Idiopathic Parkinson Disease
Conditions
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.
Interventions
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.
OTHERSaline
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.
Sponsors
Carolina Longevity Institute
The Neurology Center
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, 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.
Intervention model description
Placebo controlled trial
Eligibility
Sex/Gender
ALL
Age
45 Years to 90 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Changes in Physician assessment | Two weeks prior to infusion, at one-three-six months post-infusion | Unified Parkinson Disease Rating Scale (UPDRS) |
| Patient assessment | Two weeks prior to infusion, at one-three-six months post-infusion | Changes in Stanford Presenteeism Scale (SPS) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Stability of administration | At the time of infusions, day 1 and day 2 | Adverse events (if any) |
| Factors predicting a beneficial response | After the last participant's final contact at 6 months | Patient Global Impression of change |
Countries
United States
Outcome results
None listed