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NCT03533257 | COMPLETED | Alzheimer Disease

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

Amylyx Pharmaceuticals Inc.

Brief Summary:

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

Condition or disease

Alzheimer Disease

Intervention/treatment

AMX0035

Placebo

Phase

PHASE2

Detailed Description:

The study is a 24-week, randomized, double-blind, multi-site, placebo-controlled study in volunteers with late mild cognitive impairment (MCI) or early dementia due to Alzheimer's disease (AD). The study is designed to evaluate the safety, tolerability, drug target engagement and neurobiological effects of treatment with AMX0035 over 24 weeks. The study is designed to yield deep phenotyping insight for the purposes of demonstrating the effects of AMX0035 on mechanistic targets of engagement and disease biology. The study will evaluate diverse disease-relevant markers and produce an informative dataset that will allow for evaluation and correlation of imaging-based markers, neurobiological changes, functional measures, and cognitive outcomes.

Study Type : INTERVENTIONAL
Estimated Enrollment : 95 participants
Masking : QUADRUPLE
Primary Purpose : TREATMENT
Official Title : Phase II Study to Assess the Safety, Tolerability, and Target Engagement of AMX0035, a Fixed Combination of Sodium Phenylbutyrate and Tauroursodeoxycholic Acid for the Treatment of Alzheimer's Disease
Actual Study Start Date : 2018-08-27
Estimated Primary Completion Date : 2021-04-15
Estimated Study Completion Date : 2021-08-15

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 55 Years to 89 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers:
Criteria
Inclusion Criteria
  • 1. Ages 55-89, inclusive, male or female
  • 2. 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
  • 3. MoCA 8 - 26 inclusive
  • 4. Able to read and write in English sufficiently to complete all study procedures
  • 5. Geriatric Depression Scale \<7
  • 6. Willing and able to complete all assessments and study procedures
  • 7. Not pregnant, lactating or of child-bearing potential (women must be \>2 years post-menopausal or surgically sterile)
  • 8. Study partner with at least two days per week with contact with patient willing to accompany patient to visits and complete partner study forms
  • 9. No known hypersensitivity to Tauroursodeoxycholic acid or Phenylbutyrate
  • 10. Must have a previous biomarker supportive of AD as the underlying pathology of cognitive decline, which could include amyloid PET, CSF AD biomarkers, FDG-PET, or vMRI scan
  • 11. If on cholinesterase inhibitor and/or memantine, doses are stable for 3 months prior to baseline
Exclusion Criteria
  • 1. 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
  • 2. Abnormal liver function defined as AST and/or ALT \> 3 times the upper limit of normal
  • 3. Renal insufficiency as defined by a serum creatinine \> 1.5 times the upper limit of normal
  • 4. History of cholecystectomy or biliary disease
  • 5. 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
  • 6. Any contraindication to undergo MRI studies such as
    • 1. History of a cardiac pacemaker or pacemaker wires
    • 2. Metallic particles in the body
    • 3. Vascular clips in the head
    • 4. Prosthetic heart valves
    • 5. Severe claustrophobia impeding ability to participate in an imaging study
    • 7. Major active or chronic psychiatric illness (e.g. depression, bipolar disorder, obsessive compulsive disorder, schizophrenia) within the previous year prior to baseline
    • 8. Any significant neurodevelopmental disability
    • 9. Current suicidal ideation or history of suicide attempt within five years of baseline or significant change from the screening and baseline C-SSRS at the discretion of the Site Investigator
    • 10. History of alcohol or other substance abuse or dependence within the past two years
    • 11. Any significant systemic illness or medical condition that could affect safety or compliance with study at the discretion of the Site Investigator
    • 12. Laboratory abnormalities in B12, TSH, or other common laboratory parameters that might contribute to cognitive dysfunction
    • 13. Current use of medications with psychoactive properties that may deleteriously affect cognition (e.g., anticholinergics, centrally-acting antihistamines, antipsychotics, sedative hypnotics, anxiolytics)
    • 14. Use of any small molecule investigational therapy being used or evaluated for the treatment of AD is prohibited beginning three months (84 days) prior to the Baseline Visit and throughout the study.
    • 15. Use of any immunotherapy investigational therapy is prohibited beginning one year (365 days) prior to the Baseline Visit and throughout the study.
    • 16. Use of other investigational agents one month (28 days) prior to the Baseline Visit and for the duration of the trial.
Study to Assess the Safety and Biological Activity of AMX0035 for the Treatment of Alzheimer's Disease

Location Details

NCT03533257

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How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations

Not yet recruiting

United States, Florida

CNS Healthcare - Jacksonville

Jacksonville, Florida, United States, 32256

Not yet recruiting

United States, Florida

CNS Healthcare - Orlando

Orlando, Florida, United States, 32801

Not yet recruiting

United States, Florida

International Medical Investigational Centers (IMIC)

Palmetto Bay, Florida, United States, 33157

Not yet recruiting

United States, Illinois

Rush University Medical Center

Chicago, Illinois, United States, 60612

Not yet recruiting

United States, Kansas

University of Kansas Clinical Research Center

Fairway, Kansas, United States, 66205

Not yet recruiting

United States, Massachusetts

Massachusetts General Hospital

Boston, Massachusetts, United States, 02114

Not yet recruiting

United States, New Jersey

Rowan University

Stratford, New Jersey, United States, 08084

Not yet recruiting

United States, New York

Mount Sinai Alzheimer's Disease Research Center

New York, New York, United States, 10029

Not yet recruiting

United States, New York

Columbia University

New York, New York, United States, 10032

Not yet recruiting

United States, Pennsylvania

Penn Memory Center

Philadelphia, Pennsylvania, United States, 19104

Not yet recruiting

United States, Tennessee

Center for Biomedical Research

Knoxville, Tennessee, United States, 37909