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Badanie rejestru mające na celu obserwację długoterminowego bezpieczeństwa Vamorolonu (AGAMREE®) u pacjentów z dystrofią mięśniową Duchenne’a. (DMD-001 SUMMIT)

22 września 2026 zaktualizowane przez: Catalyst Pharmaceuticals, Inc.

Badanie rejestru mające na celu obserwację długoterminowego bezpieczeństwa stosowania Vamorolonu (AGAMREE®) u pacjentów z dystrofią mięśniową Duchenne’a

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).

Przegląd badań

Status

Rekrutacyjny

Warunki

Interwencja / Leczenie

Szczegółowy opis

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 studiów

Obserwacyjny

Zapisy (Szacowany)

250

Kontakty i lokalizacje

Ta sekcja zawiera dane kontaktowe osób prowadzących badanie oraz informacje o tym, gdzie badanie jest przeprowadzane.

Kontakt w sprawie studiów

Lokalizacje studiów

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

Kryteria uczestnictwa

Badacze szukają osób, które pasują do określonego opisu, zwanego kryteriami kwalifikacyjnymi. Niektóre przykłady tych kryteriów to ogólny stan zdrowia danej osoby lub wcześniejsze leczenie.

Kryteria kwalifikacji

Wiek uprawniający do nauki

2 lata i starsze (Dziecko, Dorosły, Starszy dorosły)

Akceptuje zdrowych ochotników

Nie

Płeć kwalifikująca się do nauki

Męski

Metoda próbkowania

Próbka prawdopodobieństwa

Badana populacja

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).

Opis

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.

Plan studiów

Ta sekcja zawiera szczegółowe informacje na temat planu badania, w tym sposób zaprojektowania badania i jego pomiary.

Jak projektuje się badanie?

Szczegóły projektu

  • Modele obserwacyjne: COHORT
  • Perspektywy czasowe: PROSPECTIVE

Co mierzy badanie?

Podstawowe miary wyniku

Miara wyniku
Opis środka
Ramy czasowe
Change in Height z-score
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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)
Ramy czasowe: 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)
Ramy czasowe: 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.

Miary wyników drugorzędnych

Miara wyniku
Opis środka
Ramy czasowe
Changes in Duchenne Muscular Dystrophy Quality of Life questionnaire score
Ramy czasowe: 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
Ramy czasowe: 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).

Współpracownicy i badacze

Tutaj znajdziesz osoby i organizacje zaangażowane w to badanie.

Sponsor

Catalyst Pharmaceuticals, Inc.

Współpracownicy

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

Śledczy

  • Główny śledczy: Aravindham Veerapandiyan, MD, Arkansas Childrens Hospital
  • Dyrektor Studium: William Andrews, MD, Catalyst Pharmaceuticals

Daty zapisu na studia

Daty te śledzą postęp w przesyłaniu rekordów badań i podsumowań wyników do ClinicalTrials.gov. Zapisy badań i zgłoszone wyniki są przeglądane przez National Library of Medicine (NLM), aby upewnić się, że spełniają określone standardy kontroli jakości, zanim zostaną opublikowane na publicznej stronie internetowej.

Główne daty studiów

Rozpoczęcie studiów (Rzeczywisty)

25 września 2024

Zakończenie podstawowe (Szacowany)

1 lutego 2032

Ukończenie studiów (Szacowany)

1 lutego 2032

Daty rejestracji na studia

Pierwszy przesłany

9 sierpnia 2024

Pierwszy przesłany, który spełnia kryteria kontroli jakości

19 sierpnia 2024

Pierwszy wysłany (Rzeczywisty)

21 sierpnia 2024

Aktualizacje rekordów badań

Ostatnia wysłana aktualizacja (Rzeczywisty)

24 września 2026

Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości

22 września 2026

Ostatnia weryfikacja

1 września 2026

Więcej informacji

Terminy związane z tym badaniem

Słowa kluczowe

Dodatkowe istotne warunki MeSH

Inne numery identyfikacyjne badania

  • DMD-001

Plan dla danych uczestnika indywidualnego (IPD)

Planujesz udostępniać dane poszczególnych uczestników (IPD)?

NIE

Informacje o lekach i urządzeniach, dokumenty badawcze

Bada produkt leczniczy regulowany przez amerykańską FDA

Tak

Bada produkt urządzenia regulowany przez amerykańską FDA

Nie

Te informacje zostały pobrane bezpośrednio ze strony internetowej clinicaltrials.gov bez żadnych zmian. Jeśli chcesz zmienić, usunąć lub zaktualizować dane swojego badania, skontaktuj się z register@clinicaltrials.gov. Gdy tylko zmiana zostanie wprowadzona na stronie clinicaltrials.gov, zostanie ona automatycznie zaktualizowana również na naszej stronie internetowej .

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