Back to resultsATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis (LIGHTHOUSE)
Primary Purpose
X-linked Retinoschisis
Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
ATSN-201
Sponsored by
About this trial
This is an interventional treatment trial for X-linked Retinoschisis focused on measuring XLRS, RS1
Eligibility 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.
Sites / Locations
- Children's Hospital of Los AngelesRecruiting
- Oregon Health Sciences UniversityRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm 5
Arm 6
Arm Type
Experimental
Experimental
Experimental
Experimental
No Intervention
Experimental
Arm Label
Cohort 1
Cohort 2
Cohort 3, High Dose
Cohort 3, Low Dose
Cohort 3, Control
Cohort 4, Pediatric
Arm Description
ATSN-201 at Low Dose
ATSN-201 at High Dose
ATSN-201 at High Volume
ATSN-201 at Low Volume
ATSN-201 at High Dose
Outcomes
Primary Outcome Measures
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).
Secondary Outcome Measures
Visual acuity as assessed by best-corrected visual acuity
Change in best-corrected visual acuity (BCVA).
Visual acuity as assessed by low-luminance visual acuity
Change in low-luminance visual acuity (LLVA).
Visual function as assessed by contrast sensitivity
Change in contrast sensitivity.
Visual function as assessed by full-field electroretinogram parameters
Change in full-field electroretinogram (ffERG) parameters.
Visual function as assessed by microperimetry
Change in microperimetry.
Visual function as assessed by static perimetry
Change in static perimetry.
Macular structure as assessed by spectral domain optical coherence tomography
Change in spectral domain optical coherence tomography (SD-OCT).
Macular structure as assessed by fundus autofluorescence
Change in fundus autofluorescence (FAF).
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.
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05878860
Brief Title
ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
Acronym
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
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Recruiting
Study Start Date
August 22, 2023 (Actual)
Primary Completion Date
October 2025 (Anticipated)
Study Completion Date
October 2029 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Atsena Therapeutics Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
X-linked Retinoschisis
Keywords
XLRS, RS1
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Masking Description
Cohort 3 will be partially masked.
Allocation
Randomized
Enrollment
18 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Cohort 1
Arm Type
Experimental
Arm Description
ATSN-201 at Low Dose
Arm Title
Cohort 2
Arm Type
Experimental
Arm Description
ATSN-201 at High Dose
Arm Title
Cohort 3, High Dose
Arm Type
Experimental
Arm Description
ATSN-201 at High Volume
Arm Title
Cohort 3, Low Dose
Arm Type
Experimental
Arm Description
ATSN-201 at Low Volume
Arm Title
Cohort 3, Control
Arm Type
No Intervention
Arm Title
Cohort 4, Pediatric
Arm Type
Experimental
Arm Description
ATSN-201 at High Dose
Intervention Type
Biological
Intervention Name(s)
ATSN-201
Intervention Description
AAV.SPR-hGRK1-hRS1syn
Primary Outcome Measure Information:
Title
Safety and tolerability as assessed by dose-limiting toxicities and treatment-emergent adverse events
Description
Incidence of dose-limiting toxicities (DLTs) and treatment-emergent adverse events (TEAEs).
Time Frame
From baseline to week 52
Secondary Outcome Measure Information:
Title
Visual acuity as assessed by best-corrected visual acuity
Description
Change in best-corrected visual acuity (BCVA).
Time Frame
From baseline to week 52
Title
Visual acuity as assessed by low-luminance visual acuity
Description
Change in low-luminance visual acuity (LLVA).
Time Frame
From baseline to week 52
Title
Visual function as assessed by contrast sensitivity
Description
Change in contrast sensitivity.
Time Frame
From baseline to week 52
Title
Visual function as assessed by full-field electroretinogram parameters
Description
Change in full-field electroretinogram (ffERG) parameters.
Time Frame
From baseline to week 52
Title
Visual function as assessed by microperimetry
Description
Change in microperimetry.
Time Frame
From baseline to week 52
Title
Visual function as assessed by static perimetry
Description
Change in static perimetry.
Time Frame
From baseline to week 52
Title
Macular structure as assessed by spectral domain optical coherence tomography
Description
Change in spectral domain optical coherence tomography (SD-OCT).
Time Frame
From baseline to week 52
Title
Macular structure as assessed by fundus autofluorescence
Description
Change in fundus autofluorescence (FAF).
Time Frame
From baseline to week 52
Title
Subject-reported visual function as assessed by the NEI VFQ-25 in adult subjects
Description
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.
Time Frame
From baseline to week 52
Title
Subject-reported visual function as assessed by the CVAQC in pediatric subjects
Description
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.
Time Frame
From baseline to week 52
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
64 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Atsena Therapeutics Clinical Trials
Phone
984-261-2001
Email
clinicaltrials@atsenatx.com
Facility Information:
Facility Name
Children's Hospital of Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hillary Schwartz, MS
Phone
323-361-1004
Email
hschwartz@chla.usc.edu
First Name & Middle Initial & Last Name & Degree
Dilshad Contractor
Phone
323-361-7194
Email
dcontractor@chla.usc.edu
First Name & Middle Initial & Last Name & Degree
Aaron Nagiel, MD, PhD
Facility Name
Oregon Health Sciences University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Stephanie Timko
Phone
503-494-9743
Email
spences@ohsu.edu
First Name & Middle Initial & Last Name & Degree
Lesley Everett, MD, PhD, MPhil
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
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