History of Changes for Study: NCT05878860

ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis (LIGHTHOUSE)

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Study Record Versions

Version	Submitted Date	Changes
1	May 24, 2023	None (earliest Version on record)
2	May 26, 2023	Outcome Measures and Study Status
3	July 7, 2023	Recruitment Status, Study Status and Contacts/Locations
4	August 1, 2023	Study Status and Contacts/Locations
5	August 28, 2023	Study Status
6	November 7, 2023	Study Status
7	November 14, 2023	Contacts/Locations and Study Status
8	February 16, 2024	Eligibility, Study Status and Study Description
9	March 5, 2024	Study Status and Contacts/Locations

Comparison Format:

Merged
Side-by-Side

Study Identification


Unique Protocol ID:	ATSN-201-1
Brief Title:	ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis (LIGHTHOUSE)
Official Title:	A Phase 1/2, Open-Label, Dose Escalation and Dose Expansion Study to Evaluate the Safety and Tolerability of ATSN-201 Gene Therapy in Male Subjects With RS1-Associated X-linked Retinoschisis
Secondary IDs:

Study Status


Record Verification:	May 2023
Overall Status:	Not yet recruiting
Study Start:	July 2023
Primary Completion:	October 2025 [Anticipated]
Study Completion:	October 2029 [Anticipated]

First Submitted:	May 12, 2023
First Submitted that Met QC Criteria:	May 24, 2023
First Posted:	May 26, 2023 [Actual]

Last Update Submitted that Met QC Criteria:	May 24, 2023
Last Update Posted:	May 26, 2023 [Actual]

Sponsor/Collaborators


Sponsor:	Atsena Therapeutics Inc.
Responsible Party:	Sponsor
Collaborators:

Oversight


U.S. FDA-regulated Drug:	Yes
U.S. FDA-regulated Device:	No
Data Monitoring:	Yes

Study Description


Brief Summary:	This study will evaluate the safety and tolerability of ATSN-201 in male subjects 6 to 64 years old with RS1-associated X-linked retinoschisis (XLRS).
Detailed Description:	Eligible patients who enroll in this study will receive a one-time subretinal injection of ATSN-201 in one eye. Safety and tolerability will be evaluated for 5 years.

Conditions


Conditions:	X-linked Retinoschisis
Keywords:	XLRS, RS1

Study Design


Study Type:	Interventional
Primary Purpose:	Treatment
Study Phase:	Phase 1/Phase 2
Interventional Study Model:	Sequential Assignment
Number of Arms:	6
Masking:	None (Open Label)
Allocation:	Randomized
Enrollment:	18 [Anticipated]

Arms and Interventions

Arms	Assigned Interventions
Experimental: Cohort 1 ATSN-201 at Low Dose	Biological: ATSN-201 AAV.SPR-hGRK1-hRS1syn
Experimental: Cohort 2 ATSN-201 at High Dose	Biological: ATSN-201 AAV.SPR-hGRK1-hRS1syn
Experimental: Cohort 3, High Dose ATSN-201 at High Volume	Biological: ATSN-201 AAV.SPR-hGRK1-hRS1syn
Experimental: Cohort 3, Low Dose ATSN-201 at Low Volume	Biological: ATSN-201 AAV.SPR-hGRK1-hRS1syn
No Intervention: Cohort 3, Control
Experimental: Cohort 4, Pediatric ATSN-201 at High Dose	Biological: ATSN-201 AAV.SPR-hGRK1-hRS1syn

Outcome Measures


Primary Outcome Measures:
1.	Safety and tolerability as assessed by dose-limiting toxicities and treatment-emergent adverse events [ Time Frame: From baseline to week 52 ] Incidence of dose-limiting toxicities (DLTs) and treatment-emergent adverse events (TEAEs).
Secondary Outcome Measures:
1.	Visual acuity as assessed by best-corrected visual acuity [ Time Frame: From baseline to week 52 ] Change in best-corrected visual acuity (BCVA).
2.	Visual acuity as assessed by low-luminance visual acuity [ Time Frame: From baseline to week 52 ] Change in low-luminance visual acuity (LLVA).
3.	Visual function as assessed by contrast sensitivity [ Time Frame: From baseline to week 52 ] Change in contrast sensitivity.
4.	Visual function as assessed by full-field electroretinogram parameters [ Time Frame: From baseline to week 52 ] Change in full-field electroretinogram (ffERG) parameters.
5.	Visual function as assessed by microperimetry [ Time Frame: From baseline to week 52 ] Change in microperimetry.
6.	Visual function as assessed by static perimetry [ Time Frame: From baseline to week 52 ] Change in static perimetry.
7.	Macular structure as assessed by spectral domain optical coherence tomography [ Time Frame: From baseline to week 52 ] Change in spectral domain optical coherence tomography (SD-OCT).
8.	Macular structure as assessed by fundus autofluorescence [ Time Frame: From baseline to week 52 ] Change in fundus autofluorescence (FAF).
9.	Subject-reported visual function as assessed by the NEI VFQ-25 or the CVAQC [ Time Frame: From baseline to week 52 ] Change in the National Eye Institute's Visual Function Questionnaire 25 (NEI VFQ-25) score for adult subjects or the Cardiff Visual Ability Questionnaire for Children (CVAQC) score for pediatric subjects.

Eligibility


Minimum Age:	6 Years
Maximum Age:	64 Years
Sex:	Male
Gender Based:
Accepts Healthy Volunteers:	No
Criteria:	Inclusion Criteria: Age ≥ 18 and < 65 years for Cohorts 1 through 3, and age ≥ 6 years and < 18 years for Cohort 4. Male patients with clinical diagnosis of XLRS caused by mutations in RS1. Best corrected visual acuity (BCVA) in both eyes of 34 to 73 Early Treatment Diabetic Retinopathy Study (ETDRS) letters (corresponding to a Snellen acuity of 20/200 to 20/40). Exclusion Criteria: Pre-existing eye conditions in the study eye that would contribute significantly to an increased risk of visual loss from a subretinal injection. Any intraocular surgery (including laser treatment) in the study eye within 6 months prior or any intraocular surgery anticipated in the study eye during the first 12 months of the study. Treatment in a prior ocular gene or cell therapy study.

Contacts/Locations


Central Contact Person:	Atsena Therapeutics Clinical Trials Telephone: 984-261-2001 Email: clinicaltrials@atsenatx.com
Locations:

IPDSharing


Plan to Share IPD:	No

References


Citations:
Links:
Available IPD/Information: