In short
Ciracigene golparvovec is an investigational gene therapy being studied for Canavan disease, a rare and serious genetic disorder that affects the brain. It is currently being tested in a Phase 1/2 clinical trial involving 32 children. The trial is authorised and is measuring the effect of the therapy on a key substance in the cerebrospinal fluid called NAA.
Ciracigene golparvovec is an investigational gene therapy being studied for Canavan disease, a rare inherited disorder that damages the brain's white matter. It is also known by the research name rAAV-Olig001-ASPA. The substance is not yet authorised as a medicine and is currently being evaluated in a clinical trial.
Key points
- Ciracigene golparvovec is an investigational gene therapy, not yet approved as a medicine.
- It is being studied in Canavan disease, a rare inherited brain disorder.
- The only registered trial is a Phase 1/2 study in children.
- The trial plans to enrol 32 participants.
- The main goal is to measure changes in NAA levels in the cerebrospinal fluid.
- The trial is currently authorised, meaning it has received regulatory approval to proceed.
What is ciracigene golparvovec?
Ciracigene golparvovec is an investigational gene therapy. It is being developed for the treatment of Canavan disease, a rare and progressive genetic condition that affects the brain. The substance is also referred to in research records as rAAV-Olig001-ASPA. It has not received marketing authorisation and is available only within the context of clinical research.
How does ciracigene golparvovec work?
Canavan disease is caused by a genetic defect that leads to a deficiency of the enzyme aspartoacylase (ASPA). This enzyme normally breaks down a substance called N-acetylaspartate (NAA) in the brain. When ASPA is missing or not working properly, NAA builds up and damages the brain's white matter. Ciracigene golparvovec is designed to deliver a functional copy of the ASPA gene to brain cells using a modified virus called adeno-associated virus (AAV). The goal is to enable the cells to produce the missing enzyme and reduce the harmful accumulation of NAA.
Which conditions is it studied for?
- Canavan disease — a rare, inherited disorder of the brain's white matter that typically appears in infancy and leads to progressive neurological decline. Ciracigene golparvovec is being studied specifically in children with this condition.
How is ciracigene golparvovec given?
In the registered clinical trial, ciracigene golparvovec is administered as a single dose by intracerebroventricular (ICV) injection. This means the therapy is injected directly into the fluid-filled spaces of the brain, known as the ventricles, so that it can reach the brain tissue.
What do the clinical trials show?
One clinical trial of ciracigene golparvovec is registered in the EU Clinical Trials Information System. The trial, identified as NCT04833907, is a Phase 1/2 study. Phase 1/2 means that the trial is investigating both the safety of the therapy in humans and early signs of how well it works.
The trial is currently authorised, which means it has received approval from the relevant regulatory authorities and can begin or continue enrolling participants. It plans to include 32 children with Canavan disease.
What does the trial measure?
The main outcome being measured is the change in NAA concentration in the cerebrospinal fluid (CSF) from the start of the study to six months after treatment. NAA is the substance that accumulates in Canavan disease, so a reduction in its level could indicate that the therapy is working. The measurement is done using a biochemical assay, a laboratory test that quantifies the amount of NAA in a sample of spinal fluid.
Who can take part in this trial?
The trial enrols children diagnosed with Canavan disease. The registry record does not list further details about specific age ranges or other inclusion criteria beyond the diagnosis.
What side effects are known?
The clinical trial registry record does not include information about side effects observed with ciracigene golparvovec. As this is an investigational therapy in early-stage research, the safety profile is still being established through the ongoing trial.