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

Clinical Trial Update: SKG0201 Gene Therapy for Spinal Muscular Atrophy Type 1 (SMA 1)

Trial ID: NCT06191354
Study Title: A Multicenter, Open, Dose-Escalation Clinical Study Evaluating the Safety, Initial Efficacy, and Immunogenicity of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1
Sponsor: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Principal Investigator: Prof. Kun Sun

🔬 Overview

This clinical trial investigates SKG0201, an investigational gene therapy developed to treat Spinal Muscular Atrophy Type 1 (SMA 1). This rare genetic disorder is marked by severe muscle weakness and is often fatal in infants without treatment.

SKG0201 is a recombinant adeno-associated virus (rAAV) vector-based in vivo gene therapy, delivered in a one-time dose.

🎯 Objectives

• Primary Goals:

  • Assess safety through incidence of adverse events (AEs) and serious adverse events (SAEs)

  • Detect dose-limiting toxicities (DLT)

• Secondary Goals:

  • Survival rate without need for permanent ventilation

  • Improvement in motor function using CHOP-INTEND scores

  • Achievement of developmental milestones (based on BSID-III criteria)

🧬 Study Design

• Type: Interventional (Gene Therapy)

• Model: Single Group Assignment (Open-Label)

• Estimated Enrollment: 12 infants

• Intervention: SKG0201 Injection

• Duration:

  • Start: June 25, 2023

  • Estimated Primary Completion: June 2025

  • Estimated Study Completion: December 2025

👶 Eligibility Criteria

Inclusion:

• Genetically confirmed SMA Type 1 (bi-allelic mutations in SMN1)

• ≤ 180 days old at time of infusion

• Clinical presentation consistent with SMA 1

• Legal guardian consent and commitment to follow study protocol

Exclusion:

• Oxygen saturation <96% without support

• Severe underweight for age

• Active serious infections or known hypersensitivity to corticosteroids

• Prior SMA treatments (e.g., Zolgensma, Spinraza)

• Anticipated major surgery during study period

📍 Trial Sites in China

1. Xinhua Hospital, Shanghai

2. National Children’s Medical Center, Shanghai (Pending Recruitment)

3. West China Second Hospital, Sichuan University, Chengdu

📞 Contact for Participation

Dr. Yongguo Yu
📧 info@cancerfax.com
📞 +86 182 1759 2149

🌐 Additional Details

• Status: Recruiting

• Regulatory Oversight: Not FDA-regulated (China-specific)

• Trial Phase: Dose-escalation without formal phase designation

✍️ Call to Action

At CancerFax.com, we bring you the most promising clinical trials in gene therapy and rare genetic disorders. If your child or a loved one is affected by SMA Type 1, consider this cutting-edge trial in China.

📩 Contact us to learn how we can help connect you to this or other trials globally.