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

X-linked Retinoschisis Clinical Trial

— 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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05878860
• Other study ID #: ATSN-201-1
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: August 22, 2023
• Est. completion date: October 2029

Study information

• Verified date: March 2024
• Source: Atsena Therapeutics Inc.
• Contact: Atsena Therapeutics Clinical Trials
• Phone: 984-261-2001
• Email: clinicaltrials[@]atsenatx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 18
• Est. completion date: October 2029
• Est. primary completion date: October 2025
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 6 Years and older

Eligibility

Inclusion Criteria:

1. Age = 18 for Cohorts 1 through 3, and age = 6 years and < 18 years for Cohort 4.

2. Male patients with clinical diagnosis of XLRS caused by mutations in RS1.

3. 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:

1. Pre-existing eye conditions in the study eye that would contribute significantly to an increased risk of visual loss from a subretinal injection.

2. 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.

3. Treatment in a prior ocular gene or cell therapy study.

Related Conditions & MeSH terms

• Retinoschisis
• X-linked Retinoschisis

Intervention

Biological:
• ATSN-201
AAV.SPR-hGRK1-hRS1syn

Locations

Country	Name	City	State
United States	Children's Hospital of Los Angeles	Los Angeles	California
United States	Bascom Palmer Eye Institute	Miami	Florida
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Oregon Health Sciences University	Portland	Oregon

Sponsors (1)

Lead Sponsor	Collaborator
Atsena Therapeutics Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Secondary	Visual acuity as assessed by best-corrected visual acuity	Change in best-corrected visual acuity (BCVA).	From baseline to week 52
Secondary	Visual acuity as assessed by low-luminance visual acuity	Change in low-luminance visual acuity (LLVA).	From baseline to week 52
Secondary	Visual function as assessed by contrast sensitivity	Change in contrast sensitivity.	From baseline to week 52
Secondary	Visual function as assessed by full-field electroretinogram parameters	Change in full-field electroretinogram (ffERG) parameters.	From baseline to week 52
Secondary	Visual function as assessed by microperimetry	Change in microperimetry.	From baseline to week 52
Secondary	Visual function as assessed by static perimetry	Change in static perimetry.	From baseline to week 52
Secondary	Macular structure as assessed by spectral domain optical coherence tomography	Change in spectral domain optical coherence tomography (SD-OCT).	From baseline to week 52
Secondary	Macular structure as assessed by fundus autofluorescence	Change in fundus autofluorescence (FAF).	From baseline to week 52
Secondary	Subject-reported visual function as assessed by the NEI VFQ-25 in adult subjects	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.	From baseline to week 52
Secondary	Subject-reported visual function as assessed by the CVAQC in pediatric subjects	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.	From baseline to week 52