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)
Enrolling By Invitation
35-75 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
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Age: Between 35 Years - 75 Years
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Gender: All
Updated on
01 Aug 2024.
Study ID: 848900
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