GC101 Gene Therapy for Spinal Muscular Atrophy Type 1 – Phase I/II Clinical Trial

Gene Therapy for Spinal Muscular Atrophy Type 1 – Phase I/II Clinical Trial

ClinicalTrials.gov Identifier: NCT05824169
Sponsor: GeneCradle Inc.
Study Title: Safety and Efficacy of GC101 Gene Therapy in SMA Type 1
Trial Phase: Phase I/II
Status: Recruiting
Start Date: February 25, 2023 (Actual)
Primary Completion Date: December 2025 (Estimated)
Study Completion Date: December 2025 (Estimated)

🔬 Objective

This trial aims to evaluate the safety and efficacy of GC101, a one-time gene therapy delivered via intrathecal injection, in infants diagnosed with Type 1 Spinal Muscular Atrophy (SMA).

🧪 Study Design

• Type: Interventional

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

• Masking: None

• Enrollment: 18 patients (estimated)

• Dose Groups:

  • Low Dose: 2.4×10¹⁴ vg/person

  • High Dose: 4.8×10¹⁴ vg/person

💉 Intervention

• GC101 Gene Therapy: AAV9-based vector carrying a codon-optimized SMN1 gene, delivered once intrathecally

📈 Outcome Measures

Primary Endpoints:

• Incidence of treatment-related adverse events (AEs/SAEs)

• Proportion of infants who can sit unassisted for ≥10 seconds by 18 months of age

Secondary Endpoints:

• Ability to thrive without mechanical support and maintain weight

• Improvement in CHOP-INTEND scores (0–64)

• Event-free survival at 14 months

Exploratory Outcomes:

• Achievement of WHO motor milestones (e.g., crawling, standing, walking) at 18 months

✅ Inclusion Criteria

• Age ≤6 months at vector infusion

• Diagnosis of Type 1 SMA (biallelic SMN1 mutation + 2 copies of SMN2)

• Onset of symptoms before 6 months

• Legal guardian consent

❌ Exclusion Criteria

• Prior gene therapy or SMA treatment with nusinersen/risdiplam

• AAV9 antibody titer ≥1:200

• Requires ventilatory support ≥16 hrs/day

• Pulse oximetry <95% while awake

• Positive for HIV, HBV, HCV, or syphilis

• Abnormal liver or hematologic parameters

• Glucocorticoid allergy

• Active infections or recent vaccinations (<2 weeks)

• Any condition deemed unsuitable by investigator

🏥 Participating Hospitals in China

• Peking University First Hospital, Beijing

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

• Shengjing Hospital, China Medical University, Shenyang

• Children’s Hospital of Soochow University, Suzhou

📞 Contact for Participation

GeneCradle Inc. (China)
📧 info@cancerfax.com
📞 +86 18217592149

🌐 About the Therapy

GC101 gene therapy offers a potentially life-saving single-dose treatment for infants with SMA Type 1 by introducing the SMN1 gene via a safe AAV9 vector. This trial assesses motor milestones and survival outcomes in the most vulnerable age group.

🟢 Join the Trial / Learn More

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