Duchenne Muscular DystrophyCDLinkNCT04972604
CureDuchenne Link®: A Resource for Research
An observational study of Duchenne Muscular Dystrophy and Becker Muscular Dystrophy, run by CureDuchenne at 10 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 4 weeks and older
- Conditions the study is for
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
- How many people
- 240 participants
- Where
- 10 sitesUnited States
- Study dates
- 9 Jul 2021to 12 Dec 2025
- How long will I take part?
- 2 Years
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 weeks and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Individuals with or carriers of Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD).
Who may be able to join
- 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.
Who cannot join
- Is a foster child or ward of the state.
- Is a prisoner.
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
Rare Disease Research
Atlanta, Georgia, 30329
Neurology Rare Disease Center
Denton, Texas, 76208
Kansas University Clinical Research Center
Fairway, Kansas, 66205
Corewell Health
Grand Rapids, Michigan, 49503
Penn State Health
Hershey, Pennsylvania, 17033
Rare Disease Research Center
Hillsborough, North Carolina, 27278
University of Iowa
Iowa City, Iowa, 52242
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
CureDuchenne
Newport Beach, California, 92660
Children's Hospital of Orange County
Orange, California, 92868
About this study
Official title: CureDuchenne Link®: A Resource to Support Research Studies in Duchenne and Becker Muscular Dystrophy (DMD/BMD)
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).
Read the detailed description
Individuals can participate through the CureDuchenne Link™ application (accessible via mobile device or web interface) and receive communications about research opportunities and community programs. Participation may be done using virtual methods, at a project site, and/or at community events nationwide.
All collected information will be stored in a secure, HIPAA-compliant data warehouse for approved researchers to use for studies relevant to DMD, BMD and other neuromuscular disorders. Combining health and outcomes data with biospecimens provides an impactful solution and novel resource for researchers, allowing for effective translational research.
- Keywords
- Carrier, DMD, BMD
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Duchenne and Becker muscular dystrophy | Individuals with Duchenne muscular dystrophy and Becker muscular dystrophy | |
| Carriers | Carriers of Duchenne muscular dystrophy and Becker muscular dystrophy |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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.
Measured: Upon study entry or when genetic testing results are available
Secondary outcomes (6)
Functional Status
Self reported data (questionnaire on ambulation and mobility) will be captured
Measured: Upon study entry and every 6-12 months thereafter for up to ten (10) years
North Star Ambulation Assessment (NSAA) Score
Clinically reported NSAA scores will be captured
Measured: Upon study entry and every 6-12 months thereafter for up to ten (10) years
6 Minute Walk Test (6MWT) Score
Clinically reported 6MWT scores will be captured
Measured: Upon study entry and every 6-12 months thereafter for up to ten (10) years
Corticosteroid Status
Self reported and clinically reported corticosteroid status (past and present) will be captured
Measured: Upon study entry and every 6-12 months thereafter for up to ten (10) years
Cardiac Status
Self reported and clinically reported cardiac status (past and present) will be captured
Measured: Upon study entry and every 6-12 months thereafter for up to ten (10) years
Respiratory Status
Self reported and clinically reported respiratory status (past and present) will be captured
Measured: Upon study entry and every 6-12 months thereafter for up to ten (10) years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jul 2021
- Study start9 Jul 2021
- Primary completion12 Dec 2025
- Study completion12 Dec 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 240 participants (actual)
- Target duration
- 2 Years
Sponsor and investigators
- Lead sponsor
- CureDuchenne
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Debra Miller, Principal investigator, CureDuchenne
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04972604
- Sponsor's protocol ID
- CD-2021-01
- Acronym
- CDLink
- Status last verified
- Jul 2026
- First submitted
- 21 Jun 2021
- Last updated
- 24 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 24 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- CureDuchenne Link®: A Resource to Support Research Studies in Duchenne and Becker Muscular Dystrophy (DMD/BMD)
- ClinicalTrials.gov ID
- NCT04972604
- Acronym / sponsor ID
- CDLink / CD-2021-01
- Keywords
- Carrier, DMD, BMD
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 240 participants (actual)
- Lead sponsor
- CureDuchenneUniversity, hospital or other
- Study start
- 9 Jul 2021 (actual)
- Primary completion
- 12 Dec 2025 (actual)
- Study completion
- 12 Dec 2025 (actual)
- First posted
- 22 Jul 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
Individuals can participate through the CureDuchenne Link™ application (accessible via mobile device or web interface) and receive communications about research opportunities and community programs. Participation may be done using virtual methods, at a project site, and/or at community events nationwide.
All collected information will be stored in a secure, HIPAA-compliant data warehouse for approved researchers to use for studies relevant to DMD, BMD and other neuromuscular disorders. Combining health and outcomes data with biospecimens provides an impactful solution and novel resource for researchers, allowing for effective translational research.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 2 Years
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Duchenne and Becker muscular dystrophy | – | –Individuals with Duchenne muscular dystrophy and Becker muscular dystrophy |
| Carriers | – | –Carriers of Duchenne muscular dystrophy and Becker muscular dystrophy |
Who may qualify
- Ages
- 4 weeks and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Individuals with or carriers of Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD).
Key inclusion criteria (5 listed)
- Any of the following are true:
- 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.
Key exclusion criteria (2 listed)
- Is a foster child or ward of the state.
- Is a prisoner.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Diagnosis
Time frame: Upon study entry
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
Genetic Mutation
Time frame: Upon study entry or when genetic testing results are available
Participants will be asked to provide genetic testing reports confirming their diagnosis, where available, which will be reviewed by a central genetic counselor.
6 secondary outcomes
Functional Status
Time frame: Upon study entry and every 6-12 months thereafter for up to ten (10) years
North Star Ambulation Assessment (NSAA) Score
Time frame: Upon study entry and every 6-12 months thereafter for up to ten (10) years
6 Minute Walk Test (6MWT) Score
Time frame: Upon study entry and every 6-12 months thereafter for up to ten (10) years
Corticosteroid Status
Time frame: Upon study entry and every 6-12 months thereafter for up to ten (10) years
Cardiac Status
Time frame: Upon study entry and every 6-12 months thereafter for up to ten (10) years
Respiratory Status
Time frame: Upon study entry and every 6-12 months thereafter for up to ten (10) years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jul 2026.
- CureDuchenne Link Information PageLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Debra Miller principal investigator
CureDuchenne
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Duchenne Muscular Dystrophy and Becker Muscular Dystrophy, run by CureDuchenne at 10 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Diagnosis (time frame: Upon study entry). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jul 2026.