Tundra Space

Tundra Space

Clinical Research Directory

Browse clinical research sites, groups, and studies.

Back to Studies
ACTIVE NOT RECRUITING
NCT07618585
PHASE1

Safety and Efficacy of Intrathecal NTF001 Injection in ALS

Sponsor: Ruijin Hospital

View on ClinicalTrials.gov

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 adverse events (SAEs). Secondary outcome measures include changes in the ALS Functional Rating Scale-Revised (ALSFRS-R), quality-of-life assessments, and neurological function. This study aims to provide preliminary clinical evidence regarding the safety and potential efficacy of intrathecal NTF001 injection in patients with ALS.

Official title: A Study of the Safety and Efficacy of Intrathecal NTF001 Injection in the Treatment of Amyotrophic Lateral Sclerosis

Key Details

Gender

All

Age Range

18 Years - 65 Years

Study Type

INTERVENTIONAL

Enrollment

12

Start Date

2026-04-01

Completion Date

2028-03-31

Last Updated

2026-06-01

Healthy Volunteers

No

Interventions

GENETIC

NTF001 Injection

NTF001 is an investigational AAV-mediated gene therapy product designed to express human neuron-derived neurotrophic factor (NDNF). It will be administered once by intrathecal injection to patients with amyotrophic lateral sclerosis (ALS). Participants will receive NTF001 according to the assigned dose cohort and will be followed for safety, tolerability, and preliminary efficacy.

Locations (1)

Ruijin Hospital, Shanghai Jiao Tong University School of Medicine

Shanghai, Shanghai Municipality, China