Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT
Part of paid clinical trials in Phoenix, Arizona.
- Sponsor
- Catalyst Pharmaceuticals, Inc.
- Study ID
- NCT06564974
- Status
- Recruiting
Conditions
Eligibility Criteria
- Sex
- MALE
- Age
- 2 Years - N/A
- Healthy Volunteers
- Not accepted
Interventions
- Vamorolone — DRUGStudy treatment is AGAMREE®, which is commercially available as an oral suspension.
Study Details
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).
Key Dates
- Start date
- Sep 25, 2024
- Status verified
- Sep 2025
- Primary completion
- Feb 29, 2032
- Completion
- Feb 29, 2032
Study Design
- Enrollment
- 250 participants (estimated)
Primary Outcome Measure
Change in Height z-score [ Time Frame: At Enrollment Visit (baseline), and at each Yearly Follow-up Visit (for up to 5 years). ]
Central Contacts
- Syune Nersisyan, PhD(305) 420-3200
Locations (25)
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