Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-24 @ 6:34 PM
Ignite Modification Date:
2025-12-24 @ 6:34 PM
NCT ID:
NCT04322357
Eligibility Criteria:
Inclusion Criteria:
* Diagnosis of DMD confirmed by 1) clinical history with features before the age of five, 2) physical examination, 3) elevated serum creatine kinase level and 4) absence of dystrophin expression, as determined by immunostain or Western blot (\<2%) and/or DNA confirmation of dystrophin mutation.
* Age 5.0 to 9 years: a lower age limit of 5.0 years is selected as children younger than that are likely unable to cooperate and comply with all of the exercise measures as needed. An upper age limit of 9 years has been set as boys with DMD tend to reach a rapid progression into a late ambulatory phase soon after this age.
* Ambulatory at the time of the first visit, defined as the ability to walk for at least 100 m without an external assistive device and able to climb four stairs.
* Aim 1 only: GC-naïve at baseline (and prior 6 months)
* Aim 2 only: on stable daily GC regimen for 6 months prior to baseline
Exclusion Criteria:
* Contraindication to an MR examination (e.g. aneurysm clip, severe claustrophobia, magnetic implants)
* Presence of unstable medical problems, significant concomitant illness including cardiomyopathy or cardiac conduction abnormalities
* Presence of a secondary condition that impacts muscle function or muscle metabolism (e.g. myasthenia gravis, endocrine disorder, mitochondrial disease)
* Presence of a secondary condition leading to developmental delay or impaired motor control (e.g. cerebral palsy)
* Presence of an unstable medical condition (e.g. uncontrolled seizure disorder)
* Behavioral problems causing an inability to cooperate during testing or understand exercise instruction
* Participation in other forms of drug or gene therapy during the period of the study
Healthy Volunteers:
False
Sex:
MALE
Minimum Age:
5 Years
Maximum Age:
9 Years
Study:
NCT04322357
Study Brief:
Protocol Section:
NCT04322357