Becker Muscular Dystrophy Clinical Trial
Official title:
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. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Terminated |
NCT01168908 -
Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy
|
Phase 2 | |
| Recruiting |
NCT01484678 -
Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
|
||
| Completed |
NCT02147639 -
Effects of Sodium Nitrate on Blood Flow in Becker Muscular Dystrophy
|
Phase 2/Phase 3 | |
| Completed |
NCT02847975 -
Sodium Nitrate to Improve Blood Flow
|
Phase 1 | |
| Recruiting |
NCT02069756 -
The Duchenne Registry
|
||
| Completed |
NCT04585464 -
A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults
|
Phase 1 | |
| Recruiting |
NCT04668716 -
Brain Involvement in Dystrophinopathies Part 2
|
||
| Completed |
NCT03236662 -
(-)- Epicatechin Becker Muscular Dystrophy
|
Phase 2 | |
| Completed |
NCT01350154 -
Effect of Modulating the nNOS System on Cardiac, Muscular and Cognitive Function in Becker Muscular Dystrophy Patients
|
Phase 2 | |
| Enrolling by invitation |
NCT06066580 -
Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy
|
Phase 2 | |
| Not yet recruiting |
NCT06363526 -
Effectiveness of 5-week Digital Respiratory Practice in a Group of Children With Duchenne Muscular Dystrophy and Becker Muscular Dystrophy.
|
N/A | |
| Recruiting |
NCT05409079 -
Schulze Muscular Dystrophy Ability Clinical Study
|
N/A | |
| Completed |
NCT01856868 -
Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study)
|
Phase 1/Phase 2 | |
| Completed |
NCT01557400 -
Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
|
Phase 3 | |
| Recruiting |
NCT02109692 -
Evaluation of Muscle miRNA as Biomarkers in Dystrophinopathies
|
N/A | |
| Recruiting |
NCT03057002 -
UTSW HP [13-C] Pyruvate Injection in HCM
|
||
| Recruiting |
NCT04583917 -
Brain Involvement in Dystrophinopathies Part 1
|
||
| Completed |
NCT02207283 -
PDE5 Inhibition to Alleviate Functional Muscle Ischemia in Becker Muscular Dystrophy
|
Phase 4 | |
| Withdrawn |
NCT03076814 -
Functional Muscle Ischemia With Tadalafil Treatment in Becker Muscular Dystrophy
|
N/A | |
| Completed |
NCT00873782 -
Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy
|
Phase 1 |