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

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ClinicalTrials.gov Identifier: NCT05878860

Recruitment Status : Recruiting

First Posted : May 26, 2023

Last Update Posted : March 6, 2024

See Contacts and Locations

View this study on the modernized ClinicalTrials.gov

Sponsor:

Information provided by (Responsible Party):

Atsena Therapeutics Inc.

Tracking Information

First Submitted Date ^ICMJE

May 12, 2023

First Posted Date ^ICMJE

May 26, 2023

Last Update Posted Date

March 6, 2024

Actual Study Start Date ^ICMJE

August 22, 2023

Estimated Primary Completion Date

October 2025 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

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).

Original Primary Outcome Measures ^ICMJE

Same as current

Change History

Current Secondary Outcome Measures ^ICMJE

Visual acuity as assessed by best-corrected visual acuity [ Time Frame: From baseline to week 52 ]
Change in best-corrected visual acuity (BCVA).
Visual acuity as assessed by low-luminance visual acuity [ Time Frame: From baseline to week 52 ]
Change in low-luminance visual acuity (LLVA).
Visual function as assessed by contrast sensitivity [ Time Frame: From baseline to week 52 ]
Change in contrast sensitivity.
Visual function as assessed by full-field electroretinogram parameters [ Time Frame: From baseline to week 52 ]
Change in full-field electroretinogram (ffERG) parameters.
Visual function as assessed by microperimetry [ Time Frame: From baseline to week 52 ]
Change in microperimetry.
Visual function as assessed by static perimetry [ Time Frame: From baseline to week 52 ]
Change in static perimetry.
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).
Macular structure as assessed by fundus autofluorescence [ Time Frame: From baseline to week 52 ]
Change in fundus autofluorescence (FAF).
Subject-reported visual function as assessed by the NEI VFQ-25 in adult subjects [ 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 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 [ Time Frame: 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.

Original Secondary Outcome Measures ^ICMJE

Visual acuity as assessed by best-corrected visual acuity [ Time Frame: From baseline to week 52 ]
Change in best-corrected visual acuity (BCVA).
Visual acuity as assessed by low-luminance visual acuity [ Time Frame: From baseline to week 52 ]
Change in low-luminance visual acuity (LLVA).
Visual function as assessed by contrast sensitivity [ Time Frame: From baseline to week 52 ]
Change in contrast sensitivity.
Visual function as assessed by full-field electroretinogram parameters [ Time Frame: From baseline to week 52 ]
Change in full-field electroretinogram (ffERG) parameters.
Visual function as assessed by microperimetry [ Time Frame: From baseline to week 52 ]
Change in microperimetry.
Visual function as assessed by static perimetry [ Time Frame: From baseline to week 52 ]
Change in static perimetry.
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).
Macular structure as assessed by fundus autofluorescence [ Time Frame: From baseline to week 52 ]
Change in fundus autofluorescence (FAF).
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.

Current Other Pre-specified Outcome Measures

Not Provided

Original Other Pre-specified Outcome Measures

Not Provided

Descriptive Information

Brief Title ^ICMJE

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

Official Title ^ICMJE

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

Brief Summary

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).

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 Type ^ICMJE

Interventional

Study Phase ^ICMJE

Phase 1
Phase 2

Study Design ^ICMJE

Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Masking Description:

Cohort 3 will be partially masked.

Primary Purpose: Treatment

Condition ^ICMJE

X-linked Retinoschisis

Intervention ^ICMJE

Biological: ATSN-201

AAV.SPR-hGRK1-hRS1syn

Study Arms ^ICMJE

Experimental: Cohort 1
ATSN-201 at Low Dose

Intervention: Biological: ATSN-201
Experimental: Cohort 2
ATSN-201 at High Dose

Intervention: Biological: ATSN-201
Experimental: Cohort 3, High Dose
ATSN-201 at High Volume

Intervention: Biological: ATSN-201
Experimental: Cohort 3, Low Dose
ATSN-201 at Low Volume

Intervention: Biological: ATSN-201
No Intervention: Cohort 3, Control
Experimental: Cohort 4, Pediatric
ATSN-201 at High Dose

Intervention: Biological: ATSN-201

Publications *

Not Provided

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Recruiting

Estimated Enrollment ^ICMJE

Original Estimated Enrollment ^ICMJE

Same as current

Estimated Study Completion Date ^ICMJE

October 2029

Estimated Primary Completion Date

October 2025 (Final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

Inclusion Criteria:

Age ≥ 18 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 study eye 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.

Sex/Gender ^ICMJE

Sexes Eligible for Study:

Male

Ages ^ICMJE

6 Years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers ^ICMJE

Contacts ^ICMJE

Contact: Atsena Therapeutics Clinical Trials

984-261-2001

clinicaltrials@atsenatx.com

Listed Location Countries ^ICMJE

United States

Removed Location Countries

Administrative Information

NCT Number ^ICMJE

NCT05878860

Other Study ID Numbers ^ICMJE

ATSN-201-1

Has Data Monitoring Committee

Yes

U.S. FDA-regulated Product

Studies a U.S. FDA-regulated Drug Product:	Yes
Studies a U.S. FDA-regulated Device Product:	No

IPD Sharing Statement ^ICMJE

Plan to Share IPD:

Current Responsible Party

Atsena Therapeutics Inc.

Original Responsible Party

Same as current

Current Study Sponsor ^ICMJE

Atsena Therapeutics Inc.

Original Study Sponsor ^ICMJE

Same as current

Collaborators ^ICMJE

Not Provided

Investigators ^ICMJE

Not Provided

PRS Account

Atsena Therapeutics Inc.

Verification Date

March 2024

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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