ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
LIGHTHOUSE
A Phase 1/2/3, Open-Label, Dose Escalation, Dose Expansion and Randomized, Controlled Study to Evaluate the Safety and Efficacy of ATSN-201 Gene Therapy in Subjects With RS1-Associated X-linked Retinoschisis (LIGHTHOUSE)
1 other identifier
interventional
97
2 countries
19
Brief Summary
This study will evaluate the safety and efficacy of ATSN-201 in subjects ≥ 6 years of age with RS1-associated X-linked retinoschisis (XLRS).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Aug 2023
Longer than P75 for phase_3
19 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 12, 2023
CompletedFirst Posted
Study publicly available on registry
May 26, 2023
CompletedStudy Start
First participant enrolled
August 22, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 1, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
April 1, 2033
August 10, 2026
August 1, 2026
4.6 years
May 12, 2023
August 7, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Part A (Dose Escalation) and Part B (Dose Expansion): Safety and tolerability as assessed by dose-limiting toxicities and treatment-emergent adverse events
Incidence of dose-limiting toxicities (DLTs) and treatment-emergent adverse events (TEAEs).
From baseline to week 52
Part C (Phase 3, Randomized, Controlled) Effect of ATSN-201 on visual function.
Proportion of subjects ≥12 years of age with improvement ≥ 7dB from baseline in microperimetry across prespecified loci in the study eye.
From baseline to week 52
Secondary Outcomes (19)
Part A and Part B: Visual acuity as assessed by best-corrected visual acuity
From baseline to week 52
Part A and Part B: Visual acuity as assessed by low-luminance visual acuity
From baseline to week 52
Part A and Part B: Visual function as assessed by contrast sensitivity
From baseline to week 52
Part A and Part B: Visual function as assessed by microperimetry
From baseline to week 52
Part A and Part B: Macular structure as assessed by spectral domain optical coherence tomography
From baseline to week 52
- +14 more secondary outcomes
Study Arms (9)
Cohort 1, Low Dose
EXPERIMENTALCohort 2, High Dose
EXPERIMENTALCohort 3, Mid Dose
EXPERIMENTALCohort 4, Low Volume
EXPERIMENTALCohort 4, High Volume
EXPERIMENTALCohort 4, Control
NO INTERVENTIONCohort 5, Pediatric
EXPERIMENTALCohort 6, Treatment
EXPERIMENTALCohort 6, Control
NO INTERVENTIONInterventions
AAV.SPR-hGRK1-hRS1syn
Eligibility Criteria
You may qualify if:
- Age ≥ 18 for Cohorts 1 through 4, and age ≥ 6 years and \< 18 years for Cohort 5.
- Male patients with clinical diagnosis of XLRS caused by mutations in RS1.
- Best corrected visual acuity (BCVA) in study eye of 34 to 73 Early Treatment Diabetic Retinopathy Study (ETDRS) letters (corresponding to a Snellen acuity of 20/200 to 20/40).
- Presence of foveal schisis and /or parafoveal/perifoveal schisis in the study eye on SD-OCT per the Principal Investigator.
You may not qualify if:
- Pre-existing eye conditions in the study eye that would contribute significantly to an increased risk of visual loss from a subretinal injection (eg, advanced glaucoma, optic neuropathy, uveitis, corneal transplants).
- Any intraocular surgery (including laser treatment) in the study eye within 6 months prior to Screening 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.
- Part C:
- General All of the following criteria must be met for unilateral or bilateral dosing.
- Age ≥ 6 years.
- Genetically male patients with clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1 OR Genetically female patients with clinical diagnosis of XLRS caused by biallelic pathogenic or likely pathogenic mutations in RS1.
- Ocular At least 1 eye must meet all of the following criteria for both unilateral and bilateral dosing.
- BCVA of 34 to 73 ETDRS letters (corresponding to a Snellen acuity of 20/200 to 20/40).
- Presence of foveal schisis on SD-OCT.
- General None of the following criteria can be met for unilateral or bilateral dosing.
- Treatment with any carbonic anhydrase inhibitor (oral or topical) within 1 month prior to Screening.
- Treatment in a prior ocular gene or cell therapy study.
- Absence of macular schisis.
- BCVA better than 75 ETDRS letters (corresponding to a Snellen acuity of 20/32).
- +2 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (19)
Associated Retina Consultants
Phoenix, Arizona, 85020, United States
Shiley Eye Institute
La Jolla, California, 92093, United States
Children's Hospital of Los Angeles
Los Angeles, California, 90027, United States
Stanford University
Palo Alto, California, 94303, United States
UC San Francisco
San Francisco, California, 94158, United States
Bascom Palmer Eye Institute
Miami, Florida, 33136, United States
Massachusetts Eye and Ear
Boston, Massachusetts, 02114, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
University of Michigan
Ann Arbor, Michigan, 48105, United States
Mayo Clinic
Rochester, Minnesota, 55905, United States
Columbia University
New York, New York, 100032, United States
Flaum Eye Institute, University of Rochester
Rochester, New York, 14642, United States
Duke University
Durham, North Carolina, 27710, United States
Oregon Health Sciences University
Portland, Oregon, 97239, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Retina Foundation of the Southwest
Dallas, Texas, 75231, United States
Moran Eye Center
Salt Lake City, Utah, 84132, United States
University of Wisconsin
Madison, Wisconsin, 53705, United States
Oxford Eye Hospital
Oxford, England, OX3 9DU, United Kingdom
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- NONE
- Masking Details
- Cohort 4 will be partially masked. Cohort 6 will be partially masked.
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 12, 2023
First Posted
May 26, 2023
Study Start
August 22, 2023
Primary Completion (Estimated)
April 1, 2028
Study Completion (Estimated)
April 1, 2033
Last Updated
August 10, 2026
Record last verified: 2026-08
Data Sharing
- IPD Sharing
- Will not share