Safety and Preliminary Efficacy of NXL-001 in Patients With Ischemic Stroke
ConditionIschemic Stroke
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age40-75
SponsorBeijing Tiantan Hospital
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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.
Trial design
Design model
Sequential
Treatments tested in this trial
intracerebral stereotactic injection of NXL-001
Geneticintracerebral 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.
Treatment groups
9 Participants
are divided into 1 treatment groupGroup A: intracerebral stereotactic injection of NXL-001Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
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 doseSecondary outcomes
1
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 dose2
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 dose3
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 dose4
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 doseSponsors and contacts
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Source ClinicalTrials.gov