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New treatment option for X-linked Retinoschisis
Official title ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
ClinicalTrials.gov ID: NCT05878860
What this study is testing
What is ATSN-201?
ATSN-201 is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for x-linked retinoschisis.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study will evaluate the safety and efficacy of ATSN-201 in subjects ≥ 6 years of age with RS1-associated X-linked retinoschisis (XLRS).
- Phase 3: a large, late-stage study
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 6 and older
You may be able to join if
- Part A and B:
- Age ≥ 18 for Cohorts 1 through 4, and age ≥ 6 years and \< 18 years for Cohort 5.
- 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...
- Presence of foveal schisis and /or parafoveal/perifoveal schisis in the study eye on SD-OCT per the Principal Investigator.
You likely can't join if
- Pre-existing eye conditions in the study eye that would contribute significantly to an increased risk of visual loss from a subretinal injection (eg...
- Any intraocular surgery (including laser treatment) in the study eye within 6 months prior to Screening or any intraocular surgery anticipated in the...
- Treatment in a prior ocular gene or cell therapy study. Part C: Inclusion Criteria: Note: For patients ineligible for bilateral dosing based on the...
- Age ≥ 6 years.
- Genetically male patients with clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1 OR Genetically female patients...
- BCVA of 34 to 73 ETDRS letters (corresponding to a Snellen acuity of 20/200 to 20/40).
See the full eligibility criteria
- Part A and B:
- Age ≥ 18 for Cohorts 1 through 4, and age ≥ 6 years and \< 18 years for Cohort 5.
- 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).
- Presence of foveal schisis and /or parafoveal/perifoveal schisis in the study eye on SD-OCT per the Principal Investigator.
- Pre-existing eye conditions in the study eye that would contribute significantly to an increased risk of visual loss from a subretinal injection (eg, advanced glaucoma, optic neuropathy, uveitis, corneal transplants).
- Any intraocular surgery (including laser treatment) in the study eye within 6 months prior to Screening 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. Part C: Inclusion Criteria: Note: For patients ineligible for bilateral dosing based on the ocular exclusion criteria, the same eye must meet all ocular inclusion...
- Age ≥ 6 years.
- Genetically male patients with clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1 OR Genetically female patients with clinical diagnosis of XLRS caused by biallelic pathogenic or...
- BCVA of 34 to 73 ETDRS letters (corresponding to a Snellen acuity of 20/200 to 20/40).
- Presence of foveal schisis on SD-OCT. Exclusion Criteria: General None of the following criteria can be met for unilateral or bilateral dosing.
- Treatment with any carbonic anhydrase inhibitor (oral or topical) within 1 month prior to Screening.
- Treatment in a prior ocular gene or cell therapy study.
- Absence of macular schisis.
- BCVA better than 75 ETDRS letters (corresponding to a Snellen acuity of 20/32).
- Pre-existing eye conditions that would contribute significantly to an increased risk of visual loss from a subretinal injection (eg, advanced glaucoma, optic neuropathy, uveitis, corneal transplants).
- Any intraocular surgery (including laser treatment) within 6 months prior to Screening or any intraocular surgery anticipated during the first 12 months of the study.
The study team makes the final eligibility decision.
Where it's taking place
- Phoenix, Arizona, United States
- La Jolla, California, United States
- Los Angeles, California, United States
- Palo Alto, California, United States
- San Francisco, California, United States
- Miami, Florida, United States
- Boston, Massachusetts, United States
- Ann Arbor, Michigan, United States
- Rochester, Minnesota, United States
- New York, New York, United States
- Rochester, New York, United States
- Durham, North Carolina, United States
- Portland, Oregon, United States
- Philadelphia, Pennsylvania, United States
- Dallas, Texas, United States
- Salt Lake City, Utah, United States
- Madison, Wisconsin, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 6 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Phoenix, Arizona, United States; La Jolla, California, United States; Los Angeles, California, United States; Palo Alto, California, United States; San Francisco, California, United States; Miami, Florida, United States and 11 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.