Tests NXL-001 gene therapy injected into the brain after ischemic stroke
Tests a brain-injected gene therapy in adults with lasting effects of ischemic stroke.
Plain-language title generated from the registry record
Registry title: Safety and Preliminary Efficacy of NXL-001 in Patients With Ischemic Stroke
Key facts
- Where
- Beijing, ChinaSee the site
- Phase
- Early phase 1
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for adults aged 40-75 who had an ischemic stroke 2-12 months ago and still have moderate to severe movement problems. The trial tests a gene therapy, NXL-001, given as one injection into the brain.
- Age
- 40 to 75 years
- Sex
- Men and women can join.
- Disease stage
- Ischemic stroke 2-12 months ago, confirmed by a brain scan (CT or MRI). The stroke area on MRI is 20-80ml, with injury to the motor cortex and the corticospinal tract. Moderate to severe movement problems remain after standard rehabilitation: NIHSS score 6-20, motor score 2-4 on the affected arm or leg.
- Earlier treatment
- Standard rehabilitation therapy must already have been tried. People who had any investigational drug within 3 months, or any other cell or gene therapy for stroke, cannot join.
- Biomarker or test result
- Blood anti-AAV9 antibody level must be below 1:1000.
Main reasons for not taking part
- Movement problems from a stroke in the back part of the brain, or from another cause
- History of epilepsy, brain infection, multiple sclerosis or bleeding in the brain
- Need for blood-thinning medicine (anticoagulants)
- Active infection such as HIV, hepatitis or syphilis
- Pregnant or breastfeeding women
Read by machine on 9 November 2026.
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 40 and 75 years old?
Source: NCT06761183, who can take part
Where in China
- Beijing Tiantan HospitalBeijing, ChinaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Beijing Tiantan Hospital
- Trial code
- NCT06761183
- Record updated
- 9 September 2026
- Original description
This is a single-arm, open-label, single center, dose-escalation exploratory clinical study to evaluate the safety and tolerability of a single intracerebral injection of NXL-001, a NeuroD1 base gene therapy, in patients with chronic neuronal deficits from ischemic stroke.Read full original descriptionShow less
Safety and Preliminary Efficacy of NXL-001 in Patients With Ischemic Stroke
This is a single-arm, open-label, single center, dose-escalation exploratory clinical study to evaluate the safety and tolerability of a single intracerebral injection of NXL-001, a NeuroD1 base gene therapy, in patients with chronic neuronal deficits from ischemic stroke.
Inclusion: 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: 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; 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); Motor deficit due to ischemic stroke of posterior circulation; 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; 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; 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; Serum anti-AAV9 antibody titers ≥ 1:1000; 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; Motor deficit due to any other causes; Any contraindications to MRI scanning (such as implanted pacemaker, infusion pump etc.); Requirement for anticoagulants; Pregnant or lactating female subjects; 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.