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Study on the Long-Term Safety of SAR422459 for Patients with Stargardt’s Disease

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What is this study about?

This clinical trial is focused on studying the long-term safety and effects of a treatment called SAR422459 in patients with Stargardt’s macular degeneration. Stargardt’s macular degeneration is a genetic eye disease that affects the retina, leading to vision loss. The treatment involves a special type of injection known as a lentiviral vector containing the human ABCA4 gene, which is designed to help address the genetic cause of the disease.

The purpose of this study is to evaluate how well patients tolerate the treatment over a long period and to observe any biological changes that occur. Participants in the study will receive the treatment as a suspension for injection. The study will monitor the occurrence of any side effects and assess any changes in eye health and the progression of retinal degeneration.

This study is open-label, meaning that both the researchers and participants know what treatment is being administered. The trial will follow patients who have previously participated in a related study and received the treatment. The study aims to gather valuable information on the treatment’s safety and its potential to slow down the progression of Stargardt’s macular degeneration.

1 joining the study

Participation begins after providing signed and dated written informed consent. This includes any locally required authorization, such as HIPAA.

Eligibility requires previous enrollment in protocol TDU13583 and completion of that protocol to Week 48 or an early discontinuation visit.

2 treatment administration

The treatment involves a subretinal injection of SAR422459, which is a suspension for injection containing a lentiviral vector with the human abca4 gene.

The specific route of administration is not detailed beyond being an injection.

3 monitoring and follow-up

The main objective is to evaluate the long-term safety and tolerability of SAR422459 in patients with Stargardt’s macular degeneration.

Monitoring includes assessing the incidence of adverse events and observing any clinically important changes in ocular safety assessments.

The study also aims to observe any delay in retinal degeneration.

4 study duration

The study is expected to continue until December 29, 2033.

The recruitment for the study began on October 3, 2013.

Who Can Join the Study?

  • Provide signed and dated written informed consent, which means you agree to participate in the study and understand what it involves.
  • Have any locally required authorization, such as HIPAA, which is a law that protects your health information.
  • Must have been enrolled in a previous study called protocol TDU13583.
  • Must have received a subretinal injection of SAR422459, which is a treatment given directly into the eye.
  • Must have completed the previous study (protocol TDU13583) up to Week 48 or have had an early discontinuation visit, which means you left the study early but followed the necessary procedures.

Who Cannot Join the Study?

  • Patients with other congenital (present from birth) or hereditary (passed down from parents) diseases that are not related to Stargardt’s macular degeneration.
  • Patients with other neonatal (conditions that occur in newborns) diseases that are not related to Stargardt’s macular degeneration.
  • Patients who are not within the specified age range for the study.
  • Patients who are not part of the specified clinical trial group.
  • Patients who are considered part of a vulnerable population, which means they might need special protection or care.

Where you can join this trial?

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Other Sites

Site Name City Country Status
Quinze-Vingts National Ophthalmology Hospital Paris France

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
France France
Not recruiting
03.10.2013

Trial locations

SAR422459 is a medication being studied for its potential to treat Stargardt’s macular degeneration, a genetic eye disorder that causes vision loss. The trial aims to assess the long-term safety and tolerability of this medication in patients. SAR422459 is designed to help improve or stabilize vision by targeting the underlying causes of the disease.

Investigated diseases:

Stargardt’s Macular Degeneration – Stargardt’s macular degeneration is a genetic eye disorder that affects the macula, the central part of the retina responsible for sharp, straight-ahead vision. It typically begins in childhood or adolescence and leads to progressive vision loss. The disease is caused by the buildup of lipofuscin, a fatty substance, in the retinal cells, which leads to their degeneration. Patients often experience difficulty seeing in low light and may notice blurry or distorted vision. Over time, the central vision becomes more impaired, although peripheral vision usually remains intact. The progression of vision loss can vary widely among individuals.

Trial ID:
2024-513501-31-00
Protocol code:
LTS13588
Trial Phase:
Therapeutic exploratory (Phase II)

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