A Safety and Efficacy Study of a One-time Intravitreal Injection of SAR446597 in Participants With Geographic Atrophy Secondary to Age-related Macular Degeneration

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

This is a sequential Phase 1/2, two-part, multicenter study on safety, tolerability, and efficacy of one-time intravitreal SAR446597 for the treatment of participants with Geographic Atrophy (GA) secondary to Age-related Macular Degeneration (AMD).

The core phase duration will be approximately 2 years for each participant. An Extended Follow-Up (EFU) phase of 3 years follows the core phase.

The treatment is a one-time intravitreal injection of SAR446597 (or sham as applicable in Part II).

Study Overview

• Status: Recruiting
• Conditions: Geographic Atrophy
• Intervention / Treatment: Drug: SAR446597; Drug: Sham Comparator

• Study Type: Interventional
• Enrollment (Estimated): 104
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Trial Transparency email recommended (Toll free for US & Canada); Phone Number: option 6 800-633-1610; Email: contact-us@sanofi.com

Study Locations

• Australia
  • New South Wales
    • Sydney, New South Wales, Australia, 2000
      • Recruiting
      • Investigational Site Number : 0360003
  • South Australia
    • Adelaide, South Australia, Australia, 5000
      • Recruiting
      • Investigational Site Number : 0360002
  • Victoria
    • East Melbourne, Victoria, Australia, 3002
      • Recruiting
      • Investigational Site Number : 0360001
• United States
  • Arizona
    • Peoria, Arizona, United States, 85381
      • Recruiting
      • Associated Retina Consultants - Peoria- Site Number : 8400011
    • Scottsdale, Arizona, United States, 85255
      • Recruiting
      • Retina Macula Institute of Arizona- Site Number : 8400028
  • Florida
    • Gainesville, Florida, United States, 32607
      • Recruiting
      • Vitreo Retinal Associates - Gainesville- Site Number : 8400004
    • St. Petersburg, Florida, United States, 33711
      • Recruiting
      • Retina Vitreous Associates of Florida - St. Petersburg- Site Number : 8400002
  • Illinois
    • Lemont, Illinois, United States, 60439
      • Recruiting
      • University Retina - Lemont- Site Number : 8400005
  • Maryland
    • Chevy Chase, Maryland, United States, 20815
      • Recruiting
      • The Retina Group of Washington - Chevy Chase- Site Number : 8400009
    • Hagerstown, Maryland, United States, 21740
      • Recruiting
      • Cumberland Valley Retina Consultants - Hagerstown- Site Number : 8400003
  • Oregon
    • Eugene, Oregon, United States, 97401
      • Recruiting
      • Oregon Retina- Site Number : 8400017
  • Pennsylvania
    • Bethlehem, Pennsylvania, United States, 18017-9412
      • Recruiting
      • Mid Atlantic Retina - Bethlehem- Site Number : 8400031
  • Texas
    • Austin, Texas, United States, 78750
      • Recruiting
      • Austin Clinical Research - Austin - Anderson Mill Road- Site Number : 8400007
    • Bellaire, Texas, United States, 77401
      • Recruiting
      • Retina Consultants of Texas - Bellaire- Site Number : 8400019
    • Dallas, Texas, United States, 75231
      • Recruiting
      • Retina Foundation of the Southwest- Site Number : 8400001
    • Dallas, Texas, United States, 75231
      • Recruiting
      • Texas Retina Associates - Dallas- Site Number : 8400006
    • San Antonio, Texas, United States, 78240
      • Recruiting
      • Retinal Consultants of Texas - San Antonio- Site Number : 8400012

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 60 years old or above
• Participants with diagnosis of GA secondary to age-related macular degeneration (AMD)
• Study eye with best corrected visual acuity (BCVA) ETDRS Snellen equivalent for dose escalation (Part I) between 20/40 and 20/320 and for expansion (Part II) equal or better than 20/200
• Study eye with GA lesion measuring between 2.5 and 17.5 mm2 for dose escalation (Part I) and between 2.5 and 14.0 mm2 for expansion (Part II). For multifocal disease, study eye with at least one single lesion of more than 1.25mm2 for both Part I and Part II

Exclusion Criteria:

• GA in the study eye caused by a disease different than AMD
• Presence of neovascularization or a history of treatment with an anti vascular endothelial growth factor agent in the study eye
• Any condition or treatment (ocular or systemic) or medical or surgical history in the study eye that may prevent visual acuity improvement or interfere with ocular safety or efficacy assessments
• Current or history of systemic complement targeting treatment in the past 12 months
• Use of ocular corticosteroids for 4 months (for ocular or periocular injections), 6 months (for intraocular implants) or 3 years (for long lasting intraocular implants) prior to screening in the study eye
• History of macular laser photocoagulation treatment, photodynamic- or thermotherapy or photo biomodulation in the study eye
• History of active ocular infection in the study eye in 6 months prior to screening
• Presence of active ocular or periocular infections
• Active uncontrolled glaucoma in the study eye
• History of uveitis or scleritis in either eye
• Previous gene therapy in either eye
• Any significant poorly controlled illness that would preclude study compliance and follow up

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Double

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Part I - SAR446597 open-label (OL); Participants will receive SAR at a dose specified for each cohort. Multiple dose levels of SAR446597 will be evaluated in successive cohorts of participants.	Drug: SAR446597; Intravitreal injection
Experimental: Part II - SAR446597 Dose A; Participants will receive SAR at a dose specified for each arm.	Drug: SAR446597; Intravitreal injection
Experimental: Part II - SAR446597 Dose B; Participants will receive SAR at a dose specified for each arm.	Drug: SAR446597; Intravitreal injection
Sham Comparator: Part II - Sham control; Participants will receive Sham at a dose specified for each arm.	Drug: Sham Comparator; Sham injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAEs)	Day 1 to Week 104
Incidence and severity of ocular and non-ocular treatment-emergent serious adverse events (TESAEs)	Day 1 to Week 104

Secondary Outcome Measures

Outcome Measure	Time Frame
Change in square root-transformed (mm) and untransformed area (mm2) of GA	Baseline, Week 52, Week 104
Change in best-corrected visual acuity (BCVA) from baseline to Week 52 and Week 104 following SAR446597 administration measured with the Early Treatment of Diabetic Retinopathy Study (ETDRS) chart	Baseline, Week 52, Week 104
Percentage of participants without loss of BCVA of ≥15 ETDRS letters from baseline in the study eye	Baseline, Week 52, Week 104
Incidence and severity of ocular and non-ocular TEAEs and TESAEs including clinically significant changes in safety parameters	Week 260 or End of Study

Collaborators and Investigators

• Sponsor: Sanofi

Publications and helpful links

Helpful Links

• DFI18231 Plain Language Results Summary

Study record dates

Study Major Dates

• Study Start (Actual): October 9, 2025
• Primary Completion (Estimated): October 13, 2027
• Study Completion (Estimated): July 22, 2032

Study Registration Dates

• First Submitted: October 1, 2025
• First Submitted That Met QC Criteria: October 9, 2025
• First Posted (Actual): October 10, 2025

Study Record Updates

• Last Update Posted (Actual): April 21, 2026
• Last Update Submitted That Met QC Criteria: April 20, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Retinal Diseases; Retinal Degeneration; Macular Degeneration; Geographic Atrophy
• Other Study ID Numbers: DFI18231; 2025-524508-31 (Registry Identifier: CTIS)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No