Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injection in the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) – Phase III ​

Phase III Trial: GC101 Gene Therapy for Type 2 Spinal Muscular Atrophy (SMA)

ClinicalTrials.gov Identifier: NCT06971094
Sponsor: GeneCradle Inc.
Study Title: Safety and Efficacy Evaluation of GC101 Gene Therapy via Intrathecal Injection in Type 2 SMA
Study Status: Recruiting
Study Start Date: May 12, 2025 (Estimated)
Primary Completion Date: December 31, 2026 (Estimated)
Study Completion Date: December 31, 2026 (Estimated)

🔬 Objective

To evaluate the safety, efficacy, and pharmacokinetics of GC101, a gene therapy using self-complementary adeno-associated virus (scAAV) carrying a codon-optimized SMN1 gene, delivered via intrathecal injection in children with Type 2 SMA.

🧪 Study Design

• Type: Interventional (Phase III)

• Model: Randomized, parallel-assignment

• Masking: None (Open-label)

• Control: Standard-of-care with nusinersen

• Enrollment: 50 participants (estimated)

💉 Intervention Arms

1. GC101 Arm (Experimental):

   • Single intrathecal injection of GC101 (1.2×10¹⁴ vg per patient)

   • Discontinue prior nusinersen treatment

2. Control Arm:

   • Continue standard-of-care nusinersen treatment

📈 Outcome Measures

Primary Endpoint:

• Change in HFMSE (Hammersmith Functional Motor Scale Expanded) score at 52 weeks

Secondary Endpoints:

• HFMSE score change at 26 weeks

• % of participants with ≥3-point increase in HFMSE

• WHO-MGRS milestone achievements (sitting, walking, crawling)

• RULM (upper limb motor) and SMAIS (independence) scores at 26 & 52 weeks

• Incidence of adverse events (AEs, SAEs, AESIs)

✅ Inclusion Criteria

• Age 2–12 years, with genetically confirmed Type 2 5q-SMA

• Received nusinersen for >1 year

• No risdiplam within 2 months before screening

• Can sit independently but not walk

• HFMSE score ≥10

• Informed consent from patient/legal guardian

❌ Exclusion Criteria

• Anti-AAV9 antibody titer >1:50

• Recent nusinersen (<2 months)

• Comorbidities like epilepsy, cerebrospinal fluid disorders, severe scoliosis

• Positive for HIV, HCV, HBV, or syphilis

• Severe hepatic impairment (ALT/AST ≥3x ULN)

• Contraindications to lumbar puncture or glucocorticoids

• Gene therapy or other trial participation within 3 months

🏥 Participating Locations in China

• The Seventh Medical Center of PLA General Hospital, Beijing

• Peking University First Hospital, Beijing

• Beijing Children’s Hospital

• National Children’s Medical Center, Shanghai

• Shenzhen Children’s Hospital

• Children’s Hospital of Soochow University, Suzhou

• Wuhan Children’s Hospital, Wuhan

📞 Contact for Participation

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

🌐 Why This Matters

GC101 aims to replace the missing SMN1 gene in Type 2 SMA patients with a single-dose intrathecal injection. This study could redefine treatment by offering a long-lasting, potentially one-time therapy alternative to ongoing maintenance drugs like nusinersen.

🟢 Join the Trial / Learn More

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