Trials / Recruiting
RecruitingNCT05878860
ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
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
- Status
- Recruiting
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 21 (estimated)
- Sponsor
- Atsena Therapeutics Inc. · Industry
- Sex
- Male
- Age
- 6 Years
- Healthy volunteers
- Not accepted
Summary
This study will evaluate the safety and tolerability of ATSN-201 in male subjects ≥ 6 years of age 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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | ATSN-201 | AAV.SPR-hGRK1-hRS1syn |
Timeline
- Start date
- 2023-08-22
- Primary completion
- 2025-10-01
- Completion
- 2029-10-01
- First posted
- 2023-05-26
- Last updated
- 2025-02-19
Locations
4 sites across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT05878860. Inclusion in this directory is not an endorsement.