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

NCT01409915 | Completed Phase 2 Results DMC

• 1 other identifier: 12-1273
• Study Type: interventional
• Target: 44
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Alzheimer's Disease

Keywords

Alzheimer's disease; neuropsychological assessment; Granulocyte-Macrophage Colony-Stimulating Factor; Leukine

Outcome Measures

Primary Outcomes (1)

• Adverse Events (AEs) by Body System

  Count of AE's from Consent to Follow-up 2 within a safety analysis set consisting of all participants who were enrolled and randomized and who received at least one injection of sargramostim or placebo

  20 weeks (From Consent to Follow-up 2)

Secondary Outcomes (2)

• MMSE (Mini Mental State Examination) From Baseline to End of Treatment (3 Weeks), Follow-Up 1 (45 Days Post Treatment) and Follow-Up 2 (90 Days Post Treatment)

  From Baseline to End of Treatment (3 weeks), Follow-Up 1 (45 days post treatment) and Follow-Up 2 (90 days post treatment)

• Alzheimer's Disease Assessment Scale - Cognitive Subscale (ADAS-Cog13) From Baseline to End of Treatment (3 Weeks), Follow-Up 1 (45 Days Post Treatment) and Follow-Up 2 (90 Days Post Treatment)

  Baseline to End of Treatment (3 weeks), Follow-Up 1 (45 days post treatment) and Follow-Up 2 (90 days post treatment)

Other Outcomes (3)

• Alzheimer's Disease Cooperative Study -Activities of Daily Living Inventory (ADCS-ADL) From Baseline to End of Treatment (3 Weeks), Follow-Up 1 (45 Days Post Treatment) and Follow-Up 2 (90 Days Post Treatment)

  Baseline to End of Treatment (3 weeks), Follow-Up 1 (45 days post treatment) and Follow-Up 2 (90 days post treatment)

• Clinical Dementia Rating Scale - Sum of Boxes (CDR-SB) From Baseline to End of Treatment (3 Weeks), Follow-Up 1 (45 Days Post Treatment) and Follow-Up 2 (90 Days Post Treatment)

  Baseline to End of Treatment (3 weeks), Follow-Up 1 (45 days post treatment) and Follow-Up 2 (90 days post treatment)

• Trail Making Test - Part A (TMT-A) From Baseline to End of Treatment (3 Weeks), Follow-Up 1 (45 Days Post Treatment) and Follow-Up 2 (90 Days Post Treatment)

  Baseline to End of Treatment (3 weeks), Follow-Up 1 (45 days post treatment) and Follow-Up 2 (90 days post treatment)

Study Arms (2)

Sagramostim (Leukine)

EXPERIMENTAL

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

Control Group

PLACEBO COMPARATOR

Saline -- placebo comparator. Given as a subcutaneous injection.

Drug: Saline -- placebo comparator

Interventions

Sagramostim DRUG

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

Also known as: Leukine, Granulocyte-Macrophage Colony-Stimulating Factor

Sagramostim (Leukine)

Saline -- placebo comparator DRUG

subcutaneous injection

Also known as: Sterile solution of sodium chloride in water

Control Group

Eligibility Criteria

• Age: 55 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• age 55 to 85 years;
• should have a mild-to-moderate AD diagnosis (MMSE 10-26 inclusive);
• 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.
• if on anti-dementia treatment should be on stable treatment for at least 2 months (i.e. cholinesterase inhibitor and/or Memantine or Axona);
• stable on all other medications for at least 30 days prior to screen;
• should be fluent in English;
• should be physically able to participate by medical history, clinical exam and tests;
• should have a study partner to accompany them to scheduled visits.

You may not qualify if:

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

Sponsors & Collaborators

• University of Colorado, Denver: lead
• The Dana Foundation: collaborator

Study Sites (1)

University of Colorado Denver, Anschutz Medical Campus

Aurora, Colorado, 80045, United States

Location

MeSH Terms

Conditions

Alzheimer Disease

Interventions

Colony-Stimulating Factors; sargramostim; Granulocyte-Macrophage Colony-Stimulating Factor; Water

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors; Hydroxides; Alkalies; Inorganic Chemicals; Anions; Ions; Electrolytes; Oxides; Oxygen Compounds

Limitations and Caveats

The main weakness is the small sample size of the study.

Results Point of Contact

• Title: John O'Shaughnessy, MS, Clinical Research Services Principal Professional
• Organization: University of Colorado Denver | Anschutz Medical Campus

john.oshaughnessy@cuanschutz.edu

3037247924

Study Officials

• Huntington Potter, PhD

  University of Colorado, Denver

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 2, 2011
• First Posted: August 4, 2011
• Study Start: March 1, 2011
• Primary Completion: December 9, 2019
• Study Completion: December 9, 2019
• Last Updated: June 2, 2021
• Results First Posted: March 23, 2021

Record last verified: 2021-05

Locations

US (1)