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Registerstudie för att observera långsiktig säkerhet av Vamorolone (AGAMREE®) hos patienter med Duchennes muskeldystrofi. (DMD-001 SUMMIT)

22 september 2026 uppdaterad av: Catalyst Pharmaceuticals, Inc.

Registerstudie för att observera långsiktig säkerhet för Vamorolone (AGAMREE®) hos patienter med Duchennes muskeldystrofi

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).

Studieöversikt

Status

Rekrytering

Betingelser

Intervention / Behandling

Detaljerad beskrivning

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.

Studietyp

Observationell

Inskrivning (Beräknad)

250

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studiekontakt

Studieorter

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

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

2 år och äldre (Barn, Vuxen, Äldre vuxen)

Tar emot friska volontärer

Nej

Kön som är behöriga för studier

Manlig

Testmetod

Sannolikhetsprov

Studera befolkning

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).

Beskrivning

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.

Studieplan

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

  • Observationsmodeller: COHORT
  • Tidsperspektiv: PROSPECTIVE

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Change in Height z-score
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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)
Tidsram: 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)
Tidsram: 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.

Sekundära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Changes in Duchenne Muscular Dystrophy Quality of Life questionnaire score
Tidsram: 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
Tidsram: 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).

Samarbetspartners och utredare

Det är här du hittar personer och organisationer som är involverade i denna studie.

Sponsor

Catalyst Pharmaceuticals, Inc.

Samarbetspartners

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

Utredare

  • Huvudutredare: Aravindham Veerapandiyan, MD, Arkansas Childrens Hospital
  • Studierektor: William Andrews, MD, Catalyst Pharmaceuticals

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart (Faktisk)

25 september 2024

Primärt slutförande (Beräknad)

1 februari 2032

Avslutad studie (Beräknad)

1 februari 2032

Studieregistreringsdatum

Först inskickad

9 augusti 2024

Först inskickad som uppfyllde QC-kriterierna

19 augusti 2024

Första postat (Faktisk)

21 augusti 2024

Uppdateringar av studier

Senaste uppdatering publicerad (Faktisk)

24 september 2026

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

22 september 2026

Senast verifierad

1 september 2026

Mer information

Termer relaterade till denna studie

Nyckelord

Ytterligare relevanta MeSH-villkor

Andra studie-ID-nummer

  • DMD-001

Plan för individuella deltagardata (IPD)

Planerar du att dela individuella deltagardata (IPD)?

NEJ

Läkemedels- och apparatinformation, studiedokument

Studerar en amerikansk FDA-reglerad läkemedelsprodukt

Ja

Studerar en amerikansk FDA-reglerad produktprodukt

Nej

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