Safety and Efficacy Study Evaluating TRx0237 in Subjects With Behavioral Variant Frontotemporal Dementia (bvFTD)

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2014 by TauRx Therapeutics Ltd
Information provided by (Responsible Party):
TauRx Therapeutics Ltd Identifier:
First received: June 20, 2012
Last updated: August 4, 2014
Last verified: August 2014

The purpose of this study is to demonstrate the safety and efficacy of TRx0237 in the treatment of patients with behavioral variant frontotemporal dementia (bvFTD).

Condition Intervention Phase
Behavioral Variant Frontotemporal Dementia (bvFTD)
Drug: TRx0237
Drug: Placebo
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Double-Blind, Placebo-Controlled, Randomized, Parallel Group, 12-Month Safety and Efficacy Trial of TRx0237 in Subjects With Behavioral Variant Frontotemporal Dementia (bvFTD)

Resource links provided by NLM:

Further study details as provided by TauRx Therapeutics Ltd:

Primary Outcome Measures:
  • Change from Baseline on Modified Alzheimer's Disease Cooperative Study - Clinical Global Impression of Change (modified ADCS-CGIC) [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
  • Change from Baseline on Addenbrooke's Cognitive Examination - Revised (ACE-R) [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change from Baseline on Unified Parkinson's Disease Rating Scale (UPDRS Parts II and III) [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
  • Change from Baseline on Frontotemporal Dementia Rating Scale (FRS) [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
  • Change from Baseline on Functional Activities Questionnaire (FAQ) [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
  • Number of study participants who tolerate oral doses of TRx0237 as determined by safety parameter changes [ Time Frame: 52 weeks ] [ Designated as safety issue: Yes ]
    Safety parameters included adverse events, vital signs, methemoglobin and oxygen saturation, physical and neurological examinations, laboratory tests (hematology, serum chemistry, and urinalysis), electrocardiograms, assessment of serotonin syndrome, brain magnetic resonance imaging (MRI) and potential for suicidal behavior and thoughts

Other Outcome Measures:
  • Early effect on modified ADCS-CGIC (change from Baseline) [ Time Frame: 8 weeks ] [ Designated as safety issue: No ]
  • Change from Baseline on whole brain volume (assessed by brain MRI) [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
  • Change from Baseline on Mini-Mental Status Examination (MMSE) [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
  • Change from Baseline on Addenbrooke's Cognitive Examination-III (ACE-III) [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
  • Determine the effect of TRx0237 in subjects with known genetic mutations associated with bvFTD [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]

Estimated Enrollment: 180
Study Start Date: August 2013
Estimated Study Completion Date: January 2016
Estimated Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: TRx0237 200 mg/day group Drug: TRx0237
TRx0237 100 mg tablet will be administered twice daily.
Placebo Comparator: Placebo Drug: Placebo
Placebo tablets will be administered twice daily. The placebo tablets include 4 mg of TRx0237 as a urinary and fecal colorant to maintain blinding; hence, the placebo group will receive a total of 8 mg/day of TRx0237.


Ages Eligible for Study:   up to 79 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Diagnosis of probable bvFTD
  • Centrally rated frontotemporal atrophy score of 2 or greater on brain MRI
  • MMSE ≥20
  • Age <80 years
  • Modified Hachinski ischemic score of ≤ 4
  • Females, if of child-bearing potential, must practice true abstinence or be competent to use adequate contraception and agree to maintain this throughout the study
  • Subject, and/or, in the case of reduced decision-making capacity, legally acceptable representative(s) consistent with national law is/are able to read, understand, and provide written informed consent
  • Has one (or more) identified adult caregiver who is willing to provide written informed consent for his/her own participation; is able to read, understand, and speak the designated language at the study site; either lives with the subject or sees the subject for ≥2 hours/day ≥3 days/week; agrees to accompany the subject to each study visit; and is able to verify daily compliance with study drug
  • If currently taking an acetylcholinesterase inhibitor and/or memantine, the subject must have been taking such medication(s) for ≥3 months. The dosage regimen must have remained stable for ≥6 weeks and it must be planned to remain stable throughout participation in the study.
  • Able to comply with the study procedures

Exclusion Criteria:

  • Significant central nervous system (CNS) disorder other than bvFTD
  • Significant focal or vascular intracranial pathology seen on brain MRI scan
  • Biomarker evidence of underlying Alzheimer's disease pathology
  • Expressive language deficits
  • Meets research criteria for Amyotrophic Lateral Sclerosis or motor neuron disease
  • Meets diagnostic criteria for probable bvFTD but has a proven mutation producing non-tau, non-TDP-43 pathology
  • Clinical evidence or history of stroke, transient ischemic attack, significant head injury or other unexplained or recurrent loss of consciousness ≥15 minutes
  • Epilepsy
  • Rapid eye movement sleep behavior disorder
  • Major depressive disorder, schizophrenia, or other psychotic disorders, bipolar disorder, substance (including alcohol) related disorders
  • Metal implants in the head (except dental), pacemaker, cochlear implants, or any other non-removable items that are contraindications to MRI; magnetic resonance compatible prosthetics, clips, stents, or any other device proven to be compatible will be allowed
  • Resides in hospital or moderate to high dependency continuous care facility
  • History of swallowing difficulties
  • Pregnant or breastfeeding
  • Glucose-6-phosphate dehydrogenase deficiency
  • History of significant hematological abnormality or current acute or chronic clinically significant abnormality
  • Abnormal serum chemistry laboratory value at Screening deemed to be clinically relevant by the investigator
  • Clinically significant cardiovascular disease or abnormal assessments
  • Preexisting or current signs or symptoms of respiratory failure
  • Concurrent acute or chronic clinically significant immunologic, hepatic, or endocrine disease (not adequately treated) and/or other unstable or major disease other than bvFTD
  • Diagnosis of cancer within the past 2 years prior to Baseline (other than basal cell or squamous cell skin cancer or Stage 1 prostate cancer) unless treatment has resulted in complete freedom from disease for at least 2 years
  • Prior intolerance or hypersensitivity to methylthioninium-containing drug, similar organic dyes, or any of the excipients
  • Treatment currently or within 90 days before Baseline with any of the following medications (unless otherwise noted):

    • Tacrine
    • Amphetamine or dexamphetamine
    • Clozapine, olanzapine (and there is no intent to initiate therapy during the course of the study)
    • Carbamazepine, primidone
    • Drugs for which there is a warning or precaution in the labeling about methemoglobinemia at approved doses
  • Current or prior participation in a clinical trial as follows:

    • Clinical trial of a product for cognition within 3 months (unless confirmed to have been randomized to placebo)
    • A clinical trial of a drug, biologic, device, or medical food in which the last dose/administration was received within 28 days prior to Baseline
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01626378

  Hide Study Locations
United States, California
David Geffen School of Medicine at UCLA, UCLA Neurological Services Recruiting
Los Angeles, California, United States, 90095
Contact: Mario Mendez, MD    310-794-2550   
The Shankle Clinic Recruiting
Newport Beach, California, United States, 92663
Contact: William Shankle, MD    949-764-8190   
Memory and Aging Centre Recruiting
San Francisco, California, United States, 94158
Contact: Adam Boxer, MD    415-476-0661   
United States, Florida
Meridien Research Recruiting
Brooksville, Florida, United States, 34601
Contact: Kelli Maw, MD    352-597-8839   
Mayo Clinic Recruiting
Jacksonville, Florida, United States, 32224
Contact: Neill Graff-Radford, MD    904-953-6523   
Compass Research, LLC Recruiting
Orlando, Florida, United States, 32806
Contact: Ira Goodman, MD    407-426-9299   
University of South Florida Recruiting
Tampa, Florida, United States, 33613
Contact: Jean Fils, MD    813-974-2832   
United States, Georgia
Department of Neurology, Emory University Recruiting
Atlanta, Georgia, United States, 30322
Contact: William Hu, MD    404-728-6443   
United States, Illinois
Alexian Brothers Neurosciences Institute Clinical Research Recruiting
Elk Grove Village, Illinois, United States, 60007
Contact: Concetta Forchetti, MD    847-593-8553   
United States, Indiana
Indiana University Department of Neurology Recruiting
Indianapolis, Indiana, United States, 46202
Contact: Brandy Matthews, MD    317-963-7404   
United States, Maryland
Johns Hopkins Hospital Recruiting
Baltimore, Maryland, United States, 21224
Contact: Chiadi Onyike, MD    410-550-9054   
United States, Massachusetts
Neurological Clinical Research Institute (NCRI) Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
Contact: Scott McGinnis, MD    617-525-9539   
United States, Minnesota
Mayo Clinic, Department of Neurology Recruiting
Rochester, Minnesota, United States, 55905
Contact: David Knopman, MD    507-293-5354   
United States, New Jersey
Memory Enhancement Center of America, Inc. Recruiting
Eatontown, New Jersey, United States, 07724
Contact: Joel Ross, MD    732-263-0101   
United States, New York
Neurological Associates of Albany, P. C. Recruiting
Albany, New York, United States, 12208
Contact: Richard Holub, MD    518-449-2662   
Integrative Clinical Trials LLC Recruiting
Brooklyn, New York, United States, 11229
Contact: Inna Yuryev-Golger, MD    718-444-7774   
United States, North Carolina
UNC Department of Neurology, Physicians Office Building Recruiting
Chapel Hill, North Carolina, United States, 27599
Contact: Daniel Kaufer, MD    919-966-8172   
United States, Ohio
University Hospitals Case Medical Center, Neurology Clinical Trials Unit Recruiting
Cleveland, Ohio, United States, 44106
Contact: Alan Lerner, MD    216-464-6412   
United States, Oklahoma
Oklahoma Clinical Research Center, Clinical Research Department Recruiting
Oklahoma City, Oklahoma, United States, 73112
Contact: Marvin Peyton, MD    405-753-4994   
United States, Pennsylvania
The Clinical Trial Center, LLC Recruiting
Jenkintown, Pennsylvania, United States, 19046
Contact: Marvin Kalafer, MD    215-884-1700   
Hospital of the University of Pennsylvania, Department of Neurology Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Murray Grossman, MD    215-662-3361   
United States, Utah
CRI Lifetree Recruiting
Salt Lake City, Utah, United States, 84106
Contact: Miroslav Backonja, MD    801-269-8200   
United States, Vermont
The Memory Clinic Recruiting
Bennington, Vermont, United States, 05201
Contact: Lisa Capatano-Friedman, MD    802-447-1409   
United States, Virginia
University of Virginia Recruiting
Charlottesville, Virginia, United States, 22903
Contact: Erin Foff, MD    434-243-5898   
Fundación para el Estudio y Tratamiento de Enfermedades Mentales (FETEM) Not yet recruiting
Buenos Aires, Argentina, C1425AHQ
Contact: Ricardo Corral, MD    54 1147727834   
CENydET Not yet recruiting
Buenos Aires, Argentina, C1058AAJ
Contact: Enrique Kuper, Dr    54 911 5820 3000   
Instituto de Neurociencias San Agustín Not yet recruiting
Buenos Aires, Argentina
Contact: Hector Fabian Lamaison, MD    54 221-4538870   
Instituto Privado Kremer Not yet recruiting
Cordoba, Argentina, X5004AOA
Contact: Leonardo Janus Kremer, MD    54 0351 4535153   
Instituto Medico Rodriguez Alfici Not yet recruiting
Mendoza, Argentina, M5501AAP
Contact: Alberto Rodriguez Alfici, MD    54 (26) 1424 2886   
Australia, New South Wales
Neuroscience Research Australia Recruiting
Randwick, New South Wales, Australia, 2031
Contact: Olivier Piguet, Dr    61 2 9399 1113   
Australia, Victoria
Box Hill Hospital Recruiting
Box Hill, Victoria, Australia, 3128
Contact: Amy Brodtman, Dr    61 3 9895 4974   
Australia, Western Australia
Neurodegenerative Disorders Research Pty Ltd Recruiting
West Perth, Western Australia, Australia, 6005
Contact: Peter Panegyres, Prof    61 8 6380 2255   
Canada, Alberta
Heritage Medical Research Clinic-University of Calgary Not yet recruiting
Calgary, Alberta, Canada, T2N 4Z6
Contact: David Patry, MD    403-220-8394   
Canada, British Columbia
University of British Columbia Hospital, Clinic for Alzheimer Disease and Related Disorders Recruiting
Vancouver, British Columbia, Canada, V6T 2B5
Contact: Ging-Yuek Robin Hsiung, MD    (604) 822-3610   
Vancouver Island Health Authority Recruiting
Victoria, British Columbia, Canada, V8R 1J8
Contact: Alex Henri-Bhargava, MD    250-370-8261   
Canada, Nova Scotia
True North Clinical Research Not yet recruiting
Halifax, Nova Scotia, Canada, B3S 1M7
Contact: Mark Johnston, MD    902-431-4433   
Canada, Ontario
Geriatric Clinical Trials Group, St. Joseph's Health Care, Parkwood Hospital Site Recruiting
London, Ontario, Canada, N6C 5J1
Contact: Elizabeth Finger, MD    (519) 646-6032   
University Health Network, Toronto Western Hospital, Memory Clinic Not yet recruiting
Toronto, Ontario, Canada, M5T 2S8
Contact: Maria Carmela Tartaglia, MD    416 603 5483   
Toronto Memory Program Recruiting
Toronto, Ontario, Canada, M3B 2S7
Contact: Sharon Cohen, MD    (416) 386-9761   
Canada, Quebec
McGill Centre for Studies in Aging, Alzheimer Disease Research Unit Not yet recruiting
Verdun, Quebec, Canada, H4H 1R3
Contact: Pedro Rosa-Neto, MD    514 766 1009   
University Psychiatric Hospital Vrapče Recruiting
Zagreb, Croatia, 10090
Contact: Ninoslav Mimica, MD    385 1 3780 678   
University Hospital Centre Zagreb Recruiting
Zagreb, Croatia, 10000
Contact: Natasa Klepac, MD    38598541880   
Clinical Research Services Turku (CRST) Not yet recruiting
Turku, Finland, 20520
Contact: Juha Rinne, MD    00358 2 3131866   
Charité-Universitätsmedizin Berlin Klinik für Psychiatrie und Psychotherapie Recruiting
Berlin, Germany, 10117
Contact: Josef Priller, Prof Dr med    49 30 450 617209   
Klinik für Psychiatrie und Psychotherapie, Charité CBF Recruiting
Berlin, Germany, 14050
Contact: Oliver Peters, PD Dr med    49 30 84458215   
Universitätsklinikum Hamburg-Eppendorf Klinik für Psychiatrie und Psychotherapie Recruiting
Hamburg, Germany, 20246
Contact: Holger Jahn, MD    49 040 7410 55657   
Klinik und Poliklinik für Psychiatrie und Psychotherapie der Technischen Universität München Recruiting
München, Germany, 81675
Contact: Janine Diehl-Schmid, PD Dr med    49 89 4140 6488   
Universitäts - und Rehabilitationskliniken Ulm, Neurologie Recruiting
Ulm, Germany, 89081
Contact: Albert Ludolph, Prof Dr med    49 731 177 1200   
Unità per il trasferimento dalla Ricerca alla Clinica Not yet recruiting
Brescia, Italy, 25125
Contact: Giovanni Frisoni, MD    39 030 3501261   
Fondazione Universita' Gabriele D'Annunzio di Chieti Not yet recruiting
Chieti Scalo, Italy, 66100
Contact: Marco Onofrj, Dr    39 328 653 7740   
Fondazione IRCCS Istituto Neurologico "Carlo Besta" Recruiting
Milano, Italy, 20133
Contact: Fabrizio Tagliavini, MD    39 0223942384   
Neurology I, Department of Neuroscience, University of Torino Not yet recruiting
Torino, Italy, 10126
Contact: Innocenzo Rainero, Prof    39 011 6334763   
Alzheimer Research Center Amsterdam Recruiting
Amsterdam, Netherlands, 1081
Contact: Niels Prins, Dr    31 203 017170   
Jeroen Bosch Ziekenhuis, afdeling geriatrie Recruiting
Den Bosch, Netherlands, 5223
Contact: Paul Dautzenberg, MD    31 73 553 8629   
Erasmus MC: University Medical Center Recruiting
Rotterdam, Netherlands, 3015
Contact: John Van Swieten, Dr    31 10 703 3274   
NZOZ Neuro-Kard Ilkowski i Partnerzy Spółka Partnerska Not yet recruiting
Poznań, Poland, 61-853
Contact: Jan Ilkowski, Dr    48 609517676   
Euromedis Sp. z o.o. Not yet recruiting
Szczecin, Poland, 70-111
Contact: Andrzej Potemkowski, Prof, MD, PhD    48 91 813 63 22   
Psychomedical Consult Recruiting
Bucharest, Romania, 024072
Contact: Catalina Tudose, PhD    40744586103   
University Emergency Central Military Hospital "Dr.Carol Davila", Psychiatry Department Not yet recruiting
Bucharest, Romania, 010816
Contact: Daniel Vasile, PhD    40722609430   
National Neuroscience Institute Department of Neurology Recruiting
Singapore, Singapore, 308433
Contact: Nagaendran Kandiah, Dr    65 6357 7171   
Fundació ACE. Institut Català de Neurociències Aplicades Not yet recruiting
Barcelona, Spain, 08028
Contact: Mercè Boada Rovira, Dr    34 93 444 73 18   
Ceuta University Hospital; Neurology Recruiting
Ceuta, Spain, 51003
Contact: Rafael Merino de Torres, MD    34 600890870   
Hospital Viamed Montecanal, Neurology Department Not yet recruiting
Zaragoza, Spain, 50012
Contact: Antonio Oliveros, Dr    34 670 492 099   
United Kingdom
NHS Grampian, OAP Directorate Recruiting
Aberdeen, United Kingdom, AB25 2ZH
Contact: Alasdair Lawrie, Dr    44 01224 557941   
The Barberry Out-Patients Department Recruiting
Birmingham, United Kingdom, B15 2FG
Contact: Peter Bentham, Dr    44 0121 301 2067   
Blackberry Hill Hospital Not yet recruiting
Bristol, United Kingdom, BS16 2EW
2gether NHS foundation trust Recruiting
Cheltenham, United Kingdom, GL53 9DZ
Contact: Tarun Kuruvilla, Dr    44 1242 634460   
Kingsway Hospital Not yet recruiting
Derby, United Kingdom, DE22 3LZ
Contact: Simon Thacker, MD    44(0)1332 623700   
St Margaret's Hospital Mental Health Unit Recruiting
Epping, United Kingdom, CM16 6TN
Contact: Zuzana Walker, Dr    44 01279 827893   
Cognition Health Ltd. Recruiting
London, United Kingdom, W1G 9JF
Contact: Emer MacSweeney, Dr    44 203 355 3536   
Imperial College Healthcare NHS Trust - Charing Cross Hospital Recruiting
London, United Kingdom, W6 8RF
Contact: Richard Perry, Dr    44 0203 3135515   
Dementia Research Center at Queens Square Recruiting
London, United Kingdom, WC1N 3BG
Contact: Catherine Mummery, Dr    44 (0) 203 4483227   
Nuffield Department of Clinical Neurosciences Recruiting
Oxford, United Kingdom, OX3 9DU
Contact: Christopher Butler, Dr    44 01865 234632   
Redwoods Centre Recruiting
Shrewsbury, United Kingdom, SY3 5DS
Contact: Ejaz Nazir, Dr    44 1743 210000 ext 4175   
Wessex Neurological Centre, Southampton General Hospital Recruiting
Southampton, United Kingdom, SO16 6YD
Contact: Christopher Kipps, Dr    44 23 8079 6781   
Sponsors and Collaborators
TauRx Therapeutics Ltd
  More Information

Additional Information:
No publications provided by TauRx Therapeutics Ltd

Additional publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: TauRx Therapeutics Ltd Identifier: NCT01626378     History of Changes
Other Study ID Numbers: TRx-237-007
Study First Received: June 20, 2012
Last Updated: August 4, 2014
Health Authority: United States: Food and Drug Administration
Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica
Australia: Department of Health and Ageing Therapeutic Goods Administration
Bulgaria: Bulgarian Drug Agency
Canada: Health Canada
Croatia: Agency for Medicinal Product and Medical Devices
Germany: Federal Institute for Drugs and Medical Devices
Italy: The Italian Medicines Agency
Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
Singapore: Health Sciences Authority
Spain: Agencia Española de Medicamentos y Productos Sanitarios
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by TauRx Therapeutics Ltd:
Behavioral Variant Frontotemporal Dementia
Frontotemporal Dementia

Additional relevant MeSH terms:
Aphasia, Primary Progressive
Frontotemporal Dementia
Pick Disease of the Brain
Brain Diseases
Central Nervous System Diseases
Communication Disorders
Delirium, Dementia, Amnestic, Cognitive Disorders
Frontotemporal Lobar Degeneration
Language Disorders
Mental Disorders
Metabolic Diseases
Nervous System Diseases
Neurobehavioral Manifestations
Neurodegenerative Diseases
Neurologic Manifestations
Proteostasis Deficiencies
Signs and Symptoms
Speech Disorders
TDP-43 Proteinopathies processed this record on October 21, 2014