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Study on the Safety and Effectiveness of rAAV.hCNGA3 for Adults and Minors with CNGA3-Linked Achromatopsia

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What is this study about?

This clinical trial is focused on studying the safety and effectiveness of a new treatment for CNGA3-linked achromatopsia, a genetic eye condition that affects color vision and visual clarity. The treatment being tested is called rAAV.hCNGA3, which is a solution for injection. It contains a special type of virus, known as an adeno-associated viral vector, that carries a human gene called CNGA3. This gene is delivered to the eye to help improve vision by targeting the specific genetic cause of the condition.

The purpose of the study is to evaluate how safe and effective this treatment is when injected into both eyes of patients, including both adults and children. Participants in the study will receive a single injection of the treatment under the retina, which is the light-sensitive layer at the back of the eye. The study is designed to compare the results of those who receive the treatment immediately with those who are placed on a waitlist, ensuring a fair assessment of the treatment’s impact.

Throughout the study, various tests will be conducted to monitor changes in vision, such as contrast sensitivity and visual acuity, which measures how well one can see details at different distances. Additionally, participants will be asked about their experiences and any changes they notice in their vision. The study aims to provide valuable information on whether this gene therapy can be a safe and effective option for improving vision in people with CNGA3-linked achromatopsia.

1 initial assessment

Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes a clinical diagnosis of achromatopsia and genetic testing to identify mutations in the CNGA3 gene.

For female participants of childbearing potential, a negative pregnancy test is required. Male participants must agree to use condoms for the first 6 months after treatment.

2 treatment administration

The treatment involves a single bilateral injection of rAAV.hCNGA3, a solution for injection, administered subretinally. This means the injection is given directly into the space beneath the retina in both eyes.

The goal of this treatment is to assess its safety and effectiveness in improving vision for those with CNGA3-linked achromatopsia.

3 post-treatment monitoring

After the injection, regular follow-up visits are scheduled to monitor the patient’s response to the treatment. These visits include various tests to evaluate vision and eye health.

Primary assessments focus on contrast sensitivity, measured 6 months after treatment. Secondary assessments include visual acuity tests, patient-reported outcomes, and other specialized eye tests.

4 long-term follow-up

The study continues to monitor participants over several years to gather long-term data on the treatment’s safety and effectiveness.

The estimated end date for the trial is April 30, 2028, allowing for comprehensive data collection and analysis.

Who Can Join the Study?

  • Have a clinical diagnosis of achromatopsia, which is a condition affecting color vision.
  • Female patients who can have children must agree to use an effective method of birth control for the first 6 months after treatment.
  • Female patients who can have children must have a negative pregnancy test. Women who are two years post-menopausal or surgically sterile are not considered to be able to have children.
  • Be between 6-12 years old or 18 years or older.
  • Have a bi-allelic pathogenic or likely pathogenic mutation in CNGA3, meaning there are changes in both copies of a specific gene that can cause disease.
  • Have a BCVA (Best Corrected Visual Acuity) of 20/400 or better, which is a measure of vision clarity.
  • Have a minimal outer nuclear layer thickness of 10 micrometers at 3 degrees eccentricity. This refers to a specific measurement of the retina, which is the light-sensitive layer at the back of the eye.
  • Be able to understand and willing to agree to the study protocol.
  • Not have an infection with Human Immunodeficiency Virus (HIV).
  • Male patients must agree to use condoms for the first 6 months after treatment.

Who Cannot Join the Study?

  • Patients who do not have CNGA3-linked achromatopsia cannot participate. This is a specific eye condition that affects color vision.
  • Patients who are not within the specified age range cannot participate. The study includes certain age groups, so if you are too young or too old, you may not be eligible.
  • Patients who are part of a vulnerable population cannot participate. This means people who might need special protection or care, like those with certain disabilities or conditions.

Where you can join this trial?

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Verified Sites

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Other Sites

Site Name City Country Status
Universitaetsklinikum Tuebingen AöR Tuebingen Germany

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

Trial status

Country Status Recruitment Start
Germany Germany
Not recruiting
01.11.2015

Trial locations

rAAV.hCNGA3 is a gene therapy being studied for its potential to treat achromatopsia, a condition that affects vision. This therapy involves a single injection into the eye, aiming to deliver a healthy copy of the CNGA3 gene to help improve vision in patients with this genetic disorder. The trial is designed to assess both the safety and effectiveness of this treatment when administered to both adults and minors.

CNGA3-linked achromatopsia – This is a genetic disorder that affects the cone cells in the retina, leading to color blindness, reduced visual acuity, and light sensitivity. Individuals with this condition often experience difficulty seeing in bright light and may have nystagmus, which is an involuntary eye movement. The disease is caused by mutations in the CNGA3 gene, which is crucial for normal cone cell function. Over time, the lack of functional cone cells can lead to a reliance on rod cells, which are more suited for low-light conditions but do not support color vision. This condition is present from birth and remains stable throughout life, without progressive worsening of symptoms.

Trial ID:
2024-515553-17-00
Protocol code:
RDC-CNGA3-01
Trial Phase:
Human Pharmacology (Phase I) – Other

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