Ignite Creation Date:
2024-06-16 @ 11:48 AM
Last Modification Date:
2025-12-17 @ 3:26 AM
Study NCT ID:
NCT06421831
Status:
None
Last Update Posted:
2025-07-03 00:00:00
First Post:
2024-05-10 00:00:00
Brief Title:
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients
Sponsor:
GeneCradle Inc
Organization:
GeneCradle Inc
Study Overview
Official Title:
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 3 Patients
Status:
None
Status Verified Date:
2025-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of this trial is to evaluate safety and efficacy of gene therapy drug GC101 in SMA 3 patients. Open-label, dose-escalation clinical trials of GC101 will be conducted in multiple centers in China.
GC101 will be administrated intrathecally. Short-term safety will be evaluated in 52 weeks and enter long-term follow-up study of 5 years at will. Patients will be tested at baseline and followed up at various time points.
The primary analysis for efficacy will be assessed at 12 months after treatment with GC101 on the changes from baseline HFMSE (Hammersmith Functional Motor Scale Expanded) and RULM（Revised Upper Limb Module) scores for patients of age ≥ 6 years old.
GC101 will be administrated intrathecally. Short-term safety will be evaluated in 52 weeks and enter long-term follow-up study of 5 years at will. Patients will be tested at baseline and followed up at various time points.
The primary analysis for efficacy will be assessed at 12 months after treatment with GC101 on the changes from baseline HFMSE (Hammersmith Functional Motor Scale Expanded) and RULM（Revised Upper Limb Module) scores for patients of age ≥ 6 years old.
Detailed Description:
The purpose of this trial is to evaluate safety and efficacy of gene therapy drug GC101 in SMA 3 patients Open-label dose-escalation clinical trials of GC101 will be conducted in multiple centers in China
GC101 will be administrated intrathecally Short-term safety will be evaluated in 52 weeks and enter long-term follow-up study of 5 years at will Patients will be tested at baseline and followed up at various time points
The primary analysis for efficacy will be assessed at 12 months after treatment with GC101 on the changes from baseline HFMSE Hammersmith Functional Motor Scale Expanded and RULMRevised Upper Limb Module scores for patients of age 6 years old
GC101 will be administrated intrathecally Short-term safety will be evaluated in 52 weeks and enter long-term follow-up study of 5 years at will Patients will be tested at baseline and followed up at various time points
The primary analysis for efficacy will be assessed at 12 months after treatment with GC101 on the changes from baseline HFMSE Hammersmith Functional Motor Scale Expanded and RULMRevised Upper Limb Module scores for patients of age 6 years old
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None