BridgeMD
New treatment option for Duchenne Muscular Dystrophy (DMD)
Official title Urinary Titin Biomarker in DMD
ClinicalTrials.gov ID: NCT07332013
What this study is testing
- What it's testing
- 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).
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 10, men only. Healthy volunteers may be eligible.
You may be able to join if
- DMD/BMD Subject Inclusion/
You likely can't join if
- Inclusion Criteria:
- Ambulatory at screening
- Genetically confirmed diagnosis of DMD/BMD
- Parental/guardian permission (informed consent) for children. Child assent will also be obtained from patients ages 7 years old and older and deemed...
- 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
See the full eligibility criteria
- DMD/BMD Subject Inclusion/
- Inclusion Criteria:
- Ambulatory at screening
- Genetically confirmed diagnosis of DMD/BMD
- 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
- 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:
- Healthy children without DMD, BMD, or other significant chronic medical disease
- Ambulatory at Screening, defined as able to walk independently without assistive devices
- 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.
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Philadelphia, Pennsylvania, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 2 years to 10 years. Healthy volunteers may be eligible. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Philadelphia, Pennsylvania, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.