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Enrolling by Invitation

NCT Number: NCT07023965

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

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.

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Key information

Conditions

Peripheral Arterial Disease(PAD) Arterial Occlusive Disease Arterial Occlusive Diseases Arteriosclerosis Arteriosclerosis Obliterans Atherosclerosis Cardiovascular Diseases Chronic Disease Chronic Limb-Threatening Ischemia Critical Limb Ischemia (CLI) Diabetes Complications Diabetes Mellitus Diabetic Angiopathies Diabetic Foot Diabetic Foot Ulcer (DFU) Diabetic Foot Ulcer Ischemic Diabetic Neuropathies Disease Attributes Endocrine System Diseases Foot Ulcer Ischemia Leg Ulcer Pathologic Processes Pathological Conditions, Signs and Symptoms Peripheral Arterial Disease Peripheral Vascular Diseases Skin Diseases Skin Ulcer Skin and Connective Tissue Diseases Thromboangiitis Obliterans Vascular Diseases Vasculitis

Age range

20 year–85 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Beijing, Beijing Municipality, China

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

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

Treatment and study plan

NL003

Drug

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

Placebo

Drug

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

Primary outcomes

  1. Incidence of adverse events (AEs) and serious adverse events (SAEs)

    Time frame: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose

  2. Incidence of benign and malignant tumors

    Time frame: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose

  3. Incidence of significant vision loss, blindness, or other obvious visual abnormalities

    Time frame: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose

  4. Incidence of major cardiovascular and cerebrovascular events

    Time frame: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose

  5. Pregnancy status and its outcomes

    Time frame: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose

  6. Amputation-free survival of the trial limb

    Time frame: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose

    Amputation-free survival of the trial limb is one of the key endpoints for efficacy assessment. The time from the first dose administration to the occurrence of amputation above the ankle of the trial limb or death due to any cause, whichever occurs first.

  7. Amputation-free survival or target vessel revascularization composite endpoint

    Time frame: 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 is one of the key endpoints for efficacy assessment. The time from the first dose administration to the occurrence of amputation above the ankle of the trial limb, death due to any cause, or target vessel revascularization* of the trial limb, whichever occurs first.*Target vessel revascularization: bypass grafting, endovascular revascularization, thrombectomy, or thrombolysis of the trial limb.

  8. Amputation-free survival or target vessel revascularization or neovascularization therapies (including stem cell or gene therapy) composite endpoint

    Time frame: 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 is one of the key endpoints for efficacy assessment. The time from the first dose administration to the occurrence of amputation above the ankle of the trial limb, death due to any cause, or target vessel revascularization and neovascularization therapies (including stem cell or gene therapy) of the trial limb, whichever occurs first.

Secondary outcomes

  1. All-cause mortality rate, time to death

    Time frame: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose

  2. Major amputation rate of the trial limb (amputation plane above the ankle)

    Time frame: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose

  3. Minor amputation rate of the trial limb (below the ankle, including toe amputation)

    Time frame: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose

  4. Incidence of target vessel revascularization of the trial limb

    Time frame: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose

  5. Rehospitalization rate for worsening ischemic symptoms of the trial limb

    Time frame: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose

  6. Incidence of death due to acute myocardial infarction or stroke

    Time frame: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose

  7. Time to amputation of the trial limb

    Time frame: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose

  8. Level of amputation of the trial limb (above the knee, knee-ankle amputation, below the ankle amputation/toe amputation)

    Time frame: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose

  9. Change in Rutherford classification of the trial limb compared to baseline at first dose

    Time frame: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose

    The Rutherford classification is a standardized system used to assess the severity of peripheral arterial disease (PAD) in the trial limb. It ranges from category 0 (asymptomatic) to category 6 (ischemic rest pain with gangrene). Higher categories indicate more severe disease. The change in Rutherford classification will be measured by comparing the classification at the first dose to the baseline classification.

  10. Change in rest pain of the trial limb assessed by Numeric Rating Scale (NRS) compared to baseline at first dose

    Time frame: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose

    The NRS is a pain assessment tool that ranges from 0 to 10, where 0 indicates no pain and 10 indicates the worst possible pain. A higher score on the NRS indicates worse pain. The change in rest pain will be determined by comparing the NRS score at the first dose to the baseline NRS score.

  11. Change in ulceration/necrosis of the trial limb compared to baseline at first dose

    Time frame: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose

Sponsors and collaborators

Lead sponsor

Beijing Northland Biotech. Co., Ltd.

Industry

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2027
Study completion
2027
First posted
Jun 17, 2025
Registry last updated
Jul 11, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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