Contact SCGE




Gene Therapy Trial Report

Summary

Safety and Preliminary Efficacy of NXL-001 in Patients with Ischemic Stroke


NCTID NCT06761183 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Ischemic Stroke
Disease Ontology Term DOID:3526
Compound Name NXL-001
Sponsor Beijing Tiantan Hospital
Funder Type Other
Recruitment Status
Not yet recruiting
Enrollment Count 9 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant NeuroD1
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Intracerebral
Drug Product Type Viral vector
Target Tissue/Cell Astrocyte
Delivery System Viral transduction
Vector Type AAV9
Editor Type none
Dose 1 Dose escalation with 3 levels, unknown concentrations

Study Record Dates


Current Stage Early phase1
Submit Date 2024-12-27
Completion Date 2026-12-31
Last Update 2025-01-07

Participation Criteria


Eligible Age 40 Years - 75 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * Age 40-75 years, inclusive, gender is not limited. * Clinical diagnosis of ischemic stroke confirmed by neuro-imaging(CT , MRI,et al). * 2-4 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 3-4 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 ≥ 1:100 * 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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
FDA Designations
Recent Updates This product is also being evaluated in Alzheimer's Disease indication in IIT trial in China

Resources/Links