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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
NCTID
NCT05878860
(View at clinicaltrials.gov)
Description
This study will evaluate the safety and tolerability of ATSN-201 in male subjects ≥ 6 years of age with RS1-associated X-linked retinoschisis (XLRS).
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Indication
X-linked Retinoschisis
Compound Name
ATSN-201 (AAV.SPR-hGRK1-hRS1syn)
Sponsor
Atsena Therapeutics Inc.
Funder Type
Industry
Status
Recruiting
Enrollment Count
21
Therapy Information
Target Gene/Variant
RS1
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Subretinal
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV.SPR
Editor Type
none
Dose 1
1.5E10 vg/eye
Dose 2
5.0E10 vg/eye
Dose 3
1.7E11 vg/eye
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2023-05-12
Completion Date
2029-10
Last Update
2025-02-19
Participation Criteria
Eligible Age
>=6 Years
Standard Ages
Child, Adult, Older adult
Eligible Sex
MALE
Locations
No.of Trial Sites
4
Locations
United States
Regulatory Information
Has US IND
True
Recent Updates
FDA granted Rare Pediatric Disease Designation
Resources/Links
Clinical Publications
(Presentation) Interim Safety and Efficacy of ATSN-201 Dose Escalation Study in Patients with X-linked Retinoschisis - AAO 2024
News and Press Releases
Atsena Therapeutics Initiates Part B of Phase I/II Clinical Trial Evaluating Gene Therapy ATSN-201 to Treat X-linked Retinoschisis
Preclinical Publications
(Presentation) IND-enabling Studies to Support the Clinical Development of ATSN-201, a Subretinally Delivered, Laterally Spreading Gene Replacement Therapy For X-linked Retinoschisis (XLRS) - ESGCT 2023