Subretinal Injection for Leber Congenital Amaurosis

Recruiting in Palo Alto (17 mi)

+1 other location

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Atsena Therapeutics Inc.

No Placebo Group

Trial Summary

What is the purpose of this trial?

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.

Eligibility Criteria

This trial is for adults and children with Leber Congenital Amaurosis (LCA) due to specific GUCY2D gene mutations. Adults must have very limited vision, while kids can have slightly better sight. Participants need some retinal structure visible on scans and agree to contraception and not donating biological materials post-treatment.

Inclusion Criteria

I have Leber congenital amaurosis due to GUCY2D gene mutations.

I am at least 18 years old for Cohorts 1-4, or between 6 and 17 years old for Cohort 5.

My tests show mutations in the GUCY2D gene from a certified lab.

Exclusion Criteria

I am not using, nor plan to use, any anti-viral therapy that could affect the trial treatment.

My eyes are healthy enough for surgery and won't affect the study's outcome.

I haven't had any eye procedures in the last 6 months that would affect my surgery.

Treatment Details

Interventions

• SAR439483 (Gene Therapy)

Trial Overview The study tests different doses of SAR439483, injected under the retina, for safety and effectiveness in LCA patients with GUCY2D mutations. It includes a single injection followed by monitoring. Other medications like steroids may be used to manage side effects or prepare the eye.

Participant Groups

Group I: ATSN-101 Experimental Treatment 6 Interventions

ATSN-101 single dose according to an ascending dose design (dose escalation phase) or ATSN-101 single dose (dose expansion phase)

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Casey Eye Institute - Oregon Health & Science University Portland, OR

Scheie Eye Institute, University of Pennsylvania Philadelphia, PA

Who Is Running the Clinical Trial?

Atsena Therapeutics Inc. Lead Sponsor

References