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Summary
A Clinical Study Evaluating the Safety and Efficacy of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1
NCTID
NCT06191354
(View at clinicaltrials.gov)
Description
This is a clinical study to evaluate the safety and efficacy of gene therapy drug SKG0201 Injection in patients with spinal muscular atrophy Type 1 (SMA 1).
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Development Status
Active
Indication
Spinal Muscular Atrophy
Disease Ontology Term
DOID:13137
Compound Name
SKG0201
Sponsor
Kun Sun
Funder Type
Other
Recruitment Status
Active not recruiting
Enrollment Count
12 (ESTIMATED)
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
Intravenous
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV
Editor Type
Dose 1
Undisclosed dose escalation
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Na
Submit Date
2023-12-19
Completion Date
2025-12
Last Update
2025-11-21
Participation Criteria
Eligible Age
<=180 Days
Standard Ages
Child
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
3
Locations
China
Regulatory Information
Has US IND
False
FDA Designations
Recent Updates
Resources/Links
News and Press Releases
Skyline Therapeutics Receives China NMPA's Approval of IND for SKG0201, a Novel SMN1 Gene Replacement Therapy Candidate for Spinal Muscular Atrophy (SMA)