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
Status: Recruiting
Location: See all (4) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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
Participation Requirements
Sex: All
Maximum Age: 6 months
Healthy Volunteers: f
View:
• Six months of age and younger on day of vector infusion with Type 1 SAM as defined by the following features:
‣ Diagnosis of SMA based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and 2 copies of SMN2;
⁃ Onset of disease before 6 months of age
• The patient's legal guardian(s) must be able to understand the purpose and risks of the study and voluntarily provide signed and dated informed consent prior to any study-related procedures being performed.
Locations
Other Locations
China
Bayi Children's Hospital, Seventh Medical Center, PLA general hospital
RECRUITING
Beijing
Peking University， First Hospital, Department of Pediatrics
RECRUITING
Beijing
Shengjing Hospital of China Medical University
RECRUITING
Shenyang
Children's Hospital of Soochow University
RECRUITING
Suzhou
Contact Information
Primary
GeneCradle, Inc. China
ind@bj-genecradle.com
86-13501380583
Time Frame
Start Date: 2023-02-25
Estimated Completion Date: 2026-12
Participants
Target number of participants: 18
Treatments
Experimental: Low dosage group
2.4x10\^14 vg/person of GC101 delivered one-time intrathecally (n=3)
Experimental: High dosage group
4.8x10\^14 vg/person of GC101 delivered one-time intrathecally (n=3)
Related Therapeutic Areas
Sponsors
Leads: GeneCradle Inc