Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy [SUpplemental Patient dMd assessMents Investigating ouTcomes (SUMMIT)]
Catalyst Pharmaceuticals, Inc.
Summary
The goal of this study is to collect additional information on the safety of long-term treatment with AGAMREE® and to explore long-term clinical impact of AGAMREE® on quality of life, as assessed by standardized patient-reported outcome measures (QoL questionnaires) in male patients aged 2 years and older with Duchenne muscular dystrophy (DMD).
Description
This is a multi-center, observational, prospective, longitudinal registry study, designed to collect safety data and to explore long-term clinical impact of AGAMREE® on quality of life, as assessed by standardized patient-reported outcome measures (QoL questionnaires) in male patients aged 2 years and older with DMD. Primary Objective is to collect additional information on the safety of long-term treatment with AGAMREE® in male patients aged 2 years and older with Duchenne muscular dystrophy (DMD) as determined by: Growth parameters Body mass index (BMI) Musculoskeletal assessments Ophthalm…
Eligibility
- Age range
- 2+ years
- Sex
- Male
- Healthy volunteers
- No
Inclusion Criteria: 1. Patient or parent/legal guardian is willing and able to provide written informed consent once the nature of the registry has been explained and prior to 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 anti- dystrophin antibodies 3, 1, or 2, or dystrophin immunohistochemistry or western blot). 5. Patient has a current, active prescription for, or i…
Interventions
- DrugVamorolone
Study treatment is AGAMREE®, which is commercially available as an oral suspension.
Locations (27)
- Phoenix Children's HospitalPhoenix, Arizona
- Arkansas Childrens HospitalLittle Rock, Arkansas
- Children's Hospital Los AngelesLos Angeles, California
- Stanford UniversityPalo Alto, California
- University of California, DavisSacramento, California
- University of Florida Clinical and Translational Science InstitueGainesville, Florida