SAR422459 Follow-Up Study for Stargardt Disease

Not currently recruiting at 3 trial locations

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Sanofi

Disqualifiers: Not received SAR422459

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial evaluates the long-term safety and tolerability of SAR422459, a new potential drug, for individuals with Stargardt Disease, a genetic eye condition affecting vision. Researchers aim to determine if the treatment has any biological effects. It is intended for those who participated in a previous study and received this treatment. Those who joined that earlier study may find this follow-up trial suitable. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group, offering valuable insights into its potential benefits.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications.

Is there any evidence suggesting that SAR422459 is likely to be safe for humans?

Research has shown that SAR422459, a gene therapy for Stargardt's disease, underwent safety testing over three years. The treatment demonstrated a strong safety record among patients. Most participants tolerated it well, with no serious side effects reported. However, one patient on the highest dose experienced a reduction in macular flecks (spots on the retina), indicating some biological activity.

These findings suggest that SAR422459 is generally safe for people with Stargardt's disease. The current study aims to monitor its long-term safety and effects, building on these positive results. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Stargardt's?

Unlike the standard treatments for Stargardt disease, which focus primarily on managing symptoms and slowing progression, SAR422459 offers a more innovative approach by using gene therapy. This treatment is designed to target the underlying genetic cause of the disease by delivering a functional copy of the ABCA4 gene directly to the retina. Researchers are excited about SAR422459 because it has the potential to address the root cause of vision loss in Stargardt disease, rather than just treating the symptoms.

What evidence suggests that SAR422459 might be an effective treatment for Stargardt Disease?

Research has shown that SAR422459, a gene therapy, is being explored as a potential treatment for Stargardt disease. In earlier studies, one patient who received a high dose of SAR422459 had fewer macular flecks (a type of eye damage) in the treated eye compared to the untreated eye. However, overall vision did not significantly improve, and some treated eyes experienced negative changes. The therapy targets a specific gene linked to the disease, aiming to slow or stop its progression. While still in early stages, these results are both promising and cautious. Participants in this follow-up study will undergo long-term monitoring to assess the effects of SAR422459 received in a previous study. ^{1 4 5 6 7}

Who Is on the Research Team?

David Wilson, MD

Principal Investigator

Oregon Health and Science University

José-Alain Sahel, MD

Principal Investigator

Centre National d'Ophtalmologie des Quinze-Vingts

Are You a Good Fit for This Trial?

This trial is for patients who have Stargardt's Macular Degeneration and were previously enrolled in the TDU13583 study. Participants must have completed that study up to Week 48 or reached an early discontinuation point, having received a subretinal injection of SAR422459.

Inclusion Criteria

Must have completed protocol TDU13583 to Week 48 or undergone an early discontinuation visit.

I have received a SAR422459 injection in my eye.

Provide signed and dated written informed consent and any locally required authorization (e.g., Health Insurance Portability and Accountability Act [HIPAA])

Exclusion Criteria

Did not receive SAR422459 as part of the TDU13583 protocol.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Long Term Follow-up

Participants are monitored for long-term safety, tolerability, and biological activity of SAR422459

15 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• SAR422459

Trial Overview The follow-up study focuses on the long-term safety and tolerability of SAR422459, which was administered to participants in a prior trial. It also aims to assess any ongoing biological activity of the treatment.

How Is the Trial Designed?

Group I: Long Term Follow up Experimental Treatment 1 Intervention

Long term follow up in all patients who received SAR422459 in previous study TDU13583

Who Is Running the Clinical Trial?

Sanofi

Lead Sponsor

Trials

2,246

Recruited

4,085,000+

Paul Hudson

Sanofi

Chief Executive Officer since 2019

Degree in Economics from Manchester Metropolitan University

Christopher Corsico

Sanofi

Chief Medical Officer

MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9308722/

Three-year safety results of SAR422459 (EIAV-ABCA4) gene ... The purpose of the study was to investigate safety of the first gene therapy (EIAV-ABCA4) in patients with ABCA4-associated Stargardt disease.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT01736592

NCT01736592 | Phase I/II Follow-up Study of SAR422459 ... A way for patients with serious diseases or conditions who cannot participate in a clinical trial to gain access to a medical product that has not been approved ...

3. researchgate.net researchgate.net/publication/359011473_Three-year_safety_results_of_SAR422459_EIAV-ABCA4_gene_therapy_in_patients_with_ABCA4-associated_Stargardt_disease_An_open-label_dose-escalation_phase_IIIa_clinical_trial_cohorts_1-5

Three-year safety results of SAR422459 (EIAV-ABCA4) ... In one patient treated with the highest dose, there was a significant decline in the number of macular flecks as compared to the untreated eye.

4. retinalphysician.com retinalphysician.com/issues/2025/october/novel-therapies-for-stargardt-disease/

Novel Therapies for Stargardt Disease No clinically significant changes in visual function were observed. However, 27% of treated eyes showed worsening hypoautofluorescent changes, ...

5. mdpi.com mdpi.com/1422-0067/25/16/8859

Emerging Therapeutic Approaches and Genetic Insights in ... This review aims to synthesize recent advancements in understanding and treating Stargardt disease. By highlighting breakthroughs in genetic therapies, stem ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT01736592

Phase I/II Follow-up Study of SAR422459 in Patients With ... An Open Label Study to Determine the Long Term Safety, Tolerability and Biological Activity of SAR422459 in Patients With Stargardt's Macular Degeneration.

7. clinicaltrials.eu clinicaltrials.eu/trial/study-on-the-long-term-safety-of-sar422459-for-patients-with-stargardts-disease/

Study on the Long-Term Safety of SAR422459 for Patients ... This clinical trial studies the long-term safety of SAR422459, a lentiviral vector containing the human ABCA4 gene, in patients with Stargardt's macular ...