PBFT02-001- Phase 1B Study to Assess a Single | Clinical Research Studies Listing

PBFT02-001- Phase 1B Study to Assess a Single Dose of PBFT02 Delivered into Adult Subjects with Fronto-Temporal Dementia (FTD) and Mutations in the Progranulin Gene (GRN)

Recruiting

35 years - 75 years

All

Phase 1

4 participants needed

1 Location

Brief description of study

This research is being done to study a new way to treat FTD in patients who have the GRN mutation. This process is called “gene therapy”. Gene therapy is an experimental technique that aims to replace the gene that is not working properly with a version of the gene that is normal.

The main purpose of this study is to find a safe dose of a specific gene therapy called PBFT02 in adults who have been diagnosed with FTD-GRN. The study will also try to determine how PBFT02 might affect your disease course over time.

Participants in the study will be asked to complete the following research procedures: Answer questionnaires, Blood, urine, saliva, and tissue collection, MRI scan, Lumbar puncture, nerve conduction test, Optical Coherence tomography (OCT), general anesthesia, and study drug injection.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

Conditions: dementia,gene therapy
Age: 35 years - 75 years
Gender: All

Updated on 06 Feb 2026. Study ID: 21-0473

Find a site

Your Information has been sent successfully.

We have submitted the contact information you provided to the research team at {{SITE_NAME}}. A copy of the message has been sent to your email for your records.

Would you like to be notified about other trials? Sign up for Patient Notification Services.

Send a message

Enter your contact details to connect with study team

View email

Phone

View phone

First name*

Last name*

Email*

Phone number*

Preferred way of contact

Please provide your preferred way of contact

Other language

By Submitting your information, you agree that your details may be shared with the selected study site and handled in accordance with this Website's Privacy Policy (see link in footer). You may be contacted by email, text message, or phone regarding this study and other research opportunities that may be of interested to you. You can unsubscribe from these communications at any time. Message and data rates may apply.

Interested in the study

Select a study center that’s convenient for you, and get in touch with the study team.

Contact a study center

PBFT02-001- Phase 1B Study to Assess a Single Dose of PBFT02 Delivered into Adult Subjects with Fronto-Temporal Dementia (FTD) and Mutations in the Progranulin Gene (GRN)

Brief description of study

Eligibility of study

Find a site

Interested in the study