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

Sponsor

Atsena Therapeutics Inc. (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05878860

Collaborator

(none)

Enrollment

Arms

Anticipated Duration (Months)

Study Details

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).

Condition or Disease	Intervention/Treatment	Phase
X-linked Retinoschisis	Biological: ATSN-201	Phase 1/Phase 2

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.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

18 participants

Allocation:

Randomized

Intervention Model:

Sequential Assignment

Masking:

None (Open Label)

Masking Description:

Cohort 3 will be partially masked.

Primary Purpose:

Treatment

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

Anticipated Study Start Date :

Jul 1, 2023

Anticipated Primary Completion Date :

Oct 1, 2025

Anticipated Study Completion Date :

Oct 1, 2029

Arms and Interventions

Arm	Intervention/Treatment
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

Safety and tolerability as assessed by dose-limiting toxicities and treatment-emergent adverse events [From baseline to week 52]
Incidence of dose-limiting toxicities (DLTs) and treatment-emergent adverse events (TEAEs).

Secondary Outcome Measures

Visual acuity as assessed by best-corrected visual acuity [From baseline to week 52]
Change in best-corrected visual acuity (BCVA).
Visual acuity as assessed by low-luminance visual acuity [From baseline to week 52]
Change in low-luminance visual acuity (LLVA).
Visual function as assessed by contrast sensitivity [From baseline to week 52]
Change in contrast sensitivity.
Visual function as assessed by full-field electroretinogram parameters [From baseline to week 52]
Change in full-field electroretinogram (ffERG) parameters.
Visual function as assessed by microperimetry [From baseline to week 52]
Change in microperimetry.
Visual function as assessed by static perimetry [From baseline to week 52]
Change in static perimetry.
Macular structure as assessed by spectral domain optical coherence tomography [From baseline to week 52]
Change in spectral domain optical coherence tomography (SD-OCT).
Macular structure as assessed by fundus autofluorescence [From baseline to week 52]
Change in fundus autofluorescence (FAF).
Subject-reported visual function as assessed by the NEI VFQ-25 in adult subjects [From baseline to week 52]
Change in the National Eye Institute's Visual Function Questionnaire 25 (NEI VFQ-25) score for adult subjects with scores from 0 to 100 where a higher score indicates a better outcome.
Subject-reported visual function as assessed by the CVAQC in pediatric subjects [From baseline to week 52]
Change in the Cardiff Visual Ability Questionnaire for Children (CVAQC) score for pediatric subjects with scores from -3.00 to +2.80 where a higher score indicates a worse outcome.

Eligibility Criteria

Criteria

Ages Eligible for Study:

6 Years to 64 Years

Sexes Eligible for Study:

Male

Accepts Healthy Volunteers:

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 and Locations

Locations

No locations specified.

Sponsors and Collaborators

Atsena Therapeutics Inc.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Atsena Therapeutics Inc.

ClinicalTrials.gov Identifier:

NCT05878860

Other Study ID Numbers:

ATSN-201-1

First Posted:

May 26, 2023

Last Update Posted:

May 31, 2023

Last Verified:

May 1, 2023

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Keywords provided by Atsena Therapeutics Inc.

XLRS, RS1

Additional relevant MeSH terms:

Retinoschisis

Study Results

No Results Posted as of May 31, 2023