Alzheimer's DiseasePhase 1/2NCT02221947
Study to Evaluate the Preliminary Safety, Efficacy, PK and PD of Bryostatin 1 in Patients With Alzheimer's Disease
A Phase 1/2 study testing Bryostatin 1 in people with Alzheimer's Disease, run by Neurotrope Bioscience, Inc. at 1 site in United States.
Trial summary
This study is being done to evaluate the safety, tolerability and potential effectiveness of a new investigational drug, bryostatin 1, in patients with Alzheimer's disease (AD). Read more about this study
- Who is it for?
- Ages 50 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
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2014to Dec 2014
- Phase
- Phase 1/2
- 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 50 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female, age 50 - 85 yrs. Females are non-childbearing potential
- Patient must have a cognitive deficit present for at least 1 year and meet diagnostic criteria for probable Alzheimer's Disease Dementia by NIA-AA criteria or prodromal Alzheimer's Disease
- Mini Mental State Exam score of 16-26
- Ability to walk, at least with an assistive device
- Vision and hearing sufficient to comply with testing
- Normal cognitive and social functioning prior to onset of dementia, with evidence of progressive symptoms from patient or informant
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- Consistent caregiver to accompany patient to visits
- Sufficient basic education to be able to complete the cognitive assessments
- Living outside an institution
Who cannot join
- Dementia due to any condition other than AD, including vascular dementia
- Significant neuroimaging abnormalities, previously known or discovered on screening MRI scan,
- Evidence of clinically significant unstable cardiovascular, renal, hepatic, gastrointestinal, neurological, or metabolic disease within the past 6 months
- Use of any drug within 14 days prior to randomization unless the dose of the drug and the condition being treated have been stable for at least 30 days and are expected to remain stable during the study
- Use of tobacco products or nicotine-containing products within 3 months before Day 1
- Use of high dose vitamin E, or valproic acid
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- Any medical or psychiatric condition that may require medication or surgical treatment during the study
- Life expectancy less than 6 months
- Use of an investigational drug within 2 months prior to the screening visit
- Clinically significant neurological disease other than AD
- Major depression, alcohol or drug dependence or suicidality
- Psychotic episodes requiring hospitalization or antipsychotic therapy for more than 2 weeks within the past 10 years, not linked to AD
- Agitation sufficient to preclude participation in this trial
- Epilepsy or anti-epileptic drug therapy
- Abnormal laboratory tests that might point to another etiology for dementia;
- Acute or poorly controlled medical illness
- Likelihood, according to clinical judgment, of being transferred to a nursing home within 6 months
Trial locations and contacts
1 site in 1 country.
United States1 site
California Clinical Trials Medical Center
Glendale, California, 91206
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Preliminary Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Bryostatin 1 in Patients With Alzheimer's Disease
This study is being done to evaluate the safety, tolerability and potential effectiveness of a new investigational drug, bryostatin 1, in patients with Alzheimer's disease (AD).
Read the detailed description
This study is a single center, randomized, double-blind, placebo-controlled, parallel groups trial in patients with AD. Each subject enrolled in the trial will be randomized to receive a single IV dose of 0 (placebo) or 25 μg/m2 bryostatin. A total of 15 subjects (5 in the placebo arm and 10 in the treatment arm) will be enrolled in the study. The study consists of screening evaluations and on study evaluations divided into two segments, an inpatient segment and an outpatient segment. The four-day inpatient segment will consist of baseline evaluations and a 1-hour IV infusion of study drug followed by evaluations at multiple evaluations over the first 72 hrs post dose. During the outpatient segment, patients will be followed for AEs and have a final evaluation at 2 weeks post dose and a 4-week telephone safety follow up. Evaluations will include safety, efficacy, pharmacokinetics, and pharmacodynamics as assessed by PKC activity
- Conditions
- Alzheimer's Disease
- Being tested
- Bryostatin 1, Placebo
- Keywords
- Alzheimer's, bryostatin, PKC epsilon
- 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
25 μg/m2 bryostatin 1, single dose via intravenous infusion over 1 hour.
PlaceboDrug
Placebo, single dose via intravenous infusion over 1 hour.
| Study group | Role | What they receive |
|---|---|---|
| Bryostatin 1 | Active comparator | single dose of 25 μg/m2 bryostatin, intravenous infusion over 1 hour Receives: Drug: Bryostatin 1 |
| placebo | Placebo comparator | single dose of placebo, intravenous infusion over 1 hour Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Adverse Events as a Measure of Safety and Tolerability
Evaluate the safety and tolerability of bryostatin 1 (hereinafter referred to as bryostatin) in patients with Alzheimer's Disease (AD) following a single intravenous (IV) dose.
Measured: Within 2 weeks of study drug dosing
Preliminary Efficacy of a Single Dose of Bryostatin in the Treatment of Patients With AD
Hopkins Verbal Learning Test - Revised (HVLT-R) delayed recall; change from baseline. HVLT consists of a 12-item word list drawn from 3 semantic categories, presented in 3 learning trials. Score range = 0-12. The lower the number, the more impaired.
Repeatable Battery of Assessments for Neuropsychological Status (RBANS) figure recall; change from baseline. Total Score Range: 0-20. Each portion of the drawing is scored 1 point for correctness and completeness and 1 point for being placed properly in relation to the rest of the drawing. Drawing and placement scores are summed for the item total. To obtain subtest total score, the drawing and placement scores are summed for each item. The lower the number, the more impaired.
Measured: 48 hours post start of study drug infusion
Secondary outcomes (1)
Preliminary Efficacy of a Single Dose of Bryostatin in the Treatment of Patients With AD
HVLT-R (Hopkins Verbal Learning Test-Revised™) delayed recall (change from baseline). A 12-item word list: 3 learning trials. Score range = 0-12. The lower the number, the more impaired.
Repeatable Battery of Assessments for Neuropsychological Status (RBANS) figure recall; change from baseline. Score Range: 0-20. The lower the number, the more impaired. Digit Symbol Coding (observed), Score range: 0-125. The lower the number, the more impaired.
Clinical Dementia Rating- Sum of Boxes (CDR-SB, observed). Sum of 6 investigated domains (Memory, Orientation, Judgment and Problem Solving, Community Affairs, Home and Hobbies, Personal Care). Each subtest range is 0-3; Sum of all 6 subtest scores gives total CDR-SB score (range= 0-18).The higher the number, the more impaired.
Mini Mental State Exam, version 2 (MMSE-2), change from baseline. The MMSE-2 measures aspects of cognitionon a scale of 0-30. Lower scores indicate greater cognitive impairment.
Measured: Specified timepoints within 2 weeks post study drug infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Aug 2014 (estimated)
- Study startJun 2014
- Primary completionDec 2014
- Study completionDec 2014
Why it stopped: Part 2 of study replaced by NTRP-101-202, assessing 3 doses of bryostatin.
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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Neurotrope Bioscience, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Blanchette Rockefeller Neurosciences Insitute
- Responsible party
- Sponsor
- Investigators
- Hakop Gevorkyan, MD, MBA, Principal investigator, California Clinical Trials Medical Group
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02221947
- Sponsor's protocol ID
- NTRP101-201
- Status last verified
- Oct 2017
- First submitted
- 2 Jul 2014
- Last updated
- 6 Nov 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Nov 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Preliminary Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Bryostatin 1 in Patients With Alzheimer's Disease
- ClinicalTrials.gov ID
- NCT02221947
- Acronym / sponsor ID
- NTRP101-201
- Conditions
- Alzheimer's Disease
- Keywords
- Alzheimer's, bryostatin, PKC epsilon
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Part 2 of study replaced by NTRP-101-202, assessing 3 doses of bryostatin.
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Neurotrope Bioscience, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Blanchette Rockefeller Neurosciences Insitute
- Study start
- Jun 2014
- Primary completion
- Dec 2014 (actual)
- Study completion
- Dec 2014 (actual)
- First posted
- 21 Aug 2014 (estimated)
- Results
- Posted 21 Apr 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is being done to evaluate the safety, tolerability and potential effectiveness of a new investigational drug, bryostatin 1, in patients with Alzheimer's disease (AD).
Detailed description
This study is a single center, randomized, double-blind, placebo-controlled, parallel groups trial in patients with AD. Each subject enrolled in the trial will be randomized to receive a single IV dose of 0 (placebo) or 25 μg/m2 bryostatin. A total of 15 subjects (5 in the placebo arm and 10 in the treatment arm) will be enrolled in the study. The study consists of screening evaluations and on study evaluations divided into two segments, an inpatient segment and an outpatient segment. The four-day inpatient segment will consist of baseline evaluations and a 1-hour IV infusion of study drug followed by evaluations at multiple evaluations over the first 72 hrs post dose. During the outpatient segment, patients will be followed for AEs and have a final evaluation at 2 weeks post dose and a 4-week telephone safety follow up. Evaluations will include safety, efficacy, pharmacokinetics, and pharmacodynamics as assessed by PKC activity
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Bryostatin 1 | Active comparator | Drug: Bryostatin 1single dose of 25 μg/m2 bryostatin, intravenous infusion over 1 hour |
| placebo | Placebo comparator | Drug: Placebosingle dose of placebo, intravenous infusion over 1 hour |
Who may qualify
- Ages
- 50 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Male or female, age 50 - 85 yrs. Females are non-childbearing potential
- Patient must have a cognitive deficit present for at least 1 year and meet diagnostic criteria for probable Alzheimer's Disease Dementia by NIA-AA criteria or prodromal Alzheimer's Disease
- Mini Mental State Exam score of 16-26
- Ability to walk, at least with an assistive device
- Vision and hearing sufficient to comply with testing
Key exclusion criteria (17 listed)
- Dementia due to any condition other than AD, including vascular dementia
- Significant neuroimaging abnormalities, previously known or discovered on screening MRI scan,
- Evidence of clinically significant unstable cardiovascular, renal, hepatic, gastrointestinal, neurological, or metabolic disease within the past 6 months
- Use of any drug within 14 days prior to randomization unless the dose of the drug and the condition being treated have been stable for at least 30 days and are expected to remain stable during the study
- Use of tobacco products or nicotine-containing products within 3 months before Day 1
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 125 μg/m2 bryostatin 1, single dose via intravenous infusion over 1 hour. | Drug | Bryostatin 1 |
| PlaceboPlacebo, single dose via intravenous infusion over 1 hour. | Drug | placebo |
Primary outcome
Number of Participants With Adverse Events as a Measure of Safety and Tolerability
Time frame: Within 2 weeks of study drug dosing
Evaluate the safety and tolerability of bryostatin 1 (hereinafter referred to as bryostatin) in patients with Alzheimer's Disease (AD) following a single intravenous (IV) dose.
Preliminary Efficacy of a Single Dose of Bryostatin in the Treatment of Patients With AD
Time frame: 48 hours post start of study drug infusion
Hopkins Verbal Learning Test - Revised (HVLT-R) delayed recall; change from baseline. HVLT consists of a 12-item word list drawn from 3 semantic categories, presented in 3 learning trials. Score range = 0-12. The lower the number, the more impaired. Repeatable Battery of Assessments for Neuropsychological Status (RBANS) figure recall; change from baseline. Total Score Range: 0-20. Each portion of the drawing is scored 1 point for correctness and completeness and 1 point for being placed properly in relation to the rest of the drawing. Drawing and placement scores are summed for the item total. To obtain subtest total score, the drawing and placement scores are summed for each item. The lower the number, the more impaired.
1 secondary outcome
Preliminary Efficacy of a Single Dose of Bryostatin in the Treatment of Patients With AD
Time frame: Specified timepoints within 2 weeks post study drug infusion
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Nov 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Hakop Gevorkyan, MD, MBA principal investigator
California Clinical Trials Medical Group
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Bryostatin 1 in people with Alzheimer's Disease, run by Neurotrope Bioscience, Inc. at 1 site 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 17 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: stopped early, as of the sponsor's last update on 6 Nov 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 21 Apr 2016). 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?
Number of Participants With Adverse Events as a Measure of Safety and Tolerability (time frame: Within 2 weeks of study drug dosing). 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 Nov 2017.