Can a single brain injection rewire stroke damage?
NCT ID NCT06761183
First seen Sep 09, 2026 · Last updated Sep 10, 2026 · Updated 1 time
Summary
Researchers are testing a gene therapy called NXL-001 in people who have lasting movement problems from an ischemic stroke. The study enrolls 9 adults, 2 to 12 months after their stroke, who have moderate to severe arm or leg weakness despite rehabilitation. Each participant receives one stereotactic injection of NXL-001 directly into the brain, at increasing doses. The main goal is to check safety and tolerability, with brain scans at 12 months to look for changes in the stroke area.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- NXL-001, an experimental gene therapy that delivers the NeuroD1 gene directly into the brain
- What this could lead to
- If it works, a one-time brain injection could help stroke survivors regain movement by turning support cells into new neurons. This could offer an option for people whose rehabilitation has plateaued.
- What could go wrong
- This is a very early, tiny safety study with only 9 participants, so most experimental gene therapies fail. Brain injections carry risks of bleeding, infection, or immune reactions, and any benefit may not last or may not generalize.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Early phase 1
The earliest testing in people: a first look at safety, in a very small group.
- Participants
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About 9 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jan 2025
- Expected to finish
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Dec 2027
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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40 to 75 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Beijing Tiantan Hospital
RECRUITINGBeijing, Beijing Municipality, 10070, China
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