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| Name | Class |
|---|---|
| Partner Therapeutics, Inc. | INDUSTRY |
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Investigators will evaluate the safety of a 48 week regimen of Leukine administered as a weight-based dose at 3 ug/kg/ day for 5 days followed by a 2-day holiday. This 48 week long study will extend the prior biomarker evaluations observed in a previous study. Clinical signs and symptoms will be measured by personal well-being, physical, and neurological examinations (UPDRS Parts I, II, III, and IV assessments) and blood tests (CBC with differential, total T cell count, and a comprehensive metabolic sera panel). Leukapheresis will be performed to collect large numbers of immune cells for biomarker testing and immune phenotyping. Additionally, the investigators will determine whether immune deficits of PD are consistent during baseline data collection, and the potential Leukine-induced motor control and mobility improvements will be determined by UPDRS part I, II, III, and IV scores off treatment and on treatment.
Primary Objectives:
There are two main study goals. First, the investigators will determine the safety of a 48 week regimen of Leukine administered as a weight-based dose at 3 µg/kg/day for 5 days, followed by a 2-day holiday. This 48 weeks (n=10) pilot study will continue to assess the safety of Leukine for treatment of Parkinson's disease (PD). Clinical signs and symptoms will be measured by personal well-being, physical, and neurological examinations (UPDRS Parts I, II, III, and IV assessments) and blood tests (CBC with differential, total T cell count, and a comprehensive metabolic sera panel). Due to fragility of the patient population and prior recorded adverse events the proposed dose reductions are justified. Second, in future preparations for an intended broader number of patient enrollments, the investigators wish to functionally examine any or all "putative" relevant biomarker assessments.
Secondary Objectives:
The investigators will examine over a time of 48 weeks, effects of treatment on defined adaptive immune deficits in PD as measured by analysis of peripheral blood mononuclear cells collected before and during Leukine therapy. The investigators will assess the individual T cell parameters that include links between T cell function and subset analyses and clinical neurological signs and symptoms. These immune parameters will be serially examined as they may contribute to the immune deficits in PD. Thus, timed analyses of changes in T cell phenotypes and/or function will be completed. In addition, the investigators will assess the functional stability of the immune deficits in PD. To this end, the investigators will examine T cell subsets in PD patients in this study against prior results. The investigators will determine whether the immune deficits of PD are consistent during baseline data collection. The investigators will evaluate the potential Leukine-induced motor control and mobility improvements by assessing UPDRS part I, II, III, and IV scores of treatment and on treatment. Additionally, over the course of this 48 week treatment study, investigators will also be assessing various biomarkers within plasma, peripheral blood, and the total lymphocyte and monocyte populations isolated from leukapheresis before Leukine therapy and after 24 and 48 weeks of treatment. Identified biomarkers will be correlated to disease severity and progression as assessed by UPDRS. Serum will also be collected to determine the generation of anti-drug neutralizing antibodies that may develop due to the dosing scheme.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Leukine Treatment | Experimental | 48 week regimen of Leukine administered as a weight-based dose at 3 µg/kg/day for 5 days (week), followed by a 2-day holiday (weekend) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Sargramostim | Drug | Recombinant human GM-CSF produced by recombinant DNA technology using a yeast (S. cerevisiae) expression system |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in incidence of adverse events over time | The safety of Leukine administration in PD will be examined by documenting abnormal results from complete blood count (CBC) with differential, total T cell count, or comprehensive metabolic panel analyses; abnormal physical and or neurological exam findings; abnormal levels of antibodies to Granulocyte-macrophage colony-stimulating factor (GM-CSF); clinically increasing Unified Parkinson's disease Rating Scale (UPDRS) part I, II, III, and IV scores as determined by the examining physician; and other adverse events. Adverse event logs will be reported every 4 weeks. | every 6 months during the course of treatment, up to 48 weeks until drug cessation |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Treatment Biomarkers over time | During the baseline visit and at 24 weeks and 48 weeks post-Leukine initiation, subjects will undergo leukapheresis to collect large amounts of monocytes and lymphocytes for biomarker evaluations. Cells will be harvested and subjected to proteomic and transcriptomic tests to assess therapeutic response and treatment-induced biomarkers. Biomarkers assessed will be proteins levels along with corresponding gene levels determined as fold change from baseline. |
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Inclusion Criteria:
Onset of bradykinesia and 1 or both of the following: rest tremor and/or rigidity
Asymmetric onset of clinical signs
Progressive motor symptoms
Age at onset 35-85 years
Duration of PD symptoms of at least 3 years
Female subjects must be either:
Must be stage 4 or less according to the Hoehn and Yahr scale
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Howard E Gendelman, MD | University of Nebraska | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Nebraska Medical Center | Omaha | Nebraska | 68198 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 34000620 | Background | Olson KE, Namminga KL, Lu Y, Schwab AD, Thurston MJ, Abdelmoaty MM, Kumar V, Wojtkiewicz M, Obaro H, Santamaria P, Mosley RL, Gendelman HE. Safety, tolerability, and immune-biomarker profiling for year-long sargramostim treatment of Parkinson's disease. EBioMedicine. 2021 May;67:103380. doi: 10.1016/j.ebiom.2021.103380. Epub 2021 May 14. | |
| 35802825 |
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No plan to share individual participant data with other researchers.
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| Type | Date | Date Unknown |
|---|---|---|
| Release | Nov 11, 2025 | |
| Reset | Nov 21, 2025 | |
| Release | Nov 26, 2025 | |
| Reset | Dec 12, 2025 | |
| Release | Jan 19, 2026 | |
| Reset | Feb 4, 2026 | |
| Release | Mar 4, 2026 | |
| Reset | Mar 25, 2026 | |
| Release | May 12, 2026 | |
| Reset | Jun 8, 2026 |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| ICF | No | No | Yes | Informed Consent Form | Feb 16, 2023 | Oct 31, 2025 | ICF_000.pdf |
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| Release Date | Unrelease Date | Unrelease Date Unknown | Reset Date | MCP Release Number |
|---|---|---|---|---|
| Nov 11, 2025 | Nov 21, 2025 | |||
| Nov 26, 2025 |
| ID | Term |
|---|---|
| D010300 | Parkinson Disease |
| D020734 | Parkinsonian Disorders |
| ID | Term |
|---|---|
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
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| ID | Term |
|---|---|
| C081222 | sargramostim |
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| every 6 months during the course of treatment, up to 48 weeks until drug cessation |
| Abdelmoaty MM, Machhi J, Yeapuri P, Shahjin F, Kumar V, Olson KE, Mosley RL, Gendelman HE. Monocyte biomarkers define sargramostim treatment outcomes for Parkinson's disease. Clin Transl Med. 2022 Jul;12(7):e958. doi: 10.1002/ctm2.958. |
| 28649610 | Background | Gendelman HE, Zhang Y, Santamaria P, Olson KE, Schutt CR, Bhatti D, Shetty BLD, Lu Y, Estes KA, Standaert DG, Heinrichs-Graham E, Larson L, Meza JL, Follett M, Forsberg E, Siuzdak G, Wilson TW, Peterson C, Mosley RL. Evaluation of the safety and immunomodulatory effects of sargramostim in a randomized, double-blind phase 1 clinical Parkinson's disease trial. NPJ Parkinsons Dis. 2017 Mar 23;3:10. doi: 10.1038/s41531-017-0013-5. eCollection 2017. |
| Dec 12, 2025 |
| Jan 19, 2026 | Feb 4, 2026 |
| Mar 4, 2026 | Mar 25, 2026 |
| May 12, 2026 | Jun 8, 2026 |
| Jun 22, 2026 |
| D009069 | Movement Disorders |
| D000080874 | Synucleinopathies |
| D019636 | Neurodegenerative Diseases |