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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified April 2010 by Washington University School of Medicine.
Recruitment status was  Recruiting
Sponsor:
Collaborators:
Nationwide Children's Hospital
Children's Hospital Boston
University of Minnesota - Clinical and Translational Science Institute
University of California, Davis
Information provided by:
Washington University School of Medicine
ClinicalTrials.gov Identifier:
NCT01098708
First received: April 1, 2010
Last updated: NA
Last verified: April 2010
History: No changes posted
  Purpose

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.


Condition
Duchenne Muscular Dystrophy

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Clinical Outcomes Validation in Non Ambulatory and Young Boys/Men With Duchenne Muscular Dystrophy (DMD)

Resource links provided by NLM:


Further study details as provided by Washington University School of Medicine:

Primary Outcome Measures:
  • Dystrophin gene mutations that predispose to early onset cardiomyopathy [ Time Frame: 3 years ] [ Designated as safety issue: No ]

Estimated Enrollment: 105
Study Start Date: March 2010
Estimated Study Completion Date: October 2012
Estimated Primary Completion Date: October 2012 (Final data collection date for primary outcome measure)
Detailed Description:

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.

  Eligibility

Ages Eligible for Study:   7 Years to 22 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Non ambulatory boys and men with Duchenne muscular dystrophy

Criteria

Inclusion Criteria:

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

Exclusion Criteria:

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

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01098708

Contacts
Contact: Pallavi Anand 314-362-2490 anandp@neuro.wustl.edu
Contact: Charlie Wulf 314-362-6980 wulfc@neuro.wustl.edu

Locations
United States, Missouri
Washington University Recruiting
St. Louis, Missouri, United States, 63110
Contact: Pallavi Anand    314-362-2490    anandp@neuro.wustl.edu   
Contact: Charlie Wulf    314-362-6980    wulfc@neuro.wustl.edu   
Principal Investigator: Anne M Connolly, M.D.         
Sponsors and Collaborators
Washington University School of Medicine
Nationwide Children's Hospital
Children's Hospital Boston
University of Minnesota - Clinical and Translational Science Institute
University of California, Davis
Investigators
Principal Investigator: Anne Connolly, M.D. Washington University School of Medicine
  More Information

No publications provided

Responsible Party: Anne Connolly, M.D., Principal investigator, Washington University
ClinicalTrials.gov Identifier: NCT01098708     History of Changes
Other Study ID Numbers: 10-0067
Study First Received: April 1, 2010
Last Updated: April 1, 2010
Health Authority: United States: Institutional Review Board

Keywords provided by Washington University School of Medicine:
DMD
non ambulatory

Additional relevant MeSH terms:
Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on August 27, 2014