Intravenous Plasma Treatment for Parkinson's Disease (yFFP)

• ClinicalTrials.gov Identifier: NCT04202757
• Recruitment Status: Completed
• First Posted: December 18, 2019
• Last Update Posted: January 5, 2022
• Sponsor: The Neurology Center
• Collaborator: Carolina Longevity Institute
• Information provided by (Responsible Party): The Neurology Center

Tracking Information

• First Submitted Date: September 21, 2018
• First Posted Date: December 18, 2019
• Last Update Posted Date: January 5, 2022
• Actual Study Start Date: September 14, 2018
• Actual Primary Completion Date: August 7, 2019 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: December 14, 2021)

• Changes in Physician assessment [ Time Frame: Two weeks prior to infusion, at one-three-six months post-infusion ]
  Unified Parkinson Disease Rating Scale (UPDRS)
• Patient assessment [ Time Frame: Two weeks prior to infusion, at one-three-six months post-infusion ]
  Changes in Stanford Presenteeism Scale (SPS)

Original Primary Outcome Measures (submitted: December 13, 2019)

• Changes in laboratory blood-test results of objective serum factors [ Time Frame: Two weeks prior to infusion, at one-three-six months post-infusion ]
  White blood cell and differential count (eosinophils, basophils, neutrophils, lymphocytes and monocytes)
• Changes in laboratory blood-test results of objective serum factors [ Time Frame: Two weeks prior to infusion, at one-three-six months post-infusion ]
  Red blood cell and indices Packed Cell Volume (PCV), Mean Corpuscular Volume (MCV), Mean Corpuscular Hemoglobin (MCH), Mean Corpuscular Hemoglobin Concentration (MCHC)
• Changes in laboratory blood-test results of objective serum factors [ Time Frame: Two weeks prior to infusion, at one-three-six months post-infusion ]
  Hemoglobin
• Changes in laboratory blood-test results of objective serum factors [ Time Frame: Two weeks prior to infusion, at one-three-six months post-infusion ]
  Platelets
• Changes in laboratory blood-test results of objective serum factors [ Time Frame: Two weeks prior to infusion, at one-three-six months post-infusion ]
  immunoglobulin A (IgA), immunoglobulin M (IgM), immunoglobulin G (IgG)
• Changes in laboratory blood-test results of objective serum factors [ Time Frame: Two weeks prior to infusion, at one-three-six months post-infusion ]
  creatinine
• Changes in laboratory blood-test results of objective serum factors [ Time Frame: Two weeks prior to infusion, at one-three-six months post-infusion ]
  C - reactive protein
• Changes in laboratory blood-test results of objective serum factors [ Time Frame: Two weeks prior to infusion, at one-three-six months post-infusion ]
  Sodium, Potassium
• Changes in laboratory blood-test results of objective serum factors [ Time Frame: Two weeks prior to infusion, at one-three-six months post-infusion ]
  Urea
• Changes in laboratory blood-test results of objective serum factors [ Time Frame: Two weeks prior to infusion, at one-three-six months post-infusion ]
  ALT (alanine transaminase), AST (aspartate aminotransferase), GGT (gamma-glutamyl transpeptidase), Bilirubin
• Changes in Physician assessment [ Time Frame: Two weeks prior to infusion, at one-three-six months post-infusion ]
  Unified Parkinson Disease Rating Scale (UPDRS)
• Patient assessment [ Time Frame: Two weeks prior to infusion, at one-three-six months post-infusion ]
  Changes in Stanford Presenteeism Scale (SPS)
• Caregiver assessment [ Time Frame: Two weeks prior to infusion, at one-three-six months post-infusion ]
  Changes in Kingston Caregiver Stress Scale (KCSS)

Current Secondary Outcome Measures (submitted: December 13, 2019)

• Stability of administration [ Time Frame: At the time of infusions, day 1 and day 2 ]
  Adverse events (if any)
• Factors predicting a beneficial response [ Time Frame: After the last participant's final contact at 6 months ]
  Patient Global Impression of change

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Intravenous Plasma Treatment for Parkinson's Disease
• Official Title: Intravenous Young Fresh Frozen Plasma (yFFP) Investigational Treatment for Parkinson's Disease
• Brief Summary: This study will do a preliminary evaluation of a possible treatment for mild-to-moderate Parkinson's disease. Patients will be treated with either transfusions of plasma from young donors. or with placebo transfusions. In the following several months, Parkinson's symptoms will be monitored and compared for the two groups.
• Detailed Description: This is a double-blind placebo controlled trial, designed to evaluate the efficacy of plasma from volunteer donors of ages 18-25. This young fresh frozen plasma (yFFP) will be given intravenously in two doses of 12.5 ml/kg each, with 40-56 hours between doses. In the following 24 weeks, Parkinson's symptoms will be monitored using the short version of the Stanford Presenteeism Scale (SPS-6), and a modified version of the UPDRS scale.
• Study Type: Interventional
• Study Phase: Early Phase 1

Study Design

Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:

Placebo controlled trial

Masking: Triple (Participant, Care Provider, Outcomes Assessor)
Masking Description:

Blinding will be achieved by preparing both yFFP and placebo (0.1% riboflavin in normal saline) solution into identical-sized, masked bags. The riboflavin is added to the saline to achieve indistinguishable color when compared to the yFFP. Physicians who evaluate patients are masked from treatment assignment.

Primary Purpose: Treatment

• Condition: Idiopathic Parkinson Disease

Intervention

• Biological: [21CFR640.30] Plasma from 18 - 25 year old volunteer donors
  12.5 ml/kg intravenous Spectrum Plasma yFFP, per each of two infusions (25 ml/kg total), in combination with ongoing normal standard treatment.
  Other Name: Young Fresh Frozen Plasma (yFFP)
• Other: Saline
  12.5 ml/kg intravenous 0.9% sodium chloride with 0.1% riboflavin , per each of two infusions (25 ml/kg total), in combination with ongoing normal standard treatment.
  Other Name: Normal saline

Study Arms

• Active Comparator: Young Fresh Frozen Plasma (yFFP)
  [21CFR640.30] Plasma from 18 - 25 year old volunteer donors
  Intervention: Biological: [21CFR640.30] Plasma from 18 - 25 year old volunteer donors
• Placebo Comparator: Saline
  0.1% riboflavin in normal saline
  Intervention: Other: Saline

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: December 14, 2021): 22
• Original Actual Enrollment (submitted: December 13, 2019): 18
• Actual Study Completion Date: August 8, 2019
• Actual Primary Completion Date: August 7, 2019 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Diagnosis of Parkinson's disease
• Disease duration of 1 to 5 years
• Willingness to confirm the use of adequate birth control while on the trial will be required in premenopausal women without evidence for an inability to become pregnant.
• Willingness to not start any other treatment for Parkinson's or Multiple Sclerosis during the parallel part of the trial.

Exclusion Criteria:

• Other significant disease, which in the view of the study doctor may make assessment of the efficacy of yFFP difficult.
• Unstable medical conditions.
• Must weigh at least 45.5 kg. Cannot weigh more than 130 kg.
• A severe disease state diagnosis
• Litigation. Patients in litigation will be excluded only if conclusion of that litigation is imminent during the course of the study.
• If patient is pregnant or breastfeeding.
• Complete IgA deficiency.
• Rare contraindications to yFFP therapy as per summary of product characteristics.
• Receiving yFFP for other reasons.
• Ongoing drug or alcohol abuse.
• Psychiatric disorder that could, in the judgement of the site investigator, interfere with successful study participation.
• Unwillingness or inability to complete the study or an inability to understand the questionnaires being used.
• Cancer other than basal cell carcinoma within the last 5 years. However, those patients who have received definitive treatment, such as curative surgery more than 6 months ago, with no known recurrence can be included.
• A history of hypercoagulable or thrombophilic clotting abnormalities.
• A history of thromboembolic events: ischaemic stroke, confirmed myocardial infarction, pulmonary embolism; deep venous thrombosis except where immobility related (for example, after injury or operation).
• Unstable angina pectoris.
• Medications that might react with yFFP such as blood thinners
• Renal failure or serum creatinine greater than 1.5 times the upper limit of normal at screening.
• Any medical condition that, in the opinion of the investigator, would make it unsafe for the patient to participate or which would interfere with assessment of the outcome measures.
• Participation in another interventional trial within 3 months of randomization. Participation in non-interventional studies is not a reason for exclusion.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 45 Years to 90 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT04202757
• Other Study ID Numbers: NeurologyCenter
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Product Manufactured in and Exported from the U.S.: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: The Neurology Center
• Original Responsible Party: Same as current
• Current Study Sponsor: The Neurology Center
• Original Study Sponsor: Same as current
• Collaborators: Carolina Longevity Institute

Investigators

• Principal Investigator: Dian Ginsberg, M.D.; The Ginstitute of Functional Medicine

• PRS Account: The Neurology Center
• Verification Date: December 2021