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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients
NCTID
NCT05824169
(View at clinicaltrials.gov)
Description
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.
(Show More)
Development Status
Active
Indication
Spinal Muscular Atrophy
Disease Ontology Term
DOID:13137
Compound Name
GC101
Compound Description
scAAV9.CBA.CMV.coSMN1
Sponsor
GeneCradle Inc
Funder Type
Industry
Recruitment Status
Recruiting
Enrollment Count
18
Results Posted
Not Available
Therapy Information
Target Gene/Variant
SMN1
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Intrathecal
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV9
Editor Type
Dose 1
2.4E14 vg
Dose 2
4.8E14 vg
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2023-04-10
Completion Date
2025-12
Last Update
2023-04-25
Participation Criteria
Eligible Age
0 Months - 6 Months
Standard Ages
Child
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
4
Locations
China
Regulatory Information
Has US IND
False
FDA Designations
Recent Updates
Resources/Links
Clinical Publications
Treatment of SMA type 1 infants using a single-dose AAV9-mediated gene therapy via intrathecal injection of GC101: An open-label, single-arm study
News and Press Releases
Genecradle Therapeutics' GC101 Injection Recognized as a Breakthrough Therapy
Related NCTID
Phase 1/2: NCT05901987 (for SMA Type 2)
Phase 1/2: NCT06421831 (for SMA Type 3)