Leukine (Sargramostim) for Parkinson's Disease

Summary

The purpose of this study is to determine if Leukine (sargramostim) can be safely administered to Parkinson's disease patients for an extended period of time (56 days) and restore immune deficits seen in Parkinson's patients compared to controls. The development of magnetoencephalography (MEG) as a monitoring tool for PD will also be explored. At enrollment and repeating again at two 4-week intervals, whole blood from PD patients and controls will be obtained for analyses and the results will be used to calculate immune response profiles as a baseline for comparison after drug treatment. Physical examinations and motor assessments will also be performed on PD patients. After the 8-week baseline data collection, control participation will end and drug treatment of PD patients will begin. PD patients will be randomized, and half will receive drug and half will receive placebo. Leukine at a dosage of 6 µg/kg or saline as placebo will be administered by subcutaneous injection daily for 56 days (8 weeks). During drug treatment, PD patients will be monitored every two weeks by physical examinations, motor assessments, and blood analyses. As follow-up, four weeks after drug administration has stopped, subjects will again have physical examinations, motor assessments, and blood analyses. MEG will be performed on PD patients and controls at the start of drug treatment, and on PD patients at the end of the drug treatment period and 4 weeks after drug is stopped. In addtion, at the second cohort of 8 PD subjects, we will evaluate the potential Leukine-induced motor control and mobility improvements. Also, levels of the neurotransmitters glutamate, glutamine, serotonin, acetylcholine, GABA, norepinephrine and epinephrine in serum/plasma will be analyzed to correlate with changes in motor function and drug treatment.

Conditions

• Parkinson's Disease

Interventions

DRUG

sargramostim

lyophilized 250 micrograms/vial 6 micrograms/kg daily subcutaneous injection 56 days

DRUG

placebo

saline solution daily subcutaneous injection 56 days

DIAGNOSTIC_TEST

Sham Comparator: Controls

Caregivers, spouse, friends, relatives of PD patients, have blood draws, MEG.

Sponsors & Collaborators

• Sanofi

  collaborator INDUSTRY

• National Institute of Neurological Disorders and Stroke (NINDS)

  collaborator NIH

• Nebraska Neuroscience Alliance

  collaborator UNKNOWN

• University of Nebraska

  lead OTHER

Principal Investigators

• Howard E Gendelman, MD · University of Nebraska

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Model

PARALLEL

Eligibility

Min Age

35 Years

Max Age

85 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2013-09-01

Primary Completion

2016-01-01

Completion

2016-01-01

FDA Drug

Yes

Countries

• United States

Study Locations