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



Status:Recruiting
Conditions:Alzheimer Disease
Therapuetic Areas:Neurology
Healthy:No
Age Range:55 - 89
Updated:1/23/2019
Start Date:August 27, 2018
End Date:September 1, 2020
Contact:Sara Thrower
Email:Sara.Thrower@mgh.harvard.edu
Phone:617-643-5374

Use our guide to learn which trials are right for you!

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

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).

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.

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.
We found this trial at
7
sites
New York, New York 10029
Phone: 212-659-8885
?
mi
from
New York, NY
Click here to add this to my saved trials
185 Cambridge Street
Boston, Massachusetts 02114
617-724-5200
Phone: 617-643-4848
?
mi
from
Boston, MA
Click here to add this to my saved trials
116th St and Broadway
New York, New York 10027
(212) 854-1754
Phone: 212-305-7661
Columbia University In 1897, the university moved from Forty-ninth Street and Madison Avenue, where it...
?
mi
from
New York, NY
Click here to add this to my saved trials
1653 W. Congress Parkway
Chicago, Illinois 60612
(312) 942-5000
Phone: 312-942-0050
Rush University Medical Center Rush University Medical Center encompasses a 664-bed hospital serving adults and...
?
mi
from
Chicago, IL
Click here to add this to my saved trials
Fairway, Kansas 66205
Phone: 913-588-0555
?
mi
from
Fairway, KS
Click here to add this to my saved trials
Philadelphia, Pennsylvania 19104
Phone: 215-349-8727
?
mi
from
Philadelphia, PA
Click here to add this to my saved trials
Stratford, New Jersey 08084
Phone: 856-566-6003
?
mi
from
Stratford, NJ
Click here to add this to my saved trials