RECRUITING

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

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Conditions

Duchenne Muscular DystrophyNeuromuscularAGAMREE®

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2024-07-31
Study Completion:2030-11
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06564974

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.

Ages Eligible for Study:2 Years
Sexes Eligible for Study:MALE
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. 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. 2. Patient and/or parent/guardian are willing and able to complete QoL questionnaires.
  3. 3. Male patients at least 2 years old.
  4. 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. 5. Currently on treatment with AGAMREE®.
  1. 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.

Contacts and Locations

Study Contact

Nadeem Khaliq, MBBS (M.D)
CONTACT
1-844-347-3277
nadeem.khaliq@catalystpharma.com
Syune Nersisyan, PhD
CONTACT
1-844-347-3277
snersisyan@catalystpharma.com

Principal Investigator

Gary Ingenito, MD, PhD
STUDY_DIRECTOR
Catalyst Pharmaceuticals
Aravindham Veerapandiyan, MD
PRINCIPAL_INVESTIGATOR
Arkansas Childrens Hospital

Study Locations (Sites)

Arkansas Childrens Hospital
Little Rock, Arkansas, 72202
United States
University of Massachusetts Memorial Medical Center
North Worcester, Massachusetts, 01655
United States
Duke University Medical Center and Childrens Health Center
Durham, North Carolina, 27710
United States
Penn State Milton Hershey Medical Center- Penn State Hershey Neuroscience Institute
Hershey, Pennsylvania, 17033
United States
Neurology Rare Disease Center - Neurology & Neuromuscular Care Center
Denton, Texas, 76208
United States

Collaborators and Investigators

Sponsor: Catalyst Pharmaceuticals, Inc.

  • Gary Ingenito, MD, PhD, STUDY_DIRECTOR, Catalyst Pharmaceuticals
  • Aravindham Veerapandiyan, MD, PRINCIPAL_INVESTIGATOR, Arkansas Childrens Hospital

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-07-31
Study Completion Date2030-11

Study Record Updates

Study Start Date2024-07-31
Study Completion Date2030-11

Terms related to this study

Keywords Provided by Researchers

  • Duchenne Muscular Dystrophy
  • Neuromuscular
  • AGAMREE®

Additional Relevant MeSH Terms

  • Duchenne Muscular Dystrophy