Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D
- Conditions
- LCALCA1Leber Congenital Amaurosis
- Interventions
- Drug: Triamcinalone AcetonideDrug: Trimethoprim/polymyxin B
- Registration Number
- NCT03920007
- Lead Sponsor
- Atsena Therapeutics Inc.
- Brief Summary
Primary Objective:
To evaluate the safety and tolerability of ascending doses of ATSN-101 administered as a unilateral subretinal injection in patients with Leber Congenital Amaurosis (LCA) caused by autosomal recessive guanylate cyclase 2D (GUCY2D) mutations (GUCY2D-LCA).
Secondary Objective:
To evaluate the efficacy of ascending doses of ATSN-101 administered as a unilateral subretinal injection in patients with GUCY2D-LCA.
- Detailed Description
Study duration per participant is approximately 112 weeks including: an approximately 56-day screening/baseline period, an approximately 52-week study observation period including 1 treatment day, and an approximately 52-week safety follow-up period. The end of study visit will be approximately 260 weeks after the Investigational Medicinal Product (IMP) administration.
After completion of the main study (ATSN-101-1), participants may have the option to enroll in a separate long-term follow-up study, in which case they would no longer continue in ATSN-101-1 and their end of study visit would be conducted at Week 52.
The study is separated into 2 parts including a dose escalation phase (Part A) and a dose expansion phase (Part B). In Part B participants will be treated at the maximum tolerated dose (MTD) or maximum administered dose (MAD) determined from Part A.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 15
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description ATSN-101 ATSN-101 Diluent Solution ATSN-101 single dose according to an ascending dose design (dose escalation phase) or ATSN-101 single dose (dose expansion phase) ATSN-101 1% Prednisolone ATSN-101 single dose according to an ascending dose design (dose escalation phase) or ATSN-101 single dose (dose expansion phase) ATSN-101 Triamcinalone Acetonide ATSN-101 single dose according to an ascending dose design (dose escalation phase) or ATSN-101 single dose (dose expansion phase) ATSN-101 Trimethoprim/polymyxin B ATSN-101 single dose according to an ascending dose design (dose escalation phase) or ATSN-101 single dose (dose expansion phase) ATSN-101 Prednisone ATSN-101 single dose according to an ascending dose design (dose escalation phase) or ATSN-101 single dose (dose expansion phase) ATSN-101 ATSN-101 ATSN-101 single dose according to an ascending dose design (dose escalation phase) or ATSN-101 single dose (dose expansion phase)
- Primary Outcome Measures
Name Time Method Number of participants with adverse events (AEs) from baseline up to the end of the observation period From baseline to week 52 Number of participants with AEs will be summarized in each cohort and overall
Number of participants with AEs from baseline up to the end of the safety follow-up period From baseline to week 260 Number of participants with AEs will be summarized in each cohort and overall
- Secondary Outcome Measures
Name Time Method Change in best -corrected visual acuity (BCVA) Baseline to week 52 and Baseline to week 260 Change in BCVA from baseline in the treated and untreated eye (control)
Change in sensitivity Baseline to week 52 and Baseline to week 260 Change in sensitivity from baseline in the treated eye and untreated eye (control) as measured by the full-field stimulus testing
Trial Locations
- Locations (2)
Casey Eye Institute - Oregon Health & Science University
🇺🇸Portland, Oregon, United States
Scheie Eye Institute, University of Pennsylvania
🇺🇸Philadelphia, Pennsylvania, United States