Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

UNKNOWN

Total Enrollment

53 participants

Study Classification

OBSERVATIONAL

Study Start Date

2012-09-30

Study Completion Date

2016-08-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

We will utilize the Cooperative International Neuromuscular Research Group (CINRG) network to collect and store tissue and blood from patients with Duchenne muscular dystrophy (DMD) with specific genetic mutations within the dystrophin gene that could be treated by antisense oligonucleotide (AO) drugs.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Muscle Tissue Bank for Muscular Dystrophy

NCT01950897

Muscular Dystrophies Muscular Dystrophy

COMPLETED

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

NCT01539772

Becker Muscular Dystrophy

UNKNOWN

Longitudinal Study of the Natural History of Duchenne Muscular Dystrophy (DMD)

NCT00468832

Duchenne Muscular Dystrophy

UNKNOWN

Study of Eteplirsen in DMD Patients

NCT02255552

Duchenne Muscular Dystrophy (DMD)

COMPLETED PHASE3

Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy

NCT02420379

Duchenne Muscular Dystrophy (DMD)

COMPLETED PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

The purpose of this tissue bank is to collect blood and skin samples from participants who are diagnosed with Duchenne muscular dystrophy (DMD) and carry one of nine specific changes in the dystrophin gene. The specific dystrophin changes that we are interested in studying are those that would work with exon-skipping therapies in patients with DMD, specifically deletions of the follow exons: 10-52, 13-50, 29-50, 43-52, 44, 43-50, 45-50, 45-52, 46, 46-47, 46-48, 46-49, 46-51, 46-53, 46-55, 46-60, 47-50, 47-52, 48-50, 49-50, 50, 52, 52-63, 48-52, 49-52, 50-52.

These blood and skin samples will be held in a tissue bank at Carolinas Medical Center for future DMD research.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Duchenne Muscular Dystrophy

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Duchenne muscular dystrophy

No interventions assigned to this group

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Age 4 and above
* Diagnosis of DMD with a confirmed out-of-frame dystrophin gene deletions that could be corrected by skipping exon 45, 51, or 53 based on past genetic testing.