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Study on Long-Term Safety and Effects of SAR421869 for Patients with Usher Syndrome Type 1B

InvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

Usher syndrome type 1B is a rare inherited condition that causes both hearing loss and progressive loss of vision due to damage to the light‑sensing cells in the eye. The study investigates a single experimental medicine called SAR421869, which is a lentiviral vector – a tiny, harmless virus that has been engineered to carry a normal copy of the human MYO7A gene and deliver it to eye cells in hopes of slowing or stopping the visual decline.

The main aim of the trial is to evaluate the long‑term safety and how well the treatment is tolerated in people with this form of Usher syndrome. Participants will receive a series of injections of the study drug into the eye over several months and will be followed for a period of years with regular check‑ups that include eye examinations and imaging to look for any adverse events and to see whether the disease‑related retinal degeneration progresses more slowly. Safety checks focus on the ocular health of the participants, and any important changes are recorded throughout the study.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Participation begins after providing signed and dated written informed consent. This includes any necessary local authorizations, such as the Health Insurance Portability and Accountability Act (HIPAA).

    Eligibility requires previous enrollment in protocol TDU13600 and having received a subretinal injection of SAR421869.

  2. Step 2

    Treatment administration

    SAR421869 is administered as a suspension for injection. The specific route of administration is not detailed in the available information.

  3. Step 3

    Monitoring and assessments

    The study aims to evaluate the long-term safety and tolerability of SAR421869 in patients with Usher syndrome Type 1B.

    Primary assessments focus on the incidence of adverse events.

    Secondary assessments include monitoring for clinically important changes in eye safety and any delay in retinal degeneration.

  4. Step 4

    Study duration

    The study is expected to continue until June 18, 2031.

Who can join the trial?

6 criteria

  • Provide signed and dated written informed consent. This means you agree to participate in the study and understand what it involves. If needed, provide assent, which is a way for younger participants to agree.
  • Have any required local authorization, such as the Health Insurance Portability and Accountability Act (HIPAA), which is a law that protects your health information.
  • Must have been enrolled in a previous study with the code TDU13600.
  • Must have received a subretinal injection of SAR421869. A subretinal injection is a procedure where a small amount of medicine is placed under the retina, which is a part of the eye.
  • Both males and females can participate.
  • Participants can be from vulnerable populations, which means groups that might need special protection or care.

Who cannot join the trial?

5 criteria

  • Patients who do not have Usher's syndrome Type 1B cannot participate. Usher's syndrome is a condition that affects hearing and vision.
  • Patients who are not within the specified age range for the study cannot participate. The age range is not specified here, but it is important for eligibility.
  • Patients who are not part of the specified clinical trial group cannot participate. This refers to a specific group of patients that the study is focusing on.
  • Patients who are not male or female cannot participate. The study includes both male and female participants.
  • Patients who are not considered part of a vulnerable population cannot participate. A vulnerable population includes groups that may need special protection or care.
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Investigated drugs

SAR421869 is a medication being studied for its long-term safety and tolerability in patients with Usher syndrome Type 1B. This trial aims to understand how well patients can handle the medication over an extended period and to observe any biological effects it may have. Usher syndrome Type 1B is a genetic condition that affects hearing and vision, and this study is focused on finding a treatment that could potentially help manage these symptoms.

What is already known about the treatment

SAR421869 – This medication is administered through an injection, specifically designed for patients with Usher syndrome Type 1B. Currently, it is under investigation in clinical trials to assess its long-term safety and tolerability. The main therapeutic indication is to address symptoms associated with Usher syndrome, a condition that affects hearing and vision. At the molecular level, SAR421869 works by targeting specific pathways involved in the disease process, although the exact mechanism is still being studied. It falls under the pharmacological classification of investigational drugs, as it is not yet approved for general medical use.

Investigated diseases

Usher Syndrome Type 1B – Usher Syndrome Type 1B is a genetic disorder characterized by hearing loss and progressive vision loss. Individuals with this condition are typically born with severe to profound hearing impairment and experience balance issues due to inner ear problems. Vision loss usually begins in childhood or adolescence and is caused by a condition called retinitis pigmentosa, which leads to the degeneration of the retina. As the disease progresses, individuals may experience night blindness and a narrowing of the field of vision. Over time, the vision impairment can become more severe, affecting daily activities. The progression of symptoms can vary among individuals, but the combination of hearing and vision loss significantly impacts communication and mobility.
Trial detailsLast updated 9 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-513500-34-00Protocol codeLTS13619Estimated enrolment9 patientsSponsorSanofi-Aventis Recherche & Developpement

sourced from the EU Clinical Trials Register and site verification

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