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Registeronderzoek om de veiligheid van Vamorolone (AGAMREE®) op de lange termijn te observeren bij patiënten met Duchenne-spierdystrofie. (DMD-001 SUMMIT)

22 september 2026 bijgewerkt door: Catalyst Pharmaceuticals, Inc.

Registeronderzoek om de veiligheid van Vamorolone (AGAMREE®) op de lange termijn te observeren bij patiënten met Duchenne-spierdystrofie

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).

Studie Overzicht

Toestand

Werving

Conditie

Interventie / Behandeling

Gedetailleerde beschrijving

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.

Studietype

Observationeel

Inschrijving (Geschat)

250

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studiecontact

Studie Locaties

  • Puerto Rico
    • Puerto Rico
      • San Juan, Puerto Rico, Puerto Rico, 00912
        • Werving
        • San Jorge Children's Hospital
        • Contact:
        • Hoofdonderzoeker:
          • Edwardo Ramos, MD
      • San Juan, Puerto Rico, Puerto Rico, 00927
        • Aanmelden op uitnodiging
        • FDI Clinical Research
  • Verenigde Staten
    • Arizona
      • Phoenix, Arizona, Verenigde Staten, 85016
    • Arkansas
      • Little Rock, Arkansas, Verenigde Staten, 72202
        • Aanmelden op uitnodiging
        • Arkansas Childrens Hospital
    • California
      • Loma Linda, California, Verenigde Staten, 92354
        • Werving
        • Loma Linda University Pediatric Subspecialty Clinics
        • Hoofdonderzoeker:
          • David Michelson, MD
        • Contact:
      • Los Angeles, California, Verenigde Staten, 90027
        • Nog niet aan het werven
        • Children's Hospital Los Angeles
        • Contact:
      • Madera, California, Verenigde Staten, 93636
        • Nog niet aan het werven
        • Valley Children's Hospital
        • Hoofdonderzoeker:
          • Raymund David, MD
        • Contact:
      • Palo Alto, California, Verenigde Staten, 94305
        • Nog niet aan het werven
        • Stanford University
        • Contact:
          • Jenna Klotz, MD
          • Telefoonnummer: 408 426 5590
      • Sacramento, California, Verenigde Staten, 95817
        • Werving
        • University of California, Davis
        • Contact:
    • District of Columbia
      • Washington D.C., District of Columbia, Verenigde Staten, 20010
        • Nog niet aan het werven
        • Children's National Health System
        • Contact:
    • Florida
      • Gainesville, Florida, Verenigde Staten, 32610
        • Werving
        • University of Florida Clinical and Translational Science Institue
        • Contact:
      • Miami, Florida, Verenigde Staten, 33136
        • Nog niet aan het werven
        • University of Miami
        • Hoofdonderzoeker:
          • Mario Saporta, MD
        • Contact:
      • Miami, Florida, Verenigde Staten, 33155
        • Aanmelden op uitnodiging
        • Nicklaus Children's Hospital
      • Orlando, Florida, Verenigde Staten, 32827
        • Aanmelden op uitnodiging
        • Nemours Children's Hospital
      • St. Petersburg, Florida, Verenigde Staten, 33701
        • Werving
        • Johns Hopkins All Children's Hospital
        • Contact:
        • Hoofdonderzoeker:
          • Matias Lopez Chacon, MD
    • Illinois
      • Chicago, Illinois, Verenigde Staten, 60611
        • Aanmelden op uitnodiging
        • Lurie Children's Hospital of Chicago
    • Indiana
      • Indianapolis, Indiana, Verenigde Staten, 46202
        • Aanmelden op uitnodiging
        • Indiana University Health - Riley Hospital for Children
    • Kansas
      • Kansas City, Kansas, Verenigde Staten, 66205
        • Aanmelden op uitnodiging
        • University of Kansas Medical Center
    • Massachusetts
      • North Worcester, Massachusetts, Verenigde Staten, 01655
        • Aanmelden op uitnodiging
        • University of Massachusetts Memorial Medical Center
    • Michigan
      • Grand Rapids, Michigan, Verenigde Staten, 49503
        • Aanmelden op uitnodiging
        • Helen DeVos Children's Hospital
    • Minnesota
      • Minneapolis, Minnesota, Verenigde Staten, 55455
        • Nog niet aan het werven
        • Masonic Children's Hospital - Discovery Clinic
        • Hoofdonderzoeker:
          • Peter Karachunski, MD
        • Contact:
    • New York
      • The Bronx, New York, Verenigde Staten, 10467-2403
        • Nog niet aan het werven
        • The Children's Hospital at Montefiore
        • Hoofdonderzoeker:
          • Carla Zingariello, DO
        • Contact:
    • North Carolina
      • Charlotte, North Carolina, Verenigde Staten, 28207
        • Aanmelden op uitnodiging
        • Atrium Health Neurosciences Institute
      • Durham, North Carolina, Verenigde Staten, 27710
        • Aanmelden op uitnodiging
        • Duke University Medical Center and Childrens Health Center
    • Ohio
      • Cincinnati, Ohio, Verenigde Staten, 45229
        • Werving
        • Cincinnati Children's Hospital Medical Center
        • Contact:
    • Pennsylvania
      • Hershey, Pennsylvania, Verenigde Staten, 17033
        • Aanmelden op uitnodiging
        • Penn State Milton S. Hershey Medical Center- Penn State Hershey Neuroscience Institute
      • Philadelphia, Pennsylvania, Verenigde Staten, 19104
        • Aanmelden op uitnodiging
        • Children's Hospital of Philadelphia (CHOP)
    • Texas
      • Dallas, Texas, Verenigde Staten, 75235
        • Aanmelden op uitnodiging
        • University of Texas Southwestern Medical Center
      • Denton, Texas, Verenigde Staten, 76208
        • Aanmelden op uitnodiging
        • Neurology Rare Disease Center - Neurology & Neuromuscular Care Center
      • San Antonio, Texas, Verenigde Staten, 78229
        • Werving
        • The University of Texas Health Science Center at San Antonio
        • Contact:
    • Virginia
      • Charlottesville, Virginia, Verenigde Staten, 22903
        • Aanmelden op uitnodiging
        • University of Virginia Health System (UVAHS) - Pediatric Neuromuscular Center
    • Washington
      • Seattle, Washington, Verenigde Staten, 98105
        • Aanmelden op uitnodiging
        • Seattle Children's Hospital
    • Wisconsin
      • Milwaukee, Wisconsin, Verenigde Staten, 53226
        • Nog niet aan het werven
        • Children's Wisconsin
        • Hoofdonderzoeker:
          • Nancy Bass, MD
        • Contact:

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

2 jaar en ouder (Kind, Volwassen, Oudere volwassene)

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Mannelijk

Bemonsteringsmethode

Kanssteekproef

Studie Bevolking

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).

Beschrijving

Inclusion Criteria:

  1. 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.
  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 anti- dystrophin antibodies 3, 1, or 2, or dystrophin immunohistochemistry or western blot).
  5. Patient has a current, active prescription for, or is on, AGAMREE®.

Exclusion Criteria:

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.

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Observatiemodellen: COHORT
  • Tijdsperspectieven: PROSPECTIVE

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Change in Height z-score
Tijdsspanne: 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.
At Enrollment Visit (baseline), and at each Yearly Follow-up Visit (for up to 5 years).
Change in BMI z-score
Tijdsspanne: 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.
At Enrollment Visit (baseline), and each Yearly Follow-up Visit (for up to 5 years)
Change in North Star Ambulatory Assessment from baseline
Tijdsspanne: 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.
At Enrollment visit, and each Yearly Follow-up Visit (for up to 5 years).
Change in Performance of Upper Limb from baseline
Tijdsspanne: 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.
At Enrollment Visit, at each Yearly Follow-up Visit (for up to 5 years).
Tanner stage documentation
Tijdsspanne: 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.
At Enrollment Visit (baseline), at each Yearly Follow-up Visit (for up to 5 years).
Percentage of patients with cataract and/or glaucoma
Tijdsspanne: 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.
At Enrollment (baseline), and during each Yearly Follow-up Visit (for up to 5 years).
Findings from lateral spine x-ray
Tijdsspanne: 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.
At Enrollment Visit (baseline), and during each Yearly Follow-up Visit (for up to 5 years).
Findings from anterior-posterior/posterior-anterior X-ray
Tijdsspanne: 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.
At Enrollment Visit (baseline), and at each Yearly Follow-Up Visit (for up to 5 years).
Findings from hand-wrist X-ray
Tijdsspanne: 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.
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
Tijdsspanne: 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.
At Enrollment Visit (baseline) and each Yearly Follow-Up Visit (for up to 5 years).
Body Composition Mass Measures
Tijdsspanne: 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.
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
Tijdsspanne: 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.
At Enrollment visit, each Yearly Follow-up visit, and End of study/Early termination (for approximately 5 years).
Findings from standard of care echocardiography
Tijdsspanne: 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.
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
Tijdsspanne: 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.
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)
Tijdsspanne: 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
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)
Tijdsspanne: 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).
At Enrollment Visit (baseline), and each Yearly Follow-up Visit (for up to 5 years) or Early Termination.

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Changes in Duchenne Muscular Dystrophy Quality of Life questionnaire score
Tijdsspanne: At Enrollment Visit (baseline) and at each Yearly Follow-up Visit for up to 5 years.

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

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

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

Catalyst Pharmaceuticals, Inc.

Medewerkers

Children's Hospital of Eastern Ontario
Medpace, Inc.
ICON plc
Veeva Systems

Onderzoekers

  • Hoofdonderzoeker: Aravindham Veerapandiyan, MD, Arkansas Childrens Hospital
  • Studie directeur: William Andrews, MD, Catalyst Pharmaceuticals

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Werkelijk)

25 september 2024

Primaire voltooiing (Geschat)

1 februari 2032

Studie voltooiing (Geschat)

1 februari 2032

Studieregistratiedata

Eerst ingediend

9 augustus 2024

Eerst ingediend dat voldeed aan de QC-criteria

19 augustus 2024

Eerst geplaatst (Werkelijk)

21 augustus 2024

Updates van studierecords

Laatste update geplaatst (Werkelijk)

24 september 2026

Laatste update ingediend die voldeed aan QC-criteria

22 september 2026

Laatst geverifieerd

1 september 2026

Meer informatie

Termen gerelateerd aan deze studie

Trefwoorden

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • DMD-001

Plan Individuele Deelnemersgegevens (IPD)

Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?

NEE

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Ja

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

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