Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients

GC101 Gene Therapy for Spinal Muscular Atrophy Type 2 – Phase I/II Clinical Trial

ClinicalTrials.gov Identifier: NCT05901987
Sponsor: GeneCradle Inc.
Study Title: Safety and Efficacy of GC101 Gene Therapy in SMA Type 2
Trial Phase: Phase I/II
Study Status: Recruiting
Start Date: August 1, 2023 (Actual)
Primary Completion Date: May 2025 (Estimated)
Study Completion Date: December 2028 (Estimated)

🔬 Objective

This clinical trial aims to evaluate the safety, efficacy, and dose response of GC101, a self-complementary AAV9-based gene therapy, in treating children with SMA Type 2, delivered via a one-time intrathecal injection.

🧪 Study Design

• Type: Interventional

• Model: Sequential, single-arm, open-label

• Masking: None

• Enrollment: 33 patients (estimated)

• Dosing Groups:

  • Low Dose: 1.2×10¹⁴ vg/person (n=3)

  • Medium Dose: 2.4×10¹⁴ vg/person (n=3)

  • High Dose: 4.8×10¹⁴ vg/person (n=3)

💉 Intervention

• GC101 Gene Therapy: AAV9 vector carrying codon-optimized SMN1 gene driven by CMV enhancer + chicken β-actin promoter, delivered intrathecally.

📈 Outcome Measures

Primary Endpoints:

• Incidence of adverse events over 52 weeks

• Proportion of patients who can stand unassisted for ≥3 seconds at Month 12 (ages 6–24 months)

• Change from baseline in HFMSE scores at Month 12 (ages 24–60 months)

Secondary & Exploratory Endpoints:

• Proportion of patients who walk independently for 5 steps

• HFMSE score improvement ≥3 points

• Improvement in ventilation time, motor milestones, and SMAIS independence scores

✅ Inclusion Criteria

• Age between 6 to 60 months

• Confirmed Type 2 SMA (biallelic SMN1 mutation + 2 SMN2 copies)

• Onset between 6 and 18 months

• Able to sit but never walked or stood alone

• Informed consent from legal guardian

❌ Exclusion Criteria

• Prior gene therapy participation

• Recent nusinersen (<4 months) or risdiplam (<15 days)

• AAV9 antibody titer ≥1:200

• Need for ventilatory support ≥12 hrs/day or O₂ sat <95%

• Positive for HIV, HBV, HCV, or syphilis

• Abnormal liver/hematologic labs (e.g., ALT/AST >3× ULN)

• Glucocorticoid allergy, active infections, recent vaccinations (<2 weeks)

🏥 Participating Locations in China

• Peking University First Hospital, Beijing

• Bayi Children’s Hospital (PLA General Hospital), Beijing

• West China Second University Hospital, Chengdu

• Children’s Hospital of Chongqing Medical University, Chongqing

• Tongji Medical College Affiliated Children’s Hospital, Wuhan

📞 Contact for Participation

GeneCradle Inc. (China)
📧 info@cancerfax.com
📞 +86 182 1759 2149

🌐 About the Therapy

GC101 offers a potentially curative one-time gene therapy for SMA Type 2 patients. It replaces the missing SMN1 gene to restore motor function and independence in affected children. This trial explores safety and efficacy across low, medium, and high-dose cohorts.

🟢 Join the Trial / Learn More

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