Набор по приглашению NCT06270719

An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy

— Наблюдательное Duchenne Muscular Dystrophy

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают: В протоколе указаны: Delandistrogene Moxeparvovec, Standard of Care.
Кому может быть актуально: Состояния в реестре: Duchenne Muscular Dystrophy. Базовые параметры: от 4 лет · Мужчины.
Что важно проверить: Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится: США
Следующий шаг: Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

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Официальное название

A Long-term Multicenter Prospective Observational Study Evaluating the Comparative Effectiveness and Safety of Sarepta Gene Transfer Therapy vs. Standard of Care in Participants With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice

Обзор

This is a multicenter, prospective, observational Phase 4 study including a post marketing safety requirement, designed to collect both medical history data and prospective data on Duchenne muscular dystrophy (DMD) treatment outcomes in participants receiving delandistrogene moxeparvovec (ELEVIDYS) as part of clinical care, compared to participants with DMD receiving or prescribed to start chronic glucocorticoid treatment at the time of study enrollment in routine clinical practice.

Вмешательства

Генная терапия Delandistrogene Moxeparvovec
No study medication will be provided by the sponsor during this study.
Препарат Standard of Care
No study medication will be provided by the sponsor during this study.

Первичные конечные точки

Mean Change From Baseline in Time to Walk/Run 10 Meters (10MWR) (Calculated Velocity) at Month 12 [Срок оценки: Baseline, Month 12]
Number of Participants Experiencing Acute Liver Injury (ALI) [Срок оценки: Baseline through Month 12]

Вторичные конечные точки (11)

Time to Rise From Floor (Supine to Stand) [Срок оценки: Up to 10 years]
Loss of Ambulation (LOA) [Срок оценки: Up to 10 years]
Performance of Upper Limb (PUL) Version 2.0 Entry Item A Score or Brooke Upper Extremity Scale Score [Срок оценки: Up to 10 years]
Patient-reported Outcomes Measurement Information (PROMIS) Domain Scores of Mobility, Upper Extremity, and Fatigue [Срок оценки: Up to 10 years]
Pulmonary Function as Measured by Forced Vital Capacity (FVC) [Срок оценки: Up to 10 years]
Pulmonary Function as Measured by FVC Percent Predicted (FVC%p) [Срок оценки: Up to 10 years]
Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF), as Measured by Echocardiogram (ECHO) or Cardiac MRI (cMRI) [Срок оценки: Up to 10 years]
Number of Participants Experiencing Serious ALI and Acute Liver Failure (ALF) [Срок оценки: Baseline through Month 12]
Number of Participants Experiencing Complications Associated with ALI and ALF [Срок оценки: Up to 10 years]
Survival Time [Срок оценки: Up to 10 years]
Time to First Vertebral Body (Spine) Fracture [Срок оценки: Up to 10 years]

Критерии участия

Критерии включения

Has an established clinical diagnosis of DMD based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test.
Is currently receiving or has been prescribed to start chronic glucocorticoid therapy at the time of this observational study enrollment.

For ELEVIDYS-treated Participants (Cohorts 1a, 1b, and 1c):

Is at least 4 years of age at the time of infusion
Will either: a) be initiating or has initiated ELEVIDYS within the last 30 days in routine clinical practice at the time of this observational study enrollment, or b) was administered ELEVIDYS in routine clinical practice and has the required minimum dataset for entry into the observational study per Sponsor approval

For Standard of Care Comparators (Cohort 2):

Is at least 4 years of age at the time of enrollment
Is unexposed to DMD gene therapy at the time of this observational study enrollment

Критерии исключения

Has any deletion of exon 8 and/or exon 9 in the DMD gene.
Is currently participating in any DMD interventional study at the time of this observational study enrollment.
Has any prior exposure to DMD gene therapy other than that described for Cohort 1c (ELEVIDYS Retrospectively Treated Cohort).
Has a medical condition or confounding circumstances (for example, prior traumatic limitation for mobility or significant behavioral comorbidity) that, in the opinion of the Investigator, might compromise: