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Progressive Supranuclear PalsyPhase 1NCT02460731

Young Plasma Transfusions for Progressive Supranuclear Palsy

A Phase 1 study testing Fresh Frozen Plasma in people with Progressive Supranuclear Palsy, run by University of California, San Francisco at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 50 to 85 years
Conditions the study is for
Progressive Supranuclear Palsy
What is being tested?
Fresh Frozen Plasma
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
6 participants
Where
1 siteUnited States
Study dates
May 2015to Dec 2019
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 50 to 85 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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);
  • Between 50 and 85 years of age (inclusive);
  • MRI at Screening is consistent with PSP (≤ 4 microhemorrhages and no large strokes or severe white matter disease);
  • MMSE score at Screening is between 14 and 30 (inclusive);
  • 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;
  • 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;
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  • Agrees to 3 MRIs;
  • Agrees to 2 lumbar punctures for CSF examination;
  • Signed and dated written informed consent obtained from the subject and subject's caregiver in accordance with local IRB regulations;
  • 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.

Who cannot join

  • Meets National Institute on Aging-Alzheimer's Association Workgroups criteria for probable AD (McKhann et al. 2011);
  • Any medical condition other than PSP that could account for cognitive deficits (e.g., active seizure disorder, stroke, vascular dementia);
  • A prominent and sustained response to levodopa therapy;
  • History of significant cardiovascular, hematologic, renal, or hepatic disease (or laboratory evidence thereof);
  • History of major psychiatric illness or untreated depression;
  • 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;
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  • 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;
  • Current or recent history (within four weeks prior to Screening) of a clinically significant bacterial, fungal, or mycobacterial infection;
  • Current clinically significant viral infection;
  • Major surgery within four weeks prior to Screening;
  • 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;
  • 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.
  • Treatment with another investigational drug or participation in another interventional clinical trial within 3 months of Screening;
  • Treatment with any human blood product, including IV immunoglobulin, during the 6 months prior to Screening or during the trial;
  • Pregnant or lactating;
  • Positive pregnancy test at Screening or Baseline (Day 1);
  • 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.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • University of California, San Francisco, Memory and Aging Center

    San Francisco, California, 94158

Other trials in United States

About this study

Official title: A 6 Month, Open-Label, Pilot Futility Clinical Trial of Monthly Young Healthy Male Donor Plasma Transfusions for Progressive Supranuclear Palsy

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.

Read the 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.

Being tested
Fresh Frozen Plasma
Keywords
PSP, Young Plasma
Medical subject headings
Supranuclear Palsy, Progressive

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Fresh Frozen PlasmaBiological

    Fresh Frozen Plasma [young (<30 years of age) healthy male donors] Solution for intravenous infusion

Study groups in NCT02460731
Study groupRoleWhat they receive
Fresh Frozen PlasmaExperimentalFresh Frozen Plasma [young (<30 years of age) healthy male donors]
Receives: Biological: Fresh Frozen Plasma

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of patients experiencing drug limiting toxicity (DLT)

    To determine the safety and tolerability of once monthly 4-unit transfusions of young (<30 years of age) healthy male donor plasma for 6 months in patients with progressive supranuclear palsy (PSP). Number of patients experiencing drug limiting toxicity (DLT), defined as: 1) any Grade 3 or higher adverse event (AE) per National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) for which there is reasonable possibility that salsalate caused the event, 2) any Grade 2 AE in the CTCAE system organ class of nervous system disorders that is considered clinically significant and for which there is reasonable possibility that salsalate caused the event, or 3) any Grade 2 or higher treatment-related adverse events during administration that do not resolve promptly with supportive treatment

    Measured: 6 months

Secondary outcomes (4)

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

    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.

    Measured: 6 months

  2. Changes in cognition as measured by Progressive Supranuclear Palsy Rating Scale (PSPRS)

    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.

    Measured: 6 months

  3. Changes in activities of daily living as measured by Progressive Supranuclear Palsy Rating Scale (PSPRS)

    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.

    Measured: 6 months

  4. Changes in behavior as measured by Progressive Supranuclear Palsy Rating Scale (PSPRS)

    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.

    Measured: 6 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted2 Jun 2015 (estimated)
  2. Study startMay 2015
  3. Primary completionDec 2017
  4. Study completionDec 2019

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
6 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02460731
Sponsor's protocol ID
PSP-YP-001
Status last verified
Aug 2020
First submitted
19 May 2015
Last updated
11 Aug 2020
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Bensimon G, Ludolph A, Agid Y, Vidailhet M, Payan C, Leigh PN; NNIPPS Study Group. Riluzole treatment, survival and diagnostic criteria in Parkinson plus disorders: the NNIPPS study. Brain. 2009 Jan;132(Pt 1):156-71. doi: 10.1093/brain/awn291. Epub 2008 Nov 23.

    PubMed 19029129

  • Boxer AL, Lang AE, Grossman M, Knopman DS, Miller BL, Schneider LS, Doody RS, Lees A, Golbe LI, Williams DR, Corvol JC, Ludolph A, Burn D, Lorenzl S, Litvan I, Roberson ED, Hoglinger GU, Koestler M, Jack CR Jr, Van Deerlin V, Randolph C, Lobach IV, Heuer HW, Gozes I, Parker L, Whitaker S, Hirman J, Stewart AJ, Gold M, Morimoto BH; AL-108-231 Investigators. Davunetide in patients with progressive supranuclear palsy: a randomised, double-blind, placebo-controlled phase 2/3 trial. Lancet Neurol. 2014 Jul;13(7):676-85. doi: 10.1016/S1474-4422(14)70088-2. Epub 2014 May 27.

    PubMed 24873720

  • Breen DP, Vuono R, Nawarathna U, Fisher K, Shneerson JM, Reddy AB, Barker RA. Sleep and circadian rhythm regulation in early Parkinson disease. JAMA Neurol. 2014 May;71(5):589-595. doi: 10.1001/jamaneurol.2014.65.

    PubMed 24687146

  • Dugbartey AT, Townes BD, Mahurin RK. Equivalence of the Color Trails Test and Trail Making Test in nonnative English-speakers. Arch Clin Neuropsychol. 2000 Jul;15(5):425-31.

    PubMed 14590218

  • Fagan AM, Shaw LM, Xiong C, Vanderstichele H, Mintun MA, Trojanowski JQ, Coart E, Morris JC, Holtzman DM. Comparison of analytical platforms for cerebrospinal fluid measures of beta-amyloid 1-42, total tau, and p-tau181 for identifying Alzheimer disease amyloid plaque pathology. Arch Neurol. 2011 Sep;68(9):1137-44. doi: 10.1001/archneurol.2011.105. Epub 2011 May 9.

    PubMed 21555603

  • Folstein MF, Folstein SE, McHugh PR. "Mini-mental state". A practical method for grading the cognitive state of patients for the clinician. J Psychiatr Res. 1975 Nov;12(3):189-98. doi: 10.1016/0022-3956(75)90026-6. No abstract available.

    PubMed 1202204

  • Gardner RC, Boxer AL, Trujillo A, Mirsky JB, Guo CC, Gennatas ED, Heuer HW, Fine E, Zhou J, Kramer JH, Miller BL, Seeley WW. Intrinsic connectivity network disruption in progressive supranuclear palsy. Ann Neurol. 2013 May;73(5):603-16. doi: 10.1002/ana.23844. Epub 2013 Mar 27.

    PubMed 23536287

  • Golbe LI, Ohman-Strickland PA. A clinical rating scale for progressive supranuclear palsy. Brain. 2007 Jun;130(Pt 6):1552-65. doi: 10.1093/brain/awm032. Epub 2007 Apr 2.

    PubMed 17405767

  • Golbe LI, Davis PH, Schoenberg BS, Duvoisin RC. Prevalence and natural history of progressive supranuclear palsy. Neurology. 1988 Jul;38(7):1031-4. doi: 10.1212/wnl.38.7.1031.

    PubMed 3386818

  • Katsimpardi L, Litterman NK, Schein PA, Miller CM, Loffredo FS, Wojtkiewicz GR, Chen JW, Lee RT, Wagers AJ, Rubin LL. Vascular and neurogenic rejuvenation of the aging mouse brain by young systemic factors. Science. 2014 May 9;344(6184):630-4. doi: 10.1126/science.1251141. Epub 2014 May 5.

    PubMed 24797482

  • Knopman DS, Kramer JH, Boeve BF, Caselli RJ, Graff-Radford NR, Mendez MF, Miller BL, Mercaldo N. Development of methodology for conducting clinical trials in frontotemporal lobar degeneration. Brain. 2008 Nov;131(Pt 11):2957-68. doi: 10.1093/brain/awn234. Epub 2008 Oct 1.

    PubMed 18829698

  • Litvan I, Agid Y, Calne D, Campbell G, Dubois B, Duvoisin RC, Goetz CG, Golbe LI, Grafman J, Growdon JH, Hallett M, Jankovic J, Quinn NP, Tolosa E, Zee DS. Clinical research criteria for the diagnosis of progressive supranuclear palsy (Steele-Richardson-Olszewski syndrome): report of the NINDS-SPSP international workshop. Neurology. 1996 Jul;47(1):1-9. doi: 10.1212/wnl.47.1.1.

    PubMed 8710059

  • Litvan I, Agid Y, Jankovic J, Goetz C, Brandel JP, Lai EC, Wenning G, D'Olhaberriague L, Verny M, Chaudhuri KR, McKee A, Jellinger K, Bartko JJ, Mangone CA, Pearce RK. Accuracy of clinical criteria for the diagnosis of progressive supranuclear palsy (Steele-Richardson-Olszewski syndrome). Neurology. 1996 Apr;46(4):922-30. doi: 10.1212/wnl.46.4.922.

    PubMed 8780065

  • McKhann GM, Knopman DS, Chertkow H, Hyman BT, Jack CR Jr, Kawas CH, Klunk WE, Koroshetz WJ, Manly JJ, Mayeux R, Mohs RC, Morris JC, Rossor MN, Scheltens P, Carrillo MC, Thies B, Weintraub S, Phelps CH. The diagnosis of dementia due to Alzheimer's disease: recommendations from the National Institute on Aging-Alzheimer's Association workgroups on diagnostic guidelines for Alzheimer's disease. Alzheimers Dement. 2011 May;7(3):263-9. doi: 10.1016/j.jalz.2011.03.005. Epub 2011 Apr 21.

    PubMed 21514250

  • Pandey S, Vyas GN. Adverse effects of plasma transfusion. Transfusion. 2012 May;52 Suppl 1(Suppl 1):65S-79S. doi: 10.1111/j.1537-2995.2012.03663.x.

    PubMed 22578374

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Fresh Frozen Plasma in people with Progressive Supranuclear Palsy, run by University of California, San Francisco at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 12 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 11 Aug 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of patients experiencing drug limiting toxicity (DLT) (time frame: 6 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02460731, which also lists 24 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Aug 2020.

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