What is Passage Bio evaluating in this trial?
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: 1 to 24 months
In GM1 gangliosidosis, 2 mutated, or non-working copies of a gene called galactosidase beta 1 (GLB1) result in little to no production of something called beta galactosidase (β-gal) enzyme. Passage Bio is studying a treatment, called gene therapy, that is intended to give the body a working copy of the GLB1 gene.
The goal of this trial is to see if the gene therapy can lead to β-gal production and, by doing so, protect nerves and prevent nerve damage for children with GM1 gangliosidosis.
This trial is for children aged 1 to 24 months with GM1 gangliosidosis
This study is designed to provide information about the safety, effectiveness, and appropriate dose of treatment
The gene therapy being studied is called PBGM01
You may hear this trial referred to as Imagine-1 or NCT04713475
In Imagine-1, 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 β-gal enzyme which is missing in people with GM1 gangliosidosis?
-
DOSING Do different doses…
- Change the effectiveness of the potential treatment?
- Change the type and intensity of side effects?
Imagine-1 study design
A study design is a blueprint of a study that outlines the steps for enrolled participants.
Imagine-1 study design for gene therapy PBGM01
Imagine-1 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: What the possible side effects are and how much they impact the participants.
Passage Bio is a genetics medicine company focused on developing transforming therapies for central nervous system (CNS) disorders
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