Krabbe Disease GALax-C (PBKR03) Clinical Trial

Information for parents and caregivers

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

Passage Bio is studying a potential new therapy called PBKR03 for the treatment of Krabbe disease

PBKR03 is a novel gene therapy intended to help replace the gene that doesn't work in people with Krabbe disease, called the GALC gene

This clinical trial, called GALax-C, is studying if PBKR03 can help children with infantile Krabbe disease

First, infants between the age of 4 and 9 months will be included in the study. After a safety review, the potential treatment will be studied in infants between 1 and 9 months old

Contact Passage Bio to learn more:

We are here to help. We're happy to answer questions you have about Krabbe disease, the treatment we are studying, and the clinical trial.

Please fill out the form to the right to contactform below to contact Passage Bio. If you are interested in trial participation, please include the information listed below:

  • Date of birth of child (month/date/year)
  • Diagnosis of child
  • Language preference