CureDuchenne Link®: A Resource for Research
CDLink
1 other identifier
observational
5,000
1 country
10
Brief Summary
CureDuchenne link is a data hub comprised of integrated biospecimens, clinical data, and self- and/or caregiver-reported information from participants. Anyone over 4 weeks old who has been diagnosed with DMD or BMD or who is a carrier of DMD or BMD can join. Parents or legal guardians can sign up their child(ren).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Jul 2021
Longer than P75 for all trials
10 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 21, 2021
CompletedStudy Start
First participant enrolled
July 9, 2021
CompletedFirst Posted
Study publicly available on registry
July 22, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 9, 2031
ExpectedStudy Completion
Last participant's last visit for all outcomes
July 9, 2031
August 20, 2024
August 1, 2024
10 years
June 21, 2021
August 19, 2024
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Diagnosis
There is no intervention in this project. Participants will provide documentation to support their diagnosis of Duchenne muscular dystrophy, Becker muscular dystrophy, or a carrier of these mutations
Upon study entry
Genetic Mutation
Participants will be asked to provide genetic testing reports confirming their diagnosis, where available, which will be reviewed by a central genetic counselor.
Upon study entry or when genetic testing results are available
Secondary Outcomes (6)
Functional Status
Upon study entry and every 6-12 months thereafter for up to ten (10) years
North Star Ambulation Assessment (NSAA) Score
Upon study entry and every 6-12 months thereafter for up to ten (10) years
6 Minute Walk Test (6MWT) Score
Upon study entry and every 6-12 months thereafter for up to ten (10) years
Corticosteroid Status
Upon study entry and every 6-12 months thereafter for up to ten (10) years
Cardiac Status
Upon study entry and every 6-12 months thereafter for up to ten (10) years
- +1 more secondary outcomes
Study Arms (2)
Duchenne and Becker muscular dystrophy
Individuals with Duchenne muscular dystrophy and Becker muscular dystrophy
Carriers
Carriers of Duchenne muscular dystrophy and Becker muscular dystrophy
Eligibility Criteria
Individuals with or carriers of Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD).
You may qualify if:
- Any of the following are true:
- Currently has a confirmed diagnosis of DMD/BMD based on genetic testing, muscle biopsy, or clinical diagnosis.
- Currently has a confirmed diagnosis of carrier status for DMD/BMD based on genetic testing.
- Parent/guardian (for minor participants) or participant gives informed consent and/or assent as required by local regulations.
- Is age 4 weeks or older at the time of consent.
You may not qualify if:
- Is a foster child or ward of the state.
- Is a prisoner.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- CureDuchennelead
Study Sites (10)
Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
CureDuchenne
Newport Beach, California, 92660, United States
Children's Hospital of Orange County
Orange, California, 92868, United States
Rare Disease Research
Atlanta, Georgia, 30329, United States
University of Iowa
Iowa City, Iowa, 52242, United States
Kansas University Clinical Research Center
Fairway, Kansas, 66205, United States
Corewell Health
Grand Rapids, Michigan, 49503, United States
Rare Disease Research Center
Hillsborough, North Carolina, 27278, United States
Penn State Health
Hershey, Pennsylvania, 17033, United States
Neurology Rare Disease Center
Denton, Texas, 76208, United States
Related Links
Biospecimen
Blood, Urine, Saliva, Muscle and Skin will be collected. Sample collection will be optional.
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Debra Miller
CureDuchenne
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Target Duration
- 10 Years
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 21, 2021
First Posted
July 22, 2021
Study Start
July 9, 2021
Primary Completion (Estimated)
July 9, 2031
Study Completion (Estimated)
July 9, 2031
Last Updated
August 20, 2024
Record last verified: 2024-08
Data Sharing
- IPD Sharing
- Will share
After a thorough application process, data will be shared to qualified researchers. Approved researchers will complete all required CDA/data transfer agreements with CureDuchenne. Once complete, they will be given access to a limited dataset with direct identifiers removed.