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

NCT06564974 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 250

Last updated 2026-02-27

No results posted yet for this study

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).

Conditions

Interventions

DRUG

Vamorolone

Study treatment is AGAMREE®, which is commercially available as an oral suspension.

Sponsors & Collaborators

  • ICON Medical Imaging

    collaborator UNKNOWN

  • ICON plc

    collaborator INDUSTRY

  • Veeva Systems

    collaborator INDUSTRY

  • Children's Hospital of Eastern Ontario

    collaborator OTHER

  • Medpace, Inc.

    collaborator INDUSTRY

  • Catalyst Pharmaceuticals, Inc.

    lead INDUSTRY

Principal Investigators

  • William Andrews, MD · Catalyst Pharmaceuticals

  • Aravindham Veerapandiyan, MD · Arkansas Childrens Hospital

Eligibility

Min Age
2 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-09-25
Primary Completion
2032-02-29
Completion
2032-02-29
FDA Drug
Yes

Countries

  • United States
  • Puerto Rico

Study Locations

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Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06564974 on ClinicalTrials.gov