What is Passage Bio evaluating in this trial?

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

In Krabbe disease, 2 mutated, or non-working copies of a gene called GALC result in little to no production of something called a galactosylceramidase (GALC) enzyme. Passage Bio is studying a treatment, called gene therapy, that is intended to give the body a working copy of the GALC gene.

The goal of this trial is to see if the gene therapy can lead to GALC production and, by doing so, prevent damage to nerves in the brain, spinal cord, and throughout the body for children with Krabbe disease.

This trial is for children aged 1 to 9 months with Krabbe disease

This study is designed to provide information about the safety, effectiveness, and appropriate dose of treatment

The gene therapy being studied is called PBKR03

You may hear this trial referred to as GALax-C or NCT04771416

In GALax-C, researchers are collecting data about:

  • SAFETY Is the treatment…

    • Safe?
    • Causing side effects? If so, what are they?
  • EFFECTIVENESS Does the treatment…

    • Help brain function?
    • Help motor and sensory nerves continue working or work better?
    • Help the body produce the GALC enzyme which is missing in people with Krabbe disease?
  • DOSING Do different doses…

    • Change the effectiveness of the potential treatment?
    • Change the type and intensity of side effects?

GALax-C study design

A study design is a blueprint of a study that outlines the steps for enrolled participants, including how many children will be included and their age range.

GALax-C study design for gene therapy PBKR03

  • CONFIRMATORY GROUP (6 patients)

    AGE: 4 months to less than 9 months old

    DOSE: Selected

  • CONFIRMATORY GROUP (6 patients)

    AGE: 1 month to less than 4 months old

    DOSE: Selected

  • GROUP 2 (3 patients)

    AGE: 4 months to less than 9 months old

    DOSE: High

  • GROUP 4 (3 patients)

    AGE: 1 month to less than 4 months old

    DOSE: High

  • GROUP 1 (4 patients)

    AGE: 4 months to less than 9 months old

    DOSE: Low

    The groups are called cohorts

  • GROUP 3 (3 patients)

    AGE: 1 month to less than 4 months old

    DOSE: Low

60 days after the last patient in each group receives the therapy, an independent committee will review all available data to determine safety before authorizing enrollment for the next cohort.
Part 1: Optimal dose selection phase
Part 2: Expansion and confirmatory dose phase
  • GROUP 1 (4 patients)

    AGE: 4 months to less than 9 months old

    DOSE: Low

    The groups are called cohorts

  • GROUP 3 (3 patients)

    AGE: 1 month to less than 4 months old

    DOSE: Low

  • GROUP 2 (3 patients)

    AGE: 4 months to less than 9 months old

    DOSE: High

  • GROUP 4 (3 patients)

    AGE: 1 month to less than 4 months old

    DOSE: High

  • CONFIRMATORY GROUP (6 patients)

    AGE: 4 months to less than 9 months old

    DOSE: Selected

  • CONFIRMATORY GROUP (6 patients)

    AGE: 1 month to less than 4 months old

    DOSE: Selected

60 days after the last patient in each group receives the therapy, an independent committee will review all available data to determine safety before authorizing enrollment for the next cohort.
Part 1: Optimal dose selection phase
Part 2: Expansion and confirmatory dose phase

GALax-C monitoring

Throughout the study, your child's progress will be monitored through different types of tests to evaluate the safety and efficacy of the treatment.

If you have any questions about this trial, our team at [email protected] can help connect you with answers.

Common clinical trial terms

Adverse events (AEs): Side effects that are documented during the trial. Most frequently when doctors use this phrase, doctors are referring to side effects from the treatment itself. However, not all side effects recorded during the trial are the result of the treatment. Some can be related to the disease or other factors.

Cohort: A group of patients who receive the same treatment.

Efficacy: The effectiveness of a treatment or the ability of it to produce a beneficial result.

Eligibility: Guidelines for who can or cannot participate in the study such as age, medical history, and current health status. Your doctor will help determine if your child is eligible for the trial.

Outcomes (primary and secondary): Results that are monitored to determine the impact of treatment on a patient population. The primary outcome is the most important result in answering the research question. This question is typically, "Is the treatment safe? What is the best dose? Does it work?" Secondary outcomes are additional results of interest and are studied to help better understand the efficacy and safety of the potential treatment.

Principal Investigators (PIs): An individual, often a doctor, who leads a trial at a trial location.

Tolerability: How much side effects can be experienced by or impacts study participants.