safety and efficacy study evaluating trx0237 in subjects with behavioral variant frontotemporal dementia (bvftd)

Conditions: Behavioral Variant Frontotemporal Dementia (bvFTD)

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).


Eligibility 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

Keywords: behavioral variant frontotemporal dementia bvftd frontotemporal dementia