NCT03920007

Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D

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

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 15 (actual)
• Sponsor: Atsena Therapeutics Inc. · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Leber Congenital Amaurosis
• LCA
• LCA1

Interventions

Type	Name	Description
DRUG	ATSN-101	Pharmaceutical form:Solution for intraocular administration Route of administration: Subretinal injection
DRUG	ATSN-101 Diluent Solution	Pharmaceutical form:Solution for parenteral use Route of administration: Subretinal injection
DRUG	Prednisone	Pharmaceutical form:Tablet Route of administration: Oral
DRUG	Triamcinalone Acetonide	Pharmaceutical form:Suspension Route of administration: Peri-ocular injection
DRUG	1% Prednisolone	Pharmaceutical form:Suspension Route of administration: Drops
DRUG	Trimethoprim/polymyxin B	Pharmaceutical form:Solution Route of administration: Topical

Timeline

• Start date: 2019-09-12
• Primary completion: 2023-05-19
• Completion: 2027-05-19
• First posted: 2019-04-18
• Last updated: 2024-02-20

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study