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

• Status: Completed
• Trial end date: 2016-02-01
• Target enrollment: 0
• Participant gender: All

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).

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: TauRx Therapeutics Ltd

Criteria

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 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

• 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 of Screening (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