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观察瓦莫洛龙 (AGAMREE®) 在杜氏肌营养不良症患者中的长期安全性的注册研究。 (DMD-001 SUMMIT)

2026年9月22日 更新者：Catalyst Pharmaceuticals, Inc.

观察瓦莫洛龙 (AGAMREE®) 在杜氏肌营养不良症患者中的长期安全性的注册研究

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).

研究概览

地位

招聘中

条件

干预/治疗

详细说明

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.

研究类型

观察性的

注册 (估计的)

250

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习联系方式

学习地点

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

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

2年 及以上 (孩子、成人、年长者)

接受健康志愿者

不

有资格学习的性别

男性

取样方法

概率样本

研究人群

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).

描述

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.

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

  • 观测模型：COHORT
  • 时间观点：PROSPECTIVE

研究衡量的是什么？

主要结果指标

结果测量
措施说明
大体时间
Change in Height z-score
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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)
大体时间：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)
大体时间：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.

次要结果测量

结果测量
措施说明
大体时间
Changes in Duchenne Muscular Dystrophy Quality of Life questionnaire score
大体时间：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
大体时间：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).

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

赞助

Catalyst Pharmaceuticals, Inc.

合作者

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

调查人员

  • 首席研究员：Aravindham Veerapandiyan, MD、Arkansas Childrens Hospital
  • 研究主任：William Andrews, MD、Catalyst Pharmaceuticals

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (实际的)

2024年9月25日

初级完成 (估计的)

2032年2月1日

研究完成 (估计的)

2032年2月1日

研究注册日期

首次提交

2024年8月9日

首先提交符合 QC 标准的

2024年8月19日

首次发布 (实际的)

2024年8月21日

研究记录更新

最后更新发布 (实际的)

2026年9月24日

上次提交的符合 QC 标准的更新

2026年9月22日

最后验证

2026年9月1日

更多信息

与本研究相关的术语

关键字

其他相关的 MeSH 术语

其他研究编号

  • DMD-001

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)？

不

药物和器械信息、研究文件

研究美国 FDA 监管的药品

是的

研究美国 FDA 监管的设备产品

不

此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.

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