#1 Clinical Trials Search in Europe

Adeno-Associated Viral Vector Serotype 8 Containing The 3′-Myo7A Gene Coding Sequence

This article discusses an ongoing clinical trial investigating the use of AAVB-081, a novel gene therapy treatment for Usher Syndrome Type 1B (USH1B) Retinitis Pigmentosa. AAVB-081 is an adeno-associated viral vector designed to deliver the MYO7A gene to the retina. The study aims to assess the safety, tolerability, and potential effectiveness of this innovative approach in treating this rare genetic eye disorder.

Table of Contents

What is AAVB-081?

AAVB-081 is an innovative gene therapy designed to treat a specific type of inherited eye disorder. It is classified as an advanced therapy medicinal product, which means it’s a cutting-edge treatment that uses genetic material to address the root cause of a disease[1].

This therapy is also known by several other names, including:

  • Dual AAV8.MYO7A
  • AAV8.hMYO7A
  • AAV8.5′MYO7A and AAV8.3′MYO7A

These names reflect the specific components and structure of the therapy[1].

Target Condition: Usher Syndrome Type 1B (USH1B) Retinitis Pigmentosa

AAVB-081 is being developed to treat a condition called Usher Syndrome Type 1B (USH1B) Retinitis Pigmentosa. This is a rare genetic disorder that affects both vision and hearing[1].

Retinitis pigmentosa is a group of inherited eye disorders that cause progressive vision loss. In people with USH1B, this condition is caused by mutations in a gene called MYO7A[1].

How AAVB-081 Works

AAVB-081 is a gene replacement therapy. It uses a modified virus called adeno-associated virus serotype 8 (AAV8) to deliver functional copies of the MYO7A gene to the retina, the light-sensitive tissue at the back of the eye[1].

The therapy consists of two parts:

  1. AAV8.5′MYO7A: This contains the first part of the MYO7A gene
  2. AAV8.3′MYO7A: This contains the second part of the MYO7A gene

Together, these two components aim to provide a complete, functional MYO7A gene to the cells in the retina, potentially slowing or stopping the progression of vision loss[1].

Clinical Trial Details

AAVB-081 is currently being studied in a Phase 1/2 clinical trial. This means it’s in the early stages of testing in humans. The main goals of this trial are:

  • To assess the safety and tolerability of AAVB-081 when injected into the space under the retina (subretinal administration)
  • To determine the best dose that balances potential benefits with risks
  • To evaluate how AAVB-081 affects the structure and function of the retina[1]

This is an open-label study, which means both the researchers and participants know which treatment is being given. It also involves dose escalation, where different groups of participants receive increasing doses of the therapy to find the optimal amount[1].

Eligibility Criteria

To participate in this clinical trial, individuals must meet certain criteria. Some key requirements include:

  • Being diagnosed with USH1B and having confirmed mutations in the MYO7A gene
  • Being between 18 and 50 years old
  • Having enough viable retinal cells for the treatment to potentially work
  • Not having certain other eye conditions that could interfere with the treatment or its evaluation[1]

There are also several conditions that would exclude someone from participating, such as a history of retinal detachment, poorly controlled diabetes, or previous participation in another gene therapy trial[1].

Safety and Monitoring

The safety of participants is a top priority in this clinical trial. The researchers will closely monitor for any side effects or adverse events. This includes:

  • Regular physical examinations and vital sign checks
  • Blood and urine tests
  • Monitoring the immune system’s response to the therapy
  • Checking for the presence of the viral vector in blood and tears
  • Comprehensive eye exams to track any changes in vision or eye structure[1]

Potential Benefits

While it’s important to note that the effectiveness of AAVB-081 is still being studied, the hope is that this therapy could potentially:

  • Slow or stop the progression of vision loss in people with USH1B
  • Improve retinal function
  • Provide a long-lasting treatment option for this rare genetic condition[1]

However, as with any experimental treatment, the actual benefits and risks will only be fully understood after thorough clinical testing[1].

Aspect Details
Study Type Phase 1/2 open-label, dose escalation, safety and efficacy study
Drug Name AAVB-081 (Dual AAV8.MYO7A)
Target Condition Usher Syndrome Type 1B (USH1B) Retinitis Pigmentosa
Administration Method Subretinal injection
Primary Objectives Assess safety, tolerability, and determine optimal dose
Secondary Objective Evaluate effects on retinal anatomy and function
Key Inclusion Criteria Adults 18-50 years old with molecular diagnosis of USH1B
Key Exclusion Criteria History of retinal detachment, prior gene therapy, certain eye conditions
Primary Endpoints Incidence and severity of adverse events, number of dose-limiting toxicities

FAQ

  • What is AAVB-081? AAVB-081 is a gene therapy treatment that uses adeno-associated viral vectors to deliver the MYO7A gene to the retina. It's being studied as a potential treatment for Usher Syndrome Type 1B Retinitis Pigmentosa.
  • How is AAVB-081 administered? AAVB-081 is administered through subretinal injection, which means it's injected directly under the retina of the eye.
  • What are the main objectives of this clinical trial? The main objectives are to assess the safety and tolerability of AAVB-081 in patients with USH1B retinitis pigmentosa, and to determine a well-tolerated dose with an optimal risk-benefit profile.
  • Who is eligible to participate in this trial? Eligible participants are adults aged 18 to 50 with a molecular diagnosis of USH1B due to MYO7A mutations. They must be willing to adhere to the study protocol and provide informed consent.
  • What are some of the exclusion criteria for this trial? Some exclusion criteria include a history of retinal detachment, poorly controlled diabetes, previous participation in gene therapy trials, and certain pre-existing eye conditions that could interfere with the study.

Ongoing Clinical Trials on Adeno-Associated Viral Vector Serotype 8 Containing The 3′-Myo7A Gene Coding Sequence

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

    Not recruiting

    2 1 1 1
    Investigated diseases:
    Investigated drugs:
    Italy

Glossary

  • Usher Syndrome Type 1B (USH1B): A rare genetic disorder characterized by hearing loss, vision loss (retinitis pigmentosa), and sometimes balance problems.
  • Retinitis Pigmentosa: A group of rare genetic disorders that involve a breakdown and loss of cells in the retina, which can lead to vision loss.
  • Adeno-Associated Viral Vector: A virus-based tool used to deliver genetic material into cells, commonly used in gene therapy.
  • MYO7A Gene: A gene that provides instructions for making a protein called myosin VIIA, which is important for the normal function of inner ear hair cells and retinal cells.
  • Subretinal Injection: A method of delivering medication or gene therapy directly under the retina of the eye.
  • Dose Escalation: A process in clinical trials where the dose of a drug is gradually increased to find the optimal balance between effectiveness and side effects.
  • Adverse Events (AEs): Any unfavorable and unintended sign, symptom, or disease temporarily associated with the use of a medical treatment or procedure.
  • Serious Adverse Events (SAEs): Adverse events that result in death, are life-threatening, require hospitalization, cause persistent or significant disability, or lead to birth defects.
  • Dose-Limiting Toxicities (DLTs): Side effects of a drug that are severe enough to prevent an increase in dose or require a dose reduction.
  • Best Corrected Visual Acuity (BCVA): The best vision a person can achieve with the help of corrective lenses, used as a measure of visual function.

References

  1. http://clinicaltrials.eu/trial/study-of-aavb-081-and-prednisolone-for-patients-with-usher-syndrome-type-1b-retinitis-pigmentosa/