Common questions about this trial, participation, and Krabbe disease
GALax-C trial key
- Name: GALax-C
- Treatment: PBKR03
- Goal: Study the effectiveness and safety of a new gene therapy, PBKR03, in infants with Krabbe disease
- Ages eligible: 1 to 9 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 GALax-C, 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 Krabbe disease, 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 GALax-C is a Phase 1/2 trial.
Krabbe disease is a rare disease with symptoms that most frequently begin soon after birth. Krabbe disease causes damage to the nerve cells and the nervous system. This process is called neurodegeneration. This is a result of your child’s body not being able to make something called galactosylceramidase (GALC) enzyme. GALC is important in protecting the body’s nerves. Neurodegeneration is progressive, meaning it worsens over time, and causes muscle weakness that affects the entire body.
Krabbe disease is a genetic, or hereditary, disease. This means it could be passed down to children. You receive 1 copy of every gene (a segment of DNA) from each parent, meaning you have 2 copies of every gene in your body.
Krabbe disease is an autosomal recessive disorder, which means that both parents had the abnormal gene and the affected child received both copies.
Currently, there is no cure for Krabbe disease and there are limited options for treatment and symptom management.
There are currently no approved disease-modifying treatments for Krabbe disease. A disease-modifying treatment is a treatment that addresses the underlying cause of a disease.
In the US, hematopoietic stem cell transplantation (HSCT) has been used in children with Krabbe disease. HSCT may be considered when diagnosed: within the first 30 days of life for a child with infantile onset, or before symptoms appear, or for those with mild symptoms.
There are risks associated with HSCT and infants who benefit from stem cell transplants may still experience disease progression such as difficulties with speech, walking, and other motor skills.
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.