An Early Phase 1 interventional study of intracerebral stereotactic injection of NXL-001 in Ischemic Stroke, sponsored by Beijing Tiantan Hospital. Recruiting at 1 site in China. Open to participants aged 40 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-09-09.
Sponsored by Beijing Tiantan Hospital · Early Phase 1, Interventional, and Treatment
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.
The death of neurons after stroke is the direct cause of brain function loss, and the difficulty of neurons to regenerate themselves in the adult brain is an important reason for the lack of effective treatment for stroke. Replacing the lost neurons and then reconstructing the functional connectivity between neurons is the key to improving stroke symptoms. NXL-001 is a gene therapy, using an AAV9 (adeno-associated virus 9) vector to deliver and express the neurodevelopmental transcription factor NeuroD1 to directly convert astrocytes into functional neurons. NXL-001 has been shown to regenerate neurons and improve motor function when administered in animal models of stroke. The primary objective of this single-arm, open-label, single center, dose-escalation study is to evaluate the safety and tolerability of intracerebral stereotactic injection of NXL-001. The secondary objective is to preliminarily evaluate the efficacy of NXL-001 in patients with chronic neuronal deficits from ischemic stroke and to determine its safe and effective dose range. This dose escalation study involves three cohorts, with 3 subjects in each group to receive a single stereo-tactically intracerebral injection of NXL-001 at escalating doses.
2,593 studies on the registry are indexed under Ischemic Stroke; 930 are open to participants now.
This study's planned enrollment of 9 is below the median of 120 across 1,752 interventional studies indexed under Ischemic Stroke.
Browse Ischemic Stroke studies →Beijing Tiantan Hospital is the lead sponsor of 465 studies on the registry; 282 are open to participants now.
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Exclusion Criteria:
intracerebral stereotactic injection of NXL-001: with 3 subjects in each group to receive a single stereo-tactically intracerebral injection of NXL-001 at escalating doses.
Genetic: intracerebral stereotactic injection of NXL-001
intracerebral stereotactic injection of NXL-001: with 3 subjects in each group to receive a single stereo-tactically intracerebral injection of NXL-001 at escalating doses.
Safety and tolerability of NXL-001
Incidence of adverse events (AE) and serious adverse events (SAE) according to CTCAE 5.0
Time frame: baseline to month 3 after dose
Change in MRI images from baseline at month 12
Changes in MRI images of cerebral infarction areas before and after treatment
Time frame: Baseline (Screening), and on day 7, month 1,3,6 and 12 after dose
Change in PET images from baseline at month 12
Changes in PET images will be used to assess metabolic changes in the infarct and its surrounding area before and after treatment
Time frame: Baseline (Screening), and at month 3 and 12 after dose
Change in the National Institutes of Health Stroke Scale (NIHSS) Total Score from baseline to month 12
The NIHSS is a standardized method used to measure the level of impairment caused by a stroke. The scale consists of 11 items that measure several aspects of brain function, including consciousness, vision, sensation, movement, speech, and language. A certain number of points are given for each impairment uncovered during a focused neurological examination. A maximal score of 42 represents the most severe and devastating stroke with 0=no stroke, 1-4=minor stroke, 5-15=moderate stroke, 15-20=moderate/severe stroke, and 21-42=severe stroke.
Time frame: Baseline (Screening), and on day1, 7, and at month 1,3,6 and 12 after dose
Change in the Modified Rankin Scale (mRS) Response from Baseline to Month 12
The mRS is a commonly used scale for measuring the degree of disability in daily activities for patients who have suffered a stroke. The mRS is an ordinal scale from 0 (no symptoms at all) to 5 (severe disability; requiring constant nursing care and attention, bedridden, incontinent) with a sixth category of death.
Time frame: Baseline (Screening), and on day1, 7, and at month 1,3,6 and 12 after dose
Change in the Fugl-Meyer Assessment (FMA) from Baseline to Month 12
The FMA is used as a clinical measure of body function impairment after stroke that assesses several dimensions of motor impairment, including range of motion in upper and lower limbs, reflex activity, volitional movement, and coordination.The Fugl-Meyer motor total score ranged from 0 (hemiplegia) to a maximum of 100 points (normal motor performance), and is comprised of a 33-item upper extremity subscale (UE-FMMS) and the 17-item lower extremity subscale (LE-FMMS). Items were scored on a 3-point ordinal scale: 0= cannot perform; 1= partial motion; 2= full motion Individual items are summed to determine scores for the 2 subscale scores, as well as a motor total score (total of the 2 subscales UE-FMMS and LE-FMMS). As a result, the UE-FMMS subscale score ranged from 0 to 66 and the LE-FMMS subscale score ranged from 0 to 34. A maximally affected person will have a score of 0 while a fully functional person will have the highest score (i.e., 34 for LE-FMMS, 66 for UE-FMMS, 100 for FMM)
Time frame: Baseline (Screening), and at month 1,3,6 and 12 after dose
Change in the modified Ashworth Scale from baseline to month 12
The Modified Ashworth Scale (MAS) is the most commonly used clinical scale to measure hypertonic or spastic muscle. The scale ranges from 0 to 4, with the muscle acting across the joint rated based on what point during flexion or extension resistance or a catch is noted. A score of 0 indicates no resistance to passive movement, with a 4 indicating a rigid joint.
Time frame: Baseline (Screening), and at month 1,3,6 and 12 after dose
Change in the gait function scale from baseline to month 12
The gait function scale consists of 4 items that measure several aspects of gait function quantitatively and semi-quantitatively, including balance, Six-meter Walk test, Tandem walking test, and Quick sit-up test.
Time frame: Baseline (Screening), and at month 1,3,6 and 12 after dose
Change in the Action Research Arm Test (ARAT) from baseline to month 12
The Action Research Arm Test (ARAT) is a 19-item observational measure to assess upper extremity performance (coordination, dexterity and functioning) in stroke recovery, brain injury and multiple sclerosis populations. Items comprising the ARAT are categorized into four subscales (grasp, grip, pinch and gross movement) and arranged in order of decreasing difficulty, with the most difficult task examined first, followed by the least difficult task. Each item is rated 4 (0-3 points), 0 = no movement；1= movement task is partially performed；2 = movement task is completed but takes abnormally long；3 = movement is performed normally. Scores on the ARAT may range from 0-57 points, with a maximum score of 57 points indicating better performance.
Time frame: Baseline (Screening), and at month 1,3,6 and 12 after dose
Change in the EQ-5D-5L scale from baseline to month 12
The EQ-5D, EuroQol Five Dimensions Questionnaire, is a set of standardized scales measuring health status. The EQ-5D-5L descriptive system consists of five dimensions (each describing a specific aspect of health): mobility, self-care, daily activities, pain or discomfort, anxiety or depression. Each dimension has five response levels: no problems, slight problems, moderate problems, severe problems, unable to/extreme problems. The respondent is asked to indicate his/her health state by checking the box next to the most appropriate response level for each of the five dimensions.
Time frame: Baseline (Screening), and at month 1,3,6 and 12 after dose
Change in the Electroencephalogram（EEG）from baseline to month 12
EEG is a simple, low-cost, non-invasive tool that can provide information about the changes occurring in the cerebral cortex during the recovery process after stroke. EEG provides data on the evolution of cortical activation patterns.
Time frame: Baseline (Screening), and at month 1,3,6 and 12 after dose
Change in the Motor-evoked potential (MEP) from baseline to month 12
Motor evoked potential (MEP) amplitude and threshold are predictors of functional outcome in the early stages after stroke, and improvement in these parameters usually accompanies motor recovery.
Time frame: Baseline (Screening), and at month 1,3,6 and 12 after dose
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Beijing Tiantan Hospital