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

Last updated: March 28, 2025
Sponsor: Catalyst Pharmaceuticals, Inc.
Overall Status: Active - Recruiting

Phase

N/A

Condition

Muscular Dystrophy

Treatment

Vamorolone

Clinical Study ID

NCT06564974
DMD-001
  • Ages > 2
  • Male

Study Summary

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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Patient or parent/guardian willing and able to provide written informed consentafter the nature of the registry has been explained and before the start of anyregistry-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 absentdystrophin staining to antidystrophin antibodies 3, 1, or 2, or dystrophinimmunohistochemistry or western blot).

  5. Currently on treatment with AGAMREE®.

Exclusion

Exclusion Criteria:

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

Study Design

Total Participants: 250
Treatment Group(s): 1
Primary Treatment: Vamorolone
Phase:
Study Start date:
July 31, 2024
Estimated Completion Date:
November 30, 2030

Study 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).

Connect with a study center

  • FDI Clinical Research

    San Juan, 00927
    Puerto Rico

    Active - Recruiting

  • Arkansas Childrens Hospital

    Little Rock, Arkansas 72202
    United States

    Active - Recruiting

  • Nicklaus Children's Hospital

    Miami, Florida 33155
    United States

    Active - Recruiting

  • Omer Hamid

    Orlando, Florida 32827
    United States

    Active - Recruiting

  • Lurie Childrens Hospital of Chicago

    Chicago, Illinois 60611
    United States

    Active - Recruiting

  • University of Massachusetts Memorial Medical Center

    North Worcester, Massachusetts 01655
    United States

    Active - Recruiting

  • Duke University Medical Center and Childrens Health Center

    Durham, North Carolina 27710
    United States

    Active - Recruiting

  • Penn State Milton Hershey Medical Center- Penn State Hershey Neuroscience Institute

    Hershey, Pennsylvania 17033
    United States

    Active - Recruiting

  • Children's Hospital of Philadelphia (CHOP)

    Philadelphia, Pennsylvania 19104
    United States

    Active - Recruiting

  • Neurology Rare Disease Center - Neurology & Neuromuscular Care Center

    Denton, Texas 76208
    United States

    Active - Recruiting

  • University of Virginia Health System (UVAHS) - Pediatric Neuromuscular Center

    Charlottesville, Virginia 22903
    United States

    Active - Recruiting

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