Overview

This trial is active, not recruiting.

Condition granulin mutation
Treatments frm-0334, placebo
Phase phase 2
Target HDAC
Sponsor FORUM Pharmaceuticals Inc
Start date October 2014
End date August 2016
Trial size 30 participants
Trial identifier NCT02149160, 2014-001489-85, FRM-0334-002

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety study
Intervention model parallel assignment
Masking double blind (subject, caregiver, investigator, outcomes assessor)
Primary purpose treatment
Arm
(Experimental)
low dose, Capsule, Once Daily, Day 1 through Day 28
frm-0334
(Experimental)
high dose, Capsule, Once Daily, Day 1 through Day 28
frm-0334
(Placebo Comparator)
Placebo, Capsule, Once Daily, Day 1 through Day 28
placebo

Primary Outcomes

Measure
Evaluate the safety and tolerability of FRM-0334
time frame: Baseline to Day 28 or Early Termination
Assess the pharmacodynamic (PD) effects of FRM-0334 on the change from baseline in plasma concentrations of progranulin (PGRN) after 28 days
time frame: Baseline to Day 28 or Early Termination

Secondary Outcomes

Measure
Assess the pharmacodynamic effects of FRM-0334 on the change from baseline in cerebrospinal fluid (CSF) concentrations of PGRN after 28 days
time frame: Baseline and Day 28
Characterize the plasma concentrations of FRM-0334 and metabolites following once daily dosing for 28 days
time frame: Day 1 to Day 28 or Early Termination
Characterize the CSF concentrations of FRM-0334 and metabolites following once daily dosing for 28 days
time frame: Day 1 to Day 28 or Early Termination

Eligibility Criteria

Male or female participants from 21 years up to 75 years old.

Inclusion Criteria: - 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) Exclusion Criteria: - 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

Additional Information

Official title A Randomized, Double-Blind, Placebo-Controlled, Dose-Escalating, Phase 2a Safety, Tolerability, and Pharmacodynamic Study of Two Doses of an Histone Deacetylase Inhibitor (FRM-0334) in Subjects With Prodromal to Moderate Frontotemporal Dementia With Granulin Mutation
Trial information was received from ClinicalTrials.gov and was last updated in March 2016.
Information provided to ClinicalTrials.gov by FORUM Pharmaceuticals Inc.