Overview

This trial is active, not recruiting.

Condition becker muscular dystrophy
Sponsor Cooperative International Neuromuscular Research Group
Start date April 2012
End date March 2017
Trial size 85 participants
Trial identifier NCT01539772, PITT0112

Summary

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Observational model cohort
Time perspective prospective
Arm
BMD participants over 4 years of age with in-frame deletions in the dystrophin gene.

Primary Outcomes

Measure
Strength and function
time frame: Annual
Quality of life
time frame: Annual
Medical history assessment - ambulation status, medication history, hospitalizations, surgeries, nutrition, fractures, and cardiac tests
time frame: Annual

Eligibility Criteria

Male participants at least 4 years old.

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

Additional Information

Official title PITT0112: Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping
Description 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.
Trial information was received from ClinicalTrials.gov and was last updated in April 2016.
Information provided to ClinicalTrials.gov by Cooperative International Neuromuscular Research Group.