Universitaetsklinikum Tuebingen AöR
Tuebingen, Germany
Not recruitingColour blindness
Study on the Safety and Effectiveness of rAAV.hCNGA3 for Adults and Minors with CNGA3-Linked Achromatopsia
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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 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.
The research process
The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.
Who can join the trial?
10 criteria
- 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 trial?
3 criteria
- 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.
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Investigated drugs
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.
What is already known about the treatment
rAAV.hCNGA3 – This medication is administered as a single subretinal injection, targeting the eye directly. It is currently under investigation in clinical trials to assess its safety and effectiveness for treating CNGA3-linked achromatopsia, a genetic condition affecting vision. The main therapeutic indication is to improve visual function in individuals with this specific type of achromatopsia. At the molecular level, the medication works by delivering a healthy copy of the CNGA3 gene to retinal cells, aiming to restore normal function. It is classified as a gene therapy, focusing on correcting genetic defects at their source.
Investigated diseases
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 detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase ITrial ID2024-515553-17-00Protocol codeRDC-CNGA3-01Estimated enrolment14 patientsSponsorUniversitaetsklinikum Tuebingen AöR
sourced from the EU Clinical Trials Register and site verification
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