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

Neurotrope Bioscience

Status and phase

Terminated

Phase 2

Phase 1

Conditions

Alzheimer's Disease

Treatments

Drug: Placebo

Drug: Bryostatin 1

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT02221947

NTRP101-201

Details and patient eligibility

About

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).

Full 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

Enrollment

9 patients

Sex

All

Ages

50 to 85 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

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

Exclusion criteria

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;
Acute or poorly controlled medical illness
Likelihood, according to clinical judgment, of being transferred to a nursing home within 6 months