What is Passage Bio evaluating in this trial?

upliFT-D trial key

  • Name: upliFT-D
  • Treatment: PBFT02
  • Goal: Study the effectiveness and safety of a new gene therapy, PBFT02, in people with FTD caused by a GRN mutation
  • Ages eligible: 35 to 75 years old

The GRN gene contains a code that tells cells how to produce the protein progranulin (PGRN), which plays an essential role in the brain. Changes in this gene, called mutations, can stop the body from producing PGRN and cause the type of FTD that Passage Bio is studying a potential treatment for: FTD-GRN. Passage Bio is studying a gene therapy that is intended to give the body a working copy of the GRN gene.

The goal of this trial is to first study the safety of the gene therapy. The trial will also study if this gene therapy can lead to increased PGRN production and, by doing so, prevent the damage to the brain caused by FTD-GRN.

If you don't know if you have a GRN genetic mutation, click here for more information.

This trial is for people with FTD-GRN who are between the ages of 35 and 75

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

The gene therapy being studied is called PBFT02

You may hear this trial referred to as upliFT-D or NCT04747431

In upliFT-D, researchers are collecting data about:

  • SAFETY Is the treatment…

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

    • Help improve cognitive functions?
    • Help improve behavioral and language symptoms?
  • DOSING Do different doses…

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

upliFT-D study design

A study design is a blueprint of a study that outlines the steps for enrolled participants, including how many patients will be included and the dose that they will be given.

upliFT-D study design for gene therapy PBFT02

  • GROUP 3* (3 patients)

    DOSE: Optional

    Two dose levels of PBFT02 will be studied in patients with FTD-GRN. An optional third dose level may be studied based on results from the first 2 groups.

  • GROUP 2 (3 patients)

    DOSE: High

  • GROUP 1 (3 patients)

    DOSE: Low

    The groups are called cohorts

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.
  • GROUP 1 (3 patients)

    DOSE: Low

    The groups are called cohorts

  • GROUP 2 (3 patients)

    DOSE: High

  • GROUP 3* (3 patients)

    DOSE: Optional

    Two dose levels of PBFT02 will be studied in patients with FTD-GRN. An optional third dose level may be studied based on results from the first 2 groups.

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.

upliFT-D monitoring

Throughout the study, 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 you or your loved one 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.