Frontotemporal Dementia Trial of LMTM

  • Study Director: Adam Boxer, MD, PhD
  • Sponsor: TauRx Therapeutics Ltd
  • Recruiting? No
  • Official Study Title: A Double-Blind, Placebo-Controlled, Randomized, Parallel Group, 12-Month Safety and Efficacy Trial of Leuco-methylthioninium bis(hydromethanesulfonate) in Subjects with Behavioral Variant Frontotemporal Dementia (bvFTD)
  • ClinicalTrials.gov Identifier: NCT01626378
  • Conditions Studied: Behavioral variant frontotemporal dementia (bvFTD)
  • Intervention: Leuco-methylthioninium bis(hydromethanesulfonate) (LMTM, TRx0237) and placebo control. Study drug (LMTM or placebo) is administered as an oral tablet, to be swallowed whole, once in the morning and once in the evening.
  • Phase: Phase 3
  • Duration of Participation: Approximately 14 months

Purpose of the Study

LMTM is postulated to dissolve, as well as prevent, further formation of tau and TDP-43 aggregates that are thought to be neurotoxic in frontotemporal lobar degeneration syndromes. The purpose of this study is to evaluate whether LMTM is efficacious, safe and well-tolerated in subjects with bvFTD.

Eligibility

Inclusion Criteria: Subjects must be below 80 years old and meet diagnostic criteria for probable behavioral variant frontotemporal dementia (bvTFD). Subjects must have a study partner who is able to accompany them to all study visits and able to verify daily compliance with the study drug. Subjects must also have a Mini Mental State Examination (MMSE) score that is 20 or above at screening. Subjects must be willing and able to have brain MRIs throughout the course of the study.

Exclusion Criteria: Subjects must not have a significant central nervous system disorder other than bvFTD and must not have expressive language deficits that would prevent cognitive and neuropsychological assessments. Subjects must not have a proven mutation producing non-tau, non-TDP43 pathology even if the diagnostic criteria for probable bvFTD have been met. Subject also should not have a history of stroke, transient ischemic attack, significant head injury, REM sleep behavior disorder, major depressive disorder, schizophrenia, bipolar disorders, other psychotic disorders or substance related disorders. Subjects should not be residing in a hospital or moderate to high dependency continuous care facility.

What to Expect

Testing: Brain MRIs, neurological and physical examinations, cognitive testing and neuropsychiatric assessments, ECGs, Pulse-Co-oximetry, blood and urine specimen collection, vital signs, study diary

The Frequency of Visits:

  • 42-day screening period
  • Visits once every 1–2 months
  • One follow-up visit, one month after the end of the study treatment period

Materials Needed Before Evaluation: Diagnosis of probable behavioral variant frontotemporal dementia according to the International Consensus Criteria for bvFTD (Rascovsky et. al. 2011; Appendix 1)

Costs: No costs will be charged for any of the study procedures. Parking will be validated at UCSF public garages for all study visits, and subjects will be reimbursed $100 per visit for meal and travel expenses.

Contact Information

Clinical Trials Nurse: Mary Koestler, RN, PhD, CCRC – Mary.Koestler@ucsf.edu, 415.476.0661