In this article
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
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.
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)
Xinhua Hospital, Shanghai
National Children’s Medical Center, Shanghai (Pending Recruitment)
West China Second Hospital, Sichuan University, Chengdu
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.
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About Dr. Nishant Mittal
Dr. Nishant Mittal is a highly accomplished researcher with over 13 years of experience in the fields of cardiovascular biology and cancer research. Significant contributions to stem cell biology, developmental biology, and innovative research techniques mark his career. Research Highlights Dr. Mittal's research has focused on several key areas: 1) Cardio…
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This article is for educational purposes only and should not replace medical advice, diagnosis, or treatment from a qualified oncology specialist. Every patient's case is different. Treatment decisions should always be made after a review of complete medical records by the treating medical team.
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