RecruitingPhase 1Phase 2NCT05824169

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients

A Multi-center, Open Label, Single-arm, Dose Ascending Clinical Trial for Evaluation of Safety and Efficacy of Gene Therapy Drug GC101 in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients

Sponsor

GeneCradle Inc

Enrollment

18 participants

Start Date

Feb 25, 2023

Study Type

INTERVENTIONAL

Conditions

Spinal Muscular Atrophy

Summary

The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 1 (SMA 1) patients.

Eligibility

Min Age: 0 MonthsMax Age: 6 Months

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying GC101 for people with spinal muscular atrophy. The study is currently recruiting participants at 4 locations. People eligible for this study include aged 0 Months to 6 Months.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

GENETICGC101

Self-complementary AAV9 carrying a codon-optimized SMN coding sequence(coSMN1) driven by CMV enhancer and chicken β-actin promoter

Locations(4)

Peking University， First Hospital, Department of Pediatrics

Beijing, China

Bayi Children's Hospital, Seventh Medical Center, PLA general hospital

Beijing, China

Shengjing Hospital of China Medical University

Shenyang, China

Children's Hospital of Soochow University

Suzhou, China

View Full Details on ClinicalTrials.gov

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NCT05824169

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