Phase 2
N=8
Efficacy and Safety of Filgrastim in Alzheimer's Disease
Alzheimer's Disease
Bottom Line
View on ClinicalTrials.gov: NCT01617577 ↗Enrolled (actual)
8
Serious AEs
0.0%
Results posted
Dec 2012
Primary outcome: Primary: Cognitive Measures Incluing ADAScog, Selected CANTABS Tests (Paired Associate Learning (PAL)and PAL ) — 22.25; 22.25; 20.5; 17.75 units on a scale
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- G-CSF; filgrastim (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 55+ yrs
- Sex
- All
- Sponsor
- University of South Florida
- Primary completion
- Feb 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Cognitive Measures Incluing ADAScog, Selected CANTABS Tests (Paired Associate Learning (PAL)and PAL ) |
24; 20.37; 2.85; 5.17; 33.88; 30. | 0.36 |
| PRIMARY Cognitive Measures Incluing ADAScog, Selected CANTABS Tests (Paired Associate Learning (PAL)and PAL ) |
24; 20.37; 2.85; 5.17; 33.88; 30. | 0.36 |
Summary
Filgrastim (G-CSF) is widely used for treatment of patients who have a deficiency of white blood cells. It is also routinely used to stimulate and mobilize stem/progenitor cells for bone marrow transplantation. In studies of thousands of healthy donor subjects treated with G-CSF, the side-effects profile has been reported to be mild and reversible. Currently, G-CSF is under investigation in clinical trials in Germany and the US that aim to enhance recovery from strokes and heart attacks. In animal studies, G-CSF has been observed to improve cognitive performance and to markedly reduce amyloid deposition in hippocampus and entorhinal cortex in a mouse model of Alzheimer's Disease (AD). Since this drug is being used safely in many people throughout the world, the investigators hypothesize that it will also be safe to give to patients with Alzheimer's disease and that it may improve some aspects of memory and thinking. The present pilot study has two goals or objectives: 1) to investigate the effects of a five day schedule of Filgrastim administration on cognitive function and 2) to assess its tolerability and safety in a small group (12 patients) with mild to moderate stage AD. Patients who are eligible for the study will be randomly assigned to one of two groups (n=6 per group). One group will receive a five-day course of Filgrastim injections and the other group of subjects will receive vehicle injections (solution without drug). At the end of the first phase of the study (week 8), the groups will cross over to receive either vehicle or Filgrastim as appropriate. In this way all subjects will have received the active medication by the end of the study. After the study is finished the investigators should know whether or not Filgrastim improves some aspects of thinking and memory. And the investigators should know whether or not it is safe to give this medication to patients with Alzheimer's disease. To ensure that the drug is safe, a Safety Monitoring Committee will oversee the entire study. They will review all laboratory data, including complete blood counts, serum chemistry, EKGs and adverse events.
Eligibility Criteria
Inclusion Criteria
- People with probable AD (by NINDS/ADRDA criteria) who are likely to be testable at the conclusion of the study period, and who do not have concurrent medical conditions or medications that might influence cognitive testing or that would increase the risk of treatment
- The participants will have a Min Mental State Examination score of between 10 and 24
- stable medical condition and stable medications for 3 months prior to screening
- study partner (spouse or caregiver) to accompany patient to all scheduled visits; able to complete baseline assessments
- physically acceptable for this study as confirmed by medical history, physical exam, neurological exam and clinical tests
Exclusion Criteria
- clinically significant cardiac arrhythmia
- history of clinically significant stroke
- use of another investigational drug within 2 months of screening
- current evidence or history in the past 2 years of epilepsy, focal brain lesion, head injury with loss of consciousness or DSM-IV criteria for any major psychiatric disorder including psychosis, major depression, bipolar disorder alcohol or substance abuse; residence in a skilled nursing facility (but patients in assisted living facility are acceptable)
Data sourced from ClinicalTrials.gov (NCT01617577). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.