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Study of the Safety & Efficacy of Leukine® in the Treatment of Alzheimer's Disease

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01409915
First Posted: August 4, 2011
Last Update Posted: May 24, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
The Dana Foundation
Information provided by (Responsible Party):
University of Colorado, Denver
  Purpose
A medicine that is FDA-approved for bone marrow stimulation (called Leukine) will be tested for its ability to be tolerated by Alzheimer's disease patients and potentially to improve their memory.

Condition Intervention Phase
Alzheimer's Disease Drug: Sagramostim Drug: Saline -- placebo comparator Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: Pilot Phase 2 Trial of the Safety & Efficacy of Granulocyte-Macrophage Colony-Stimulating Factor (Leukine®) in the Treatment of Alzheimer's Disease

Resource links provided by NLM:


Further study details as provided by University of Colorado, Denver:

Primary Outcome Measures:
  • Ability of Alzheimer's Disease subjects to tolerate Leukine treatment will be assessed [ Time Frame: Up to 5 months ]
    Various tests of well being and toxicity will be monitored for 3 months after treatment


Secondary Outcome Measures:
  • Ability of Leukine treatment to improve cognition of Alzheimer's Disease subjects [ Time Frame: Up to 5 months ]
    Neuropsychological measures will be assessed at various intervals up to 3 months following treatment (or placebo)


Estimated Enrollment: 40
Study Start Date: March 2011
Estimated Study Completion Date: December 2017
Estimated Primary Completion Date: November 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Sagramostim (Leukine)
5 subjects 250 mcg /m2/day Leukine subcutaneously for 5 days/week for three weeks. Data and Safety Monitoring Board will then review data and recommend whether to continue at the same current recommended dose for additional subjects or to reduce the dose by half if excessive leukocytosis occurs
Drug: Sagramostim
5 subjects 250 mcg /m2/day Leukine subcutaneously for 5 days/week for three weeks. Data and Safety Monitoring Board will then review data and recommend whether to continue at the same current recommended dose for additional subjects or to reduce the dose by half if excessive leukocytosis occurs
Other Names:
  • Leukine
  • Granulocyte-Macrophage Colony-Stimulating Factor
Placebo Comparator: Control Group
Saline -- placebo comparator. Given as a subcutaneous injection.
Drug: Saline -- placebo comparator
subcutaneous injection
Other Name: Sterile solution of sodium chloride in water

Detailed Description:
Preliminary preclinical results demonstrated that GM-CSF (Granulocyte macrophage colony-stimulating factor, e.g. Leukine®/Sargramostim) rapidly reduced cerebral amyloid deposition and completely reversed memory deficits in transgenic mouse models of Alzheimer's Disease (AD). To assess the efficacy of GM-CSF in humans, the investigators performed a retrospective analysis of a cognition study of human patients undergoing hematopoietic cell transplantation for cancer and who garner cognitive impairments from the chemotherapy or irradiation. In the patients that received a colony-stimulating factor (CSF) to stimulate the bone marrow and recover immune system function, the investigators found that those who received GM-CSF (Leukine®/Sargramostim) plus G-CSF (Filigrastim) significantly improved in cognitive function as compared to those who received G-CSF alone. These findings combined with over two decades of accrued safety data using recombinant human GM-CSF, Leukine®/Sargramostim, in elderly leukopenic patients, suggested that Leukine® should be tested as a treatment to reverse cerebral amyloid pathology and cognitive impairment in AD.
  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   55 Years to 85 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. age 55 to 85 years;
  2. should have a mild-to-moderate AD diagnosis (MMSE 10-26 inclusive);
  3. should have evidence of elevated cortical amyloid by PET using florbetapir F18 (Amyvid) [i.e. a positive scan], assessed qualitatively according to the Amyvid product label.
  4. if on anti-dementia treatment should be on stable treatment for at least 2 months (i.e. cholinesterase inhibitor and/or Memantine or Axona);
  5. stable on all other medications for at least 30 days prior to screen;
  6. should be fluent in English;
  7. should be physically able to participate by medical history, clinical exam and tests;
  8. should have a study partner to accompany them to scheduled visits.

Exclusion Criteria:

  1. clinically relevant arrhythmias;
  2. a resting pulse less than 50;
  3. active cancer other than non-melanoma skin cancers;
  4. use of another investigatory drug within 2 months of screening;
  5. significant stroke or head trauma by history or MRI;
  6. contraindication for having a MRI;
  7. diagnostic and Statistical Manual of Mental Disorders-IV criteria for a current major psychiatric disorder;
  8. sensitivity to yeast or yeast products;
  9. impaired kidney function as measured by a Glomerular Filtration Rate less than 60 milliliters/min;
  10. preexisting fluid retention, pulmonary infiltrates, or congestive heart failure;
  11. history of moderate-to-severe lung disease;
  12. history of moderate-to-severe liver disease;
  13. pregnant women, or any women who feel they are likely to become pregnant during the study;
  14. prisoners.
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01409915


Locations
United States, Colorado
University of Colorado Denver, Anschutz Medical Campus
Aurora, Colorado, United States, 80045
Sponsors and Collaborators
University of Colorado, Denver
The Dana Foundation
Investigators
Principal Investigator: Huntington Potter, PhD University of Colorado, Denver
  More Information

Responsible Party: University of Colorado, Denver
ClinicalTrials.gov Identifier: NCT01409915     History of Changes
Other Study ID Numbers: 12-1273
First Submitted: August 2, 2011
First Posted: August 4, 2011
Last Update Posted: May 24, 2017
Last Verified: May 2017

Keywords provided by University of Colorado, Denver:
Alzheimer's disease
neuropsychological assessment
Granulocyte-Macrophage Colony-Stimulating Factor
Leukine

Additional relevant MeSH terms:
Alzheimer Disease
Dementia
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Tauopathies
Neurodegenerative Diseases
Neurocognitive Disorders
Mental Disorders