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

Description

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.

Conditions

Duchenne Muscular Dystrophy

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Study Overview

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Study Details

Study overview

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.

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

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

Condition
Duchenne Muscular Dystrophy
Intervention / Treatment

-

Contacts and Locations

Little Rock

Arkansas Childrens Hospital, Little Rock, Arkansas, United States, 72202

North Worcester

University of Massachusetts Memorial Medical Center, North Worcester, Massachusetts, United States, 01655

Durham

Duke University Medical Center and Childrens Health Center, Durham, North Carolina, United States, 27710

Hershey

Penn State Milton Hershey Medical Center- Penn State Hershey Neuroscience Institute, Hershey, Pennsylvania, United States, 17033

Denton

Neurology Rare Disease Center - Neurology & Neuromuscular Care Center, Denton, Texas, United States, 76208

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • 1. Patient or parent/guardian willing and able to provide written informed consent after the nature of the registry has been explained and before 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 antidystrophin antibodies 3, 1, or 2, or dystrophin immunohistochemistry or western blot).
  • 5. Currently on treatment with AGAMREE®.
  • 1. Any contraindication to AGAMREE® or medical condition, which, in the opinion of the investigator, would affect registry participation, performance, or interpretation of registry assessments.

Ages Eligible for Study

2 Years to

Sexes Eligible for Study

MALE

Accepts Healthy Volunteers

No

Collaborators and Investigators

Catalyst Pharmaceuticals, Inc.,

Gary Ingenito, MD, PhD, STUDY_DIRECTOR, Catalyst Pharmaceuticals

Aravindham Veerapandiyan, MD, PRINCIPAL_INVESTIGATOR, Arkansas Childrens Hospital

Study Record Dates

2030-11