CureDuchenne Link®: A Resource for Research

Duchenne Muscular Dystrophy Clinical Trial

— CDLink  

Official title:

CureDuchenne Link®: A Resource to Support Research Studies in Duchenne and Becker Muscular Dystrophy (DMD/BMD)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04972604
• Other study ID #: CD-2021-01
• Status: Recruiting
• Start date: July 9, 2021
• Est. completion date: July 9, 2031

Study information

• Verified date: December 2023
• Source: CureDuchenne
• Contact: Erica Rudoff
• Phone: 888-235-4655
• Email: support[@]cureduchennelink.org
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical 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).

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 5000
• Est. completion date: July 9, 2031
• Est. primary completion date: July 9, 2031
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Weeks and older

Eligibility

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.

Related Conditions & MeSH terms

• Becker Muscular Dystrophy
• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Locations

Country	Name	City	State
United States	Rare Disease Research	Atlanta	Georgia
United States	Neurology Rare Disease Center	Denton	Texas
United States	Kansas University Clinical Research Center	Fairway	Kansas
United States	Corewell Health	Grand Rapids	Michigan
United States	Penn State Health	Hershey	Pennsylvania
United States	University of Iowa	Iowa City	Iowa
United States	Arkansas Children's Hospital	Little Rock	Arkansas
United States	CureDuchenne	Newport Beach	California

Sponsors (1)

Lead Sponsor	Collaborator
CureDuchenne

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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	Functional Status	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
Secondary	North Star Ambulation Assessment (NSAA) Score	Clinically reported NSAA scores will be captured	Upon study entry and every 6-12 months thereafter for up to ten (10) years
Secondary	6 Minute Walk Test (6MWT) Score	Clinically reported 6MWT scores will be captured	Upon study entry and every 6-12 months thereafter for up to ten (10) years
Secondary	Corticosteroid Status	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
Secondary	Cardiac Status	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
Secondary	Respiratory Status	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

External Links

•   CureDuchenne Link Information Page
•   CureDuchenne Link Registration Page