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Study of AAVB-081 and Prednisolone for Patients with Usher Syndrome Type 1B Retinitis Pigmentosa

Verified siteRegistered drugNo placebo
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What is this trial about?

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

This clinical trial is focused on studying a condition called Usher Syndrome Type 1B (USH1B) Retinitis Pigmentosa. This is a genetic disorder that affects both hearing and vision, leading to progressive vision loss. The study is testing a new treatment called AAVB-081, which is a type of gene therapy. Gene therapy involves using a specially designed virus to deliver a healthy copy of a gene to cells in the body. In this case, the treatment uses an adeno-associated viral vector to deliver the MYO7A gene, which is important for normal vision, directly to the eye.

The purpose of the study is to evaluate the safety and tolerability of this new treatment when it is injected under the retina, which is the light-sensitive layer at the back of the eye. Participants will receive the treatment through a procedure called subretinal administration. The study will also aim to find the best dose that balances benefits and risks. Throughout the study, participants will undergo various health checks, including eye exams and blood tests, to monitor their response to the treatment.

Participants in the study will be monitored over a period of time to assess any side effects and to determine how well the treatment works in improving or stabilizing vision. The study will help researchers understand if this gene therapy can be a safe and effective option for people with Usher Syndrome Type 1B Retinitis Pigmentosa.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes a review of medical history and a molecular diagnosis of Usher Syndrome Type 1B due to MYO7A mutations.

    Participants must be between 18 and 50 years old and provide written informed consent to adhere to the study protocol.

  2. Step 2

    Baseline evaluations

    Baseline evaluations are performed to gather initial health data. This includes physical examinations, vital signs, and laboratory tests such as biochemistry, hematology, and urinalysis.

    Ophthalmic exams are conducted to assess vision, including tests like visual field perimetry and indirect ophthalmoscopy.

  3. Step 3

    Medication administration

    The study involves the administration of two medications: prednisone and AAVB-081.

    Prednisone is taken orally. The dosage and frequency are determined by the study protocol.

    AAVB-081 is administered through a subretinal injection, which is a procedure where the medication is injected into the space beneath the retina.

  4. Step 4

    Monitoring and follow-up

    Participants are monitored for any adverse effects. This includes regular physical examinations and laboratory tests to check for changes in health status.

    Ophthalmic exams are repeated to monitor any changes in vision and eye health.

  5. Step 5

    Completion of study

    The study is estimated to conclude by May 28, 2030. Participants will undergo final evaluations to assess the overall impact of the treatment.

    Data collected throughout the study will be analyzed to determine the safety and effectiveness of the treatment.

Who can join the trial?

3 criteria

  • All participants must be willing to follow the study rules and show this by signing a written agreement called informed consent.
  • Participants must be adults, both men and women, who are diagnosed with **Usher Syndrome Type 1B (USH1B)**. They should be between 18 and 50 years old.
  • Participants need to have a specific genetic diagnosis of USH1B caused by changes in a gene called **MYO7A**. This diagnosis must be confirmed by a special laboratory approved by the study sponsor.

Who cannot join the trial?

10 criteria

  • Patients who are not diagnosed with Usher Syndrome Type 1B cannot participate.
  • Patients who do not have Retinitis Pigmentosa associated with Usher Syndrome Type 1B cannot participate.
  • Patients who are younger than 3 years old cannot participate.
  • Patients who are not part of the specified clinical trial groups cannot participate.
  • Patients who are not willing to follow the study procedures cannot participate.
  • Patients who have other medical conditions that might interfere with the study cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who have participated in another clinical trial recently may not be eligible.
  • Patients who have allergies to the study medication or its components cannot participate.
  • Patients who have a history of certain eye surgeries or conditions that affect the eyes cannot participate.
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Investigated drugs

AAVB-081 is a gene therapy product used in this clinical trial. It is administered directly into the subretinal space of the eye. The therapy is designed to deliver a healthy copy of the MYO7A gene using a viral vector, specifically AAV8, to patients with Usher Syndrome Type 1B, which is a form of retinitis pigmentosa. The goal of this treatment is to improve or stabilize vision by addressing the genetic cause of the disease.

What is already known about the treatment

AAVB-081 – This medication is administered through a subretinal injection, specifically targeting the eye. It is currently being studied in clinical trials for its safety and effectiveness in treating Usher Syndrome Type 1B, which leads to a form of retinitis pigmentosa. The main therapeutic indication is to address vision loss associated with this genetic condition. AAVB-081 works by delivering a gene therapy using a viral vector to introduce a functional copy of the MYO7A gene, which is defective in patients with this syndrome. It is classified as a gene therapy product, aiming to correct the underlying genetic cause of the disease.

Investigated diseases

Usher Syndrome Type 1B (USH1B) Retinitis Pigmentosa – This is a genetic disorder that affects both hearing and vision. Individuals with this condition are born with severe hearing loss and begin to experience vision problems in childhood. The vision issues are due to retinitis pigmentosa, which causes progressive degeneration of the retina. This leads to night blindness and a gradual loss of peripheral vision, often resulting in tunnel vision. Over time, the condition can lead to significant visual impairment. The progression of vision loss varies among individuals.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase I/IITrial ID2024-518489-29-00Protocol code081-101Estimated enrolment15 patientsSponsorAavantgarde Bio S.r.l.

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