Actively Recruiting
CureDuchenne Link4: A Resource to Support Research Studies in Duchenne and Becker Muscular Dystrophy (DMD/BMD)
Led by CureDuchenne · Updated on 2024-08-20
5000
Participants Needed
10
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are gathering data and samples from individuals diagnosed with Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD), or carriers of these conditions. This observational study collects clinical information and self- or caregiver-reported data to support research on these neuromuscular disorders. The study aims to provide a valuable resource for researchers by integrating health outcomes with biospecimens for translational research. Participants join through the CureDuchenne Link application, accessible via mobile devices or web, and may participate virtually, at project sites, or community events nationwide. The data collected includes diagnosis information and genetic mutation status upon study entry. Follow-up assessments of functional status, ambulation, walking ability, corticosteroid use, heart and lung health occur every 6 to 12 months for up to ten years. Individuals or their guardians provide consent to share this information, which is stored securely in a HIPAA-compliant data warehouse. Researchers use this data to study DMD and BMD. The study tracks detailed clinical outcomes over time, including the North Star Ambulation Assessment and 6 Minute Walk Test scores. Participants may remain involved for many years, contributing to long-term understanding of these conditions.
CONDITIONS
Brief Title
CureDuchenne Link®: A Resource for Research
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Currently has a confirmed diagnosis of Duchenne or Becker muscular dystrophy based on genetic testing, muscle biopsy, or clinical diagnosis
- Currently has a confirmed diagnosis of carrier status for Duchenne or Becker muscular dystrophy based on genetic testing
- Parent or guardian (for minors) or participant provides informed consent and/or assent as required
- Is 4 weeks of age or older at the time of consent
You will not qualify if you...
- Is a foster child or ward of the state
- Is a prisoner
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (virtual or in-person)
Duration - Up to 1 month
Participants provide information about their diagnosis and genetic mutation status upon study entry or when genetic testing results are available.
1 visit (virtual or in-person)
Duration - Up to 10 years
Participants are monitored with assessments of functional status, ambulation, corticosteroid use, cardiac and respiratory status every 6 to 12 months for up to 10 years.
Assessments every 6 to 12 months
Trial Site Locations
Total: 10 locations
1
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Actively Recruiting
2
CureDuchenne
Newport Beach, California, United States, 92660
Actively Recruiting
3
Children's Hospital of Orange County
Orange, California, United States, 92868
Actively Recruiting
4
Rare Disease Research
Atlanta, Georgia, United States, 30329
Actively Recruiting
5
University of Iowa
Iowa City, Iowa, United States, 52242
Actively Recruiting
6
Kansas University Clinical Research Center
Fairway, Kansas, United States, 66205
Actively Recruiting
7
Corewell Health
Grand Rapids, Michigan, United States, 49503
Actively Recruiting
8
Rare Disease Research Center
Hillsborough, North Carolina, United States, 27278
Actively Recruiting
9
Penn State Health
Hershey, Pennsylvania, United States, 17033
Actively Recruiting
10
Neurology Rare Disease Center
Denton, Texas, United States, 76208
Actively Recruiting
Research Team
E
Erica Rudoff
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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