Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy.
NCT ID: NCT06564974
Last Updated: 2025-08-05
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
250 participants
OBSERVATIONAL
2024-07-31
2030-11-30
Brief Summary
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Detailed Description
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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).
Conditions
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Study Design
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COHORT
PROSPECTIVE
Interventions
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Vamorolone
Patients on treatment with Vamorolone (AGAMREE®) Dosage: Oral Suspension: 40 mg/mL . Per package insert, the recommended dosage is 6 mg/kg taken orally once daily, up to a maximum daily dosage of 300 mg for patients weighing more than 50 kg.
Decrease dosage gradually when administered for more than one week.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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®.
Exclusion Criteria
2 Years
MALE
No
Sponsors
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Catalyst Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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William Andrews, MD
Role: STUDY_DIRECTOR
Catalyst Pharmaceuticals
Aravindham Veerapandiyan, MD
Role: PRINCIPAL_INVESTIGATOR
Arkansas Childrens Hospital
Locations
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Phoenix Children's Hospital
Phoenix, Arizona, United States
Arkansas Childrens Hospital
Little Rock, Arkansas, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
Stanford University
Palo Alto, California, United States
University of California, Davis
Sacramento, California, United States
University of Florida Clinical and Translational Science Institue
Gainesville, Florida, United States
Nicklaus Children's Hospital
Miami, Florida, United States
Nemours Children's Hospital
Orlando, Florida, United States
Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Indiana University Health - Riley Hospital for Children
Indianapolis, Indiana, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
University of Massachusetts Memorial Medical Center
North Worcester, Massachusetts, United States
Helen DeVos Children's Hospital
Grand Rapids, Michigan, United States
Atrium Health Neurosciences Institute
Charlotte, North Carolina, United States
Duke University Medical Center and Childrens Health Center
Durham, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Penn State Milton S. Hershey Medical Center- Penn State Hershey Neuroscience Institute
Hershey, Pennsylvania, United States
Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
Neurology Rare Disease Center - Neurology & Neuromuscular Care Center
Denton, Texas, United States
The University of Texas Health Science Center at San Antonio
San Antonio, Texas, United States
University of Virginia Health System (UVAHS) - Pediatric Neuromuscular Center
Charlottesville, Virginia, United States
Seattle Children's Hospital
Seattle, Washington, United States
San Jorge Children's Hospital
San Juan, Puerto Rico, Puerto Rico
FDI Clinical Research
San Juan, Puerto Rico, Puerto Rico
Countries
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Central Contacts
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Facility Contacts
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Jenna Klotz, MD
Role: primary
Other Identifiers
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DMD-001
Identifier Type: -
Identifier Source: org_study_id
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