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Clinical Studies


  • Biofluid Studies in Neurodegenerative Dementias Rochester, Minn.

    The purpose of this study is to collect blood samples for DNA analysis from patients clinically diagnosed with Alzheimers disease, Lewy Body disease, and Frontotemporal degeneration.

Closed for Enrollment

  • 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) Rochester, Minn., Jacksonville, Fla.

    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).

  • A Phase 3 Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Aducanumab (BIIB037) in Subjects With Early Alzheimer's Disease (ENGAGE) Rochester, Minn.

    The primary objective of the study is to evaluate the efficacy of monthly doses of aducanumab in slowing cognitive and functional impairment as measured by changes in the Clinical Dementia Rating-Sum of Boxes (CDR-SB) score as compared with placebo in participants with early AD. Secondary objectives are to assess the effect of monthly doses of aducanumab as compared with placebo on clinical progression as measured by Mini-Mental State Examination (MMSE), AD Assessment Scale-Cognitive Subscale (13 items) [ADAS-Cog 13], and AD Cooperative Study-Activities of Daily Living Inventory (Mild Cognitive Impairment version) [ADCS-ADL-MCI].

  • An Open-Label, Extension Study of the Effects of TRx0237 in Subjects With Alzheimer's Disease or Behavioral Variant Frontotemporal Dementia (bvFTD) Rochester, Minn., Jacksonville, Fla.

    The purpose of this study is to provide subjects who have completed participation in a Phase 2 or Phase 3 trial with TRx0237 continued access to therapy and to evaluate the long-term safety of TRx0237.

  • Anti-Amyloid Treatment in Asymptomatic Alzheimer's Disease (A4 Study) (A4) Rochester, Minn., Jacksonville, Fla.

    The purpose of this study is to test whether an investigational drug called solanezumab can slow the progression of memory problems associated with brain amyloid (protein that forms plaques in the brains of people with Alzheimer Disease [AD]).

  • Longitudinal Evaluation of Amyloid Risk and Neurodegeneration - The LEARN Study a Companion Observational Study to Anti-Amyloid Treatment in Asymptomatic Alzheimer's Disease (A4) Trial (LEARN) Rochester, Minn., Jacksonville, Fla.

    The purpose of this study is to evaluate the rate of cognitive change in clinically normal older individuals who "screen-failed" for the A4 trial on the basis of their screening PET imaging not demonstrating evidence of elevated amyloid accumulation ( were Aβ negative) but met all other A4 study eligibility criteria. While long term data suggests that older individuals with elevated Aβ burden are at increased risk of cognitive decline, it is important to demonstrate the different rate of clinical decline between Aβe ("Aβ elevated") and Aβne ("Aβ not elevated") individuals.  

  • Phase 3b Open-Label, Multicenter, Safety Study of BIIB037 (Aducanumab) in Subjects With Alzheimer's Disease Who Had Previously Participated in the Aducanumab Studies 221AD103, 221AD301, 221AD302 and 221AD205 Rochester, Minn., Jacksonville, Fla.

    The primary objective is to evaluate the long-term safety and tolerability of aducanumab after a wash-out period imposed by discontinuation of feeder studies in participants who had previously received aducanumab (i.e., previously treated participants) or who had previously received placebo (i.e, treatment-naïve participants).

  • Rare Diseases Clinical Research Network Advancing Research and Treatment for Frontotemporal Lobar Degeneration ARTFL: Research Projects 1 & 2 (ARTFL) Jacksonville, Fla., Rochester, Minn.

    Frontotemporal Lobar Degeneration (FTLD) is the neuropathological term for a collection of rare neurodegenerative diseases that correspond to four main overlapping clinical syndromes: frontotemporal dementia (FTD), primary progressive aphasia (PPA), corticobasal degeneration syndrome (CBS) and progressive supranuclear palsy syndrome (PSPS). The goal of this study is to build a FTLD clinical research consortium to support the development of FTLD therapies for new clinical trials. The consortium, referred to as Advancing Research and Treatment for Frontotemporal Lobar Degeneration (ARTFL), will be headquartered at UCSF and will partner with six patient advocacy groups to manage the consortium. Participants will be evaluated at 14 clinical sites throughout North America and a genetics core will genotype all individuals for FTLD associated genes.

  • Therapeutic Effects of Intranasally-Administered Insulin in Adults With Amnestic Mild Cognitive Impairment (aMCI) or Mild Alzheimer's Disease (AD) (SNIFF) Rochester, Minn., Jacksonville, Fla.

    An urgent need exists to find effective treatments for Alzheimer's disease (AD) that can arrest or reverse the disease at its earliest stages. The emotional and financial burden of AD to patients, family members, and society is enormous, and is predicted to grow exponentially as the median population age increases. Current FDA-approved therapies are modestly effective at best. This study will examine a novel therapeutic approach using intranasal insulin (INI) that has shown promise in short-term clinical trials. If successful, information gained from the study has the potential to move INI forward rapidly as a therapy for AD. The study will also provide evidence for the mechanisms through which INI may produce benefits by examining key cerebral spinal fluid (CSF) biomarkers and hippocampal/entorhinal atrophy. These results will have considerable clinical and scientific significance, and provide therapeutically-relevant knowledge about insulin's effects on AD pathophysiology. Growing evidence has shown that insulin carries out multiple functions in the brain, and that insulin dysregulation may contribute to AD pathogenesis.

    This study will examine the effects of intranasally-administered insulin on cognition, entorhinal cortex and hippocampal atrophy, and cerebrospinal fluid (CSF) biomarkers in amnestic mild cognitive impairment (aMCI) or mild AD. It is hypothesized that after 12 months of treatment with INI compared to placebo, subjects will improve performance on a global measure of cognition, on a memory composite and on daily function. In addition to the examination of CSF biomarkers and hippocampal and entorhinal atrophy, the study aims to examine whether baseline AD biomarker profile, gender, or Apolipoprotein epsilon 4 (APOE-ε4) allele carriage predict treatment response.

    In this study, 240 people with aMCI or AD will be given either INI or placebo for 12 months, following an open-label period of 6 months where all participants will be given active drug. The study uses insulin as a therapeutic agent and intranasal administration focusing on nose to brain transport as a mode of delivery.

  • Veri-T: A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy Study of Oral Verdiperstat (BHV-3241) in Patients With Semantic Variant Primary Progressive Aphasia (svPPA) Due to Frontotemporal Lobar Degeneration With TDP-43 Pathology (FTLD-TDP) (Veri-T) Rochester, Minn.

    Veri-T is a phase 1, randomized, double-blind, placebo-controlled study of the safety, tolerability, preliminary efficacy and pharmcokinetics and pharmacodynamics of oral verdiperstat in patients with Semantic Variant Primary Progressive Aphasia (svPPA) Due to Frontotemporal Lobar Degeneration With TDP-43 Pathology (FTLD-TDP).  

    This study is an NIH R01 investigator-sponsor study, the PI and holder of IND 157, 370 is Peter Ljubenkov, MD, at UCSF.  The UCSF IRB will serve as the IRB of Record.

    This is a multicenter study with all sites located in the US. About 64 patients will participate. The study includes about 12 visits over about 8 months. The screening period is held over 6 weeks followed by 24 weeks of study medication. There is one follow-up visit 4 weeks after completing study medication.  Study tests and procedures include physical and neruological exams, neurognitive tests, questionnaires, blood and urine laboratory tests including pregnancy tests for females of child-bearing potential, ECGs, MRIs and two lumbar punctures.

    Participants will be randomized 3:1 to 300 mg extended-release verdiperstant or placebo. Participants will take one tablet daily for the first week, two tablets daily for the second week and two tablets twice daily during weeks 3-24.