ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
Public ClinicalTrials.gov record NCT05878860. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 1/2/3, Open-Label, Dose Escalation, Dose Expansion and Randomized, Controlled Study to Evaluate the Safety and Efficacy of ATSN-201 Gene Therapy in Subjects With RS1-Associated X-linked Retinoschisis (LIGHTHOUSE)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This study will evaluate the safety and efficacy of ATSN-201 in subjects ≥ 6 years of age with RS1-associated X-linked retinoschisis (XLRS).
Study identification
- NCT ID
- NCT05878860
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 97 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 6 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Aug 21, 2023
- Primary completion
- Mar 31, 2028
- Completion
- Mar 31, 2033
- Last update posted
- Aug 20, 2026
2023 – 2033
United States locations
- U.S. sites
- 18
- U.S. states
- 13
- U.S. cities
- 17
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Associated Retina Consultants | Phoenix | Arizona | 85020 | Recruiting |
| Shiley Eye Institute | La Jolla | California | 92093 | Not yet recruiting |
| Children's Hospital of Los Angeles | Los Angeles | California | 90027 | Recruiting |
| Stanford University | Palo Alto | California | 94303 | Not yet recruiting |
| UC San Francisco | San Francisco | California | 94158 | Recruiting |
| Bascom Palmer Eye Institute | Miami | Florida | 33136 | Recruiting |
| Massachusetts Eye and Ear | Boston | Massachusetts | 02114 | Recruiting |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | Not yet recruiting |
| University of Michigan | Ann Arbor | Michigan | 48105 | Not yet recruiting |
| Mayo Clinic | Rochester | Minnesota | 55905 | Recruiting |
| Columbia University | New York | New York | 100032 | Recruiting |
| Flaum Eye Institute, University of Rochester | Rochester | New York | 14642 | Not yet recruiting |
| Duke University | Durham | North Carolina | 27710 | Recruiting |
| Oregon Health Sciences University | Portland | Oregon | 97239 | Recruiting |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | Recruiting |
| Retina Foundation of the Southwest | Dallas | Texas | 75231 | Recruiting |
| Moran Eye Center | Salt Lake City | Utah | 84132 | Recruiting |
| University of Wisconsin | Madison | Wisconsin | 53705 | Not yet recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 1 non-U.S. site.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05878860, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Aug 20, 2026 · Synced Sep 5, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT05878860 live on ClinicalTrials.gov.