A resource hub for Duchenne and Becker muscular dystrophy research
CureDuchenne Link®: A Resource to Support Research Studies in Duchenne and Becker Muscular Dystrophy (DMD/BMD)
This study is creating a resource hub to gather and share important health information from people with Duchenne and Becker muscular dystrophy to help researchers find better treatments.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 5000 (estimated) |
| Ages | 4 Weeks and up |
| Sex | All |
| Sponsor | CureDuchenne Academic / other |
| Locations | 10 sites (Little Rock, Arkansas and 9 other locations) |
| Trial ID | NCT04972604 on ClinicalTrials.gov |
What this trial studies
CureDuchenne Link is an observational initiative that creates a comprehensive data hub integrating biospecimens, clinical data, and self-reported information from individuals diagnosed with Duchenne Muscular Dystrophy (DMD) or Becker Muscular Dystrophy (BMD), as well as carriers. Participants, including children over 4 weeks old, can join through a mobile app or web interface, facilitating access to research opportunities and community programs. The collected data will be securely stored in a HIPAA-compliant warehouse for researchers to utilize in studies related to DMD, BMD, and other neuromuscular disorders, promoting effective translational research.
Who should consider this trial
Good fit: Ideal candidates include individuals diagnosed with DMD or BMD, as well as carriers of these conditions, aged 4 weeks and older.
Not a fit: Patients who are foster children, wards of the state, or prisoners will not benefit from this study.
Why it matters
Potential benefit: If successful, this initiative could significantly enhance research efforts and lead to improved treatments for DMD and BMD.
How similar studies have performed: Other studies utilizing similar data integration approaches have shown promise in advancing research, making this initiative a valuable resource.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Any of the following are true: 1. Currently has a confirmed diagnosis of DMD/BMD based on genetic testing, muscle biopsy, or clinical diagnosis. 2. Currently has a confirmed diagnosis of carrier status for DMD/BMD based on genetic testing. 2. Parent/guardian (for minor participants) or participant gives informed consent and/or assent as required by local regulations. 3. Is age 4 weeks or older at the time of consent. Exclusion Criteria: 1. Is a foster child or ward of the state. 2. Is a prisoner.
Where this trial is running
Little Rock, Arkansas and 9 other locations
- Arkansas Children's Hospital — Little Rock, Arkansas, United States (Recruiting)
- CureDuchenne — Newport Beach, California, United States (Recruiting)
- Children's Hospital of Orange County — Orange, California, United States (Recruiting)
- Rare Disease Research — Atlanta, Georgia, United States (Recruiting)
- University of Iowa — Iowa City, Iowa, United States (Recruiting)
- Kansas University Clinical Research Center — Fairway, Kansas, United States (Recruiting)
- Corewell Health — Grand Rapids, Michigan, United States (Recruiting)
- Rare Disease Research Center — Hillsborough, North Carolina, United States (Recruiting)
- Penn State Health — Hershey, Pennsylvania, United States (Recruiting)
- Neurology Rare Disease Center — Denton, Texas, United States (Recruiting)
Study contacts
- Principal investigator: Debra Miller — CureDuchenne
- Study coordinator: Erica Rudoff
- Email: support@cureduchennelink.org
- Phone: 888-235-4655
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.