Safety and early test of NXL-001 for people with chronic deficits after ischemic stroke

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a dose-escalation, open-label, single-arm study conducted at one center to assess safety and tolerability of a single intracerebral injection of the NeuroD1-based gene therapy NXL-001 delivered via AAV9 in adults with chronic deficits after ischemic stroke. The trial uses three cohorts with escalating doses, each enrolling three participants, to identify a safe dose range and collect preliminary signals of efficacy on motor function and neural connectivity. Primary objectives focus on safety and tolerability; secondary objectives seek initial evidence of functional improvement. Eligible participants are adults aged 40-75 years with 2–12 months since stroke, motor impairment, specific lesion characteristics on MRI, and ability to participate in rehabilitation. The study is sponsored by Beijing Tiantan Hospital and conducted at that site in Beijing.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Age 40-75 years, inclusive, gender is not limited.
* Clinical diagnosis of ischemic stroke confirmed by neuro-imaging（CT , MRI，et al）.
* 2-12 months after the onset of ischemic stroke.
* MRI scan shows that the stroke lesion is 20-80ml in size, with cerebral motor cortex injury, and DTI shows corticospinal tract injury.
* Persisting moderate to severe motor function impairment due to stroke after standardized and guide-recommended rehabilitation therapy, characterized by baseline NIHSS score of 6-20 points, and a motor score of 2-4 on the affected upper or lower limb.
* Expected survival ≥ 12 months.
* The patient or his/her legal representative clearly understands, voluntarily participates in the study and signs the informed consent form.
* The subject is willing and able to return for follow-up visits as required by the trial protocol.
* Able to undergo rehabilitation training and treatment;
* Male and female subjects participating in the clinical study must agree to use an adequate birth control method for at least 6 months after administration

Exclusion Criteria:

* Motor deficit due to ischemic stroke of posterior circulation.
* Motor deficit due to any other causes.
* History of epilepsy.
* History of encephalitis, meningitis, multiple sclerosis or other central nervous system infections.
* History of intracranial hemorrhage and subarachnoid hemorrhage.
* History of severe head trauma within the past 5 years.
* Any contraindications to MRI scanning (such as implanted pacemaker, infusion pump etc.).
* Serum anti-AAV9 antibody titers ≥ 1:1000
* History of malignant tumors within 5 years before screening (except for adequately treated cervical carcinoma in situ, papillary thyroid cancer, basal cell or squamous epithelial cell skin cancer, localized prostate cancer after radical surgery, and breast ductal carcinoma in situ).
* Active infections, including but not limited to human immunodeficiency virus (HIV), hepatitis A, B or C, syphilis, etc.
* Received any investigational drugs within 3 months (or 5 half-lives of the investigational drug, whichever is longer) of initial screening.
* Received any other cell and/or gene therapy for stroke.
* Requirement for anticoagulants.
* Intermittent use of oral anti-spasticity medications (stop/start date from 1-month prior-to and 3-month post- NXL-001 administration). Use of oral anti-spasticity medications are acceptable if they have been taken regularly for at least one month prior to NXL-001 administration).
* Pregnant or lactating female subjects.
* Insufficient reserved functions of liver, kidney and bone marrow: Neutrophil count \<1,500/mm 3 ; platelets \<100, 000/mm 3 ; hemoglobin \<9.0 g/dL; serum creatinine \>1.5 times the upper limit of normal range (ULN) ; renal function eGFR \< 60mL/min/ 1.73m2 ; Bilirubin, aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \>2.5 times ULN; activated partial prothrombin time ( APTT ) or international normalized ratio ( INR ) \>1.3 times ULN.
* Poorly controlled illness judged by the investigator at screening, including cardiovascular system (decompensated heart failure (NYHA classification III and IV), unstable angina, acute myocardial infarction), Respiratory system, digestive system, endocrine metabolic system, neuropsychiatric system, blood system and immune system diseases, etc.
* Based on medical history and investigator's judgment, the subject is at significant risk of suicide.
* In the investigator's judgment, the subject has any other factors deemed inappropriate for participation in this trial.

Where it's running

• Beijing Tiantan Hospital
  Beijing, Beijing Municipality, China Recruiting

Study contacts

• Study coordinator: Zhu Yahui, MD
• Email: zhuyahuidr@sina.com
• Phone: 0086-13439182912

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.