Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy.

Catalyst Pharmaceuticals

Status: Enrolling

Conditions: Duchenne Muscular Dystrophy

Treatments: Drug: Vamorolone

Study type: Observational

Funder types: Industry

Identifiers

NCT06564974

DMD-001

Details and patient eligibility

About

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.

Full 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).

Enrollment: 250 estimated patients

Sex: Male

Ages: 2+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. 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.
2. Patient and/or parent/guardian are willing and able to complete QoL questionnaires.
3. Male patients at least 2 years old.
4. 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).
5. Currently on treatment with AGAMREE®.

Exclusion criteria

1. Any contraindication to AGAMREE® or medical condition, which, in the opinion of the investigator, would affect registry participation, performance, or interpretation of registry assessments.