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

DRUG

Vamorolone

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

Change in Height z-score

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

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.

Change in BMI z-score

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

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.

Change in North Star Ambulatory Assessment from baseline

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

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.

Change in Performance of Upper Limb from baseline

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

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.

Tanner stage documentation

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

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.

Percentage of patients with cataract and/or glaucoma

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

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

Findings from lateral spine x-ray

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

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

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

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

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

Findings from hand-wrist X-ray

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

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

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

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

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.

Body Composition Mass Measures

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

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

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

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

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.

Findings from standard of care echocardiography

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

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.

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

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

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.

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

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

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

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

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

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).

What is a clinical trial?

A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.

Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.

Will I be compensated?

Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.

Will I receive a placebo instead of treatment?

When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.

Can I leave a trial if I change my mind?

Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.

How long does a clinical trial last?

Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.