Ongoing, recruitingPhase I and Phase II (Integrated)- First administration to humansInterventional

Phase 1b Study to Assess the Safety, Tolerability and Pharmacodynamics of PBFT02 in Adult Participants with Frontotemporal Dementia (FTD) and GRN or C9orf72 Mutations

This research study is looking at a new medicine called PBFT02 for adults who have Frontotemporal Dementia (FTD). FTD is a form of dementia that affects the parts of the brain controlling personality, behaviour, and language. This particular study is for people with FTD caused by specific changes in their genes, either GRN or C9orf72. It's an early-stage study, meaning it's one of the first times this medicine is being tested in humans. The main goals are to check if the medicine is safe to use, how well people can cope with it (tolerability), and how it starts to work in the body. By understanding these things early on, researchers hope to develop new ways to help people living with FTD.

At a glance

Status

Ongoing, recruiting

Phase

Phase I and Phase II (Integrated)- First administration to humans

Sponsor

Passage Bio Inc.

Enrolment target

Start

11 Oct 2023

FRONTOTEMPORAL DEMENTIA (FTD)

What is this study about?

This study is about a new medicine called PBFT02, which is being tested for Frontotemporal Dementia (FTD). FTD is a group of brain disorders that slowly get worse over time, mainly affecting areas of the brain involved in personality, behaviour, and language. Unlike some other dementias, FTD often starts at a younger age. This study is specifically focused on people with FTD who have particular genetic changes, either in a gene called GRN or another one called C9orf72.

This is a 'Phase 1b' study, which means it's an early step in testing a new medicine. Think of it like taking a new car design out for its first test drive – you want to make sure it handles well and that everything works safely before you start making lots of them. Similarly, in this study, doctors want to find out if PBFT02 is safe for people to take, what side effects it might cause, and how much of it the body can comfortably handle. They also want to see if the medicine starts to have any expected effects on the body.

The information gathered from this study is really important. It helps researchers understand if PBFT02 could be a promising treatment for FTD in the future. If these early tests go well, the medicine might move on to larger studies to see if it can slow down or stop the progression of FTD symptoms. These steps are crucial for developing new ways to help people living with this challenging condition.

Key takeaways

This study tests a new medicine (PBFT02) for Frontotemporal Dementia (FTD).
It's for adults with FTD caused by specific gene changes (GRN or C9orf72).
The main goals are to check safety, tolerability, and how the medicine affects the body.
This is an early-stage study, aiming to gather key information for future treatments.
Participation involves taking the medicine and attending regular check-ups.

Who may be eligible?

To join this study, participants must be adults, aged 18 years or older, with no upper age limit. The study is open to both men and women.

Crucially, you must have a diagnosis of Frontotemporal Dementia (FTD) that is linked to specific genetic changes. This means you would need to have had tests that show you have either the GRN gene mutation or the C9orf72 gene mutation.

The research team will have a full list of things they look for, including your overall health, other medicines you might be taking, and your FTD symptoms. They will review all this information carefully to make sure the study is right for you and that you meet all the necessary requirements.

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.

Are you an adult (18 years or older)?
Do you have a diagnosis of Frontotemporal Dementia (FTD)?
Have you been told your FTD is linked to GRN or C9orf72 gene changes?
Are you able to attend regular study visits?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you would receive the new study medicine, PBFT02. This being an early-stage study, you would attend a number of study visits at a hospital or clinic. During these visits, doctors and nurses would perform various checks. This could include physical exams, blood and urine tests, and possibly brain scans or other assessments to see how your body is reacting to the medicine and if it's having any effects. They would carefully monitor you for any side effects. The total duration of your participation, including follow-up after receiving the medicine, would be explained to you in detail by the study team.

Potential risks and benefits

Taking part in any clinical study comes with potential benefits and risks. A potential benefit of this study is contributing to our understanding of FTD and helping to develop new treatments, which could eventually help you or others. There's also a chance you might experience some improvement in your condition, although this is not guaranteed, especially in early-stage studies. However, there are risks, as the study medicine PBFT02 is new and its full effects, including potential side effects, are not yet completely known. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (2)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Portugal
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Unverified
Spain

Common questions

What is Frontotemporal Dementia (FTD)?

FTD is a type of dementia that mainly affects parts of the brain controlling personality, behaviour, and language, often starting at a younger age than other dementias.

What does 'Phase 1b' mean?

It means this is an early study to check if a new medicine is safe for people, how much they can tolerate, and how it starts to work in the body.

What are GRN and C9orf72 mutations?

These are specific changes in certain genes that can increase someone's risk of developing FTD. This study is for people with these specific genetic changes.

Will I definitely get the new medicine?

Yes, in this study, all participants who are eligible and join will receive the study medicine, PBFT02.

How long will I be in the study?

The full duration of your participation, including follow-up, will be clearly explained by the study team if you are found to be eligible.