Study to Assess the Safety and Biological Activity of AMX0035 for the Treatment of Alzheimer's Disease

Completed · Phase 2 · Has a placebo group

Conditions studied: Alzheimer Disease

In brief

The study is a 24-week, randomized, double-blind, multi-site, placebo-controlled study in participants with mild cognitive impairment (MCI) or early dementia due to Alzheimer's disease (AD).

Key facts

Study ID

NCT03533257

Run by

Amylyx Pharmaceuticals Inc.

People needed

95

Starts

2018-09-14

Expected to finish

2020-11-06

Last updated by the study team

2025-03-07

Who can join

Age: 55 and older, up to 89. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Ages 55-89, inclusive, male or female
• Diagnosis of "Probable Alzheimer's Disease" or Mild Cognitive Impairment (amnestic or amnestic plus other) with biomarkers that suggest intermediate or high likelihood that the syndrome is due to AD, according to 2011 NIA-AA Workgroup criteria
• MoCA score >/=8
• Able to read and write in English sufficiently to complete all study procedures
• Geriatric Depression Scale <7
• Willing and able to complete all assessments and study procedures
• Not pregnant, lactating or of child-bearing potential (women must be >2 years post-menopausal or surgically sterile)
• Study partner with at least two days per week with contact with patient willing to accompany patient to visits and complete partner study forms
• No known hypersensitivity to TURSO or Phenylbutyrate
• If on cholinesterase inhibitor and/or memantine, treatment must have started for no less than 3 months (84 days) prior to baseline and the dosing regimen must have remained stable for 6 weeks (42 days) prior to baseline. The Investigator anticipated that the dosing regimen at baseline would remain unchanged throughout participation in the study.

You may not qualify if…

• Any CNS disease other than suspected AD, such as clinical stroke, brain tumor, normal pressure hydrocephalus, multiple sclerosis, significant head trauma with persistent neurological cognitive deficits or complaints, Parkinson's disease, frontotemporal dementia, or other neurodegenerative diseases
• Abnormal liver function defined as AST and/or ALT > 3 times the upper limit of normal
• Renal insufficiency as defined by a serum creatinine > 1.5 times the upper limit of normal
• Recent (less than 1 year) cholecystectomy or the presence of post-cholecystectomy syndrome or biliary obstruction
• Clinically significant unstable medical condition (other than AD) that in the Site Investigator opinion would pose a risk to the participant if they were to participate in the study
• Any contraindication to undergo MRI studies such as:
• History of a cardiac pacemaker or pacemaker wires
• Metallic particles in the body
• Vascular clips in the head
• Prosthetic heart valves
• Severe claustrophobia impeding ability to participate in an imaging study, or
• MRI findings that show one or more of the following:
• More than 4 incidental microhemorrhages
• Incidental lacunar infarct with attributable signs or symptoms and with history of stroke
• Incidental meningiomas with attributable signs or symptoms
• Newly recognized meningioma
• Major active or chronic psychiatric illness (e.g. depression, bipolar disorder, obsessive compulsive disorder, schizophrenia) that is not stable or well controlled within the previous year prior to baseline
• Any significant neurodevelopmental disability
• Current suicidal ideation or history of suicide attempt within 5 years of baseline or significant change from the screening and baseline C-SSRS at the discretion of the Site Investigator
• History of alcohol or other substance abuse or dependence within the past 2 years
• Any significant systemic illness or medical condition that could affect safety or compliance with study at the discretion of the Site Investigator
• Laboratory abnormalities in B12, TSH, or other common laboratory parameters that might contribute to cognitive dysfunction
• Current use of medications with psychoactive properties that may deleteriously affect cognition (e.g., anticholinergics, centrally-acting antihistamines, antipsychotics, sedative hypnotics, anxiolytics)
• Use of any investigational therapy being used or evaluated for the treatment of AD is prohibited beginning 3 months (90 days) prior to the Baseline Visit and throughout the study.
• Use of other investigational agents 1 month (28 days) prior to the Baseline Visit and for the duration of the trial.

Where it is running

• Clinical Neuroscience Solutions, Inc. - Jacksonville — Jacksonville, Florida, United States
• Clinical Neuroscience Solutions, Inc. - Orlando — Orlando, Florida, United States
• International Medical Investigational Centers (IMIC) — Palmetto Bay, Florida, United States
• Rush University Medical Center — Chicago, Illinois, United States
• University of Kansas Clinical Research Center — Fairway, Kansas, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• Rowan University School of Osteopathic Medicine — Stratford, New Jersey, United States
• Mount Sinai Alzheimer's Disease Research Center — New York, New York, United States
• Columbia University — New York, New York, United States
• Hospital of the University of Pennsylvania, Penn Memory Center — Philadelphia, Pennsylvania, United States
• Genesis NeuroScience Clinic — Knoxville, Tennessee, United States

Full record on ClinicalTrials.gov

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