Follow-up Study Using Gene Therapy for Critical Limb Ischemia (NL003-CLI-III-L)

Официальное название

Multicenter Cohort Long-term Follow-up Study of Participants From Phase III Clinical Trials of Donaperminogene Seltoplasmid Injection for Critical Limb Ischemia

Обзор

The goal of this observational study is to learn about the long-term effects of Donaperminogene Seltoplasmid Injection (NL003) in participants who have been received drug NL003 or placebo at least one dose from the parent phase III clinical study to treat their critical limb ischemia (CLI). The main questions it aims to answer are: * First, what medical problems do participants have after taking drug NL003 to treat CLI? * Second, does drug NL003 make CLI participants live longer without serious problems (amputations or target vessel revascularizations) ? Participants who have already received drug NL003 for CLI will complete online surveys about their health conditions. This study will continue until at least 36 months after the participant's first dose.

Подробное описание

This study is a multicenter, open-label, long-term follow-up cohort study. A remote follow-up system is recommended as a tool for the study, however, in-person visits or phone calls at the research center will be conducted if remote follow-up is not feasible. The study will retrospectively collect data from the last visit in the parent Phase III study and prospectively gather data following informed consent. It will continue until at least 36 months after the first dose administered to the last enrolled participant.

Вмешательства

• Препарат NL003
  This is an observational study, and the interventions have already been administered in the preceding Phase III clinical trials.
• Препарат Placebo
  This is an observational study, and the interventions have already been administered in the preceding Phase III clinical trials.

Первичные конечные точки

• Incidence of adverse events (AEs) and serious adverse events (SAEs) [Срок оценки: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose]
• Incidence of benign and malignant tumors [Срок оценки: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose]
• Incidence of significant vision loss, blindness, or other obvious visual abnormalities [Срок оценки: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose]
• Incidence of major cardiovascular and cerebrovascular events [Срок оценки: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose]
• Pregnancy status and its outcomes [Срок оценки: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose]
• Amputation-free survival of the trial limb [Срок оценки: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose]
• Amputation-free survival or target vessel revascularization composite endpoint [Срок оценки: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose]
• Amputation-free survival or target vessel revascularization or neovascularization therapies (including stem cell or gene therapy) composite endpoint [Срок оценки: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose]

Вторичные конечные точки (11)

• All-cause mortality rate, time to death [Срок оценки: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose]
• Major amputation rate of the trial limb (amputation plane above the ankle) [Срок оценки: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose]
• Minor amputation rate of the trial limb (below the ankle, including toe amputation) [Срок оценки: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose]
• Incidence of target vessel revascularization of the trial limb [Срок оценки: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose]
• Rehospitalization rate for worsening ischemic symptoms of the trial limb [Срок оценки: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose]
• Incidence of death due to acute myocardial infarction or stroke [Срок оценки: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose]
• Time to amputation of the trial limb [Срок оценки: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose]
• Level of amputation of the trial limb (above the knee, knee-ankle amputation, below the ankle amputation/toe amputation) [Срок оценки: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose]
• Change in Rutherford classification of the trial limb compared to baseline at first dose [Срок оценки: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose]
• Change in rest pain of the trial limb assessed by Numeric Rating Scale (NRS) compared to baseline at first dose [Срок оценки: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose]
• Change in ulceration/necrosis of the trial limb compared to baseline at first dose [Срок оценки: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose]

Критерии участия

Критерии включения

• Participants who have previously enrolled in Phase III clinical trials of recombinant human hepatocyte growth factor plasmid injection (Donaperminogene Seltoplasmid Injection) for the treatment of critical limb ischemia (including ulcers and rest pain) and have received at least one dose of the treatment.

Критерии исключения

• Participants who refused to provide written informed consent;
• Participants who refused to cooperate with the retrospective or prospective data collection.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Китай · 5 центров

• Peking Union Medical College Hospital, Chinese Academy of Medical Sciences — Пекин
• Chifeng Municipal Hospital — Chifeng
• zhongshan Hospital Affiliated of Dalian University — Dalian
• The First Affiliated Hospital, Zhejiang University School of Medicine — Ханчжоу
• The First Affiliated Hospital of Xi 'an Jiaotong University — Сиань

Идентификаторы

NCT: NCT07023965 · NL003-CLI-III-L

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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