NCT01882010

Leukine (Sargramostim) for Parkinson's Disease

Completed Phase 1 Last updated 6 October 2023

What this trial tests

Phase 1 trial testing sargramostim in Parkinson's Disease in 37 participants. Completed in 1 January 2016.

Timeline

1 September 2013

Primary endpoint
1 January 2016

1 January 2016

Quick facts

Lead sponsor	University of Nebraska
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	triple
Primary purpose	treatment
Enrollment	37
Start date	1 September 2013
Primary completion	1 January 2016
Estimated completion	1 January 2016
Sites	2 locations across United States

Drugs / interventions tested

• sargramostim (SARGRAMOSTIM) — full drug profile →
• placebo
• Sham Comparator: Controls

Conditions studied

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

Sponsor

University of Nebraska

Who can join

Adults 35 to 85, any sex, with Parkinson's Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

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

1. Inflammation and immune dysfunction in Parkinson disease.
   Tansey MG, Wallings RL, Houser MC, Herrick MK, et al · · 2022 · cited 895× · PMID 35246670 · DOI 10.1038/s41577-022-00684-6
2. Microglial phenotypes in Parkinson's disease and animal models of the disease.
   Joers V, Tansey MG, Mulas G, Carta AR. · · 2017 · cited 221× · PMID 27107797 · DOI 10.1016/j.pneurobio.2016.04.006
3. Evaluation of the safety and immunomodulatory effects of sargramostim in a randomized, double-blind phase 1 clinical Parkinson's disease trial.
   Gendelman HE, Zhang Y, Santamaria P, Olson KE, et al · · 2017 · cited 106× · PMID 28649610 · DOI 10.1038/s41531-017-0013-5
4. Harnessing regulatory T cell neuroprotective activities for treatment of neurodegenerative disorders.
   Machhi J, Kevadiya BD, Muhammad IK, Herskovitz J, et al · · 2020 · cited 96× · PMID 32503641 · DOI 10.1186/s13024-020-00375-7
5. Dual destructive and protective roles of adaptive immunity in neurodegenerative disorders.
   Anderson KM, Olson KE, Estes KA, Flanagan K, et al · · 2014 · cited 68× · PMID 25671101 · DOI 10.1186/2047-9158-3-25
6. Immunotherapies for Neurodegenerative Diseases.
   Mortada I, Farah R, Nabha S, Ojcius DM, et al · · 2021 · cited 62× · PMID 34163421 · DOI 10.3389/fneur.2021.654739
7. Regulatory T cells in ischemic stroke.
   Wang HY, Ye JR, Cui LY, Chu SF, et al · · 2022 · cited 53× · PMID 33772140 · DOI 10.1038/s41401-021-00641-4
8. Immunomodulation as a neuroprotective and therapeutic strategy for Parkinson's disease.
   Olson KE, Gendelman HE. · · 2016 · cited 47× · PMID 26571205 · DOI 10.1016/j.coph.2015.10.006