Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT01539772 |
Recruitment Status
:
Active, not recruiting
First Posted
: February 28, 2012
Last Update Posted
: January 24, 2017
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Condition or disease |
---|
Becker Muscular Dystrophy |
Study Type : | Observational |
Actual Enrollment : | 85 participants |
Observational Model: | Cohort |
Time Perspective: | Prospective |
Official Title: | PITT0112: Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping |
Study Start Date : | April 2012 |
Estimated Primary Completion Date : | March 2017 |
Estimated Study Completion Date : | March 2017 |

Group/Cohort |
---|
Becker
BMD participants over 4 years of age with in-frame deletions in the dystrophin gene. |
- Strength and function [ Time Frame: Annual ]
- Quality of life [ Time Frame: Annual ]
These questionnaires include:
- Pediatric Quality of Life Inventory (PedsQL)
- Pediatrics and Adult Neuromuscular module Quality of Life (NeuroQOL)
- Medical history assessment - ambulation status, medication history, hospitalizations, surgeries, nutrition, fractures, and cardiac tests [ Time Frame: Annual ]

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Ages Eligible for Study: | 4 Years and older (Child, Adult, Senior) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Sampling Method: | Non-Probability Sample |
Inclusion Criteria:
- Male
- Age 4 or older
- Diagnosis of BMD with an in-frame deletion in the dystrophin gene, where the boundaries of the mutations are confirmed.
Exclusion Criteria:
• Investigator assessment of inability to comply with protocol

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01539772
United States, California | |
University of California Davis | |
Sacramento, California, United States | |
United States, District of Columbia | |
Children's National Health System | |
Washington, District of Columbia, United States | |
United States, Florida | |
University of Florida | |
Gainesville, Florida, United States | |
United States, Georgia | |
Children's Healthcare of Atlanta | |
Atlanta, Georgia, United States | |
United States, Illinois | |
Ann & Robert H. Lurie Children's Hospital of Chicago | |
Chicago, Illinois, United States | |
United States, Minnesota | |
University of Minnesota | |
Minneapolis, Minnesota, United States | |
United States, Missouri | |
Washington University | |
St. Louis, Missouri, United States | |
United States, North Carolina | |
Carolinas Medical Center | |
Charlotte, North Carolina, United States | |
Duke Children's Hospital and Health Center | |
Durham, North Carolina, United States | |
United States, Pennsylvania | |
Penn State Hershey Medical Center | |
Hershey, Pennsylvania, United States, 17033 | |
University of Pittsburgh | |
Pittsburgh, Pennsylvania, United States | |
United States, Tennessee | |
University of Tennessee | |
Memphis, Tennessee, United States | |
Canada, Alberta | |
Alberta Children's Hospital | |
Calgary, Alberta, Canada | |
Italy | |
Centro Clinico Nemo | |
Milan, Italy | |
United Kingdom | |
Institute of Genetic Medicine - Newcastle University | |
Newcastle upon Tyne, United Kingdom |
Study Chair: | Paula R Clemens, MD | University of Pittsburgh |
Responsible Party: | Cooperative International Neuromuscular Research Group |
ClinicalTrials.gov Identifier: | NCT01539772 History of Changes |
Other Study ID Numbers: |
PITT0112 |
First Posted: | February 28, 2012 Key Record Dates |
Last Update Posted: | January 24, 2017 |
Last Verified: | January 2017 |
Additional relevant MeSH terms:
Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked |