A Phase 1/2 Dose Escalation Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D
Overview
- Phase
- Phase 1
- Intervention
- ATSN-101
- Conditions
- Leber Congenital Amaurosis
- Sponsor
- Atsena Therapeutics Inc.
- Enrollment
- 15
- Locations
- 2
- Primary Endpoint
- Number of participants with adverse events (AEs) from baseline up to the end of the observation period
- Status
- Active, not recruiting
- Last Updated
- 2 years ago
Overview
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.
Investigators
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
ATSN-101
ATSN-101 single dose according to an ascending dose design (dose escalation phase) or ATSN-101 single dose (dose expansion phase)
Intervention: 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)
Intervention: ATSN-101 Diluent Solution
ATSN-101
ATSN-101 single dose according to an ascending dose design (dose escalation phase) or ATSN-101 single dose (dose expansion phase)
Intervention: Prednisone
ATSN-101
ATSN-101 single dose according to an ascending dose design (dose escalation phase) or ATSN-101 single dose (dose expansion phase)
Intervention: Triamcinalone Acetonide
ATSN-101
ATSN-101 single dose according to an ascending dose design (dose escalation phase) or ATSN-101 single dose (dose expansion phase)
Intervention: 1% Prednisolone
ATSN-101
ATSN-101 single dose according to an ascending dose design (dose escalation phase) or ATSN-101 single dose (dose expansion phase)
Intervention: Trimethoprim/polymyxin B
Outcomes
Primary Outcomes
Number of participants with adverse events (AEs) from baseline up to the end of the observation period
Time Frame: 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
Time Frame: From baseline to week 260
Number of participants with AEs will be summarized in each cohort and overall
Secondary Outcomes
- Change in best -corrected visual acuity (BCVA)(Baseline to week 52 and Baseline to week 260)
- Change in sensitivity(Baseline to week 52 and Baseline to week 260)