ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
Part of paid clinical trials in Phoenix, Arizona.
- Sponsor
- Atsena Therapeutics Inc.
- Study ID
- NCT05878860
- Phase
- PHASE3
- Status
- Recruiting
Conditions
- X-linked Retinoschisis
Eligibility Criteria
- Sex
- ALL
- Age
- 6 Years - N/A
- Healthy Volunteers
- Not accepted
Interventions
- ATSN-201 — BIOLOGICALAAV.SPR-hGRK1-hRS1syn
Study Details
This study will evaluate the safety and efficacy of ATSN-201 in subjects ≥ 6 years of age with RS1-associated X-linked retinoschisis (XLRS).
Key Dates
- First listed
- May 26, 2023
- Start date
- Aug 22, 2023
- Status verified
- Aug 2026
- Primary completion
- Apr 30, 2028
- Completion
- Apr 30, 2033
Study Design
- Enrollment
- 97 participants (estimated)
- Allocation
- RANDOMIZED
- Intervention model
- SEQUENTIAL
- Primary purpose
- TREATMENT
Arms
- Experimental: Cohort 1, Low Dose
- Experimental: Cohort 2, High Dose
- Experimental: Cohort 3, Mid Dose
- Experimental: Cohort 4, Low Volume
- Experimental: Cohort 4, High Volume
- No Intervention: Cohort 4, Control
- Experimental: Cohort 5, Pediatric
- Experimental: Cohort 6, Treatment
- No Intervention: Cohort 6, Control
Primary Outcome Measure
Part A (Dose Escalation) and Part B (Dose Expansion): Safety and tolerability as assessed by dose-limiting toxicities and treatment-emergent adverse events [ Time Frame: From baseline to week 52 ]
Central Contacts
- Atsena Therapeutics Clinical Trials984-261-2001
Locations (18)
| Facility | City | State | ZIP | Site coordinators |
|---|---|---|---|---|
| Associated Retina Consultants | Phoenix | Arizona | 85020 | Benjamin Bakall, MD (PRINCIPAL_INVESTIGATOR) |
| Shiley Eye Institute | La Jolla | California | 92093 | Shyamanga Borooah, MD (PRINCIPAL_INVESTIGATOR) |
| Children's Hospital of Los Angeles | Los Angeles | California | 90027 | Aaron Nagiel, MD, PhD (PRINCIPAL_INVESTIGATOR) |
| Stanford University | Palo Alto | California | 94303 | Vinit Mahajan, MD (PRINCIPAL_INVESTIGATOR) |
| UC San Francisco | San Francisco | California | 94158 | Jacque Duncan, MD (PRINCIPAL_INVESTIGATOR) |
| Bascom Palmer Eye Institute | Miami | Florida | 33136 | Byron Lam, MD (PRINCIPAL_INVESTIGATOR) |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | Anne Fulton, MD (PRINCIPAL_INVESTIGATOR) |
| Massachusetts Eye and Ear | Boston | Massachusetts | 02114 | Rachel Huckfeldt, MD (PRINCIPAL_INVESTIGATOR) |
| University of Michigan | Ann Arbor | Michigan | 48105 | Thomas Wubben, MD (PRINCIPAL_INVESTIGATOR) |
| Mayo Clinic | Rochester | Minnesota | 55905 | Raymond Iezzi, MD (PRINCIPAL_INVESTIGATOR) |
| Columbia University | New York | New York | 100032 | Aliaa Abdelhakim, MD (PRINCIPAL_INVESTIGATOR) |
| Flaum Eye Institute, University of Rochester | Rochester | New York | 14642 | Alex Levin, MD (PRINCIPAL_INVESTIGATOR) |
| Duke University | Durham | North Carolina | 27710 | Lejla Vajzovic, MD (PRINCIPAL_INVESTIGATOR) |
| Oregon Health Sciences University | Portland | Oregon | 97239 | Lesley Everett, MD, PhD, MPhil (PRINCIPAL_INVESTIGATOR) |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | Tomas Aleman, MD (PRINCIPAL_INVESTIGATOR) |
| Retina Foundation of the Southwest | Dallas | Texas | 75231 | Mark Pennesi, MD (PRINCIPAL_INVESTIGATOR) |
| Moran Eye Center | Salt Lake City | Utah | 84132 | Eileen Hwang, MD (PRINCIPAL_INVESTIGATOR) |
| University of Wisconsin | Madison | Wisconsin | 53705 | Melanie Schmitt, MD (PRINCIPAL_INVESTIGATOR) |
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