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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Safety and Preliminary Efficacy of NXL-001 in Patients with Ischemic Stroke
NCTID
NCT06761183
(View at clinicaltrials.gov)
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.
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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
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
Dose 2
Dose 3
Dose 4
Dose 5
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
Locations
No.of Trial Sites
1
Locations
China
Regulatory Information
Has US IND
False
FDA Designations
Recent Updates
Resources/Links
Preclinical Publications
Differential neuronal reprogramming induced by NeuroD1 from astrocytes in grey matter versus white matter
Transcriptomic analyses of NeuroD1-mediated astrocyte-to-neuron conversion
New AAV tools fail to detect Neurod1-mediated neuronal conversion of Müller glia and astrocytes in vivo
Regeneration of Functional Neurons After Spinal Cord Injury via in situ NeuroD1-Mediated Astrocyte-to-Neuron Conversion
A NeuroD1 AAV-Based Gene Therapy for Functional Brain Repair after Ischemic Injury through In Vivo Astrocyte-to-Neuron Conversion