Urinary Titin Biomarker in DMD
Recruiting
N/A
Interventional Study
Duchenne Muscular Dystrophy (DMD)
Becker's Muscular Dystrophy (BMD)
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Healthy Volunteers Welcome
You do not need to have the condition being studied to take part.
At a Glance
- Age
- 2 – 10
- Sex
- Male
- Study type
- Interventional
- Purpose
- Basic Science
- Participants needed
- 50 (estimated)
- Sponsor
- Children's Hospital of Philadelphia · Other
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About This Trial
A universal challenge in clinical investigation of novel therapeutics is the need for quantitative, objective biomarkers that directly address the mechanisms of disease and provide information relevant to clinically meaningful functional improvement. This has been a particular challenge in rare and slowly progressive diseases such as Duchenne Muscular Dystrophy (DMD).
The investigators hypothesiz…
Trial Locations
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Eligibility Criteria
DMD/BMD Subject Inclusion/Exclusion Criteria
Inclusion Criteria:
1. Ambulatory at screening
2. Genetically confirmed diagnosis of DMD/BMD
3. Parental/guardian permission (informed consent) for children. Child assent will also be obtained from patients ages 7 years old and older and deemed by the i…
Contacts