Common questions about this trial, participation, and GM1 gangliosidosis
Imagine-1 trial key
- Name: Imagine-1
- Treatment: PBGM01
- Goal: Study the effectiveness and safety of a new gene therapy, PBGM01, in children with GM1 gangliosidosis
- Ages eligible: 4 to 36 months
Clinical trials are research studies where doctors see if a potential treatment is safe and effective in people.
All of the prescription therapies available to you today have been studied in clinical trials before approval.
- Clinical trials study potential new treatments
- Clinical trials are voluntary
- Participation could help others with the disease by advancing research
- People with an illness or disease also take part to help others, but also to possibly receive the newest treatment and to have added (or extra) care and attention from the clinical trial staff
- Clinical trials offer hope for many people and a chance to help researchers find better treatments for others in the future
For the clinical trial Imagine-1, there are clinical trial sites around the world. Should you qualify for the trial, Passage Bio will work to assist you with travel to make sure you can participate in the trial. You do not need to live close to a clinical trial site in order to participate in the trial.
Participants and their families are not able to choose which cohort they participate in. This helps scientists avoid any potential bias or imbalance by making sure each group is treated similarly outside of the differences outlined in the trial design.
There are often 3 phases of clinical trials. Phase 1 tests safety and helps determine optimal dose. Phase 2 tests effectiveness and safety. Phase 3 also tests effectiveness and safety in a larger group of patients.
In rare diseases, such as GM1 gangliosidosis, where there are low numbers of patients and limited treatment options, researchers often combine phases to answer more questions in a smaller group of patients. This is why Imagine-1 is a Phase 1/2 trial.
GM1 gangliosidosis is a genetic disorder that occurs when a person has very low amounts of the vital enzyme β-galactosidase (also called β-gal or beta-galactosidase). This enzyme affects nerve cells (called neurons) in the brain and spinal cord, which are parts of the central nervous system (CNS). This results in a number of symptoms such as poor feeding, difficulty sitting up and crawling, difficulty breathing, poor muscle strength, and seizures.
GM1 gangliosidosis is most common and severe in infants less than a year old. It is also important to understand that anyone born with GM1 gangliosidosis has low amounts of β-gal, even if symptoms do not appear until later in childhood. GM1 gangliosidosis is estimated to affect 1 in 100,000 to 200,000 babies born worldwide.
GM1 gangliosidosis is a genetic, or hereditary, disease. This means it could be passed down to children. Each person receives 1 copy of every gene (a segment of DNA) from each parent, meaning they have 2 copies of every gene in their body.
GM1 gangliosidosis is an autosomal recessive disease, meaning only those with 2 mutated copies of the gene get the disease. This can happen when someone inherits 2 mutated copies, 1 from each parent. People with 1 mutated copy are called carriers.
Currently, there are no approved disease-modifying treatments available for GM1 gangliosidosis. A disease-modifying treatment is a treatment that addresses the underlying cause of a disease.
There is a difference between treating a disease and managing symptoms. In GM1 gangliosidosis, doctors manage symptoms through supportive care or palliative care. Supportive or palliative care focuses on relieving symptoms of a disease and giving the child the best quality of life.
Gene therapy is a type of treatment where doctors introduce a new, working gene to the body to replace a gene that isn’t working. The body reads the new gene and produces a protein to help correct a disease.