NCT02221947

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

Terminated Phase 1/Phase 2 Results posted Last updated 3 October 2017

What this trial tests

Phase 1/Phase 2 trial testing Bryostatin 1 in Alzheimer's Disease in 9 participants. Terminated before completion.

Timeline

1 June 2014

Primary endpoint
1 December 2014

1 December 2014

Quick facts

Lead sponsor	Neurotrope Bioscience, Inc.
Phase	Phase 1/Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	randomized
Design	single group
Masking	quadruple
Primary purpose	treatment
Enrollment	9
Start date	1 June 2014
Primary completion	1 December 2014
Estimated completion	1 December 2014
Sites	1 location across United States

Drugs / interventions tested

• Bryostatin 1 — full drug profile →
• Placebo

Conditions studied

• Alzheimer's Disease — all drugs for Alzheimer's Disease →

Sponsor

Neurotrope Bioscience, Inc. — full company profile →

Who can join

Adults 50 to 85, any sex, with Alzheimer's Disease. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• 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

Sponsor's own description

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).

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

1. Marine Natural Products in Clinical Use.
   Haque N, Parveen S, Tang T, Wei J, et al · · 2022 · cited 101× · PMID 36005531 · DOI 10.3390/md20080528
2. Bryostatin Effects on Cognitive Function and PKCɛ in Alzheimer's Disease Phase IIa and Expanded Access Trials.
   Nelson TJ, Sun MK, Lim C, Sen A, et al · · 2017 · cited 80× · PMID 28482641 · DOI 10.3233/jad-170161
3. New Drugs from Marine Organisms in Alzheimer's Disease.
   Russo P, Kisialiou A, Lamonaca P, Moroni R, et al · · 2015 · cited 41× · PMID 26712769 · DOI 10.3390/md14010005
4. HMGB1 signaling phosphorylates Ku70 and impairs DNA damage repair in Alzheimer's disease pathology.
   Tanaka H, Kondo K, Fujita K, Homma H, et al · · 2021 · cited 26× · PMID 34635772 · DOI 10.1038/s42003-021-02671-4
5. Protein kinases in neurodegenerative diseases: current understandings and implications for drug discovery.
   Wu X, Yang Z, Zou J, Gao H, et al · · 2025 · cited 18× · PMID 40328798 · DOI 10.1038/s41392-025-02179-x
6. Application of Marine Natural Products against Alzheimer's Disease: Past, Present and Future.
   Hu D, Jin Y, Hou X, Zhu Y, et al · · 2023 · cited 18× · PMID 36662216 · DOI 10.3390/md21010043
7. Protein Kinase C Family: Structures, Biological Functions, Diseases, and Pharmaceutical Interventions.
   Li Y, Jiang Y, Hu Z, Yang S, et al · · 2025 · cited 3× · PMID 41244006 · DOI 10.1002/mco2.70474