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

FORUM Pharmaceuticals

Status and phase: Unknown; Phase 2

Conditions: Granulin Mutation

Treatments: Drug: Placebo; Drug: FRM-0334

Study type: Interventional

Funder types: Industry

Identifiers

NCT02149160

2014-001489-85 (EudraCT Number)

FRM-0334-002

Details and patient eligibility

About

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.

Enrollment: 30 estimated patients

Sex: All

Ages: 21 to 75 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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30 participants in 3 patient groups, including a placebo group

FRM-0334; Arm 1

Experimental group

Description:

low dose, Capsule, Once Daily, Day 1 through Day 28

Treatment:

Drug: FRM-0334

FRM-0334; Arm 2

Experimental group

Description:

high dose, Capsule, Once Daily, Day 1 through Day 28

Treatment:

Drug: FRM-0334

Placebo Comparator; Arm 3

Placebo Comparator group

Description:

Placebo, Capsule, Once Daily, Day 1 through Day 28

Treatment:

Drug: Placebo