NCT06761183

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

• Status: Not Yet Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 9 (estimated)
• Sponsor: Beijing Tiantan Hospital · Academic / Other
• Sex: All
• Age: 40 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Ischemic Stroke

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2025-01-31
• Primary completion: 2026-12-31
• Completion: 2026-12-31
• First posted: 2025-01-07
• Last updated: 2025-01-07

Locations

1 site across 1 country: China