Intravenous Plasma Treatment for Parkinson's Disease (yFFP)

Intravenous Young Fresh Frozen Plasma (yFFP) Investigational Treatment for Parkinson's Disease

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.

Study Overview

• Status: Completed
• Conditions: Idiopathic Parkinson Disease
• Intervention / Treatment: Biological: [21CFR640.30] Plasma from 18 - 25 year old volunteer donors; Other: Saline

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
• Enrollment (Actual): 22
• Phase: Early Phase 1

Contacts and Locations

Study Locations

• United States
  • Texas
    • Houston, Texas, United States, 77030
      • The Neurology Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 45 years to 90 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Young Fresh Frozen Plasma (yFFP); [21CFR640.30] Plasma from 18 - 25 year old volunteer donors	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 Names: Young Fresh Frozen Plasma (yFFP)
Placebo Comparator: Saline; 0.1% riboflavin in normal saline	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 Names: Normal saline

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: The Neurology Center
• Collaborators: Carolina Longevity Institute
• Investigators: Principal Investigator: Dian Ginsberg, M.D., The Ginstitute of Functional Medicine

Study record dates

Study Major Dates

• Study Start (Actual): September 24, 2018
• Primary Completion (Actual): August 7, 2019
• Study Completion (Actual): August 8, 2019

Study Registration Dates

• First Submitted: September 21, 2018
• First Submitted That Met QC Criteria: December 13, 2019
• First Posted (Actual): December 18, 2019

Study Record Updates

• Last Update Posted (Actual): May 31, 2024
• Last Update Submitted That Met QC Criteria: May 29, 2024
• Last Verified: May 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Parkinsonian Disorders; Basal Ganglia Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases; Parkinson Disease
• Other Study ID Numbers: NeurologyCenter

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• 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