This is a Study to Get More Information About Non Ambulatory Boys & Men With Duchenne Muscular Dystrophy

NCT01098708 | Completed

• 1 other identifier: 10-0067
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

Many therapeutic trials in DMD exclude non-ambulatory boys and men. Rate of progression in these non-ambulatory patients has been studied but consensus has not been reached for what measures are most reliable and reproducible. Furthermore, any treatment trial would be expected to demonstrate improved function and improvement in quality of life. Therefore, function, strength, and quality of life must be understood and standardized. While the goal of this proposal is to standardize clinical outcomes for therapeutic trials, careful understanding of the progression of DMD in non ambulatory boys may also lead to better medical treatment.

Conditions

Duchenne Muscular Dystrophy

Keywords

DMD; non ambulatory

Outcome Measures

Primary Outcomes (1)

• Dystrophin gene mutations that predispose to early onset cardiomyopathy

  3 years

Eligibility Criteria

• Age: 7 Years - 22 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Non ambulatory boys and men with Duchenne muscular dystrophy

You may qualify if:

• For Non-ambulatory study, boys must be unable to walk without assistive devices for greater than or equal to one year.
• Age at onset of study for non-ambulatory boys and men: greater than or equal to 7 years through age 22 years.
• Genetic or biopsy confirmation of dystrophinopathy or clinical diagnosis of DMD with biopsy/genetic confirmation in a primary relative.

You may not qualify if:

• \. For Non-ambulatory study, the inability to understand and cooperate with the testing would exclude a subject. -

Sponsors & Collaborators

• Washington University School of Medicine: lead
• Nationwide Children's Hospital: collaborator
• Boston Children's Hospital: collaborator
• University of Minnesota: collaborator
• University of California, Davis: collaborator

Study Sites (1)

Washington University

St Louis, Missouri, 63110, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

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

Study Officials

• Anne Connolly, M.D.

  Washington University School of Medicine

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Neurology

Study Record Dates

• First Submitted: April 1, 2010
• First Posted: April 5, 2010
• Study Start: March 1, 2010
• Primary Completion: November 1, 2013
• Study Completion: November 1, 2013
• Last Updated: January 18, 2017

Record last verified: 2017-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)