Clinical trial NCT04972604: CureDuchenne Link®: A Resource for Research
Official title: CureDuchenne Link®: A Resource to Support Research Studies in Duchenne and Becker Muscular Dystrophy (DMD/BMD)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Duchenne Muscular Dystrophy
Becker Muscular Dystrophy
Keywords: Carrier
DMD
BMD
Lead sponsor: CureDuchenne
Sponsor class: OTHER
First posted: 2021-07-22
Enrollment: 5000
Sex: ALL
Minimum age (years): 4
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Individuals with or carriers of Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD).

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).

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.

Eligibility 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.

Primary outcomes:
measure: Diagnosis
timeFrame: Upon study entry
measure: Genetic Mutation
timeFrame: Upon study entry or when genetic testing results are available
Secondary outcomes:
measure: Functional Status
timeFrame: Upon study entry and every 6-12 months thereafter for up to ten (10) years
measure: North Star Ambulation Assessment (NSAA) Score
timeFrame: Upon study entry and every 6-12 months thereafter for up to ten (10) years
measure: 6 Minute Walk Test (6MWT) Score
timeFrame: Upon study entry and every 6-12 months thereafter for up to ten (10) years
measure: Corticosteroid Status
timeFrame: Upon study entry and every 6-12 months thereafter for up to ten (10) years
measure: Cardiac Status
timeFrame: Upon study entry and every 6-12 months thereafter for up to ten (10) years
measure: Respiratory Status
timeFrame: Upon study entry and every 6-12 months thereafter for up to ten (10) years
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Newport Beach
country: United States
facility: CureDuchenne
state: California
city: Orange
country: United States
facility: Children's Hospital of Orange County
state: California
city: Atlanta
country: United States
facility: Rare Disease Research
state: Georgia
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Fairway
country: United States
facility: Kansas University Clinical Research Center
state: Kansas
city: Grand Rapids
country: United States
facility: Corewell Health
state: Michigan
city: Hillsborough
country: United States
facility: Rare Disease Research Center
state: North Carolina
city: Hershey
country: United States
facility: Penn State Health
state: Pennsylvania
city: Denton
country: United States
facility: Neurology Rare Disease Center
state: Texas
Study officials:
affiliation: CureDuchenne
name: Debra Miller
role: PRINCIPAL_INVESTIGATOR
References: