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

NCT06564974 | Recruiting FDA Drug

• 1 other identifier: DMD-001
• Study Type: observational
• Target: 250
• Locations: 2 countries
• Sites: 27

Brief 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

Duchenne Muscular Dystrophy

Keywords

Duchenne Muscular Dystrophy; Neuromuscular; AGAMREE®; Vamorolone; DMD; Muscular Dystrophy

Outcome Measures

Primary Outcomes (16)

• Change in Height z-score

  Standing height in participants 2 years of age and older will be measured using a stadiometer mounted at a right angle between a level floor and against a straight, vertical surface. When transitioning from recumbent length to standing height measurements in participants between 2 to 3 years of age, measure both length and height. If collecting standing height measurements is not feasible, height will be estimated using arm span measurements. To estimate the height, the arm span will be measured from fingertip to fingertip with arms fully extended horizontally. The same standardized technique will be used at all study visits.

  At Enrollment Visit (baseline), and at each Yearly Follow-up Visit (for up to 5 years).

• Change in BMI z-score

  Weight will be measured according to the site's standard of care procedures. For ambulatory patients, this typically involves a standing scale; for non-ambulatory patients, a wheelchair or bed scale may be used. Sites should document the method used at each visit.

  At Enrollment Visit (baseline), and each Yearly Follow-up Visit (for up to 5 years)

• Change in North Star Ambulatory Assessment from baseline

  The North Star Ambulatory Assessment is a clinical assessment scale specifically designed to measure functional ability in ambulant male patients with DMD. The North Star Ambulatory Assessment consists of 17 scored items and 2 timed tests, including the Time to Run/Walk Test and the Time to Stand Test. The Time to Run/Walk Test measures the time (in seconds) that it takes the patient to run or walk 10 meters. The Time to Stand Test measures the time (in seconds) required for the patient to stand in an erect position from supine (floor). Patients should be barefoot and comfortably clothed. North Star Ambulatory Assessment score range is 0 to 34, with higher scores indicating better ambulatory function.

  At Enrollment visit, and each Yearly Follow-up Visit (for up to 5 years).

• Change in Performance of Upper Limb from baseline

  The Performance of Upper Limb assessment is an observer-rated measure of upper-limb function in individuals with Duchenne muscular dystrophy. It includes an entry item to determine the participant's starting functional level and a series of tasks evaluating shoulder, mid-level (elbow), and distal (wrist/hand) abilities. Total scores reflect the participant's ability to perform defined functional movements, with higher scores indicating better function.

  At Enrollment Visit, at each Yearly Follow-up Visit (for up to 5 years).

• Tanner stage documentation

  Pubertal development will be assessed by a physician using Tanner Stages assessment. This assessment uses Male External Genitalia Scale. Stage 1: Testicular volume \< 4 ml or long axis \< 2.5 cm Stage 2: 4 ml-8 ml (or 2.5 to 3.3 cm long), 1st pubertal sign in males Stage 3: 9 ml-12 ml (or 3.4 to 4.0 cm long) Stage 4: 15-20 ml (or 4.1 to 4.5 cm long) Stage 5: \> 20 ml (or \> 4.5 cm long) Pubic Hair Scale Stage 1: No hair Stage 2: Downy hair Stage 3: Scant terminal hair Stage 4: Terminal hair that fills the entire triangle overlying the pubic region Stage 5: Terminal hair that extends beyond the inguinal crease onto the thigh Pubertal development assessments will be discontinued when the patient has completed puberty.

  At Enrollment Visit (baseline), at each Yearly Follow-up Visit (for up to 5 years).

• Percentage of patients with cataract and/or glaucoma

  Presence of cataract and glaucoma will be assessed by an optometrist or ophthalmologist.

  At Enrollment (baseline), and during each Yearly Follow-up Visit (for up to 5 years).

• Findings from lateral spine x-ray

  Lateral spine x-ray will be performed to evaluate vertebral fractures and for spine deformities.

  At Enrollment Visit (baseline), and during each Yearly Follow-up Visit (for up to 5 years).

• Findings from anterior-posterior/posterior-anterior X-ray

  Anterior-posterior/Posterior-anterior spine x-ray will be performed to evaluate spine deformities.

  At Enrollment Visit (baseline), and at each Yearly Follow-Up Visit (for up to 5 years).

• Findings from hand-wrist X-ray

  Posteroanterior hand x-ray will be performed for the bone age assessment.

  At Enrollment Visit (baseline), and each Yearly Follow-Up Visit (for up to 5 years).

• Summary statistics of bone mineral density measures assessed by dual x-ray absorptiometry

  Bone mineral content, bone mineral density, bone area, and bone mineral density Z-score will be assesses using dual x-ray absorptiometry (DXA). Summary statistics will be tabulated by anatomical location at each visit.

  At Enrollment Visit (baseline) and each Yearly Follow-Up Visit (for up to 5 years).

• Body Composition Mass Measures

  Fat mass, lean mass, and fat-free mass will be assessed using DXA. Summary statistics will be tabulated by visit.

  At Enrollment Visit, each Yearly Follow-Up and End of Study/Early Termination (for approximately 5 years).

• Summary statistics of body fat percentage and regional fat distribution ratios assessed by DXA

  Body fat percentage, tissue fat percentage, android/gynoid body fat percent ratio, and android/gynoid tissue fat percent ratio will be assessed using DXA. Summary statistics will be tabulated by visit.

  At Enrollment visit, each Yearly Follow-up visit, and End of study/Early termination (for approximately 5 years).

• Findings from standard of care echocardiography

  If available as per standard of care, findings from echocardiography will be collected. This is not required if not available as part of the standard of care.

  At Enrollment Visit, and at each Yearly Follow-up Visit (for up to 5 years).

• Frequency and percentage of participants experiencing any adverse events and serious adverse events

  Adverse events (AEs) and serious adverse events (SAEs) will be collected after signing the informed consent through the end of study participation. The frequency and percentage of patients experiencing any AE or SAE will be summarized. Adverse events will be assessed and documented by the investigator based on routine clinical evaluations, including vital signs, physical examinations, laboratory assessments, and medical record review. For each adverse event, the Investigator will document relevant clinical details, including timing, severity, outcome, and assessments of the seriousness and causality. Adverse events will be assessed by the current version of the CTCAE at the time the adverse event is identified, and will be summarized by MedDRA system organ class and preferred term using the current version of MedDRA.

  From informed consent, Enrollment visit, each Yearly follow-up visit, and End of Study/Early Termination (for up to 5 years).

• Findings from cardiac magnetic resonance imaging (subset of patients taking part in the cardiac sub-study only)

  Myocardial damage will be assessed through magnetic resonance imaging using late gadolinium enhancement. Late gadolinium enhancement is a technique used in cardiac MRI for cardiac tissue characterization, in particular, the assessment of myocardial scar formation and regional myocardial fibrosis

  Enrollment Visit, and at each Yearly Follow-Up Visit (for up to 5 years).

• Presence of cardiomyopathy assessed by echocardiography using transmural strain profile (only in a subset of patients taking part in the cardiac sub-study)

  Presence of cardiomyopathy will be assessed by echocardiography using transmural strain profile (only in a subset of patients taking part in the cardiac sub-study).

  At Enrollment Visit (baseline), and each Yearly Follow-up Visit (for up to 5 years) or Early Termination.

Secondary Outcomes (2)

• Changes in Duchenne Muscular Dystrophy Quality of Life questionnaire score

  At Enrollment Visit (baseline) and at each Yearly Follow-up Visit for up to 5 years.

• Change in Patient Reported Outcome Measure for the Upper Limb score

  At Enrollment Visit (baseline), and each Yearly Follow-Up Visit (for up to 5 years).

Interventions

Vamorolone DRUG

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

Also known as: AGAMREE®

Eligibility Criteria

• Age: 2 Years+
• Gender Eligibility Details: Male subjects 2 years of age and older.
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Male patients at least 2 years old with confirmed diagnosis of DMD (via genetic testing or muscle biopsy with absent dystrophin staining to anti-dystrophin antibodies 3, 1, or 2, or dystrophin immunohistochemistry or western blot).

You may qualify if:

• Patient or parent/legal guardian is willing and able to provide written informed consent once the nature of the registry has been explained and prior to the start of any registry-related procedures.
• Patient and/or parent/guardian are willing and able to complete QoL questionnaires.
• Male patients at least 2 years old.
• Confirmed diagnosis of DMD (via genetic testing or muscle biopsy with absent dystrophin staining to anti- dystrophin antibodies 3, 1, or 2, or dystrophin immunohistochemistry or western blot).
• Patient has a current, active prescription for, or is on, AGAMREE®.

You may not qualify if:

• \. Any contraindication to AGAMREE® or medical condition, which, in the opinion of the Investigator, would affect registry participation, performance, or interpretation of registry assessments.

Sponsors & Collaborators

• Catalyst Pharmaceuticals, Inc.: lead
• ICON Medical Imaging: collaborator
• ICON plc: collaborator
• Veeva Systems: collaborator
• Children's Hospital of Eastern Ontario: collaborator
• Medpace, Inc.: collaborator

Study Sites (27)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

RECRUITING

Arkansas Childrens Hospital

Little Rock, Arkansas, 72202, United States

RECRUITING

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

NOT YET RECRUITING

Stanford University

Palo Alto, California, 94305, United States

NOT YET RECRUITING

University of California, Davis

Sacramento, California, 95817, United States

NOT YET RECRUITING

University of Florida Clinical and Translational Science Institue

Gainesville, Florida, 32610, United States

NOT YET RECRUITING

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

RECRUITING

Nemours Children's Hospital

Orlando, Florida, 32827, United States

RECRUITING

Johns Hopkins All Children's Hospital

St. Petersburg, Florida, 33701, United States

NOT YET RECRUITING

Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

RECRUITING

Indiana University Health - Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

NOT YET RECRUITING

University of Kansas Medical Center

Kansas City, Kansas, 66205, United States

RECRUITING

University of Massachusetts Memorial Medical Center

North Worcester, Massachusetts, 01655, United States

RECRUITING

Helen DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States

RECRUITING

Atrium Health Neurosciences Institute

Charlotte, North Carolina, 28207, United States

RECRUITING

Duke University Medical Center and Childrens Health Center

Durham, North Carolina, 27710, United States

RECRUITING

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

RECRUITING

Penn State Milton S. Hershey Medical Center- Penn State Hershey Neuroscience Institute

Hershey, Pennsylvania, 17033, United States

RECRUITING

Children's Hospital of Philadelphia (CHOP)

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

University of Texas Southwestern Medical Center

Dallas, Texas, 75235, United States

RECRUITING

Neurology Rare Disease Center - Neurology & Neuromuscular Care Center

Denton, Texas, 76208, United States

RECRUITING

The University of Texas Health Science Center at San Antonio

San Antonio, Texas, 78229, United States

RECRUITING

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

Charlottesville, Virginia, 22903, United States

RECRUITING

Seattle Children's Hospital

Seattle, Washington, 98105, United States

RECRUITING

Children's Wisconsin

Milwaukee, Wisconsin, 53226, United States

NOT YET RECRUITING

San Jorge Children's Hospital

San Juan, Puerto Rico, 00912, Puerto Rico

RECRUITING

FDI Clinical Research

San Juan, Puerto Rico, 00927, Puerto Rico

RECRUITING

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Muscular Dystrophies

Interventions

VBP15 compound

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• William Andrews, MD

  Catalyst Pharmaceuticals

  STUDY DIRECTOR

• Aravindham Veerapandiyan, MD

  Arkansas Childrens Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Syune Nersisyan, PhD

CONTACT

(305) 420-3200; snersisyan@catalystpharma.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 5 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 9, 2024
• First Posted: August 21, 2024
• Study Start: September 25, 2024
• Primary Completion (Estimated): February 1, 2032
• Study Completion (Estimated): February 1, 2032
• Last Updated: February 27, 2026

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (25); PR (2)