NCT01626378

Brief Summary

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).

Trial Health

98
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
220

participants targeted

Target at P25-P50 for phase_3

Timeline
Completed

Started May 2013

Typical duration for phase_3

Geographic Reach
12 countries

67 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 20, 2012

Completed
2 days until next milestone

First Posted

Study publicly available on registry

June 22, 2012

Completed
10 months until next milestone

Study Start

First participant enrolled

May 1, 2013

Completed
2.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2016

Completed
Last Updated

March 14, 2018

Status Verified

March 1, 2018

Enrollment Period

2.8 years

First QC Date

June 20, 2012

Last Update Submit

March 12, 2018

Conditions

Keywords

Behavioral Variant Frontotemporal DementiabvFTDFrontotemporal Dementia

Outcome Measures

Primary Outcomes (3)

  • Change from Baseline on Addenbrooke's Cognitive Examination - Revised (ACE-R)

    52 weeks

  • Change from Baseline on Functional Activities Questionnaire (FAQ)

    52 weeks

  • Change from Baseline on whole brain volume (assessed by brain MRI)

    52 weeks

Secondary Outcomes (4)

  • Change from Baseline on Unified Parkinson's Disease Rating Scale (UPDRS Parts II and III)

    52 weeks

  • Change from Baseline on Frontotemporal Dementia Rating Scale (FRS)

    52 weeks

  • Change from Baseline on Modified Alzheimer's Disease Cooperative Study - Clinical Global Impression of Change (modified ADCS-CGIC)

    52 weeks

  • Number of study participants who tolerate oral doses of TRx0237 as determined by safety parameter changes

    52 weeks

Other Outcomes (5)

  • Early effect on modified ADCS-CGIC (change from Baseline)

    8 weeks

  • Change from Baseline on the rate of atrophy in frontal and temporal lobes as well as ventricular volume (assessed by brain MRI)

    52 weeks

  • Change from Baseline on Mini-Mental Status Examination (MMSE)

    52 weeks

  • +2 more other outcomes

Study Arms (2)

TRx0237 200 mg/day group

EXPERIMENTAL
Drug: TRx0237

Placebo

PLACEBO COMPARATOR
Drug: Placebo

Interventions

TRx0237 100 mg tablet will be administered twice daily.

TRx0237 200 mg/day group

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.

Placebo

Eligibility Criteria

AgeUp to 79 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • Significant central nervous system (CNS) disorder other than bvFTD
  • Significant 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
  • Resides in hospital or moderate to high dependency continuous care facility
  • History of swallowing difficulties
  • Pregnant or breastfeeding
  • Glucose-6-phosphate dehydrogenase deficiency
  • +16 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (67)

David Geffen School of Medicine at UCLA, UCLA Neurological Services

Los Angeles, California, 90095, United States

Location

The Shankle Clinic

Newport Beach, California, 92663, United States

Location

Memory and Aging Centre

San Francisco, California, 94158, United States

Location

Meridien Research

Brooksville, Florida, 34601, United States

Location

Mayo Clinic

Jacksonville, Florida, 32224, United States

Location

Compass Research, LLC

Orlando, Florida, 32806, United States

Location

University of South Florida

Tampa, Florida, 33613, United States

Location

Department of Neurology, Emory University

Atlanta, Georgia, 30329, United States

Location

Alexian Brothers Neurosciences Institute Clinical Research

Elk Grove Village, Illinois, 60007, United States

Location

Indiana University Department of Neurology

Indianapolis, Indiana, 46202, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21224, United States

Location

Neurological Clinical Research Institute (NCRI) Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Mayo Clinic, Department of Neurology

Rochester, Minnesota, 55905, United States

Location

Memory Enhancement Center of America, Inc.

Eatontown, New Jersey, 07724, United States

Location

Neurological Associates of Albany, P. C.

Albany, New York, 12208, United States

Location

Integrative Clinical Trials LLC

Brooklyn, New York, 11229, United States

Location

UNC Department of Neurology, Physicians Office Building

Chapel Hill, North Carolina, 27599, United States

Location

University Hospitals Case Medical Center, Neurology Clinical Trials Unit

Cleveland, Ohio, 44106, United States

Location

Rivers Wellness and Research Institute

Oklahoma City, Oklahoma, 73112, United States

Location

The Clinical Trial Center, LLC

Jenkintown, Pennsylvania, 19046, United States

Location

Hospital of the University of Pennsylvania, Department of Neurology

Philadelphia, Pennsylvania, 19104, United States

Location

PRA Health Sciences, Phase 2/3 Outpatient and CNS Clinic

Salt Lake City, Utah, 84106, United States

Location

The Memory Clinic

Bennington, Vermont, 05201, United States

Location

University of Virginia

Charlottesville, Virginia, 22903, United States

Location

Neuroscience Research Australia

Randwick, New South Wales, 2031, Australia

Location

Box Hill Hospital

Box Hill, Victoria, 3128, Australia

Location

Neurodegenerative Disorders Research Pty Ltd

West Perth, Western Australia, 6005, Australia

Location

Heritage Medical Research Clinic-University of Calgary

Calgary, Alberta, T2N 4Z6, Canada

Location

University of British Columbia Hospital, Clinic for Alzheimer Disease and Related Disorders

Vancouver, British Columbia, V6T 2B5, Canada

Location

Vancouver Island Health Authority

Victoria, British Columbia, V8R 1J8, Canada

Location

True North Clinical Research

Halifax, Nova Scotia, B3S 1M7, Canada

Location

Geriatric Clinical Trials Group, Parkwood Institute

London, Ontario, N6C 0A7, Canada

Location

Toronto Memory Program

Toronto, Ontario, M3B 2S7, Canada

Location

University Health Network, Toronto Western Hospital, Memory Clinic

Toronto, Ontario, M5T 2S8, Canada

Location

McGill Centre for Studies in Aging, Alzheimer Disease Research Unit

Verdun, Quebec, H4H 1R3, Canada

Location

University Hospital Centre Zagreb

Zagreb, 10000, Croatia

Location

University Psychiatric Hospital Vrapče

Zagreb, 10090, Croatia

Location

Charité-Universitätsmedizin Berlin Klinik für Psychiatrie und Psychotherapie

Berlin, 10117, Germany

Location

Memory Clinic, ECRC

Berlin, 13125, Germany

Location

Universitätsklinikum Hamburg-Eppendorf Klinik für Psychiatrie und Psychotherapie

Hamburg, 20246, Germany

Location

Klinik und Poliklinik für Psychiatrie und Psychotherapie der Technischen Universität München

München, 81675, Germany

Location

Universitäts - und Rehabilitationskliniken Ulm, Neurologie

Ulm, 89081, Germany

Location

Unità di Neuroimmagine e Epidemiologia Alzheimer

Brescia, 25125, Italy

Location

Fondazione Universita' Gabriele D'Annunzio di Chieti

Chieti Scalo, 66100, Italy

Location

Fondazione IRCCS Istituto Neurologico "Carlo Besta"

Milan, 20133, Italy

Location

Neurology I, Department of Neuroscience, University of Torino

Torino, 10126, Italy

Location

Jeroen Bosch Ziekenhuis, afdeling geriatrie

's-Hertogenbosch, 5223, Netherlands

Location

Alzheimer Research Center Amsterdam

Amsterdam, 1081, Netherlands

Location

Erasmus University Medical Center

Rotterdam, 3015, Netherlands

Location

NZOZ Neuro-Kard Ilkowski i Partnerzy Spółka Partnerska

Poznan, 61-853, Poland

Location

Euromedis Sp. z o.o.

Szczecin, 70-111, Poland

Location

Psychomedical Consult

Bucharest, 024072, Romania

Location

National Neuroscience Institute Department of Neurology

Singapore, 308433, Singapore

Location

Fundació ACE. Institut Català de Neurociències Aplicades

Barcelona, 08028, Spain

Location

Ceuta University Hospital; Neurology

Ceuta, 51003, Spain

Location

Hospital Viamed Montecanal, Neurology Department

Zaragoza, 50012, Spain

Location

NHS Grampian, OAP Directorate

Aberdeen, AB25 2ZH, United Kingdom

Location

The Barberry Out-Patients Department

Birmingham, B15 2FG, United Kingdom

Location

2gether NHS foundation trust

Cheltenham, GL53 9DZ, United Kingdom

Location

Kingsway Hospital

Derby, DE22 3LZ, United Kingdom

Location

St Margaret's Hospital Mental Health Unit

Epping, CM16 6TN, United Kingdom

Location

Cognition Health Ltd.

London, W1G 9JF, United Kingdom

Location

Imperial College Healthcare NHS Trust - Charing Cross Hospital

London, W6 8RF, United Kingdom

Location

Dementia Research Center at Queens Square

London, WC1N 3BG, United Kingdom

Location

Nuffield Department of Clinical Neurosciences

Oxford, OX3 9DU, United Kingdom

Location

Redwoods Centre

Shrewsbury, SY3 5DS, United Kingdom

Location

Wessex Neurological Centre, Southampton General Hospital

Southampton, SO16 6YD, United Kingdom

Location

Related Publications (1)

  • Pletnikova O, Sloane KL, Renton AE, Traynor BJ, Crain BJ, Reid T, Zu T, Ranum LP, Troncoso JC, Rabins PV, Onyike CU. Hippocampal sclerosis dementia with the C9ORF72 hexanucleotide repeat expansion. Neurobiol Aging. 2014 Oct;35(10):2419.e17-21. doi: 10.1016/j.neurobiolaging.2014.04.009. Epub 2014 Apr 18.

MeSH Terms

Conditions

Frontotemporal Dementia

Interventions

hydromethylthionine

Condition Hierarchy (Ancestors)

Frontotemporal Lobar DegenerationDementiaBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesTDP-43 ProteinopathiesNeurodegenerative DiseasesProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic DiseasesNeurocognitive DisordersMental Disorders

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 20, 2012

First Posted

June 22, 2012

Study Start

May 1, 2013

Primary Completion

February 1, 2016

Study Completion

February 1, 2016

Last Updated

March 14, 2018

Record last verified: 2018-03

Locations