Progressive Supranuclear Palsy Trial of Plasma Transfusions

  • Study Director: Richard Tsai, MD, MBA
  • Sponsor: Tau Consortium
  • Recruiting? No
  • Official Study Title: A 6-Month, Open-Label, Pilot Futility Clinical Trial of Monthly Young Healthy Male Donor Plasma Transfusions for Progressive Supranuclear Palsy
  • ClinicalTrials.gov Identifier: NCT02460731
  • Conditions Studied: Progressive supranuclear palsy (PSP)
  • Intervention: Young healthy male donor plasma will be administered monthly for six months. Young plasma is lawfully marketed in the US and is commonly used to correct clotting deficiencies, but this is the first time young plasma will be studied in people with PSP.
  • Phase: Phase 1
  • Duration of Participation: Approximately 10 visits in 7½ months

Purpose of the Study

The purpose of this study is to determine the safety and tolerability of young healthy male donor plasma transfusions in patients with PSP.

Eligibility

Inclusion Criteria: Subjects must be between 50–85 years of age (inclusive). Subjects must also have a Mini Mental State Examination (MMSE) score of 14–30 at the screening visit. Subjects must be willing and able to have three brain MRIs as well as two lumbar punctures performed. Subjects must have a reliable caregiver who has at least five hours of contact with them per week and is willing to accompany the subject to study visits. Subjects must have stable medications for two months prior to screening.

Exclusion Criteria: Subjects must not have any medical condition other than PSP that could account for cognitive deficits (such as Alzheimer’s disease, active seizure disorder, stroke or vascular dementia). Subjects must not have a prominent and sustained response to levodopa therapy. Subjects must not have a history of significant cardiovascular, hematologic, renal, or hepatic disease, major psychiatric illness or untreated depression. Subjects must not have had previous treatment with a human blood product, including IV immunoglobulin therapy during six months prior to screening. Subjects must not have participated in another interventional clinical trial within three months of screening.

What to Expect

Testing: Three brain MRIs, two lumbar punctures, neurological and physical examinations, cognitive testing and neuropsychiatric assessments, ECGs, blood and urine specimen collection, vital signs, eye movement testing, and actigraphy measurements (wearing a device around your wrist to monitor your movements)

The Frequency of Visits:

  • Screening phase: Two visits and one week of actigraphy within 28 days
  • Plasma transfusion phase: Monthly transfusions for six months
  • Follow-up: One follow-up visit 30–37 days after the last transfusion followed by one week of actigraphy

Materials Needed Before Evaluation: Participants meet criteria for probable or possible PSP (based on the National Institute of Neurological Disorders and Stroke & Society for Progressive Supranuclear Palsy).

Costs: No costs will be charged for any of the study procedures. Parking will be validated at UCSF public garages for all study visits, and subjects will be reimbursed $75 for each scheduled visit and $50 for additional MRI scanning and/or lumbar puncture visits to help defray any incidental expenses incurred in the course of participating in this study.

Contact Information

Clinical Trials Nurse: Mary Koestler, RN, PhD, CCRCMary.Koestler@ucsf.edu, 415.476.0661