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デュシェンヌ型筋ジストロフィー患者におけるバモロロン（AGAMREE®）の長期安全性を観察するための登録研究。 (DMD-001 SUMMIT)

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

デュシェンヌ型筋ジストロフィー患者におけるバモロロン（AGAMREE®）の長期安全性を観察するための登録研究

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).

調査の概要

状態

募集

条件

介入・治療

詳細な説明

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.

研究の種類

観察的

入学 (推定)

250

連絡先と場所

このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。

研究連絡先

研究場所

  • アメリカ
    • Arizona
      • Phoenix、Arizona、アメリカ、85016
        • 募集
        • Phoenix Children's Hospital
        • コンタクト：
    • Arkansas
      • Little Rock、Arkansas、アメリカ、72202
        • 招待による登録
        • Arkansas Childrens Hospital
    • California
      • Loma Linda、California、アメリカ、92354
        • 募集
        • Loma Linda University Pediatric Subspecialty Clinics
        • 主任研究者：
          • David Michelson, MD
        • コンタクト：
      • Los Angeles、California、アメリカ、90027
        • まだ募集していません
        • Children's Hospital Los Angeles
        • コンタクト：
      • Madera、California、アメリカ、93636
        • まだ募集していません
        • Valley Children's Hospital
        • 主任研究者：
          • Raymund David, MD
        • コンタクト：
      • Palo Alto、California、アメリカ、94305
        • まだ募集していません
        • Stanford University
        • コンタクト：
          • Jenna Klotz, MD
          • 電話番号：408 426 5590
      • Sacramento、California、アメリカ、95817
        • 募集
        • University of California, Davis
        • コンタクト：
    • District of Columbia
      • Washington D.C.、District of Columbia、アメリカ、20010
        • まだ募集していません
        • Children's National Health System
        • コンタクト：
    • Florida
      • Gainesville、Florida、アメリカ、32610
        • 募集
        • University of Florida Clinical and Translational Science Institue
        • コンタクト：
      • Miami、Florida、アメリカ、33136
        • まだ募集していません
        • University of Miami
        • 主任研究者：
          • Mario Saporta, MD
        • コンタクト：
      • Miami、Florida、アメリカ、33155
        • 招待による登録
        • Nicklaus Children's Hospital
      • Orlando、Florida、アメリカ、32827
        • 招待による登録
        • Nemours Children's Hospital
      • St. Petersburg、Florida、アメリカ、33701
        • 募集
        • Johns Hopkins All Children's Hospital
        • コンタクト：
        • 主任研究者：
          • Matias Lopez Chacon, MD
    • Illinois
      • Chicago、Illinois、アメリカ、60611
        • 招待による登録
        • Lurie Children's Hospital of Chicago
    • Indiana
      • Indianapolis、Indiana、アメリカ、46202
        • 招待による登録
        • Indiana University Health - Riley Hospital for Children
    • Kansas
      • Kansas City、Kansas、アメリカ、66205
        • 招待による登録
        • University of Kansas Medical Center
    • Massachusetts
      • North Worcester、Massachusetts、アメリカ、01655
        • 招待による登録
        • University of Massachusetts Memorial Medical Center
    • Michigan
      • Grand Rapids、Michigan、アメリカ、49503
        • 招待による登録
        • Helen DeVos Children's Hospital
    • Minnesota
      • Minneapolis、Minnesota、アメリカ、55455
        • まだ募集していません
        • Masonic Children's Hospital - Discovery Clinic
        • 主任研究者：
          • Peter Karachunski, MD
        • コンタクト：
    • New York
      • The Bronx、New York、アメリカ、10467-2403
        • まだ募集していません
        • The Children's Hospital at Montefiore
        • 主任研究者：
          • Carla Zingariello, DO
        • コンタクト：
    • North Carolina
      • Charlotte、North Carolina、アメリカ、28207
        • 招待による登録
        • Atrium Health Neurosciences Institute
      • Durham、North Carolina、アメリカ、27710
        • 招待による登録
        • Duke University Medical Center and Childrens Health Center
    • Ohio
      • Cincinnati、Ohio、アメリカ、45229
        • 募集
        • Cincinnati Children's Hospital Medical Center
        • コンタクト：
    • Pennsylvania
      • Hershey、Pennsylvania、アメリカ、17033
        • 招待による登録
        • Penn State Milton S. Hershey Medical Center- Penn State Hershey Neuroscience Institute
      • Philadelphia、Pennsylvania、アメリカ、19104
        • 招待による登録
        • Children's Hospital of Philadelphia (CHOP)
    • Texas
      • Dallas、Texas、アメリカ、75235
        • 招待による登録
        • University of Texas Southwestern Medical Center
      • Denton、Texas、アメリカ、76208
        • 招待による登録
        • Neurology Rare Disease Center - Neurology & Neuromuscular Care Center
      • San Antonio、Texas、アメリカ、78229
        • 募集
        • The University of Texas Health Science Center at San Antonio
        • コンタクト：
    • Virginia
      • Charlottesville、Virginia、アメリカ、22903
        • 招待による登録
        • University of Virginia Health System (UVAHS) - Pediatric Neuromuscular Center
    • Washington
      • Seattle、Washington、アメリカ、98105
        • 招待による登録
        • Seattle Children's Hospital
    • Wisconsin
      • Milwaukee、Wisconsin、アメリカ、53226
        • まだ募集していません
        • Children's Wisconsin
        • 主任研究者：
          • Nancy Bass, MD
        • コンタクト：
  • プエルトリコ
    • Puerto Rico
      • San Juan、Puerto Rico、プエルトリコ、00912
        • 募集
        • San Jorge Children's Hospital
        • コンタクト：
        • 主任研究者：
          • Edwardo Ramos, MD
      • San Juan、Puerto Rico、プエルトリコ、00927
        • 招待による登録
        • FDI Clinical Research

参加基準

研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。

適格基準

就学可能な年齢

2年歳以上 (子、大人、高齢者)

健康ボランティアの受け入れ

いいえ

受講資格のある性別

男

サンプリング方法

確率サンプル

調査対象母集団

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).

説明

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.

研究計画

このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。

研究はどのように設計されていますか？

デザインの詳細

  • 観測モデル：COHORT
  • 時間の展望：PROSPECTIVE

この研究は何を測定していますか？

主要な結果の測定

結果測定
メジャーの説明
時間枠
Change in Height z-score
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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)
時間枠：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)
時間枠：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.

二次結果の測定

結果測定
メジャーの説明
時間枠
Changes in Duchenne Muscular Dystrophy Quality of Life questionnaire score
時間枠：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
時間枠：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).

協力者と研究者

ここでは、この調査に関係する人々や組織を見つけることができます。

スポンサー

Catalyst Pharmaceuticals, Inc.

協力者

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

捜査官

  • 主任研究者：Aravindham Veerapandiyan, MD、Arkansas Childrens Hospital
  • スタディディレクター：William Andrews, MD、Catalyst Pharmaceuticals

研究記録日

これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。

主要日程の研究

研究開始 (実際)

2024年9月25日

一次修了 (推定)

2032年2月1日

研究の完了 (推定)

2032年2月1日

試験登録日

最初に提出

2024年8月9日

QC基準を満たした最初の提出物

2024年8月19日

最初の投稿 (実際)

2024年8月21日

学習記録の更新

投稿された最後の更新 (実際)

2026年9月24日

QC基準を満たした最後の更新が送信されました

2026年9月22日

最終確認日

2026年9月1日

詳しくは

本研究に関する用語

キーワード

追加の関連 MeSH 用語

その他の研究ID番号

  • DMD-001

個々の参加者データ (IPD) の計画

個々の参加者データ (IPD) を共有する予定はありますか?

いいえ

医薬品およびデバイス情報、研究文書

米国FDA規制医薬品の研究

はい

米国FDA規制機器製品の研究

いいえ

この情報は、Web サイト clinicaltrials.gov から変更なしで直接取得したものです。研究の詳細を変更、削除、または更新するリクエストがある場合は、register@clinicaltrials.gov。 までご連絡ください。 clinicaltrials.gov に変更が加えられるとすぐに、ウェブサイトでも自動的に更新されます。

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