Study to Assess the Safety, Tolerability, and Pharmacodynamic (PD) Effects of FRM-0334 in Subjects With Prodromal to Moderate Frontotemporal Dementia With Granulin Mutation
- Registration Number
- NCT02149160
- Lead Sponsor
- FORUM Pharmaceuticals Inc
- Brief Summary
The purposes of this study are to investigate the safety, tolerability, and pharmacodynamics of FRM-0334 in subjects with prodromal to moderate frontotemporal dementia with granulin mutation.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 30
- Male or female ages aged ≥21 and ≤75 years
- Genotyped positive for a FTD-GRN mutation, and aware of it
- Prodromal to moderate FTD-GRN
- Resides in a stable living situation, living at home, senior residential setting, or an institutional setting without the need for continuous (ie, 24-hour) nursing care
- Proficiency (oral and written) in the language in which study-related documents, including the ICF and standardized tests, will be administered
- Able to swallow capsules
- Be in good general health, willing and able to comply with the protocol requirements, and expected to complete the study as designed (in the judgment of the investigator)
- Clinically significant abnormalities on physical examination, medical history, ECG, vital signs, laboratory values, or unstable medical or psychiatric illness
- Females who are pregnant, breastfeeding, or planning to become pregnant during the study
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Comparator; Arm 3 Placebo Placebo, Capsule, Once Daily, Day 1 through Day 28 FRM-0334; Arm 1 FRM-0334 low dose, Capsule, Once Daily, Day 1 through Day 28 FRM-0334; Arm 2 FRM-0334 high dose, Capsule, Once Daily, Day 1 through Day 28
- Primary Outcome Measures
Name Time Method Evaluate the safety and tolerability of FRM-0334 Baseline to Day 28 or Early Termination * Number and percentage of subjects with AEs
* Number and percentage of subjects with SAEs
* Number and percentage of subjects who discontinue due to AEs
* Number and percentage of subject deathsAssess the pharmacodynamic (PD) effects of FRM-0334 on the change from baseline in plasma concentrations of progranulin (PGRN) after 28 days Baseline to Day 28 or Early Termination
- Secondary Outcome Measures
Name Time Method Assess the pharmacodynamic effects of FRM-0334 on the change from baseline in cerebrospinal fluid (CSF) concentrations of PGRN after 28 days Baseline and Day 28 Change in CSF progranulin concentration from baseline
Characterize the plasma concentrations of FRM-0334 and metabolites following once daily dosing for 28 days Day 1 to Day 28 or Early Termination Cmax, Ctrough, tmax, t1/2, λz, AUC(0-τ), CL/F, Cav in plasma
Characterize the CSF concentrations of FRM-0334 and metabolites following once daily dosing for 28 days Day 1 to Day 28 or Early Termination Cmax, Ctrough, tmax, t1/2, AUC0-τ, CL/F, Cav in CSF
Trial Locations
- Locations (13)
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
IRCCS - Centro S. Giovanni di Dio FateBeneFratelli
🇮🇹Brescia, Italy
Neurological Clinic, University of Brescia, AO Civil Hospital of Brescia
🇮🇹Brescia, Italy
Fondazione Universita Gabriele D'Annunzio di Chieti
🇮🇹Chieti Scalo, Italy
The National Hospital for Neurology and Neuroscience
🇬🇧London, United Kingdom
Compass Research, LLC
🇺🇸Orlando, Florida, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
University Hospitals Leuven
🇧🇪Leuven, Belgium
CHU Bordeaux Hospital Pellegrin
🇫🇷Bordeaux Cedex, France
CHU Rouen, Charles Nicolle Hospital
🇫🇷Rouen, France
Erasmus Medical Center
🇳🇱Rotterdam, Zuid-Holland, Netherlands
UCSF Memory and Aging Center
🇺🇸San Francisco, California, United States
Perelman School of Medicine, University of Pennsylvania
🇺🇸Philadelphia, Pennsylvania, United States