MedTrace Logo

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

NCT03920007 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2024-02-20

No results posted yet for this study

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.

Conditions

  • Leber Congenital Amaurosis
  • LCA
  • LCA1

Interventions

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

Sponsors & Collaborators

  • Atsena Therapeutics Inc.

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-09-12
Primary Completion
2023-05-19
Completion
2027-05-19
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03920007 on ClinicalTrials.gov