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
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
X-linked Retinoschisis | Biological: ATSN-201 | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated 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 |
Actual Study Start Date : | August 22, 2023 |
Estimated Primary Completion Date : | October 2025 |
Estimated Study Completion Date : | October 2029 |
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 |
- 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).
- 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.
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Ages Eligible for Study: | 6 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05878860
Contact: Atsena Therapeutics Clinical Trials | 984-261-2001 | clinicaltrials@atsenatx.com |
United States, California | |
Children's Hospital of Los Angeles | Recruiting |
Los Angeles, California, United States, 90027 | |
Contact: Hillary Schwartz, MS 323-361-1004 hschwartz@chla.usc.edu | |
Contact: Dilshad Contractor 323-361-7194 dcontractor@chla.usc.edu | |
Principal Investigator: Aaron Nagiel, MD, PhD | |
United States, Florida | |
Bascom Palmer Eye Institute | Recruiting |
Miami, Florida, United States, 33136 | |
Contact: Tamara Riesgo 305-482-4584 txj362@miami.edu | |
Principal Investigator: Byron Lam, MD | |
United States, Oregon | |
Oregon Health Sciences University | Recruiting |
Portland, Oregon, United States, 97239 | |
Contact: Stephanie Timko 503-494-9743 spences@ohsu.edu | |
Principal Investigator: Lesley Everett, MD, PhD, MPhil | |
United States, Pennsylvania | |
Children's Hospital of Philadelphia | Recruiting |
Philadelphia, Pennsylvania, United States, 19104 | |
Contact: Sarah Galal 267-991-3912 galals@chop.edu | |
Principal Investigator: Tomas Aleman, MD |
Responsible Party: | Atsena Therapeutics Inc. |
ClinicalTrials.gov Identifier: | NCT05878860 |
Other Study ID Numbers: |
ATSN-201-1 |
First Posted: | May 26, 2023 Key Record Dates |
Last Update Posted: | March 6, 2024 |
Last Verified: | March 2024 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
XLRS, RS1 |
Retinoschisis Retinal Degeneration Retinal Diseases Eye Diseases |