Biomarker Assessments of Leukine During Treatment of Parkinson's Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 27 November 2023
• Main ID: NCT03790670
• Date of registration: 18/12/2018
• Prospective Registration: Yes
• Primary sponsor: University of Nebraska
• Public title: Biomarker Assessments of Leukine During Treatment of Parkinson's Disease
• Scientific title: Safety, Tolerability and Biomarker Assessments of Leukine (Sargramostim) During Extended Timed Treatment for Parkinson's Disease: A Phase I Pilot Study
• Date of first enrolment: January 30, 2019
• Target sample size: 7
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT03790670
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Howard Gendelman, MD
• Affiliation: University of Nebraska

Key inclusion & exclusion criteria

Inclusion Criteria:

- 1. Onset of bradykinesia and 1 or both of the following: rest tremor and/or rigidity

2. Asymmetric onset of clinical signs

3. Progressive motor symptoms

4. Age at onset 35-85 years

5. Duration of PD symptoms of at least 3 years

6. Female subjects must be either:

1. Not pregnant, not breastfeeding, and not planning on becoming pregnant during the
study;

2. 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

3. 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 (ß-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.

7. Must be stage 4 or less according to the Hoehn and Yahr scale

Exclusion Criteria:

- 1. 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

2. Neuroleptic treatment at time of onset of parkinsonism

3. Active treatment with a neuroleptic at time of study entry

4. History of repeated strokes with stepwise progression of parkinsonism

5. History of repeated head injury

6. History of definite encephalitis

7. More than one blood relative diagnosed with PD

8. Prominent gait imbalance early in the course (< 5 years)

9. Mini-mental state examination score <26

10. Hematological malignancy or coagulopathy

11. Abnormal blood analyses: hematocrit <30; WBC>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

12. Serious medical illness or co-morbidity that may interfere with participation in
the study

13. Brain surgery for parkinsonism (DBS, cell implantation, gene therapy)

14. History of an autoimmune disorder or systemic inflammatory disorder deemed
significant by physician

15. Immunostimulatory or immunosuppressive treatment (including amphet-amines or
systemic corticosteroids) within 90 days

16. Exclusively unilateral parkinsonism for longer than 3 years

17. Known hypersensitivity to GM-CSF, yeast-derived products

18. Current lithium treatment

19. Individuals with current diagnoses of alcohol or substance abuse/dependence

20. Anyone who is not appropriate for participation in this research protocol as
deemed by the principal or co-investigator

21. Anyone who has previously been treated with GM-CSF as an immunomodulatory therapy

22. Anyone with poor venous access



Age minimum: 35 Years
Age maximum: 85 Years
Gender: All

Health Condition(s) or Problem(s) studied

Parkinson Disease

Intervention(s)

Drug: sargramostim

Primary Outcome(s)

Incidence of Treatment-Associated Adverse Events [Time Frame: monthly during the course of treatment, up to 36 months, followed by 1 month drug cessation]

Secondary Outcome(s)

Determination of Immune Cell Number [Time Frame: 36 months of treatment, followed by 1 month drug cessation]

Determination of Immune Cell Function [Time Frame: 36 months of treatment, followed by 1 month drug cessation]

Determination of Immune Cell Phenotype [Time Frame: 36 months of treatment, followed by 1 month drug cessation]

Secondary ID(s)

0839-18-FB

Source(s) of Monetary Support

Please refer to primary and secondary sponsors