Active not recruitingPHASE1, PHASE2INTERVENTIONAL

A Study of PBFT02 in Participants With FTD and Mutations in the Granulin Precursor (GRN) or C9ORF72 Genes

This research is looking at a new treatment called PBFT02 for a serious condition called Frontotemporal Dementia (FTD). FTD affects behaviour and language and is often caused by faulty genes. PBFT02 is a 'gene therapy' designed to deliver a working copy of a specific gene (GRN) into the brain to try and correct the problem. This study will carefully check if PBFT02 is safe for people with FTD who have mutations in their GRN or C9ORF72 genes. Researchers will also be looking to see if it's well-tolerated and if it shows any signs of being effective in slowing down or improving symptoms. The study involves a single treatment and will monitor participants for a total of five years, with a main two-year period followed by a three-year safety check.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

Passage Bio, Inc.

Enrolment target

Start

14 Sep 2021

Estimated completion

01 Aug 2031

Frontotemporal Dementia FTD FTD-GRN Dementia Frontotemporal C9orf72

What is this study about?

This study is investigating a new type of treatment called gene therapy, named PBFT02, for a condition called Frontotemporal Dementia (FTD). FTD is a form of dementia that mainly affects parts of the brain that control behaviour, personality, and language, often starting at a younger age than Alzheimer's disease. We know that in some people, FTD is caused by specific faulty genes, particularly those known as GRN or C9ORF72.

PBFT02 is designed to address the root cause of FTD in people with these genetic mutations. It works by delivering a healthy version of the GRN gene directly into the brain. The hope is that by providing this working gene, the brain can start producing a protein it needs, which is lacking in people with these specific genetic forms of FTD. This study aims to find out if this treatment is safe for people to receive, how well their body tolerates it, and if it shows any promise in helping with the symptoms of their FTD.

This is a careful and structured study that will involve people aged between 35 and 75 who have FTD caused by either the GRN or C9ORF72 gene mutations. Participants will receive a single treatment with PBFT02. The study will then closely monitor them over five years – two years of main study and a further three years to keep a close eye on their long-term health and any effects of the treatment. This phased approach helps researchers understand the treatment's impact over time.

Key takeaways

Tests a new gene therapy (PBFT02) for Frontotemporal Dementia (FTD).
Only for people with FTD caused by GRN or C9ORF72 gene mutations.
Aims to check safety, how well it's tolerated, and if it helps symptoms.
Involves a single treatment given directly to the brain area.
Study lasts for 5 years, with close monitoring throughout.
You must have a reliable helper (carer/informant) who sees you regularly.

Who may be eligible?

To be considered for this study, you would need to have a confirmed diagnosis of Frontotemporal Dementia (FTD) and genetic testing proving that your FTD is caused by a specific problem with either your GRN or C9ORF72 genes. You should be between 35 and 75 years old and have someone reliable, like a family member or friend, who sees or speaks with you at least once a week and can help provide information about your health.

There are also some important things that would mean you couldn't join the study. For example, if you've already had a gene therapy treatment before, or if your specific gene mutation isn't thought to be the cause of FTD. Certain other health conditions, heart problems, severe kidney or liver issues, or problems with your blood tests might also prevent you from taking part.

It's important that you live independently or with support in the community rather than in a nursing home. The doctors will also check your overall health to make sure you're well enough to participate in the study.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Do I have a confirmed diagnosis of Frontotemporal Dementia?
Have genetic tests shown I have a GRN or C9ORF72 gene mutation?
Am I between 35 and 75 years old?
Do I have a reliable family member or friend who sees me weekly?
Do I live in my own home or in assisted living, not a nursing home?
Have I ever had gene therapy before? (If yes, you likely can't join).

Answer every question to see your result.

What does participation involve?

If you join this study, you will receive a single dose of the PBFT02 treatment. This treatment is given as an injection into a specific area near the brain in your back. The study will last for a total of five years. For the first two years, there will be more frequent visits to the study centre, involving various tests and assessments to closely monitor your safety, how well you tolerate the treatment, and any changes in your FTD symptoms. After these first two years, there will be a further three-year period where you'll still be monitored, but probably with fewer visits, to check on your long-term health and the lasting effects of the treatment. You'll need to have a trusted family member or carer who can attend appointments with you and provide information about your health and daily life.

Potential risks and benefits

Participating in a clinical trial offers potential benefits, such as gaining access to a new treatment before it's widely available, contributing to medical knowledge, and receiving close medical monitoring. However, there are also potential risks, including side effects from the treatment, the possibility that the treatment may not be effective, or the need for frequent hospital visits and medical procedures. You will be fully informed of all known and potential risks before deciding to join. It is very important to remember that participating is entirely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (10)

Michigan Alzheimer's Disease Center
Verified postcode
Ann Arbor, United States
University of Pennsylvania
Verified postcode
Philadelphia, United States
Vanderbilt University Medical Center
Verified postcode
Nashville, United States
University of Texas at Houston
Verified postcode
Houston, United States
Eastern Health-Box Hill Hospital
Verified postcode
Melbourne, Australia
Hospital das Clinicas da Universidade Federal de Minas Gerais (UFMG)
Verified postcode
Minas Gerais, Brazil
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo (HCFMUSP)
Verified postcode
São Paulo, Brazil
University of Toronto, Toronto Western Hospital
Verified postcode
Toronto, Canada
Montreal Neurological Institute-Hospital
Verified postcode
Montreal, Canada
Centro Hospitalar e Universitário de Coimbra
Verified postcode
Coimbra, Portugal

Common questions

What is Frontotemporal Dementia (FTD)?

FTD is a type of dementia that often affects parts of the brain controlling personality, behaviour, and language. It can start at a younger age than Alzheimer's disease.

What is a 'gene therapy' like PBFT02?

Gene therapy aims to treat diseases caused by faulty genes by introducing a healthy, working copy of that gene into the body. PBFT02 specifically tries to add a missing gene to the brain for people with FTD caused by certain genetic problems.

Why is this study looking at GRN or C9ORF72 genes?

Problems with the GRN or C9ORF72 genes are known causes of FTD in some people. This study is testing a treatment designed to specifically help people with FTD caused by these particular gene mutations.

How long will the study last if I join?

The study will last for a total of five years. This includes two years of more frequent monitoring and then another three years to follow up on your long-term health and the treatment's effects.

Will I receive the actual treatment or a placebo?

This particular study is 'single-arm,' meaning everyone who participates and qualifies will receive the PBFT02 treatment, rather than a placebo (a dummy treatment).