Ignite Creation Date:
2025-12-25 @ 2:06 AM
Ignite Modification Date:
2026-02-11 @ 6:31 PM
Study NCT ID:
NCT02149160
Status:
UNKNOWN
Last Update Posted:
2016-03-23
First Post:
2014-05-20
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study to Assess the Safety, Tolerability, and Pharmacodynamic (PD) Effects of FRM-0334 in Subjects With Prodromal to Moderate Frontotemporal Dementia With Granulin Mutation
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D057180', 'term': 'Frontotemporal Dementia'}], 'ancestors': [{'id': 'D057174', 'term': 'Frontotemporal Lobar Degeneration'}, {'id': 'D003704', 'term': 'Dementia'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D057177', 'term': 'TDP-43 Proteinopathies'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D057165', 'term': 'Proteostasis Deficiencies'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D019965', 'term': 'Neurocognitive Disorders'}, {'id': 'D001523', 'term': 'Mental Disorders'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000721055', 'term': 'FRM-0334'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 30}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2014-10'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2015-05', 'lastUpdateSubmitDate': '2016-03-22', 'studyFirstSubmitDate': '2014-05-20', 'studyFirstSubmitQcDate': '2014-05-23', 'lastUpdatePostDateStruct': {'date': '2016-03-23', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2014-05-29', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2016-08', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Evaluate the safety and tolerability of FRM-0334', 'timeFrame': 'Baseline to Day 28 or Early Termination', 'description': '* Number and percentage of subjects with AEs\n* Number and percentage of subjects with SAEs\n* Number and percentage of subjects who discontinue due to AEs\n* Number and percentage of subject deaths'}, {'measure': 'Assess the pharmacodynamic (PD) effects of FRM-0334 on the change from baseline in plasma concentrations of progranulin (PGRN) after 28 days', 'timeFrame': 'Baseline to Day 28 or Early Termination'}], 'secondaryOutcomes': [{'measure': 'Assess the pharmacodynamic effects of FRM-0334 on the change from baseline in cerebrospinal fluid (CSF) concentrations of PGRN after 28 days', 'timeFrame': 'Baseline and Day 28', 'description': 'Change in CSF progranulin concentration from baseline'}, {'measure': 'Characterize the plasma concentrations of FRM-0334 and metabolites following once daily dosing for 28 days', 'timeFrame': 'Day 1 to Day 28 or Early Termination', 'description': 'Cmax, Ctrough, tmax, t1/2, λz, AUC(0-τ), CL/F, Cav in plasma'}, {'measure': 'Characterize the CSF concentrations of FRM-0334 and metabolites following once daily dosing for 28 days', 'timeFrame': 'Day 1 to Day 28 or Early Termination', 'description': 'Cmax, Ctrough, tmax, t1/2, AUC0-τ, CL/F, Cav in CSF'}]}, 'conditionsModule': {'keywords': ['Histone deacetylase inhibitor', 'Frontotemporal dementia', 'Granulin'], 'conditions': ['Granulin Mutation']}, 'referencesModule': {'references': [{'pmid': '34559230', 'type': 'DERIVED', 'citation': '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.'}]}, 'descriptionModule': {'briefSummary': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '75 Years', 'minimumAge': '21 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Male or female ages aged ≥21 and ≤75 years\n* Genotyped positive for a FTD-GRN mutation, and aware of it\n* Prodromal to moderate FTD-GRN\n* 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\n* Proficiency (oral and written) in the language in which study-related documents, including the ICF and standardized tests, will be administered\n* Able to swallow capsules\n* 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)\n\nExclusion Criteria:\n\n* Clinically significant abnormalities on physical examination, medical history, ECG, vital signs, laboratory values, or unstable medical or psychiatric illness\n* Females who are pregnant, breastfeeding, or planning to become pregnant during the study'}, 'identificationModule': {'nctId': 'NCT02149160', 'briefTitle': 'Study to Assess the Safety, Tolerability, and Pharmacodynamic (PD) Effects of FRM-0334 in Subjects With Prodromal to Moderate Frontotemporal Dementia With Granulin Mutation', 'organization': {'class': 'INDUSTRY', 'fullName': 'FORUM Pharmaceuticals Inc'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': 'FRM-0334-002'}, 'secondaryIdInfos': [{'id': '2014-001489-85', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'FRM-0334; Arm 1', 'description': 'low dose, Capsule, Once Daily, Day 1 through Day 28', 'interventionNames': ['Drug: FRM-0334']}, {'type': 'EXPERIMENTAL', 'label': 'FRM-0334; Arm 2', 'description': 'high dose, Capsule, Once Daily, Day 1 through Day 28', 'interventionNames': ['Drug: FRM-0334']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo Comparator; Arm 3', 'description': 'Placebo, Capsule, Once Daily, Day 1 through Day 28', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'FRM-0334', 'type': 'DRUG', 'armGroupLabels': ['FRM-0334; Arm 1', 'FRM-0334; Arm 2']}, {'name': 'Placebo', 'type': 'DRUG', 'armGroupLabels': ['Placebo Comparator; Arm 3']}]}, 'contactsLocationsModule': {'locations': [{'city': 'San Francisco', 'state': 'California', 'country': 'United States', 'facility': 'UCSF Memory and Aging Center', 'geoPoint': {'lat': 37.77493, 'lon': -122.41942}}, {'city': 'Orlando', 'state': 'Florida', 'country': 'United States', 'facility': 'Compass Research, LLC', 'geoPoint': {'lat': 28.53834, 'lon': -81.37924}}, {'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Massachusetts General Hospital', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'city': 'Rochester', 'state': 'Minnesota', 'country': 'United States', 'facility': 'Mayo Clinic', 'geoPoint': {'lat': 44.02163, 'lon': -92.4699}}, {'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Perelman School of Medicine, University of Pennsylvania', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'city': 'Leuven', 'country': 'Belgium', 'facility': 'University Hospitals Leuven', 'geoPoint': {'lat': 50.87959, 'lon': 4.70093}}, {'city': 'Bordeaux', 'country': 'France', 'facility': 'CHU Bordeaux Hospital Pellegrin', 'geoPoint': {'lat': 44.84124, 'lon': -0.58046}}, {'city': 'Rouen', 'country': 'France', 'facility': 'CHU Rouen, Charles Nicolle Hospital', 'geoPoint': {'lat': 49.44313, 'lon': 1.09932}}, {'city': 'Brescia', 'country': 'Italy', 'facility': 'IRCCS - Centro S. Giovanni di Dio FateBeneFratelli', 'geoPoint': {'lat': 45.53558, 'lon': 10.21472}}, {'city': 'Brescia', 'country': 'Italy', 'facility': 'Neurological Clinic, University of Brescia, AO Civil Hospital of Brescia', 'geoPoint': {'lat': 45.53558, 'lon': 10.21472}}, {'city': 'Chieti Scalo', 'country': 'Italy', 'facility': "Fondazione Universita Gabriele D'Annunzio di Chieti"}, {'city': 'Rotterdam', 'state': 'South Holland', 'country': 'Netherlands', 'facility': 'Erasmus Medical Center', 'geoPoint': {'lat': 51.9225, 'lon': 4.47917}}, {'city': 'London', 'country': 'United Kingdom', 'facility': 'The National Hospital for Neurology and Neuroscience', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'FORUM Pharmaceuticals Inc', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}