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

TauRx Pharmaceuticals

Status and phase

Completed

Phase 3

Conditions

Behavioral Variant Frontotemporal Dementia (bvFTD)

Treatments

Drug: TRx0237

Drug: Placebo

Study type

Interventional

Funder types

Industry

Identifiers

NCT01626378

TRx-237-007

Details and patient eligibility

About

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).

Enrollment

220 patients

Sex

All

Ages

Under 79 years old

Volunteers

No Healthy Volunteers

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