Clinical Trial: Young Plasma Transfusions for Progressive Supranuclear Palsy
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: A 6 Month, Open-Label, Pilot Futility Clinical Trial of Monthly Young Healthy Male Donor Plasma Transfusions for Progressive Supranuclear Palsy
Brief Summary: This is a phase 1, multi-center, open-label study of the safety, tolerability, pharmacodynamics, and preliminary efficacy of young (<30 years of age) healthy male donor plasma transfusions in patients with PSP. Up to 10 subjects will receive once monthly 4-unit transfusions of young healthy male donor plasma for 6 months.
Detailed Summary:
This is a phase 1, multi-center, open-label study of the safety, tolerability, pharmacodynamics, and preliminary efficacy of young (<30 years of age) healthy male donor plasma transfusions in patients with PSP. Up to 10 subjects will receive once monthly 4-unit transfusions of young healthy male donor plasma for 6 months.
If ≥3 subjects experience drug limiting toxicity (DLT), as defined in Section 7.19, the study will be terminated. Any subject that experiences a DLT will be discontinued from further treatment with the study drug.
An interim futility analysis will be performed after five subjects have completed 6 months of study drug treatment. If the criteria listed in Section 9.3 of this protocol are met, an additional 5 subjects will be enrolled in the trial. If not, the trial will be terminated.
Sponsor: University of California, San Francisco
Current Primary Outcome: Number of patients experiencing drug limiting toxicity (DLT) [ Time Frame: 6 months ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Changes in motor function as measured by Progressive Supranuclear Palsy Rating Scale (PSPRS) [ Time Frame: 6 months ]Motor function as measured by Progressive Supranuclear Palsy Rating Scale (PSPRS) comprising 28 items in six categories: daily activities (by history), behavior, bulbar, ocular motor, limb motor and gait/midline The available total score ranges from 0 to 100; lower scores reflect better outcome.
- Changes in cognition as measured by Progressive Supranuclear Palsy Rating Scale (PSPRS) [ Time Frame: 6 months ]Cognitive function as measured by Progressive Supranuclear Palsy Rating Scale (PSPRS) comprising 28 items in six categories: daily activities (by history), behavior, bulbar, ocular motor, limb motor and gait/midline The available total score ranges from 0 to 100; lower scores reflect better outcome.
- Changes in activities of daily living as measured by Progressive Supranuclear Palsy Rating Scale (PSPRS) [ Time Frame: 6 months ]Activities of daily living as measured by Progressive Supranuclear Palsy Rating Scale (PSPRS) comprising 28 items in six categories: daily activities (by history), behavior, bulbar, ocular motor, limb motor and gait/midline The available total score ranges from 0 to 100; lower scores reflect better outcome.
- Changes in behavior as measured by Progressive Supranuclear Palsy Rating Scale (PSPRS) [ Time Frame: 6 months ]Behavior as measured by Progressive Supranuclear Palsy Rating Scale (PSPRS) comprising 28 items in six categories: daily activities (by history), behavior, bulbar, ocular motor, limb motor and gait/midline The available total score ranges from 0 to 100; lower scores reflect better outcome.
Original Secondary Outcome: Same as current
Information By: University of California, San Francisco
Dates:
Date Received: May 19, 2015
Date Started: May 2015
Date Completion: July 2017
Last Updated: December 2, 2016
Last Verified: December 2016