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Registrační studie k pozorování dlouhodobé bezpečnosti vamorolonu (AGAMREE®) u pacientů s Duchennovou svalovou dystrofií. (DMD-001 SUMMIT)

22. září 2026 aktualizováno: Catalyst Pharmaceuticals, Inc.

Registrační studie k pozorování dlouhodobé bezpečnosti vamorolonu (AGAMREE®) u pacientů s Duchennovou svalovou dystrofií

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).

Přehled studie

Postavení

Nábor

Podmínky

Intervence / Léčba

Detailní popis

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.

Typ studie

Pozorovací

Zápis (Odhadovaný)

250

Kontakty a umístění

Tato část poskytuje kontaktní údaje pro ty, kteří studii provádějí, a informace o tom, kde se tato studie provádí.

Studijní kontakt

Studijní místa

  • Portoriko
    • Puerto Rico
      • San Juan, Puerto Rico, Portoriko, 00912
        • Nábor
        • San Jorge Children's Hospital
        • Kontakt:
        • Vrchní vyšetřovatel:
          • Edwardo Ramos, MD
      • San Juan, Puerto Rico, Portoriko, 00927
        • Zápis na pozvánku
        • FDI Clinical Research
  • Spojené státy
    • Arizona
      • Phoenix, Arizona, Spojené státy, 85016
    • Arkansas
      • Little Rock, Arkansas, Spojené státy, 72202
        • Zápis na pozvánku
        • Arkansas Childrens Hospital
    • California
      • Loma Linda, California, Spojené státy, 92354
        • Nábor
        • Loma Linda University Pediatric Subspecialty Clinics
        • Vrchní vyšetřovatel:
          • David Michelson, MD
        • Kontakt:
      • Los Angeles, California, Spojené státy, 90027
        • Zatím nenabíráme
        • Children's Hospital Los Angeles
        • Kontakt:
      • Madera, California, Spojené státy, 93636
        • Zatím nenabíráme
        • Valley Children's Hospital
        • Vrchní vyšetřovatel:
          • Raymund David, MD
        • Kontakt:
      • Palo Alto, California, Spojené státy, 94305
        • Zatím nenabíráme
        • Stanford University
        • Kontakt:
          • Jenna Klotz, MD
          • Telefonní číslo: 408 426 5590
      • Sacramento, California, Spojené státy, 95817
        • Nábor
        • University of California, Davis
        • Kontakt:
    • District of Columbia
      • Washington D.C., District of Columbia, Spojené státy, 20010
        • Zatím nenabíráme
        • Children's National Health System
        • Kontakt:
    • Florida
      • Gainesville, Florida, Spojené státy, 32610
        • Nábor
        • University of Florida Clinical and Translational Science Institue
        • Kontakt:
          • Julie Berthy, NP
          • Telefonní číslo: 352 273 8700
          • E-mail: jberthy@ufl.edu
      • Miami, Florida, Spojené státy, 33136
        • Zatím nenabíráme
        • University of Miami
        • Vrchní vyšetřovatel:
          • Mario Saporta, MD
        • Kontakt:
      • Miami, Florida, Spojené státy, 33155
        • Zápis na pozvánku
        • Nicklaus Children's Hospital
      • Orlando, Florida, Spojené státy, 32827
        • Zápis na pozvánku
        • Nemours Children's Hospital
      • St. Petersburg, Florida, Spojené státy, 33701
        • Nábor
        • Johns Hopkins All Children's Hospital
        • Kontakt:
        • Vrchní vyšetřovatel:
          • Matias Lopez Chacon, MD
    • Illinois
      • Chicago, Illinois, Spojené státy, 60611
        • Zápis na pozvánku
        • Lurie Children's Hospital of Chicago
    • Indiana
      • Indianapolis, Indiana, Spojené státy, 46202
        • Zápis na pozvánku
        • Indiana University Health - Riley Hospital for Children
    • Kansas
      • Kansas City, Kansas, Spojené státy, 66205
        • Zápis na pozvánku
        • University of Kansas Medical Center
    • Massachusetts
      • North Worcester, Massachusetts, Spojené státy, 01655
        • Zápis na pozvánku
        • University of Massachusetts Memorial Medical Center
    • Michigan
      • Grand Rapids, Michigan, Spojené státy, 49503
        • Zápis na pozvánku
        • Helen DeVos Children's Hospital
    • Minnesota
      • Minneapolis, Minnesota, Spojené státy, 55455
        • Zatím nenabíráme
        • Masonic Children's Hospital - Discovery Clinic
        • Vrchní vyšetřovatel:
          • Peter Karachunski, MD
        • Kontakt:
    • New York
      • The Bronx, New York, Spojené státy, 10467-2403
        • Zatím nenabíráme
        • The Children's Hospital at Montefiore
        • Vrchní vyšetřovatel:
          • Carla Zingariello, DO
        • Kontakt:
    • North Carolina
      • Charlotte, North Carolina, Spojené státy, 28207
        • Zápis na pozvánku
        • Atrium Health Neurosciences Institute
      • Durham, North Carolina, Spojené státy, 27710
        • Zápis na pozvánku
        • Duke University Medical Center and Childrens Health Center
    • Ohio
      • Cincinnati, Ohio, Spojené státy, 45229
        • Nábor
        • Cincinnati Children's Hospital Medical Center
        • Kontakt:
    • Pennsylvania
      • Hershey, Pennsylvania, Spojené státy, 17033
        • Zápis na pozvánku
        • Penn State Milton S. Hershey Medical Center- Penn State Hershey Neuroscience Institute
      • Philadelphia, Pennsylvania, Spojené státy, 19104
        • Zápis na pozvánku
        • Children's Hospital of Philadelphia (CHOP)
    • Texas
      • Dallas, Texas, Spojené státy, 75235
        • Zápis na pozvánku
        • University of Texas Southwestern Medical Center
      • Denton, Texas, Spojené státy, 76208
        • Zápis na pozvánku
        • Neurology Rare Disease Center - Neurology & Neuromuscular Care Center
      • San Antonio, Texas, Spojené státy, 78229
        • Nábor
        • The University of Texas Health Science Center at San Antonio
        • Kontakt:
    • Virginia
      • Charlottesville, Virginia, Spojené státy, 22903
        • Zápis na pozvánku
        • University of Virginia Health System (UVAHS) - Pediatric Neuromuscular Center
    • Washington
      • Seattle, Washington, Spojené státy, 98105
        • Zápis na pozvánku
        • Seattle Children's Hospital
    • Wisconsin
      • Milwaukee, Wisconsin, Spojené státy, 53226
        • Zatím nenabíráme
        • Children's Wisconsin
        • Vrchní vyšetřovatel:
          • Nancy Bass, MD
        • Kontakt:

Kritéria účasti

Výzkumníci hledají lidi, kteří odpovídají určitému popisu, kterému se říká kritéria způsobilosti. Některé příklady těchto kritérií jsou celkový zdravotní stav osoby nebo předchozí léčba.

Kritéria způsobilosti

Věk způsobilý ke studiu

2 roky a starší (Dítě, Dospělý, Starší dospělý)

Přijímá zdravé dobrovolníky

Ne

Pohlaví způsobilá ke studiu

Mužský

Metoda odběru vzorků

Ukázka pravděpodobnosti

Studijní populace

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).

Popis

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.

Studijní plán

Tato část poskytuje podrobnosti o studijním plánu, včetně toho, jak je studie navržena a co studie měří.

Jak je studie koncipována?

Detaily designu

  • Observační modely: COHORT
  • Časové perspektivy: PROSPECTIVE

Co je měření studie?

Primární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
Change in Height z-score
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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)
Časové okno: 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)
Časové okno: 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.

Sekundární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
Changes in Duchenne Muscular Dystrophy Quality of Life questionnaire score
Časové okno: 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
Časové okno: 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).

Spolupracovníci a vyšetřovatelé

Zde najdete lidi a organizace zapojené do této studie.

Sponzor

Catalyst Pharmaceuticals, Inc.

Spolupracovníci

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

Vyšetřovatelé

  • Vrchní vyšetřovatel: Aravindham Veerapandiyan, MD, Arkansas Childrens Hospital
  • Ředitel studie: William Andrews, MD, Catalyst Pharmaceuticals

Termíny studijních záznamů

Tato data sledují průběh záznamů studie a předkládání souhrnných výsledků na ClinicalTrials.gov. Záznamy ze studií a hlášené výsledky jsou před zveřejněním na veřejné webové stránce přezkoumány Národní lékařskou knihovnou (NLM), aby se ujistily, že splňují specifické standardy kontroly kvality.

Hlavní termíny studia

Začátek studia (Aktuální)

25. září 2024

Primární dokončení (Odhadovaný)

1. února 2032

Dokončení studie (Odhadovaný)

1. února 2032

Termíny zápisu do studia

První předloženo

9. srpna 2024

První předloženo, které splnilo kritéria kontroly kvality

19. srpna 2024

První zveřejněno (Aktuální)

21. srpna 2024

Aktualizace studijních záznamů

Poslední zveřejněná aktualizace (Aktuální)

24. září 2026

Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality

22. září 2026

Naposledy ověřeno

1. září 2026

Více informací

Termíny související s touto studií

Klíčová slova

Další relevantní podmínky MeSH

Další identifikační čísla studie

  • DMD-001

Plán pro data jednotlivých účastníků (IPD)

Plánujete sdílet data jednotlivých účastníků (IPD)?

NE

Informace o lécích a zařízeních, studijní dokumenty

Studuje lékový produkt regulovaný americkým FDA

Ano

Studuje produkt zařízení regulovaný americkým úřadem FDA

Ne

Tyto informace byly beze změn načteny přímo z webu clinicaltrials.gov. Máte-li jakékoli požadavky na změnu, odstranění nebo aktualizaci podrobností studie, kontaktujte prosím register@clinicaltrials.gov. Jakmile bude změna implementována na clinicaltrials.gov, bude automaticky aktualizována i na našem webu .

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