Alzheimer's DiseasePhase 2NCT02431468
A Study Assessing Bryostatin in the Treatment of Moderately Severe to Severe Alzheimer's Disease
A Phase 2 study testing Bryostatin 1 in people with Alzheimer's Disease, run by Neurotrope Bioscience, Inc. at 29 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 55 to 85 years
- Conditions the study is for
- Alzheimer's Disease
- What is being tested?
- Bryostatin 1
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 147 participants
- Where
- 29 sitesUnited States
- Study dates
- Nov 2015to Feb 2017
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 55 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
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- 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.
Who cannot join
- 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
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- 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
Trial locations and contacts
29 sites in 1 country.
29 sites in 1 country
United States29 sites
Neurological Associates of Albany, PC
Albany, New York, 12208
Atlanta Center for Medical Research
Atlanta, Georgia, 30331
JEM Research
Atlantis, Florida, 33462
Neurobehavioral Clinical Research
Canton, Ohio, 44718
Alzheimer's Memory Center
Charlotte, North Carolina, 28270
Neurology Clinic, PC
Cordova, Tennessee, 38018
ATP Clinical Research, Inc.
Costa Mesa, California, 92626
Millennium Psychiatric Associates
Creve Coeur, Missouri, 63141
iResearch Atlanta
Decatur, Georgia, 30030
Brain Matters Research
Delray Beach, Florida, 33445
Alexian Brothers Neurosciences Institute Clinical Research
Elk Grove Village, Illinois, 60007
University of Kansas Alzheimer's Disease Center
Fairway, Kansas, 66205
Neuropsychiatric Research Center of South Florida
Fort Myers, Florida, 33912
The Clinical Trial Center, LLC
Jenkintown, Pennsylvania
Lake Charles Clinical Trials
Lake Charles, Louisiana, 70629
Alzheimer's Research and Treatment Center
Lake Worth, Florida, 33449
Nader Pharmacology Research Institute
Los Alamitos, California, 90720
Sunstone Clinical Research
Medford, Oregon, 97504
Miami Jewish Health System
Miami, Florida, 33137
Parker Jewish Institute for Health Care and Rehabilitation
New Hyde Park, New York, 11040
Oklahoma Clinical Research Center
Oklahoma City, Oklahoma, 73112
Medical Research Group of Central Florida
Orange City, Florida, 32763
Compass Research, LLC
Orlando, Florida, 32806
Xenoscience, Inc/ 21st Century Neurology
Phoenix, Arizona, 85004
San Francisco Clinical Research Center
San Francisco, California, 94109
J. Gary Booker, MD APMC Clinical Drug Trials
Shreveport, Louisiana, 71104
Atlantic Neuroscience Institute
Springfield, New Jersey, 07801
Axiom Clinical Research of Florida
Tampa, Florida, 33609
Compass Research
The Villages, Florida, 32162
About this study
Official title: 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
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.
Read the detailed description
This study will enroll 150 moderately severe to severe Alzheimer's disease subjects. Subjects will be randomly assigned 1:1:1 to treatment with two different doses of bryostatin 1 or placebo. The primary analysis will take place after 12 weeks of treatment (7 doses).
- Conditions
- Alzheimer's Disease
- Being tested
- Bryostatin 1, Placebo
- Medical subject headings
- Alzheimer Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Bryostatin 1Drug
Also called bryostatin
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.
PlaceboOther
The placebo is a sterile, pyrogen-free, lyophilized powder identical in appearance to the active drug, intended for IV infusion upon reconstitution and dilution.
| Study group | Role | What they receive |
|---|---|---|
| Bryostatin 1 20ug | Experimental | Bryostatin 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. Receives: Drug: Bryostatin 1 |
| Bryostatin 1 40ug | Experimental | Bryostatin 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. Receives: Drug: Bryostatin 1 |
| Placebo | Placebo comparator | Placebo administered IV over 45 minutes every other week after 2 initial doses administered weekly. A total of 7 doses administered over 12 weeks. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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.
Measured: Primary analysis at Week 13 (day 91) after 12 weeks of treatment (up to day 107)
Secondary outcomes (1)
Secondary Efficacy Endpoints
- Change from baseline in the Severe Impairment Battery (SIB) at Weeks 5 and 9. Assesses cognition. Score range 0-100. Lower scores indicate greater cognitive impairment.
- Change from baseline in Alzheimer Disease Cooperative Study Activities of Daily Living Inventory-Severe Impairment Version (ADCS-ADL-SEV) at Weeks 5, 9,13. A 19-item test of the performance of activities of daily living. Total score range 0-54; lower scores indicate greater functional impairment.
- Change from baseline in MMSE-2 at Weeks 5, 9 and 13. Tests selected aspects of cognition on a scale of 0-30. Lower scores indicate greater cognitive impairment.
- Change from baseline in Neuropsychiatric Inventory (NPI) at Weeks 5, 9,13. Caregiver interview assesses 12 behavioral disturbances. Scores range from 0-144; higher scores indicate greater behavioral disturbances.
- Clinical Global Impression of Improvement (CGI-I) at Weeks 5, 9, 13. A 7-point scale range from (1) very much improved to (7) very much worse.
Measured: Week 5, Week 9, Week 13
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 May 2015 (estimated)
- Study startNov 2015
- Primary completionFeb 2017
- Study completionFeb 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Alzheimer's DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 147 participants (actual)
Sponsor and investigators
- Lead sponsor
- Neurotrope Bioscience, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02431468
- Sponsor's protocol ID
- NTRP-101-202
- Status last verified
- Jun 2018
- First submitted
- 22 Apr 2015
- Last updated
- 6 Jul 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 6 Jul 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT02431468
- Acronym / sponsor ID
- NTRP-101-202
- Conditions
- Alzheimer's Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 147 participants (actual)
- Lead sponsor
- Neurotrope Bioscience, Inc.Industry (pharma, biotech, devices)
- Study start
- Nov 2015
- Primary completion
- Feb 2017 (actual)
- Study completion
- Feb 2017 (actual)
- First posted
- 1 May 2015 (estimated)
- Results
- Posted 6 Jul 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This study will enroll 150 moderately severe to severe Alzheimer's disease subjects. Subjects will be randomly assigned 1:1:1 to treatment with two different doses of bryostatin 1 or placebo. The primary analysis will take place after 12 weeks of treatment (7 doses).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Bryostatin 1 20ug | Experimental | Drug: Bryostatin 1Bryostatin 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 | Experimental | Drug: Bryostatin 1Bryostatin 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 comparator | Other: PlaceboPlacebo administered IV over 45 minutes every other week after 2 initial doses administered weekly. A total of 7 doses administered over 12 weeks. |
Who may qualify
- Ages
- 55 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (12 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Bryostatin 1Also: bryostatinThe 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. | Drug | Bryostatin 1 20ug, Bryostatin 1 40ug |
| PlaceboThe placebo is a sterile, pyrogen-free, lyophilized powder identical in appearance to the active drug, intended for IV infusion upon reconstitution and dilution. | Other | Placebo |
Primary outcome
Safety: Number of Subjects With Treatment-emergent Adverse Events and Serious Adverse Events
Time frame: Baseline through 30 days post end of treatment (up to Day 107)
Evaluations of adverse events (AEs), serious adverse events (SAEs), Adverse event of special interest - myalgia
Efficacy: Change From Baseline in Severe Impairment Battery (SIB) in the Full Analysis Set (FAS)
Time frame: Primary analysis at Week 13 (day 91) after 12 weeks of treatment (up to day 107)
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.
1 secondary outcome
Secondary Efficacy Endpoints
Time frame: Week 5, Week 9, Week 13
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jul 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
29 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Bryostatin 1 in people with Alzheimer's Disease, run by Neurotrope Bioscience, Inc. at 29 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Jul 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 29 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 6 Jul 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety: Number of Subjects With Treatment-emergent Adverse Events and Serious Adverse Events (time frame: Baseline through 30 days post end of treatment (up to Day 107)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Jul 2018.