An observational study in Duchenne Muscular Dystrophy, sponsored by Catalyst Pharmaceuticals, Inc.. Recruiting at 33 sites in 2 countries. Open to male participants aged 2 Years and older. Per ClinicalTrials.gov, last updated 2026-09-24.
Sponsored by Catalyst Pharmaceuticals, Inc. (part of Angelini Pharma) · Observational
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).
This is a multi-center, observational, prospective, longitudinal registry study, designed to collect safety data 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 DMD.
Primary Objective is to collect additional information on the safety of long-term treatment with AGAMREE® in male patients aged 2 years and older with Duchenne muscular dystrophy (DMD) as determined by:
Growth parameters Body mass index (BMI) Musculoskeletal assessments Ophthalmological assessments Cardiovascular assessments Pubertal development assessments Adverse events (AEs)
Secondary Objective is to explore long-term clinical impact of AGAMREE® on quality of life, as assessed by standardized patient-reported outcome measures (QoL questionnaires).
This registry will be conducted in the US, at approximately 40 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.
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 assessments. Information on some historical and ongoing standard of care treatment and procedures for management of DMD will also be collected.
481 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 108 are open to participants now.
This study's planned enrollment of 250 is above the median of 60 across 132 observational studies indexed under Muscular Dystrophy, Duchenne.
Browse Muscular Dystrophy, Duchenne studies →Catalyst Pharmaceuticals, Inc. is the lead sponsor of 13 studies on the registry; 1 is open to participants now.
First submitted before Catalyst Pharmaceuticals, Inc. became part of Angelini Pharma.
Counted across the registry records on this site, refreshed daily.
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).
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
1. Any to AGAMREE® or medical condition, which, in the opinion of the Investigator, would affect registry participation, performance, or interpretation of registry assessments.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Study treatment is AGAMREE®, which is commercially available as an oral suspension.
Also known as: AGAMREE®
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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
Time frame: 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).
Time frame: At Enrollment Visit (baseline), and each Yearly Follow-up Visit (for up to 5 years) or Early Termination.
Changes in Duchenne Muscular Dystrophy Quality of Life questionnaire score
Duchenne Muscular Dystrophy Quality of Life questionnaire is a patient-reported outcome measure designed to assess how Duchenne muscular dystrophy affects physical, emotional, and social aspects of daily life. Duchenne Muscular Dystrophy Quality of Life questionnaire proxy report will be completed by a parent/guardian for patients aged 7 years and older. For patients aged 10 years and older, a self-report will be completed by the patient, and, if feasible, a proxy report will also be completed by the parent or guardian.
Time frame: 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
Patient Reported Outcome Measure for the Upper Limb is a questionnaire for young males/children with DMD from the age of 7 years. The PROM UL will be completed by parent/legal guardian up to the age of 16 years and by the patient from the age of 17 years. It consists of 32 items covering four domains of activities of daily living: (1) food, (7 items, max score 14); (2) self-care, (8 items, max score 16); (3) household and environment, (6 items, max score 12); (4) leisure and communication, (11 items, max score 22). For each question, the patients and/or their parent/legal guardian are asked to report their perceived difficulty in performing the activity on a 3-level scale: Cannot do (0); Can do with difficulty (1); Can do easily (2). The maximum total score is 64, with higher score indicating better function.
Time frame: At Enrollment Visit (baseline), and each Yearly Follow-Up Visit (for up to 5 years).
Plan to share: No
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Muscular Dystrophy, Duchenne →
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