A Study Assessing Bryostatin in the Treatment of Moderately Severe to Severe Alzheimer's Disease
A Randomized, Double-Blind,Placebo-Controlled, Phase 2 Study Assessing the Safety, Tolerability and Efficacy of Bryostatin in the Treatment of Moderately Severe to Severe Alzheimer's Disease
1 other identifier
interventional
147
1 country
29
Brief Summary
This is a randomized double-blind placebo-controlled study comparing different doses of bryostatin for the treatment of moderately severe to severe Alzheimer's disease. The study is 15 weeks in duration, including a safety and efficacy evaluation 30 days after the last dose of study drug.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Nov 2015
Shorter than P25 for phase_2
29 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
April 22, 2015
CompletedFirst Posted
Study publicly available on registry
May 1, 2015
CompletedStudy Start
First participant enrolled
November 1, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 1, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
February 1, 2017
CompletedResults Posted
Study results publicly available
July 6, 2018
CompletedJuly 6, 2018
June 1, 2018
1.3 years
April 22, 2015
April 30, 2018
June 6, 2018
Conditions
Outcome Measures
Primary Outcomes (2)
Safety: Number of Subjects With Treatment-emergent Adverse Events and Serious Adverse Events
Evaluations of adverse events (AEs), serious adverse events (SAEs), Adverse event of special interest - myalgia
Baseline through 30 days post end of treatment (up to Day 107)
Efficacy: Change From Baseline in Severe Impairment Battery (SIB) in the Full Analysis Set (FAS)
The primary statistical objective for efficacy was to estimate the effect of bryostatin on the mean change in the total SIB score after 12 weeks of treatment, assessed at Week 13 (day 91). Efficacy analyses were conducted according to randomized groups. The SIB is used to assess cognition in subjects with moderate and severe AD. It is divided into nine subscales that include attention, language, orientation, memory, praxis, visuospatial ability, construction, social skills, orienting head to name. Non-verbal responses are allowed, thus decreasing the need for language output. Forty questions are included with a point score range of 0-100. Lower scores indicate greater cognitive impairment.
Primary analysis at Week 13 (day 91) after 12 weeks of treatment (up to day 107)
Secondary Outcomes (1)
Secondary Efficacy Endpoints
Week 5, Week 9, Week 13
Study Arms (3)
Bryostatin 1 20ug
EXPERIMENTALBryostatin 20 micrograms administered IV over 45 minutes every other week after 2 initial loading doses of 24 micrograms administered weekly. A total of 7 doses administered over 12 weeks.
Bryostatin 1 40ug
EXPERIMENTALBryostatin 40 micrograms administered IV over 45 minutes every other week after 2 initial loading doses of 48 micrograms administered weekly. A total of 7 doses administered over 12 weeks.
Placebo
PLACEBO COMPARATORPlacebo administered IV over 45 minutes every other week after 2 initial doses administered weekly. A total of 7 doses administered over 12 weeks.
Interventions
The investigational drug product, bryostatin, is a sterile, pyrogen-free, lyophilized powder intended for IV infusion upon reconstitution and dilution. Placebo is a sterile, pyrogen-free, lyophilized powder intended for IV infusion upon reconstitution and dilution.
The placebo is a sterile, pyrogen-free, lyophilized powder identical in appearance to the active drug, intended for IV infusion upon reconstitution and dilution.
Eligibility Criteria
You may qualify if:
- Written informed consent from caregiver and subject (if possible) or legally acceptable representative if different from caregiver
- Male and female subjects 55-85 years of age inclusive
- Cognitive deficit present for at least 2 years that meet the diagnostic criteria for probable Alzheimer's
- Mini Mental State Exam (MMSE-2) score of 4-15
- Patients must be able to perform at least one item on the Severe Impairment Battery Scale
- Neuroimaging (computerized tomography (CT) or Magnetic Resonance Imaging (MRI)) within the last 24 months consistent with a diagnosis of probable Alzheimer's disease (AD)
- Reliable caregiver(s) or informant(s) who attends the subject at least an average of 3 hours or more per day for 3 or more days per week
- Adequate vision and motor function to comply with testing
- If taking drugs approved for treatment of Alzheimer's disease (e.g. cholinesterase inhibitors, memantine), must be on a stable dose for at least 3 months prior to entry into study and the dose must not change during the study unless a change is required due to an adverse event or a clinically significant change in the patient's status.
You may not qualify if:
- Dementia due to any condition other than AD, including vascular dementia (Rosen-modified Hachinski lschemic score ≥ 5)
- Evidence of significant central nervous system (CNS) vascular disease on previous neuroimaging including but not limited to: cortical stroke, multiple infarcts, localized single infarcts in the thalamus, angular gyrus, multiple lacunar infarcts or extensive white matter injury
- Clinically significant neurologic disease or condition other than AD, such as cerebral tumor, chronic subdural fluid collections, Huntington's Disease, Parkinson's Disease, normal pressure hydrocephalus, or any other diagnosis that could interfere with assessment of safety and efficacy
- Evidence of clinically significant unstable cardiovascular, pulmonary, renal, hepatic, gastrointestinal, neurologic, or metabolic disease within the 6 months prior to enrollment
- Poorly controlled diabetes, at the discretion of the Principal Investigator
- Creatinine clearance (CL) of \<45ml/min
- Use of an active Alzheimer's vaccine within 2 years prior to screening
- Use of a monoclonal antibody for treatment of AD within 1 year prior to screening
- Any medical or psychiatric condition that is likely to require initiation of additional medication or surgical intervention during the course of the study
- Use of an investigational drug within 30 days prior to screening
- Prior exposure to bryostatin, or known sensitivity to bryostatin or any ingredient in the study drug
- Any other concurrent medical condition, which in the opinion of the PI makes the subject unsuitable for the clinical study
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (29)
Xenoscience, Inc/ 21st Century Neurology
Phoenix, Arizona, 85004, United States
ATP Clinical Research, Inc.
Costa Mesa, California, 92626, United States
Nader Pharmacology Research Institute
Los Alamitos, California, 90720, United States
San Francisco Clinical Research Center
San Francisco, California, 94109, United States
JEM Research
Atlantis, Florida, 33462, United States
Brain Matters Research
Delray Beach, Florida, 33445, United States
Neuropsychiatric Research Center of South Florida
Fort Myers, Florida, 33912, United States
Alzheimer's Research and Treatment Center
Lake Worth, Florida, 33449, United States
Miami Jewish Health System
Miami, Florida, 33137, United States
Medical Research Group of Central Florida
Orange City, Florida, 32763, United States
Compass Research, LLC
Orlando, Florida, 32806, United States
Axiom Clinical Research of Florida
Tampa, Florida, 33609, United States
Compass Research
The Villages, Florida, 32162, United States
Atlanta Center for Medical Research
Atlanta, Georgia, 30331, United States
iResearch Atlanta
Decatur, Georgia, 30030, United States
Alexian Brothers Neurosciences Institute Clinical Research
Elk Grove Village, Illinois, 60007, United States
University of Kansas Alzheimer's Disease Center
Fairway, Kansas, 66205, United States
Lake Charles Clinical Trials
Lake Charles, Louisiana, 70629, United States
J. Gary Booker, MD APMC Clinical Drug Trials
Shreveport, Louisiana, 71104, United States
Millennium Psychiatric Associates
Creve Coeur, Missouri, 63141, United States
Atlantic Neuroscience Institute
Springfield, New Jersey, 07801, United States
Neurological Associates of Albany, PC
Albany, New York, 12208, United States
Parker Jewish Institute for Health Care and Rehabilitation
New Hyde Park, New York, 11040, United States
Alzheimer's Memory Center
Charlotte, North Carolina, 28270, United States
Neurobehavioral Clinical Research
Canton, Ohio, 44718, United States
Oklahoma Clinical Research Center
Oklahoma City, Oklahoma, 73112, United States
Sunstone Clinical Research
Medford, Oregon, 97504, United States
The Clinical Trial Center, LLC
Jenkintown, Pennsylvania, United States
Neurology Clinic, PC
Cordova, Tennessee, 38018, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- David Crockford, Vice President, Regulatory Affairs
- Organization
- Neurotrope BioScience, Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- GT60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 22, 2015
First Posted
May 1, 2015
Study Start
November 1, 2015
Primary Completion
February 1, 2017
Study Completion
February 1, 2017
Last Updated
July 6, 2018
Results First Posted
July 6, 2018
Record last verified: 2018-06