Clinical Trial: Safety and Efficacy of Intrathecal NTF001 Injection in ALS

U.S., June 2 -- ClinicalTrials.gov registry received information related to the study (NCT07618585) titled 'Safety and Efficacy of Intrathecal NTF001 Injection in ALS' on May 25.

Brief Summary: This is a single-arm, open-label, early-phase clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of intrathecal NTF001 injection, an AAV-mediated human neuron-derived neurotrophic factor gene therapy, in patients with amyotrophic lateral sclerosis (ALS).

12 patients with ALS will be enrolled. Each participant will receive a single intrathecal administration of NTF001 and will be followed for 52 weeks after treatment. The primary outcome measures include treatment-emergent adverse events (TEAEs) and serious advers...