Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT
← catalyst calendarNCT06564974 · readout ≤ 2,019 d
Sponsored by Catalyst Pharmaceuticals, Inc. (industry) · CPRX — their whole pipeline →. With ICON plc, Veeva Systems, Children's Hospital of Eastern Ontario, Medpace, Inc..
Phase
—
Status
Recruiting
Study type
Observational
Enrollment
250estimated
Sites
33
Countries
Puerto Rico, United States
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2024-09-25 | actual | When the study began enrolling |
| Primary completion | Feb 2032 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Feb 2032 | estimated | The whole study's end, after follow-up |
| First posted | 2024-08-21 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-07-20 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Duchenne Muscular Dystrophy
Intervention
- Drug: Vamorolone — also filed as AGAMREE®
Primary outcomes
what the primary-completion date above is the date OFChange in Height z-score
measured At Enrollment Visit (baseline), and at each Yearly Follow-up Visit (for up to 5 years).
Change in BMI z-score
measured At Enrollment Visit (baseline), and each Yearly Follow-up Visit (for up to 5 years)
Change in North Star Ambulatory Assessment from baseline
measured At Enrollment visit, and each Yearly Follow-up Visit (for up to 5 years).
Change in Performance of Upper Limb from baseline
measured At Enrollment Visit, at each Yearly Follow-up Visit (for up to 5 years).
Tanner stage documentation
measured At Enrollment Visit (baseline), at each Yearly Follow-up Visit (for up to 5 years).
Percentage of patients with cataract and/or glaucoma
measured At Enrollment (baseline), and during each Yearly Follow-up Visit (for up to 5 years).
Findings from lateral spine x-ray
measured At Enrollment Visit (baseline), and during each Yearly Follow-up Visit (for up to 5 years).
Findings from anterior-posterior/posterior-anterior X-ray
measured At Enrollment Visit (baseline), and at each Yearly Follow-Up Visit (for up to 5 years).
Findings from hand-wrist X-ray
measured At Enrollment Visit (baseline), and each Yearly Follow-Up Visit (for up to 5 years).
Summary statistics of bone mineral density measures assessed by dual x-ray absorptiometry
measured At Enrollment Visit (baseline) and each Yearly Follow-Up Visit (for up to 5 years).
Body Composition Mass Measures
measured At Enrollment Visit, each Yearly Follow-Up and End of Study/Early Termination (for approximately 5 years).
Summary statistics of body fat percentage and regional fat distribution ratios assessed by DXA
measured At Enrollment visit, each Yearly Follow-up visit, and End of study/Early termination (for approximately 5 years).
Findings from standard of care echocardiography
measured At Enrollment Visit, and at each Yearly Follow-up Visit (for up to 5 years).
Frequency and percentage of participants experiencing any adverse events and serious adverse events
measured From informed consent, Enrollment visit, each Yearly follow-up visit, and End of Study/Early Termination (for up to 5 years).
Findings from cardiac magnetic resonance imaging (subset of patients taking part in the cardiac sub-study only)
measured Enrollment Visit, and at each Yearly Follow-Up Visit (for up to 5 years).
Presence of cardiomyopathy assessed by echocardiography using transmural strain profile (only in a subset of patients taking part in the cardiac sub-study)
measured At Enrollment Visit (baseline), and each Yearly Follow-up Visit (for up to 5 years) or Early Termination.
Permalink · CPRX's whole pipeline · Catalyst calendar · Every registered study · What changed