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A Study of the Safety and Preliminary Efficacy of NKG001 in Pediatric Patients With Type 1 or Type 2 Spinal Muscular Atrophy

NCT07617779 · Status: ACTIVE_NOT_RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 21

Last updated 2026-06-01

No results posted yet for this study

Summary

This study is a single-center, open-label, single-arm, non-randomized, single-dose, dose-escalation investigator-initiated trial (IIT) designed to evaluate the safety, tolerability, and preliminary efficacy of NKG001 Injection administered via different dosing regimens (intravenous \[IV\] alone or intravenous combined with intrathecal \[IV+IT\]) in subjects with Type 1 or Type 2 spinal muscular atrophy (SMA).

A total of 13-21 SMA subjects aged ≤60 months are planned to be enrolled. Based on age at enrollment, subjects will be stratified into two age cohorts for independent evaluation:

Age Cohort 1: subjects aged \<24 months at dosing; Age Cohort 2: subjects aged ≥24 months and ≤60 months at dosing.

Eligible subjects must carry 2 or 3 copies of the SMN2 gene.

Note: Subjects with 3 SMN2 copies must be able to sit independently but unable to walk independently.

Four dose cohorts are planned as follows:

S1: 6.0 × 10\^13 vg/kg, IV S2: 1.2 × 10\^14 vg/kg, IV S3: 6.0 × 10\^13 vg/kg, IV + 6 × 10\^13 vg/person, IT S4: 6.0 × 10\^13 vg/kg, IV + 1.2 × 10\^14 vg/person, IT

Subjects in the S1 cohort (2 SMN2 copies and aged \<24 months at dosing) and the S2 cohort (2 or 3 SMN2 copies and aged ≤60 months at dosing) will receive a single intravenous administration of NKG001 Injection.

Subjects in the S3 and S4 cohorts will receive a single administration of NKG001 Injection via combined intravenous and intrathecal routes. In each of these two cohorts, the first enrolled subject must have 2 SMN2 copies and be aged \<24 months at dosing, while the remaining subjects may have either 2 or 3 SMN2 copies and be aged ≤60 months at dosing.

Conditions

  • Spinal Muscular Atrophy (SMA)

Interventions

GENETIC

NKG001

NKG001 Injection

Sponsors & Collaborators

  • Xiangya Hospital of Central South University

    collaborator OTHER

  • Nikegen Pharmaceutical (Hangzhou) Company Limited

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
60 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-04-21
Primary Completion
2028-04-23
Completion
2030-04-23

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07617779 on ClinicalTrials.gov