MedTrace Logo

A Clinical Study Evaluating the Safety and Efficacy of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1

NCT06191354 · Status: ACTIVE_NOT_RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2025-11-21

No results posted yet for this study

Summary

This is a clinical study to evaluate the safety and efficacy of gene therapy drug SKG0201 Injection in patients with spinal muscular atrophy Type 1 (SMA 1).

Conditions

  • Spinal Muscular Atrophy 1

Interventions

GENETIC

SKG0201 Injection

SKG0201 is a recombinant adeno-associated virus (rAAV) vector-based in vivo gene therapeutic product.

Sponsors & Collaborators

  • Kun Sun

    lead OTHER

Principal Investigators

  • Kun Sun, Doctor · Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

  • Yongguo Yu, Doctor · Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
180 Days
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-06-25
Primary Completion
2025-12-31
Completion
2025-12-31

Countries

  • China

Study Locations

More Related Trials

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 NCT06191354 on ClinicalTrials.gov