CureDuchenne Link®: A Resource for Research (CDLink)

December 8, 2023 updated by: CureDuchenne

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).

Study Overview

Status

Recruiting

Conditions

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 Type

Observational

Enrollment (Estimated)

5000

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Recruiting
        • Arkansas Children's Hospital
        • Contact:
    • California
      • Newport Beach, California, United States, 92660
        • Recruiting
        • CureDuchenne
        • Contact:
        • Principal Investigator:
          • Debra Miller
    • Georgia
      • Atlanta, Georgia, United States, 30329
    • Iowa
    • Kansas
      • Fairway, Kansas, United States, 66205
        • Recruiting
        • Kansas University Clinical Research Center
        • Principal Investigator:
          • Jeffrey Statland, MD
        • Contact:
    • Michigan
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
    • Texas
      • Denton, Texas, United States, 76208

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

4 weeks and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Individuals with or carriers of Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD).

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
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 the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Upon study entry
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.
Upon study entry or when genetic testing results are available

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Functional Status
Time Frame: Upon study entry and every 6-12 months thereafter for up to ten (10) years
Self reported data (questionnaire on ambulation and mobility) will be captured
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
Clinically reported NSAA scores will be captured
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
Clinically reported 6MWT scores will be captured
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
Self reported and clinically reported corticosteroid status (past and present) will be captured
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
Self reported and clinically reported cardiac status (past and present) will be captured
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
Self reported and clinically reported respiratory status (past and present) will be captured
Upon study entry and every 6-12 months thereafter for up to ten (10) years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

CureDuchenne

Investigators

  • Principal Investigator: Debra Miller, CureDuchenne

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 9, 2021

Primary Completion (Estimated)

July 9, 2031

Study Completion (Estimated)

July 9, 2031

Study Registration Dates

First Submitted

June 21, 2021

First Submitted That Met QC Criteria

July 12, 2021

First Posted (Actual)

July 22, 2021

Study Record Updates

Last Update Posted (Estimated)

December 12, 2023

Last Update Submitted That Met QC Criteria

December 8, 2023

Last Verified

December 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CD-2021-01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

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.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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