Young Plasma Transfusions for Progressive Supranuclear Palsy

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Brief Title

Young Plasma Transfusions for Progressive Supranuclear Palsy

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 Description

      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.
    

Study Phase

Phase 1

Study Type

Interventional


Primary Outcome

Number of patients experiencing drug limiting toxicity (DLT)

Secondary Outcome

 Changes in motor function as measured by Progressive Supranuclear Palsy Rating Scale (PSPRS)

Condition

Progressive Supranuclear Palsy

Intervention

Fresh Frozen Plasma

Study Arms / Comparison Groups

 Fresh Frozen Plasma
Description:  Fresh Frozen Plasma [young (<30 years of age) healthy male donors]

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Biological

Estimated Enrollment

6

Start Date

May 2015

Completion Date

December 2019

Primary Completion Date

December 2017

Eligibility Criteria

        Inclusion Criteria:

          1. Meets National Institute of Neurological Disorders and Stroke - Society for
             Progressive Supranuclear Palsy (NINDS-SPSP) probable or possible PSP criteria (Litvan
             et al. 1996b), as modified for the AL-108-231 davunetide trial (Boxer et al. 2014);

          2. Between 50 and 85 years of age (inclusive);

          3. MRI at Screening is consistent with PSP (≤ 4 microhemorrhages and no large strokes or
             severe white matter disease);

          4. MMSE score at Screening is between 14 and 30 (inclusive);

          5. Stable medications for 2 months prior to Screening, including Food and Drug
             Administration- (FDA-) approved Alzheimer's disease (AD) medications and Parkinson's
             disease medications;

          6. Availability of a study partner who knows the subject well and is willing to accompany
             the subject to all trial visits and to participate in questionnaires;

          7. Agrees to 3 MRIs;

          8. Agrees to 2 lumbar punctures for CSF examination;

          9. Signed and dated written informed consent obtained from the subject and subject's
             caregiver in accordance with local IRB regulations;

         10. Males and all WCBP agree to abstain from sex or use an adequate method of
             contraception for the duration of the study and for 30 days after the last dose of
             study drug.

               -  Adequate contraceptive methods include those with a low failure rate, i.e., less
                  than 1% per year, when used consistently and correctly, such as complete
                  abstinence from sexual intercourse with a potentially fertile partner, and some
                  double barrier methods (condom with spermicide) in conjunction with use by the
                  partner of an intrauterine device (IUD), diaphragm with spermicide, oral
                  contraceptives, birth control patch or vaginal ring, oral, or injectable or
                  implanted contraceptives;

               -  For this study, a woman who has been surgically sterilized or who has been in a
                  state of amenorrhea for more than two years will be deemed not to be of
                  childbearing potential.

        Exclusion Criteria:

          1. Meets National Institute on Aging-Alzheimer's Association Workgroups criteria for
             probable AD (McKhann et al. 2011);

          2. Any medical condition other than PSP that could account for cognitive deficits (e.g.,
             active seizure disorder, stroke, vascular dementia);

          3. A prominent and sustained response to levodopa therapy;

          4. History of significant cardiovascular, hematologic, renal, or hepatic disease (or
             laboratory evidence thereof);

          5. History of major psychiatric illness or untreated depression;

          6. Neutrophil count <1,500/mm3, platelets <100,000/mm3, serum creatinine >1.5 x upper
             limit of normal (ULN), total bilirubin >1.5 x ULN, alanine aminotransferase (ALT) >3 x
             ULN, aspartate aminotransferase (AST) >3 x ULN, or INR >1.2 at Screening evaluations;

          7. Evidence of any clinically significant findings on Screening or baseline evaluations
             which, in the opinion of the Investigator would pose a safety risk or interfere with
             appropriate interpretation of study data;

          8. Current or recent history (within four weeks prior to Screening) of a clinically
             significant bacterial, fungal, or mycobacterial infection;

          9. Current clinically significant viral infection;

         10. Major surgery within four weeks prior to Screening;

         11. Any contraindication to or unable to tolerate lumbar puncture at Screening, including
             use of anti-coagulant medications such as warfarin. Daily administration of 81 mg
             aspirin will be allowed as long as the dose is stable for 30 days prior to Screening;

         12. Any contraindication to monthly plasma transfusions, including but not limited to: a.
             History of significant transfusion complications; b. Lack of a competent adult in the
             home to summon medical assistance if needed; c. Lack of a telephone to contact
             emergency personnel or lack of easy access for emergency vehicles; d. Compatible
             plasma units not available; e. Prior intolerance to intravenous (IV) fluids; f. IgA
             deficiency by history or laboratory evidence at Screening; g. Uremia or bleeding; h.
             Any concurrent use of an anti-coagulant therapy. Daily administration of 81 mg aspirin
             will be allowed as long as the dose is stable for 30 days prior to Screening.
             Anti-platelet drugs are acceptable.

         13. Treatment with another investigational drug or participation in another interventional
             clinical trial within 3 months of Screening;

         14. Treatment with any human blood product, including IV immunoglobulin, during the 6
             months prior to Screening or during the trial;

         15. Pregnant or lactating;

         16. Positive pregnancy test at Screening or Baseline (Day 1);

         17. Cancer within 5 years of Screening, except for non-metastatic skin cancer or
             non-metastatic prostate cancer not expected to cause significant morbidity or
             mortality within one year of Baseline.
      

Gender

All

Ages

50 Years - 85 Years

Accepts Healthy Volunteers

No

Contacts

Richard Tsai, MD, MBA, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT02460731

Organization ID

PSP-YP-001


Responsible Party

Sponsor

Study Sponsor

University of California, San Francisco


Study Sponsor

Richard Tsai, MD, MBA, Principal Investigator, University of California, San Francisco Memory and Aging Center


Verification Date

August 2020