Набор по приглашению NCT06491927

Long Term Follow-up for RGX-202

— 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: No Intervention.
Кому может быть актуально: Состояния в реестре: Duchenne Muscular Dystrophy. Базовые параметры: Без ограничений · 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 Follow-up Study to Evaluate the Safety and Efficacy of RGX-202 Gene Therapy in Males With Duchenne Muscular Dystrophy (DMD)

Обзор

RGX-202-5101 is a long-term follow up study that evaluates the long-term safety and efficacy of RGX-202 in participants who have received RGX-202 (an investigative gene therapy designed to deliver a transgene for novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain) in a separate parent study.

Подробное описание

This is a multicenter, prospective, observational study to evaluate the long-term safety and efficacy of investigational RGX-202. Eligible participants are those who have undergone evaluation in a previous (parent) clinical study following a single intravenous infusion of RGX-202 for the treatment of DMD. Enrollment in the current long-term follow-up (LTFU) study will occur after the participant has completed or discontinued from the parent study. Participants will be followed in this study cumulatively for up to 5 years after RGX-202 administration (inclusive of the parent study). No investigational treatment will be administered under this protocol. The total study duration for each participant may vary depending on when he enrolls in the current study following RGX-202 administration in the parent study.

Вмешательства

Генная терапия No Intervention
Observational study

Первичные конечные точки

Evaluation of long-term safety of RGX-202 [Срок оценки: Baseline, 5 years inclusive of parent study]

Вторичные конечные точки (9)

Time to Stand (TTSTAND) [Срок оценки: Baseline, 5 years inclusive of parent study]
Time to Run/Walk (TTRW) [Срок оценки: Baseline, 5 years inclusive of parent study]
Time to Climb (TTCLIMB) [Срок оценки: Baseline, 5 years inclusive of parent study]
North Start Ambulatory Assessment (NSAA) [Срок оценки: Baseline, 5 years inclusive of parent study]
Peabody Developmental Motor Scale, Third Edition (PDMS-3); Body Control Subtest [Срок оценки: Baseline, 5 years inclusive of parent study]
Peabody Developmental Motor Scale, Third Edition (PDMS-3); Body Transport Subtest [Срок оценки: Baseline, 5 years inclusive of parent study]
Stride velocity 95th centile (SV95C) [Срок оценки: Baseline, 5 years inclusive of parent study]
Pharmacokinetics (PK) [Срок оценки: Baseline, 5 years inclusive of parent study]
Vector Shedding [Срок оценки: Baseline, 5 years inclusive of parent study]

Критерии участия

Критерии включения

The parent(s) or legal guardian(s) of the participant has/(have) provided written informed consent and (where applicable) Health Insurance Portability and Accountability Act (HIPAA) authorization after the nature of the study has been explained, prior to any research-related procedures; and, where applicable, the minor participant has provided written or verbal assent according to local requirements.
Must have undergone evaluation in a previous clinical study following a single IV infusion of RGX-202 for the treatment of DMD and either completed or withdrawn early from that study.
Participant and parent(s)/legal guardian(s) are willing and able to comply with scheduled visits, and study procedures.
Sexually active participants must be willing to use a medically accepted method of contraception from the time of the screening visit through 5 years after RGX-202 administration.