DFI18231 - AMD
Research type
Research Study
Full title
A Phase 1/2, study to evaluate the safety, tolerability, and efficacy of one-time intravitreal dose of SAR446597 in participants with geographic atrophy secondary to age-related macular degeneration
IRAS ID
1013379
Contact name
Patrick Maury
Contact email
Sponsor organisation
Sanofi-Aventis Recherche & Développement
Clinicaltrials.gov Identifier
Research summary
This international study is planned to take place in 4 countries and has begun in October 2025. Age-related macular degeneration (AMD) is a common eye disease affecting older adults that causes blurred or distorted vision. Geographic atrophy (GA) is an advanced form of AMD where cells at the back of the eye gradually die, leading to permanent vision loss. This study is testing SAR446597, an experimental gene therapy given as a one-time injection into the eye. The treatment aims to modify the immune system, which researchers believe contributes to Geographic atrophy (GA) worsening over time. The goal is to determine if this one-time treatment is safe and can slow down both the growth of GA and vision loss.
Current treatments require repeated injections into the eye every month or two and have not yet shown they can maintain vision.
This is a Phase 1/2 study with two parts:
-Part I, open-label, tests different doses of SAR446597 in a small group, starting with the lowest dose.
-Part II randomly assigns participants to receive either a low dose, high dose, or a pretend injection (sham) where they have a feeling of receiving the injection but do not receive the gene therapy. Participants will not know which group they are in during Part II.
The primary endpoint is to assess safety and tolerability of SAR446597 through Week 104 and beyond.
The secondary endpoints will evaluate impact on retinal cells and vision (GA size changes and vision at 52 & 104 weeks).
Who Can Participate? Adults aged 60 years or older with geographic atrophy caused by age-related macular degeneration.
What's Involved? Participants receive steroid injections and eye drops to prevent eye inflammation. The study monitors safety and vision changes for at least 2 years.
Benefits? One-time injection potentially slowing GA, avoiding monthly/bi-monthly injections.
Risks? Eye pain, foreign body sensation, increased eye pressure, rare risks of infection, retinal damage, or permanent vision loss.REC name
London - West London & GTAC Research Ethics Committee
REC reference
26/LO/0009
Date of REC Opinion
22 May 2026
REC opinion
Further Information Favourable Opinion