Sweden

Location

Hacettepe University Medical Faculty

Ankara, Turkey (Türkiye)

Location

Related Links

• BioMarin website

Biospecimen

Retention: SAMPLES WITHOUT DNA

Blood sampling at 4 time points (first visit then once a year). Urinalysis sampling at 4 time points (first visit then once a year).

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Muscular Diseases; Muscular Dystrophies

Interventions

Observation

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Methods; Investigative Techniques

Study Officials

• Nathalie Goemans, MD

  UZ Leuven, Belgium

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 13, 2012
• First Posted: December 20, 2012
• Study Start: September 1, 2012
• Primary Completion: October 1, 2016
• Study Completion: October 1, 2016
• Last Updated: December 8, 2017

Record last verified: 2017-12

Locations

SE (1); TU (1); DE (2); IT (2); BE (2); US (3); AR (1); BR (1); NL (2); FR (1)