Gene therapy for improving blood flow in patients with Peripheral Artery Disease

A Double-Blind, Randomized, Placebo-Controlled, Single Center Study to Assess the Angiogenesis and Blood Perfusion Effect of NL003 in Patients With Critical Limb Ischemia by PETCT-RGD and MIBI

This study is testing a new gene therapy to see if it can improve blood flow and help people with Peripheral Artery Disease feel better.

Quick facts

• Phase: PHASE3
• Study type: Interventional
• Enrollment: 36 (estimated)
• Ages: 18 Years to 80 Years
• Sex: All
• Sponsor: Beijing Northland Biotech. Co., Ltd. (industry)
• Locations: 1 site (Beijing, Beijing)
• Trial ID: NCT05968118 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the effectiveness of NL003, a hepatocyte growth factor gene therapy, in patients with chronic lower limb ischemia due to Peripheral Artery Disease (PAD). The study utilizes advanced imaging techniques, including PET/CT-RGD and MIBI SPECT, to assess angiogenesis and blood perfusion in the affected limbs. Participants will be randomly assigned to receive either the investigational drug or a placebo, with injections administered at specified intervals. The trial aims to determine both the safety and efficacy of NL003 in enhancing blood flow and tissue health in PAD patients.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18-80 diagnosed with chronic lower limb ischemia and severe arterial stenosis or occlusion.

Not a fit: Patients with uncontrolled infections in the lower limb or recent serious cardiovascular events may not benefit from this study.

Why it matters

Potential benefit: If successful, this therapy could significantly improve blood flow and quality of life for patients suffering from severe PAD.

How similar studies have performed: Previous studies have shown promising results with similar gene therapy approaches in treating chronic limb ischemia, indicating potential for success in this trial.

Eligibility criteria

Inclusion Criteria:

1. 18-80 years, diagnosed with chronic lower limb ischemia;
2. According to DSA or CTA，severe stenosis (≥70%) or occlusion of one or more branches of superficial femoral artery, popliteal artery, anterior tibial artery, posterior tibial artery, and peroneal artery.
3. Patients with obvious symptoms of lower limb ischemia, including severe claudication, resting pain, ulcers, or gangrene.
4. Agree to use appropriate contraceptive methods during the trial; Female subjects of reproductive age, blood pregnancy test negative.
5. Signing the informed consent.
6. Can complete PETCT-RGD check and MIBI check;

Exclusion Criteria:

1. The foot or lower limb infection cannot be controlled, and amputation may be accepted within 3 months.
2. Those with cerebral infarction, cerebral hemorrhage, heart failure, unstable angina, acute myocardial infarction, and serious arrhythmia within 3 months;
3. Tumor patients diagnosed with malignant tumor or suspected of having malignant tumor after tumor screening;
4. Main iliac artery stenosis ≥ 70%;
5. Refractory hypertension (taking three or more antihypertensive drugs, systolic blood pressure ≥ 180mmHg or diastolic blood pressure ≥ 110mmHg);
6. Anti HIV antibody positive person, anti hepatitis C antibody positive person and RNA positive person, hepatitis B surface antigen positive person (if the subject is HBsAg positive, and judging by peripheral blood HBV DNA, the researcher believes that the subject is in a stable period of chronic hepatitis B, which will not increase the risk of the subject, the subject can be selected)
7. Patients with poor blood glucose control after treatment (HbA1c\>10%);
8. Persons allergic to contrast medium
9. The investigator believes that the patient's complications affect the evaluation of safety and effectiveness, or the expected survival period is less than 12 months;
10. Those who regularly drink more than 14 units of alcohol (1 unit=360 mL beer or 45 mL liquor or 150 mL wine with 40% alcohol) or drug abusers within 12 months before signing the informed consent form;
11. Acute lower limb ischemia or acute aggravation of chronic lower limb ischemia;
12. Those who received vascular reconstruction of the affected limb within 4 weeks before the informed consent form and whose blood supply improved after the operation
13. Serious liver or kidney disease or severe anemia judged by the investigator;
14. Those who cannot correctly describe symptoms and emotions;
15. Those who participated in other clinical trials within 3 months before signing the informed consent form.

Where this trial is running

Beijing, Beijing

• Peking Union Medical College Hospital — Beijing, Beijing, China (RECRUITING)

Study contacts

• Study coordinator: Yue Liu, MM

Conditions: Peripheral Artery Disease, Hepatocyte Growth Factor, NL003, Gene therapy, PET/CT-RGD, MIBI