UnknownPhase 2

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

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

Lead sponsor

FORUM Pharmaceuticals Inc

Asset

FRM-0334

Listed sites

Recruiting sites

Enrollment

estimated

Study population

Frontotemporal dementia

Key I/E criterion

•GRN mutation required

Primary endpoints

•Evaluate the safety and tolerability of FRM-0334•Assess the pharmacodynamic (PD) effects of FRM-0334 on the change from baseline

Footprint

Where this trial recruits

Site locations as reported to ClinicalTrials.gov. Site count is not enrollment count; per-site enrollment is not available from source.

Identifiers

Registered as

Eudract number2014-001489-85

Org study IDFRM-0334-002

NCT IDNCT02149160

Timeline

Milestones

Study first posted2014-05-29estimated

Study start2014-10 (month precision)

Last update posted2016-03-23estimated

Primary completion2016-08estimated (month precision)

Assets

Drug assets

FRM-0334

Study populations

Who this study enrolls

Frontotemporal dementia

Eligibility

Who can enroll

Minimum age21 Years

Maximum age75 Years

SexAll

Healthy volunteersNot accepted

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

Endpoints (5)

What's being measured

Protocol endpoints and posted registry outcome measures, grouped into outcome categories. Composite endpoints show their component event types. Standard codes (LOINC, SNOMED CT) are shown where available.

Coverage by outcome category

Fluid / digital biomarkers

Safety / tolerability / PK

Other (unclassified)

Fluid / digital biomarkers

2 endpoints

Secondary/protocol endpoint

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

change from baseline, improvement

Secondary/protocol endpoint

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

concentration, descriptive

Safety / tolerability / PK

2 endpoints

Primary/protocol endpoint

Evaluate the safety and tolerability of FRM-0334

Time frame:Baseline to Day 28 or Early Termination

threshold achievement, event

Secondary/protocol endpoint

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

concentration, descriptive

Other (unclassified)

1 endpoint

Primary/protocol endpoint/low confidence

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

change from baseline, improvement

Publications (1)

Bibliography

Records linked to this trial through ClinicalTrials.gov references, PubMed NCT search, and curated study seeds. 'Canonical' marks design/result papers; others are registry references or candidates.

Registry references + supporting bibliography

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 Rand…
PMID34559230via DERIVED

Provenance

Sources

Trial identity, design, statusClinicalTrials.gov API v2

Snapshot dateJuly 21, 2026

Endpoint classificationDelfa ADRD endpoint taxonomy

Results tableno registry results posted yet

Trial facts come from public ClinicalTrials.gov records. Endpoint categories are Delfa's classification of those records, not a ClinicalTrials.gov field. All figures reflect the July 21, 2026 snapshot.