Parkinson's Disease and ParkinsonismPhase 1NCT05677633
Biomarker Validation Following Sargramostim Treatment in Parkinson's Disease
A Phase 1 study testing Sargramostim in people with Parkinson's Disease and Parkinsonism, run by University of Nebraska at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 35 to 85 years
- Conditions the study is for
- Parkinson's Disease and Parkinsonism
- What is being tested?
- Sargramostim
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 1 siteUnited States
- Study dates
- 19 Jan 2023to 1 Nov 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 35 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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:
Show all 10Show fewer
- Not pregnant, not breastfeeding, and not planning on becoming pregnant during the study;
- Not of childbearing potential, defined as one who has been postmenopausal for at least 1 year and with follicle stimulating hormone (FSH) levels in the laboratory defined postmenopausal range, or has been surgically sterilized, or has had a hysterectomy at least 3 months prior to the start of this trial; or
- If of childbearing potential, must agree to use an effective method of avoiding pregnancy to the end of the trial and must have a negative serum beta-human chorionic gonadotropin (β-HCG) test. Effective methods of avoiding pregnancy are contraceptive methods used consistently and correctly (including implantable contraceptives, injectable contraceptives, oral contraceptives, transdermal contraceptives, intrauterine devices, diaphragm with spermicide, male or female condoms with spermicide, or cervical cap), abstinence, or a sterile sexual partner.
- Must be stage 4 or less according to the Hoehn and Yahr scale
Who cannot join
- Atypical features indicative of a Parkinson-Plus disorder (Progressive Supranuclear Palsy (PSP), Multiple System Atrophy (MSA), Corticobasal Degeneration (CBD)) including cerebellar signs, supranuclear gaze palsy, apraxia and other cortical signs, or prominent autonomic failure
- Neuroleptic treatment at time of onset of parkinsonism
- Active treatment with a neuroleptic at time of study entry
- History of repeated strokes with stepwise progression of parkinsonism
- History of repeated head injury
- History of definite encephalitis
Show all 25Show fewer
- More than one blood relative diagnosed with PD
- Prominent gait imbalance early in the course (< 5 years)
- Mini-mental state examination score <26
- Hematological malignancy or coagulopathy
- Abnormal blood analyses: hematocrit <30; white blood cell count >11.5; clinically significant laboratory data (e.g. alanine aminotransferase (ALT) or aspartate aminotransferase (AST) 3x the upper limit of normal (ULN), or any abnormal laboratory value that could interfere with the assessment of safety in the judgment of the investigator; significant abnormalities on the clinical examination, vital signs, and clinical chemistry or hematology results (excluding findings of Parkinson's disease), that may interfere with the study or present a safety risk for the subject as judged by the clinical investigator charged in the care of study participants
- Serious medical illness or co-morbidity that may interfere with participation in the study
- Brain surgery for parkinsonism (DBS, cell implantation, gene therapy)
- History of an autoimmune disorder or systemic inflammatory disorder deemed significant by physician
- Immunostimulatory or immunosuppressive treatment (including amphetamines or systemic corticosteroids) within 90 days
- Exclusively unilateral parkinsonism for longer than 3 years
- Known hypersensitivity to granulocyte-macrophage colony-stimulating factor (GM-CSF), yeast-derived products
- Current lithium treatment
- Individuals with current diagnoses of alcohol or substance abuse/dependence
- Anyone who is not appropriate for participation in this research protocol as deemed by the principal or co-investigator
- Anyone who has previously been treated with granulocyte-macrophage colony-stimulating factor (GM-CSF) as an immunomodulatory therapy
- Anyone with poor venous access
- Anyone who has any illnesses or events that would cause a neurological abnormality, apart from Parkinson's disease.
- Subjects with allergies or sensitivities to yeast products.
- Subjects that have received a flu shot within the past 3 weeks.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Nebraska Medical Center
Omaha, Nebraska, 68198
About this study
Official title: A Phase 1, Open-label Study to Validate Treatment-induced Biomarkers Following Sargramostim Treatment in Parkinson's Disease
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.
Read the detailed description
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.
- Conditions
- Parkinson's Disease and Parkinsonism
- Being tested
- Sargramostim
- Keywords
- Leukine, sargramostim, biomarker
- Medical subject headings
- Parkinson Disease, Parkinsonian Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SargramostimDrug
Also called Leukine
Recombinant human GM-CSF produced by recombinant DNA technology using a yeast (S. cerevisiae) expression system
| Study group | Role | What they receive |
|---|---|---|
| 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) Receives: Drug: Sargramostim |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: every 6 months during the course of treatment, up to 48 weeks until drug cessation
Secondary outcomes (1)
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.
Measured: every 6 months during the course of treatment, up to 48 weeks until drug cessation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jan 2023
- Study start19 Jan 2023
- Primary completion12 Apr 2024
- Study completion1 Nov 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Parkinson's Disease and ParkinsonismStudies of the same condition, open and under way.
- More trials run by University of NebraskaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Nebraska
- Sponsor type
- University, hospital or other
- Collaborators
- Partner Therapeutics, Inc.
- Responsible party
- Sponsor
- Investigators
- Howard E Gendelman, MD, Principal investigator, University of Nebraska
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05677633
- Sponsor's protocol ID
- 0748-22-FB
- Status last verified
- Oct 2025
- First submitted
- 15 Dec 2022
- Last updated
- 4 Nov 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: No plan to share individual participant data with other researchers.
Publications
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.
PubMed 34000620Abdelmoaty 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.
PubMed 35802825Gendelman 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.
PubMed 28649610
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Open-label Study to Validate Treatment-induced Biomarkers Following Sargramostim Treatment in Parkinson's Disease
- ClinicalTrials.gov ID
- NCT05677633
- Acronym / sponsor ID
- 0748-22-FB
- Conditions
- Parkinson's Disease and Parkinsonism
- Keywords
- Leukine, sargramostim, biomarker
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- University of NebraskaUniversity, hospital or other
- Collaborators
- Partner Therapeutics, Inc.
- Study start
- 19 Jan 2023 (actual)
- Primary completion
- 12 Apr 2024 (actual)
- Study completion
- 1 Nov 2024 (actual)
- First posted
- 10 Jan 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Leukine Treatment | Experimental | Drug: Sargramostim48 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) |
Who may qualify
- Ages
- 35 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- 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
Key exclusion criteria (25 listed)
- Atypical features indicative of a Parkinson-Plus disorder (Progressive Supranuclear Palsy (PSP), Multiple System Atrophy (MSA), Corticobasal Degeneration (CBD)) including cerebellar signs, supranuclear gaze palsy, apraxia and other cortical signs, or prominent autonomic failure
- Neuroleptic treatment at time of onset of parkinsonism
- Active treatment with a neuroleptic at time of study entry
- History of repeated strokes with stepwise progression of parkinsonism
- History of repeated head injury
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SargramostimAlso: LeukineRecombinant human GM-CSF produced by recombinant DNA technology using a yeast (S. cerevisiae) expression system | Drug | Leukine Treatment |
Primary outcome
Change in incidence of adverse events over time
Time frame: every 6 months during the course of treatment, up to 48 weeks until drug cessation
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.
1 secondary outcome
Change in Treatment Biomarkers over time
Time frame: every 6 months during the course of treatment, up to 48 weeks until drug cessation
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Nov 2025.
- Background: PubMed 34000620Olson 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.
- Background: PubMed 35802825Abdelmoaty 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.
- Background: PubMed 28649610Gendelman 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.
- Individual participant dataSharing planned: No. No plan to share individual participant data with other researchers.
Related trials for Parkinson's Disease and Parkinsonism
Enrolling by invitationObservational study
Early Molecular Biomarkers for Differentiating Parkinsonian Syndromes
PARKINSON DISEASE (Disorder), Atypical Parkinsonism
Active, not recruitingPhase 1/2
Clinical Safety and Efficacy of Umbilical Cord Mesenchymal Stromal Cells-derived Exosomes in Patients With Parkinson's Disease
Parkinson Disease (PD)
Active, not recruitingObservational study
Adaptive DBS to Offset STN Dysregulation During Exercise in PD
Parkinson Disease
Not yet recruiting
Compulsive Eating and Brain's Reward System in Different Pathologies
PARKINSON DISEASE (Disorder), Bipolar 1 Disorder
Not yet recruiting
Evaluating the Effect of the Zeen Mobility Device on Clinical Practicality, Safety, and Impact (Via Assessments of Walking, Standing Tolerance, Balance, Endurance, Effort, and Participation in Daily Tasks) in an Outpatient Rehabilitation Setting for Individuals With Mobility Impairments.
Stroke, Parkinson's Disease (PD)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Howard E Gendelman, MD principal investigator
University of Nebraska
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Sargramostim in people with Parkinson's Disease and Parkinsonism, run by University of Nebraska at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 25 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in incidence of adverse events over time (time frame: every 6 months during the course of treatment, up to 48 weeks until drug cessation). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05677633, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Nov 2025.