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

NCT ID: NCT02149160

Last Updated: 2016-03-23

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE2

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-10-31

Brief Summary

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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

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Conditions

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Granulin Mutation

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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FRM-0334; Arm 1

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

Group Type EXPERIMENTAL

FRM-0334

Intervention Type DRUG

FRM-0334; Arm 2

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

Group Type EXPERIMENTAL

FRM-0334

Intervention Type DRUG

Placebo Comparator; Arm 3

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

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Interventions

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FRM-0334

Intervention Type DRUG

Placebo

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

21 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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FORUM Pharmaceuticals Inc

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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UCSF Memory and Aging Center

San Francisco, California, United States

Site Status

Compass Research, LLC

Orlando, Florida, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Mayo Clinic

Rochester, Minnesota, United States

Site Status

Perelman School of Medicine, University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

University Hospitals Leuven

Leuven, , Belgium

Site Status

CHU Bordeaux Hospital Pellegrin

Bordeaux, , France

Site Status

CHU Rouen, Charles Nicolle Hospital

Rouen, , France

Site Status

IRCCS - Centro S. Giovanni di Dio FateBeneFratelli

Brescia, , Italy

Site Status

Neurological Clinic, University of Brescia, AO Civil Hospital of Brescia

Brescia, , Italy

Site Status

Fondazione Universita Gabriele D'Annunzio di Chieti

Chieti Scalo, , Italy

Site Status

Erasmus Medical Center

Rotterdam, South Holland, Netherlands

Site Status

The National Hospital for Neurology and Neuroscience

London, , United Kingdom

Site Status

Countries

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United States Belgium France Italy Netherlands United Kingdom

References

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Ljubenkov PA, Edwards L, Iaccarino L, La Joie R, Rojas JC, Koestler M, Harris B, Boeve BF, Borroni B, van Swieten JC, Grossman M, Pasquier F, Frisoni GB, Mummery CJ, Vandenberghe R, Le Ber I, Hannequin D, McGinnis SM, Auriacombe S, Onofrj M, Goodman IJ, Riordan HJ, Wisniewski G, Hesterman J, Marek K, Haynes BA, Patzke H, Koenig G, Hilt D, Moebius H, Boxer AL. Effect of the Histone Deacetylase Inhibitor FRM-0334 on Progranulin Levels in Patients With Progranulin Gene Haploinsufficiency: A Randomized Clinical Trial. JAMA Netw Open. 2021 Sep 1;4(9):e2125584. doi: 10.1001/jamanetworkopen.2021.25584.

Reference Type DERIVED
PMID: 34559230 (View on PubMed)

Other Identifiers

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2014-001489-85

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

FRM-0334-002

Identifier Type: -

Identifier Source: org_study_id

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