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

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What is this study about?

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.

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 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 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 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 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 Study?

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 Study?

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.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

Site Name	City	Country	Status
Azienda Ospedaliera Universitaria Universita’ Degli Studi Della Campania Luigi Vanvitelli	Naples	Italy

Other Sites

No sites found in this category

Trial status

Country	Status	Recruitment Start
Italy	Not recruiting	02.07.2024

Trial locations

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.

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 ID:

2024-518489-29-00

Protocol code:

081-101

NCT ID:

NCT06591793

Trial Phase:

Phase I and Phase II (Integrated) – First administration to humans

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

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?