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Gene Therapy

Subretinal Injection for Leber Congenital Amaurosis

Phase 1 & 2
Waitlist Available
Research Sponsored by Atsena Therapeutics Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to week 52 and baseline to week 260
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new drug for Leber Congenital Amaurosis, a rare disease that causes blindness. The drug is being injected into the retina in order to see if it is safe and effective.

Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
Potential side effects might include discomfort from the injection, inflammation inside the eye, increased eye pressure, cataracts, or other changes affecting vision. Systemic side effects could occur due to steroid use such as mood swings, weight gain, or increased blood sugar.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to week 52 and baseline to week 260
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to week 52 and baseline to week 260 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Number of participants with AEs from baseline up to the end of the safety follow-up period
Number of participants with adverse events (AEs) from baseline up to the end of the observation period
Secondary study objectives
Change in best -corrected visual acuity (BCVA)
Change in sensitivity

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: ATSN-101Experimental Treatment6 Interventions
ATSN-101 single dose according to an ascending dose design (dose escalation phase) or ATSN-101 single dose (dose expansion phase)
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Prednisone
2014
Completed Phase 4
~2500

Find a Location

Who is running the clinical trial?

Atsena Therapeutics Inc.Lead Sponsor
1 Previous Clinical Trials
21 Total Patients Enrolled

Media Library

SAR439483 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03920007 — Phase 1 & 2
Leber Congenital Amaurosis Research Study Groups: ATSN-101
Leber Congenital Amaurosis Clinical Trial 2023: SAR439483 Highlights & Side Effects. Trial Name: NCT03920007 — Phase 1 & 2
SAR439483 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03920007 — Phase 1 & 2
~2 spots leftby Dec 2025
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