RecruitingRare disease

A study to evaluate the effectiveness of ciracigene golparvovec in children with Canavan disease

Verified siteInvestigationalNo placebo

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What is this trial about?

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

This study focuses on children with Canavan disease, which is a rare genetic disorder that affects how the brain develops and functions. This condition causes a loss of myelin, the protective coating that surrounds nerve fibers in the brain, leading to various developmental challenges. The purpose of the study is to evaluate the effects of ciracigene golparvovec, also known as rAAV-Olig001-ASPA, a type of gene therapy designed to address the underlying cause of the disease. This treatment is administered as a single injection into the cerebrospinal fluid, which is the clear liquid that surrounds the brain and spinal cord.

During the study, researchers will monitor levels of NAA, a specific substance in the body that is often low in individuals with this condition, by testing the fluid around the brain and urine samples. The study will also look at changes in the brain using SyMRI, a specialized type of magnetic resonance imaging, to observe the amount of protective coating in the brain over time. Additionally, participants will be assessed on their physical movements, such as sitting, crawling, and walking, as well as their communication and fine motor skills to understand how the treatment might influence their development.

The research process

The trial runs in 3 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Administration of gene therapy
A single dose of ciracigene golparvovec is administered through intracerebroventricular injection. this means the solution for injection is delivered directly into the fluid-filled spaces of the brain.
Step 2
Follow-up assessments
At month 6, tests are performed to measure changes in naa concentrations (a specific substance) in the cerebrospinal fluid, which is the clear liquid that surrounds the brain and spinal cord, as well as in the urine.
Through month 12, brain imaging is conducted to monitor changes in myelin volume. myelin is the protective layer that covers nerve fibers in the brain.
Step 3
Motor and developmental monitoring
Assessments are conducted to track changes in gross motor function, which includes abilities such as lying/rolling, sitting, crawling, standing, and walking.
Developmental progress is monitored using msel domains, which evaluate skills like visual reception, fine motor (small movements), receptive language (understanding speech), and expressive language (using speech).

Who can join the trial?

6 criteria

A person must have a confirmed diagnosis of Canavan disease, which is a rare condition, by a neurologist (a doctor who specializes in the brain and nervous system).
The child must be between 3 and 60 months old at the time they receive the treatment through brain surgery.
Patients who are part of a specific group called Cohort 4 and have not received treatment can be older than 60 months.
A parent or legal guardian must provide written informed consent, which means they have read and signed a document agreeing to let the child participate in the study and follow all rules, such as attending follow-up appointments.
The patient must have had an MRI (a type of medical imaging that uses magnets to take pictures of the inside of the body) or a CT scan (a medical test that uses X-rays to create detailed pictures of the body) within the last 3 months to help doctors plan the surgery.
If an MRI or CT scan was not done in the last 3 months, an MRI can be performed during the screening process (the initial period where doctors check if a person is suitable for the study).

Who cannot join the trial?

9 criteria

Having any major long-term health problem involving the brain and nerves (neurological), heart (cardiac), liver (hepatic), kidneys (renal), blood (hematological), digestive system (gastrointestinal), hormones (endocrine), lungs (pulmonary), or infections (infectious) that could make surgery risky or prevent participation in the study.
A history of anaphylaxis, which is a severe and life-threatening allergic reaction.
Having taken part in gene therapy trials or used other experimental medicines within the last 6 months.
Having a medical reason that prevents the use of immunosuppression, which refers to medicines that lower the body's immune system response.
Having a medical reason that prevents the use of an MRI, which is a way to take detailed pictures of the inside of the body using magnets.
Receiving any vaccination less than 1 month before starting the gene therapy.
An expected life expectancy of less than 12 months for any reason.
A total score of more than 35% on the GMFM-88, which is a test used to measure a child's gross motor function, such as movement and physical skills.
Having lab values (results from blood or other body fluid tests) that are outside of the normal range and are considered important by the lead doctor.

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Verified sites

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Bicetre Hospital
Verified
Le Kremlin-Bicetre, France

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Investigated drugs

Ciracigene golparvovec is a gene therapy designed to treat children with Canavan disease. It is administered as a single injection directly into the fluid that surrounds the brain and spinal cord to help address the underlying cause of the condition.

What is already known about the treatment

Ciracigene golparvovec - This medication is an experimental gene therapy delivered as a single injection directly into the fluid-filled spaces of the brain. Currently being studied in clinical trials, it is being developed to treat Canavan disease, a rare genetic disorder that affects the brain's development. The treatment works by using a modified, harmless virus to carry a healthy copy of a specific gene into the patient's cells, helping the body produce a missing enzyme needed for brain health. This therapy is classified as a gene therapy medicinal product.

Investigated diseases

Canavan disease - This is a rare genetic disorder that affects the brain and the nervous system. It is caused by a lack of an enzyme needed to process certain substances in the brain. As the condition progresses, the protective coating around nerve cells, known as myelin, begins to break down. This loss of myelin affects how signals are sent throughout the brain. Over time, this leads to difficulties with physical movements and various developmental milestones.