Have you or someone you know tested positive for the Granulin Mutation?

If so, you may be eligible to participate in a clinical trial.

Trial Name: Study to Assess the Safety, Tolerability, and Pharmacodynamic (PD) Effects of FRM-0334 in Subjects With Prodromal to Moderate Frontotemporal Dementia With Granulin Mutation

What is it?

This is a clinical trial sponsored by the company Forum Pharmaceuticals.This study is being done to see if an investigational “study drug”, FRM-0334, is effective at increasing the progranulin (a type of protein in your brain) level in your cerebral spinal fluid (CSF).  The investigational study drug is a pill that you will take daily at home for the duration of the study.

What do I have to do to participate?

You will be asked to come to the University of Pennsylvania for a series of 9 visits over approximately 28 days. After two screening visits to determine eligibility, there are five in-person visits at the Hospital of the University of Pennsylvania and two phone contact visits that will occur one to four weeks apart. There is one overnight visit. Study visits and screening include: physical examination, vital signs, MRI or CT scan of the brain, PET scan of the brain, lumbar puncture, ECG, blood draw, urine sample, cognitive testing, and patient and caregiver questionnaires.

Who is eligible for this trial?

Individuals 21-75 years old with prodromal (no current FTD symptoms) to moderate Frontotemporal degeneration and who have tested positive for the Granulin mutation

For more information, please feel free to contact:

Courtney Igne at 215.349.5863

Email her at  cigne @mail.med.upenn.edu

Check out https://www.clinicaltrials.gov/ and search the identifier: NCT02149160 for more information

 

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