Study to Evaluate the Preliminary Safety, Efficacy, PK and PD of Bryostatin 1 in Patients With Alzheimer's Disease

NCT02221947 | Terminated Phase 1 Results

• 1 other identifier: NTRP101-201
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief 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).

Conditions

Alzheimer's Disease

Keywords

Alzheimer's; bryostatin; PKC epsilon

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  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

  Specified timepoints within 2 weeks post study drug infusion

Other Outcomes (1)

• Pharmacokinetic Parameters of Bryostatin.

  Bryostatin plasma concentration pre-dose and at 15 min, 30 min, 1 hr, 1.5 hr, 2hr, 3hr and 6rs post dose.

Study Arms (2)

Bryostatin 1

ACTIVE COMPARATOR

single dose of 25 μg/m2 bryostatin, intravenous infusion over 1 hour

Drug: Bryostatin 1

placebo

PLACEBO COMPARATOR

single dose of placebo, intravenous infusion over 1 hour

Drug: Placebo

Interventions

Bryostatin 1 DRUG

25 μg/m2 bryostatin 1, single dose via intravenous infusion over 1 hour.

Bryostatin 1

Placebo DRUG

Placebo, single dose via intravenous infusion over 1 hour.

placebo

Eligibility Criteria

• Age: 50 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• Consistent caregiver to accompany patient to visits
• Sufficient basic education to be able to complete the cognitive assessments
• Living outside an institution

You may not qualify if:

• 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
• 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;

Sponsors & Collaborators

• Neurotrope Bioscience, Inc.: lead
• Blanchette Rockefeller Neurosciences Insitute: collaborator

Study Sites (1)

California Clinical Trials Medical Center

Glendale, California, 91206, United States

Location

MeSH Terms

Conditions

Alzheimer Disease

Interventions

bryostatin 1

Condition Hierarchy (Ancestors)

Dementia; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Tauopathies; Neurodegenerative Diseases; Neurocognitive Disorders; Mental Disorders

Results Point of Contact

• Title: Vice President, Regulatory Affairs
• Organization: Neurotrope BioScience, Inc

dcrockford@neurotropebioscience.com

973-826-5109

Study Officials

• Hakop Gevorkyan, MD, MBA

  California Clinical Trials Medical Group

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 2, 2014
• First Posted: August 21, 2014
• Study Start: June 1, 2014
• Primary Completion: December 1, 2014
• Study Completion: December 1, 2014
• Last Updated: November 6, 2017
• Results First Posted: April 21, 2016

Record last verified: 2017-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)