• A Clinical Study of MK-2214 in People with Early Alzheimer’s Disease
• The Alzheimer’s Tau Platform Trial is coming in late 2025/early 2026

• Phase 1/2 Study of DNL593 for Frontotemporal Dementia (FTD-GRN)
• Veri-T: A Trial of Verdiperstat in Patients with svPPA due to TDP-43 Pathology
• The Progressive Supranuclear Palsy (PSP) Platform Trial is coming in late 2025/early 2026

These trials are no longer enrolling new participants but follow-up and data analysis may be ongoing.

• AHEAD 3-45 Study: A Study to Evaluate Efficacy and Safety of Treatment with Lecanemab in Participants with Preclinical Alzheimer’s Disease
• BHV-4157 (Troriluzole) in Adult Subjects with Spinocerebellar Ataxia
• Frontotemporal Dementia Trial of Intranasal Oxytocin (FOXY)
• Phase 2 Infusion Study of BMS-986446, an Anti-MTBR Tau Monoclonal Antibody
• Phase 2 Study of Oral FNP-223, an O-GlcNAcase (OGA) Inhibitor
• Proof-of-Concept Study with AMT-130 in Adults with Early Manifest Huntington's Disease
• Study to Confirm Safety and Efficacy of BAN2401 in Participants with Early Alzheimer's Disease (Clarity AD)
• Treatment of Disturbed Sleep in Progressive Supranuclear Palsy

The clinical trials we have completed here at the UCSF Memory and Aging Center highlight some of our experience with a range of treatments, populations, diseases and environments.

Alzheimer’s Disease

• A Phase 1b, 12-Month, Randomized, Double-Blind, Placebo-Controlled Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of Salsalate in Patients with Mild to Moderate Alzheimer’s Disease
  The purpose of this study is to evaluate the safety and tolerability of salsalate in comparison to placebo in patients with prodromal to mild Alzheimer’s disease.
• A Study to Evaluate Safety and Tolerability of Aducanumab in Participants With Alzheimer's Disease Who Had Previously Participated in the Aducanumab Studies 221AD103, 221AD301, 221AD302 and 221AD205
  The primary objective was to evaluate the safety and tolerability of aducanumab over 100 weeks of treatment 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).
• A Study of LY3303560 in Participants With Early Symptomatic Alzheimer's Disease
  The purpose of this study was to evaluate the safety and efficacy of a study drug that targets an abnormal protein in the brain found in people with Alzheimer's Disease (AD).
• Clinical Trial of Solanezumab for Older Individuals Who May be at Risk for Memory Loss (A4)
  The purpose of this study was 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's disease [AD]).
• A Study to Evaluate the Efficacy and Safety of ABBV-8E12 in Participants With Early Alzheimer's Disease
  This study evaluated the efficacy and safety of ABBV-8E12 in subjects with early Alzheimer’s disease.
• CREAD Study: A Study of Crenezumab Versus Placebo to Evaluate the Efficacy and Safety in Participants With Prodromal to Mild Alzheimer's Disease (AD)
  The purpose of this study was to evaluate the efficacy and safety of crenezumab in patients with prodromal to mild Alzheimer’s disease.
• 221AD301 Phase 3 Study of Aducanumab (BIIB037) in Early Alzheimer's Disease (ENGAGE)
  The primary purpose of this study was to find out whether aducanumab had the potential to slow down disease progression in subjects with early Alzheimer’s disease (AD) by comparing it to placebo and to evaluate its safety (side effects) and to find out more about aducanumab.
• A Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy Study of TPI-287 in Alzheimer’s Disease
  The purpose of this study was to determine the dose of TPI-287 that is safe and tolerable in people with mild to moderate AD, as well as to measure the properties and preliminary efficacy of TPI-287.
• Progress of Mild Alzheimer’s Disease in Participants on Solanezumab Versus Placebo (EXPEDITION 3)
  The primary purpose of the study was to determine if solanezumab slows cognitive and functional decline in participants with mild Alzheimer’s disease.
• A Study to Evaluate the Efficacy and Safety of MABT5102A in Patients with Mild to Moderate Alzheimer’s Disease (ABBY)
  The purpose of this study was to evaluate the efficacy and safety of crenezumab in patients with mild to moderate Alzheimer’s disease.
• Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Amyloid-Imaging Positron Emission Tomography (PET) and Safety Study of ACC-001 and QS-21 Adjuvant in Subjects with Mild to Moderate Alzheimer’s Disease
  The purpose of this study was to assess the safety, tolerability, and efficacy of ACC-001, an active immunization, in patients with mild to moderate Alzheimer’s disease.
• Multicenter, Randomized, Third-Party Unblinded, Multiple Ascending Dose, Safety, Tolerability, and Immunogenicity Trial of ACC-001 in Subjects with Mild to Moderate Alzheimer’s Disease
  The purpose of this study was to assess the safety, tolerability, and immunogenicity of ACC-001, an active immunization, in patients with mild to moderate Alzheimer’s disease.
• Study to Evaluate the Safety, Tolerability and the Effect of BMS-241027 on Cerebrospinal Fluid Biomarkers in Subjects with Mild Alzheimer’s Disease
  The purpose of this study was to evaluate whether BMS-241027 is safe and well-tolerated in subjects with Alzheimer’s disease. In addition, this study also tested the effects of BMS-241027 on cerebrospinal fluid (CSF) tau proteins, brain magnetic resonance imaging (MRI), and cognitive (thinking and memory) tests in subjects with mild Alzheimer’s disease.
• CONCERT: A Phase 3 Multicenter, Randomized, Placebo-Controlled, Double-Blind Twelve-Month Safety and Efficacy Study Evaluating Dimebon in Patients with Mild-to-Moderate Alzheimer’s Disease on Donepezil
  The purpose of this study was to determine if Dimebon® (latrepirdine) is safe and effective in patients with mild to moderate Alzheimer’s disease on Aricept® (donepezil).
• A Phase 2 Study Evaluating the Efficacy and Safety of PF 04494700 in Mild to Moderate Alzheimer’s Disease
  The purpose of this study was to evaluate the efficacy and safety of PF04494700 in participants with mild to moderate Alzheimer’s disease.
• ELND005 in Patients with Mild to Moderate Alzheimer’s Disease
  The purpose of this study was to evaluate the dose-related safety and efficacy of multiple oral dosages of ELND005 as a treatment for Alzheimer’s disease.
• A Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Anti-Abeta (MABT5102A) in Patients with Alzheimer’s Disease
  This was a Phase I, multicenter, randomized, double-blind, placebo-controlled study in the United States and consisted of a single ascending-dose stage followed by a multidose, parallel-treatment stage. This study was conducted in approximately 50 adult patients between 50-85 years old who have mild to moderate Alzheimer’s disease.
• Acute Effects of Donepezil on Brain Perfusion and Memory in Subjects with Cognitive Impairment and Mild Alzheimer’s Disease
  The study hypothesized that donepezil would have a positive impact on brain blood flow deficits in subjects with memory deficits and/or mild dementia and that improvements in brain blood flow would be accompanied by improvements in memory.
• Bapineuzumab in Patients with Mild to Moderate Alzheimer’s Disease (ApoE4 Non-Carrier)
  The purpose of this study was to determine the safety and efficacy of multiple doses of bapineuzumab in ApoE4 carriers and non-carriers.

Corticobasal Syndrome (CBS)

• BIIB092 in Primary Tauopathies: CBS, nfvPPA, sMAPT, and TES (TauBasket)
  The purpose of this study was to evaluate the safety and tolerability of BIBB092 in comparison to placebo in patients with four different primary tauopathy syndromes: CBS, nonfluent variant primary progressive aphasia (nfvPPA), symptomatic MAPT mutation carriers (sMAPT), and traumatic encephalopathy syndrome (TES).
• Safety Study of TPI-287 to Treat CBS and PSP (TPI-287-4RT)
  The purpose of this study was to determine the safety and tolerability of intravenous (IV) infusions of TPI-287 in patients with four-repeat tauopathies (4RT), CBS or PSP.

Frontotemporal Dementia (FTD)

• A Study of Long-term AL001 Dosing in FTD Patients
  This Phase 2 open-label study evaluated the safety, tolerability, pharmacokinetics, and pharmacodynamics of AL001 in participants with a Granulin mutation or C9orf72 ;mutation causative of frontotemporal dementia (FTD).
• BIIB092 in Primary Tauopathies: CBS, nfvPPA, sMAPT, and TES (TauBasket)
  The purpose of this study was to evaluate the safety and tolerability of BIBB092 in comparison to placebo in patients with four different primary tauopathy syndromes: CBS, nonfluent variant primary progressive aphasia (nfvPPA), symptomatic MAPT mutation carriers (sMAPT), and traumatic encephalopathy syndrome (TES).
• Dose Finding Study of Nimodipine for the Treatment of Progranulin Insufficiency from GRN Gene Mutations
  The purpose of this study was to determine the maximum tolerated dose of nimodipine as well as the safety and tolerability of oral nimodipine in progranulin mutation carriers.
• Study to Assess the Safety, Tolerability, and Pharmacodynamic (PD) Effects of FRM-0334 in Subjects with Prodromal to Moderate Frontotemporal Dementia with Granulin Mutation
  The purpose of this study was to evaluate the safety and tolerability of two fixed doses of FRM-0334 (300 or 500mg) over 28 days in subjects with prodromal to moderate frontotemporal dementia with granulin gene mutation (FTD-GRN). Additionally, we assessed the pharmacodynamic effects of FRM-0334 on the change from baseline in plasma concentrations of progranulin after 28 days.
• Safety and Efficacy Study Evaluating TRx0237 in Subjects with Behavioral Variant Frontotemporal Dementia (bvFTD)
  The purpose of this study was to evaluate whether LMTM is efficacious, safe and well-tolerated in subjects with bvFTD.
• A Prospective, Randomized, Multi-Center, Double-Blind, 26-Week, Placebo-Controlled Trial of Memantine (10mg BID) for the Frontal and Temporal Subtypes of Frontotemporal Dementia
  The study was designed to determine whether memantine is effective in slowing the rate of behavioral decline in frontotemporal dementia. The study

1. Assessed the safety and tolerability of long-term treatment with memantine in patients with frontotemporal dementia (FTD) or semantic dementia (SD)
2. Determined whether memantine is effective in slowing the rate of cognitive decline in frontotemporal dementia
3. Evaluated whether memantine delays or decreases the emergence of parkinsonism in frontotemporal dementia.

Lastly, the study was designed to determine whether treatment with memantine affects change in weight.

Progressive Supranuclear Palsy (PSP)

• Study of BIIB092 in Participants With Progressive Supranuclear Palsy (PASSPORT)
  The primary purpose of this study is to find out whether BIIB092 has the potential to slow disease progression in subjects with progressive supranuclear palsy (PSP) by comparing it to placebo and to characterize its safety/tolerability profile.
• Multiple Ascending Dose Study of Intravenously Administered BMS-986168 (BIIB092) in Patients With Progressive Supranuclear Palsy (CN002-003)
  The purpose of this study was to evaluate the safety and tolerability of multiple ascending intravenous infusions of BMS-986168 and to assess the pharmacokinetics and immunogenicity of BIIB092, and pharmacodynamics of BIIB092 on cerebrospinal fluid (CSF) extracellular tau (eTau) concentrations in participants with Progressive Supranuclear Palsy.
• Young Plasma Transfusions for Progressive Supranuclear Palsy
  The purpose of this study is to determine the safety and tolerability of young healthy male donor plasma transfusions in patients with PSP.
• A Study to Assess Efficacy, Safety, Tolerability, and Pharmacokinetics of ABBV-8E12 in Subjects with Progressive Supranuclear Palsy (PSP)
  The purpose of this study was to evaluate the efficacy and safety of ABBV-8E12 in patients with progressive supranuclear palsy (PSP).
• Safety, Tolerability and Pharmacokinetics of C2N-8E12 in Subjects with Progressive Supranuclear Palsy
  The primary purpose of this study was to determine the safety, tolerability and maximally tolerated dose of C2N-8E12, an anti-tau monoclonal antibody. C2N-8E12 is an immunotherapy (antibody) drug designed to bind to and to remove tau protein.
• Safety Study of TPI-287 to Treat CBS and PSP (TPI-287-4RT)
  The purpose of this study was to determine the safety and tolerability of intravenous (IV) infusions of TPI-287 in patients with four-repeat tauopathies (4RT), CBS or PSP.
• A 6-Month, Open-Label, Pilot Futility Clinical Trial of Oral Salsalate for Progressive Supranuclear Palsy
  The purpose of this study was to determine the safety and tolerability of oral salsalate in people with PSP.
• A Phase 2/3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Davunetide for the Treatment of Progressive Supranuclear Palsy
  The purpose of the study was to evaluate the safety and efficacy of davunetide for the treatment of progressive supranuclear palsy (PSP). The multinational trial enrolled 313 subjects definitively diagnosed with PSP and had co-primary outcome measures: the Progressive Supranuclear Palsy Rating Scale (PSPRS) and the Schwab and England Activities of Daily Living (SEADL), as well as a series of secondary and exploratory endpoints.

Spinocerebellar Ataxia

• Trial in Adult Subjects with Spinocerebellar Ataxia
  The primary purpose of this study was to compare the efficacy of BHV-4157 (Troriluzole) 140 milligrams (mg) once daily versus placebo after 8 weeks of treatment in subjects with spinocerebellar ataxia (SCA).