이 페이지는 자동 번역되었으며 번역의 정확성을 보장하지 않습니다. 참조하십시오 영문판 원본 텍스트의 경우.

Duchenne 근이영양증 환자에서 Vamorolone(AGAMREE®)의 장기 안전성을 관찰하기 위한 등록 연구. (DMD-001 SUMMIT)

2026년 9월 22일 업데이트: Catalyst Pharmaceuticals, Inc.

뒤시엔느 근이영양증 환자에서 바모롤론(AGAMREE®)의 장기 안전성을 관찰하기 위한 등록 연구

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).

연구 개요

상태

모병

정황

뒤시엔 근이영양증

개입 / 치료

의약품: Vamorolone

상세 설명

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.

연구 유형

관찰

등록 (추정된)

250

연락처 및 위치

이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.

연구 연락처

이름: Mark Wingertzahn, PhD
전화번호: (305) 420-3200
이메일: mark.wingertzahn@catalystpharma.com

연구 장소

미국
- Arizona
  - Phoenix, Arizona, 미국, 85016
    - 모병
    - Phoenix Children's Hospital
    - 연락하다:
      
      Clara Chow, MD
      
      전화번호: 602 933 0956
      
      이메일: cchow@phoenixchildrens.com
- Arkansas
  - Little Rock, Arkansas, 미국, 72202
    - 초대로 등록
    - Arkansas Childrens Hospital
- California
  - Loma Linda, California, 미국, 92354
    - 모병
    - Loma Linda University Pediatric Subspecialty Clinics
    - 수석 연구원:
      
      David Michelson, MD
    - 연락하다:
      
      Monica Pizana
      
      전화번호: 909-558-5830
      
      이메일: mpizana@llu.edu
  - Los Angeles, California, 미국, 90027
    - 아직 모집하지 않음
    - Children's Hospital Los Angeles
    - 연락하다:
      
      Dana O'Rourke, MD
      
      전화번호: 323 660 2450
      
      이메일: dorourke@chla.usc.edu
  - Madera, California, 미국, 93636
    - 아직 모집하지 않음
    - Valley Children's Hospital
    - 수석 연구원:
      
      Raymund David, MD
    - 연락하다:
      
      Claudia Nayares
      
      전화번호: (559) 353-3000
      
      이메일: cnayares@valleychildrens.org
  - Palo Alto, California, 미국, 94305
    - 아직 모집하지 않음
    - Stanford University
    - 연락하다:
      
      Jenna Klotz, MD
      
      전화번호: 408 426 5590
  - Sacramento, California, 미국, 95817
    - 모병
    - University of California, Davis
    - 연락하다:
      
      Craig McDonald, MD
      
      전화번호: 916 734 7041
      
      이메일: cmmcdonald@ucdavis.edu
- District of Columbia
  - Washington D.C., District of Columbia, 미국, 20010
    - 아직 모집하지 않음
    - Children's National Health System
    - 연락하다:
      
      Christopher Spurney, MD
      
      전화번호: 202-476-2185
      
      이메일: cspurney@childrensnational.org
- Florida
  - Gainesville, Florida, 미국, 32610
    - 모병
    - University of Florida Clinical and Translational Science Institue
    - 연락하다:
      
      Julie Berthy, NP
      
      전화번호: 352 273 8700
      
      이메일: jberthy@ufl.edu
  - Miami, Florida, 미국, 33136
    - 아직 모집하지 않음
    - University of Miami
    - 수석 연구원:
      
      Mario Saporta, MD
    - 연락하다:
      
      Branden Couto
      
      이메일: bxc612@miami.edu
  - Miami, Florida, 미국, 33155
    - 초대로 등록
    - Nicklaus Children's Hospital
  - Orlando, Florida, 미국, 32827
    - 초대로 등록
    - Nemours Children's Hospital
  - St. Petersburg, Florida, 미국, 33701
    - 모병
    - Johns Hopkins All Children's Hospital
    - 연락하다:
      
      Susan Dvojack
      
      전화번호: 727-898-7451
      
      이메일: sdvojac1@jhmi.edu
    - 수석 연구원:
      
      Matias Lopez Chacon, MD
- Illinois
  - Chicago, Illinois, 미국, 60611
    - 초대로 등록
    - Lurie Children's Hospital of Chicago
- Indiana
  - Indianapolis, Indiana, 미국, 46202
    - 초대로 등록
    - Indiana University Health - Riley Hospital for Children
- Kansas
  - Kansas City, Kansas, 미국, 66205
    - 초대로 등록
    - University of Kansas Medical Center
- Massachusetts
  - North Worcester, Massachusetts, 미국, 01655
    - 초대로 등록
    - University of Massachusetts Memorial Medical Center
- Michigan
  - Grand Rapids, Michigan, 미국, 49503
    - 초대로 등록
    - Helen DeVos Children's Hospital
- Minnesota
  - Minneapolis, Minnesota, 미국, 55455
    - 아직 모집하지 않음
    - Masonic Children's Hospital - Discovery Clinic
    - 수석 연구원:
      
      Peter Karachunski, MD
    - 연락하다:
      
      Jenny Myhre
      
      전화번호: 612-365-6777
      
      이메일: marz0031@umn.edu
- New York
  - The Bronx, New York, 미국, 10467-2403
    - 아직 모집하지 않음
    - The Children's Hospital at Montefiore
    - 수석 연구원:
      
      Carla Zingariello, DO
    - 연락하다:
      
      Channelle Mooyoung
      
      전화번호: (718) 920-4378
      
      이메일: cmooyoung@montefiore.org
- North Carolina
  - Charlotte, North Carolina, 미국, 28207
    - 초대로 등록
    - Atrium Health Neurosciences Institute
  - Durham, North Carolina, 미국, 27710
    - 초대로 등록
    - Duke University Medical Center and Childrens Health Center
- Ohio
  - Cincinnati, Ohio, 미국, 45229
    - 모병
    - Cincinnati Children's Hospital Medical Center
    - 연락하다:
      
      Meghan McAnally, MD
      
      전화번호: 513 636 9285
      
      이메일: meghan.mcanally@cchmc.org
- Pennsylvania
  - Hershey, Pennsylvania, 미국, 17033
    - 초대로 등록
    - Penn State Milton S. Hershey Medical Center- Penn State Hershey Neuroscience Institute
  - Philadelphia, Pennsylvania, 미국, 19104
    - 초대로 등록
    - Children's Hospital of Philadelphia (CHOP)
- Texas
  - Dallas, Texas, 미국, 75235
    - 초대로 등록
    - University of Texas Southwestern Medical Center
  - Denton, Texas, 미국, 76208
    - 초대로 등록
    - Neurology Rare Disease Center - Neurology & Neuromuscular Care Center
  - San Antonio, Texas, 미국, 78229
    - 모병
    - The University of Texas Health Science Center at San Antonio
    - 연락하다:
      
      Matthew Wicklund, MD
      
      전화번호: 210 450 0500
      
      이메일: wicklund@uthscsa.edu
- Virginia
  - Charlottesville, Virginia, 미국, 22903
    - 초대로 등록
    - University of Virginia Health System (UVAHS) - Pediatric Neuromuscular Center
- Washington
  - Seattle, Washington, 미국, 98105
    - 초대로 등록
    - Seattle Children's Hospital
- Wisconsin
  - Milwaukee, Wisconsin, 미국, 53226
    - 아직 모집하지 않음
    - Children's Wisconsin
    - 수석 연구원:
      
      Nancy Bass, MD
    - 연락하다:
      
      Rebecca Rehborg
      
      전화번호: 877-607-5280
      
      이메일: rrehborg@mcw.edu
푸에르토 리코
- Puerto Rico
  - San Juan, Puerto Rico, 푸에르토 리코, 00912
    - 모병
    - San Jorge Children's Hospital
    - 연락하다:
      
      Yaritza Berrios
      
      전화번호: 787-758-2525
      
      이메일: yartiza.berrios@upr.edu
    - 수석 연구원:
      
      Edwardo Ramos, MD
  - San Juan, Puerto Rico, 푸에르토 리코, 00927
    - 초대로 등록
    - FDI Clinical Research

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

공부할 수 있는 나이

2년 이상 (어린이, 성인, 고령자)

건강한 자원 봉사자를 받아들입니다

아니

연구 대상 성별

남성

샘플링 방법

확률 샘플

연구 인구

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).

설명

Inclusion Criteria:

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.
Patient and/or parent/guardian are willing and able to complete QoL questionnaires.
Male patients at least 2 years old.
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).
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.

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

연구는 어떻게 설계됩니까?

디자인 세부사항

관찰 모델: COHORT
시간 관점: PROSPECTIVE

연구는 무엇을 측정합니까?

주요 결과 측정

2차 결과 측정

결과 측정	측정값 설명	기간
Changes in Duchenne Muscular Dystrophy Quality of Life questionnaire score 기간: 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 기간: 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).

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

스폰서

Catalyst Pharmaceuticals, Inc.

협력자

Children's Hospital of Eastern Ontario

Medpace, Inc.

ICON plc

Veeva Systems

수사관

수석 연구원: Aravindham Veerapandiyan, MD, Arkansas Childrens Hospital
연구 책임자: William Andrews, MD, Catalyst Pharmaceuticals

연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작 (실제)

2024년 9월 25일

기본 완료 (추정된)

2032년 2월 1일

연구 완료 (추정된)

2032년 2월 1일

연구 등록 날짜

최초 제출

2024년 8월 9일

QC 기준을 충족하는 최초 제출

2024년 8월 19일

처음 게시됨 (실제)

2024년 8월 21일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 9월 24일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 9월 22일

마지막으로 확인됨

2026년 9월 1일

추가 정보

이 연구와 관련된 용어

키워드

추가 관련 MeSH 약관

기타 연구 ID 번호

DMD-001

개별 참가자 데이터(IPD) 계획

개별 참가자 데이터(IPD)를 공유할 계획입니까?

아니요

약물 및 장치 정보, 연구 문서

미국 FDA 규제 의약품 연구

예

미국 FDA 규제 기기 제품 연구

아니

이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .

결과 측정	측정값 설명	기간
Change in Height z-score 기간: 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 기간: 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 기간: 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 기간: 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 기간: 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 기간: 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 기간: 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 기간: 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 기간: 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 기간: 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 기간: 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 기간: 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 기간: 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 기간: 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) 기간: 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) 기간: 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.