| NCT ID | NCT07332013 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Children's Hospital of Philadelphia |
| Condition | Duchenne Muscular Dystrophy (DMD) |
| Study Type | INTERVENTIONAL |
| Enrollment | 50 participants |
| Start Date | 2026-03-04 |
| Primary Completion | 2029-12 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
This trial targets 50 participants in total. It began in 2026-03-04 with a primary completion date of 2029-12.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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 hypothesize that urinary N-terminal fragment of titin (NTFT) corresponding to activity level/intensity will define a high-precision, non-invasive biomarker of systemic muscle injury to enable serial measurements of efficacy and safety in the clinical investigation of gene therapy for DMD and other myopathies. This should provide a valuable exploratory, secondary and eventually primary outcome measure of therapeutic efficacy to minimize the enrollment size in informative early phase and pivotal clinical trials.
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 investigator to be neurodevelopmentally appropriate 4. Access to electricity and a freezer in the home, in order to utilize the provided device and store collected samples Exclusion Criteria: * Non-ambulatory at Screening, defined as unable to walk independently and needing assistive devices * Female patients * Parental/guardian unable to provide informed consent Healthy Control Subject Inclusion/Exclusion Criteria Inclusion criteria: 1. Healthy children without DMD, BMD, or other significant chronic medical disease 2. Ambulatory at Screening, defined as able to walk independently without assistive devices 3. Parental/guardian permission (informed consent). Child assent will also be obtained from patients aged 7 years and older and deemed by the investigator to be neurodevelopmentally appropriate. 4. Access to electricity and a freezer in the home, in order to utilize the provided device and store collected samples Exclusion criteria: * Non-ambulatory at Screening, defined as unable to walk independently and needing assistive devices * Female patients * Parental/guardian unable to provide informed consent
Contact & Investigator
Sabrina Yum, MD
PRINCIPAL INVESTIGATOR
Children's Hospital of Philadelphia
Frequently Asked Questions
Who can join the NCT07332013 clinical trial?
This trial is open to male participants only, aged 2 Years or older, up to 10 Years, studying Duchenne Muscular Dystrophy (DMD). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT07332013 currently recruiting?
Yes, NCT07332013 is actively recruiting participants. Contact the research team at odonnellcs@chop.edu for enrollment information.
Where is the NCT07332013 trial being conducted?
This trial is being conducted at Philadelphia, United States.
Who is sponsoring the NCT07332013 clinical trial?
NCT07332013 is sponsored by Children's Hospital of Philadelphia. The principal investigator is Sabrina Yum, MD at Children's Hospital of Philadelphia. The trial plans to enroll 50 participants.