Overview
The goal of this observational study is to follow patients being treated with the FDA approved drug AGAMREE® in male patients 2 years of age or older with Duchenne's Muscular Dystrophy for long term safety and quality of life.
Description
This is a multi-center, observational, prospective, longitudinal registry designed to collect data in male patients aged 2 years and older with DMD treated with AGAMREE®.
This registry will be conducted in the US, at approximately 25 sites known to treat and follow patients with DMD. The registry plans to enroll approximately 250 male patients aged 2 years and older with DMD.
Evaluations will include:
- Growth parameters
- Body mass index (BMI)
- Vital Signs
- Physical Exam
- Laboratory (Chemistry and Hematology)
- North Star Ambulatory Assessment (NSAA)
- Performance of Upper Limb (PUL)
- Cardiovascular status
- Fractures
- Bone density
- Puberty
- Quality of life (QoL)
- Adverse events (AEs)
Patients will be followed for approximately 5 years in the registry and will return to the site for Yearly Follow-up Visits (+/- 30 days) for registry assessments. Information on standard of care treatment and procedures for management of DMD will also be collected. Patients and/or their parents/legal guardians will be asked to complete paper QoL questionnaires at enrollment and at each Yearly Follow-up Visit (+/- 30 days).
Eligibility
Inclusion Criteria:
- Patient or parent/guardian willing and able to provide written informed consent after the nature of the registry has been explained and before the start of any registry-related procedures.
- Patient and/or parent/guardian are willing and able to complete QoL questionnaires.
- Male patients at least 2 years old.
- Confirmed diagnosis of DMD (via genetic testing or muscle biopsy with absent dystrophin staining to antidystrophin antibodies 3, 1, or 2, or dystrophin immunohistochemistry or western blot).
- Currently on treatment with AGAMREE®.
Exclusion Criteria:
- Any contraindication to AGAMREE® or medical condition, which, in the opinion of the investigator, would affect registry participation, performance, or interpretation of registry assessments.