Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping



Status:Active, not recruiting
Conditions:Neurology, Orthopedic
Therapuetic Areas:Neurology, Orthopedics / Podiatry
Healthy:No
Age Range:4 - Any
Updated:6/17/2018
Start Date:April 2012
End Date:August 2018

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PITT0112: Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping

This is a multi-center natural history study that will be conducted at participating centers
in the Cooperative International Neuromuscular Research Group (CINRG). Following a baseline
evaluation, participants will have three follow-up visits over a three-year period. The
investigators will characterize the Becker muscular dystrophy phenotype, and correlate
specific abnormal dystrophin proteins with the range of clinical outcomes.

We will utilize the Cooperative International Neuromuscular Research group (CINRG) network to
collect cohorts of Becker muscular dystrophy (BMD) patients with in-frame deletions in the
dystrophin gene. We will collect clinical data across multiple body systems and correlate
these findings to the high-resolution deletion break-point mapping performed from the tissue
samples. We will investigate the observed variability to deepen our understanding of
molecular mechanisms relevant to the optimization of exon skipping therapeutic approaches.

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
We found this trial at
14
sites
4200 Fifth Ave
Pittsburgh, Pennsylvania 15260
(412) 624-4141
Principal Investigator: Paula Clemens, MD
Phone: 412-383-7207
University of Pittsburgh The University of Pittsburgh is a state-related research university, founded as the...
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1000 Blythe Blvd
Charlotte, North Carolina 28203
(704) 355-2000
Principal Investigator: Amy Harper, MD
Phone: 704-403-2660
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Charlotte, NC
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Gainesville, Florida 32610
(352) 392-3261
Principal Investigator: Barry J Byrne, MD, PhD
Phone: 352-273-7573
University of Florida The University of Florida (UF) is a major, public, comprehensive, land-grant, research...
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920 Madison Ave
Memphis, Tennessee 38163
Principal Investigator: Tulio Bertorini, MD
Phone: 901-725-8920
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Minneapolis, Minnesota 55455
(612) 625-5000
Principal Investigator: Peter Karachunski, MD
Phone: 612-625-7967
Univ of Minnesota With a flagship campus in the heart of the Twin Cities, and...
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1405 Clifton Road NE
Atlanta, Georgia 30322
404-785-6000
Principal Investigator: Han Phan, MD
Phone: 404-785-9893
Children's Healthcare of Atlanta Whether treating a toddler in an emergency or supporting a teen...
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Calgary, Alberta
Principal Investigator: Jean Mah, MD
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225 E Chicago Ave
Chicago, Illinois 60611
(312) 227-4000
Principal Investigator: Nancy Kuntz, MD
Phone: 312-227-4483
Ann & Robert H. Lurie Children's Hospital of Chicago Ann & Robert H. Lurie Children
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2201 Inwood Rd
Dallas, Texas 75235
(214) 645-8300
U.T. Southwestern Medical Center The story of UT Southwestern Medical Center is one of commitment...
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Durham, North Carolina
Principal Investigator: Edward Smith, MD
Phone: 919-684-1143
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Hershey, Pennsylvania 17033
Principal Investigator: Matthew Wicklund, MD
Phone: 717-531-0003
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Hershey, PA
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1 Shields Ave
Sacramento, California 95616
(530) 752-1011
Principal Investigator: Craig McDonald, MD
Phone: 916-734-8898
University of California-Davis As we begin our second century, UC Davis is poised to become...
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Saint Louis, Missouri 63110
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Washington, District of Columbia 20010
Principal Investigator: Mathula Thangarajh, MD
Phone: 202-476-4802
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