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Trial record 8 of 203 for:    MIRASOL

Efficacy of Mirasol-treated Apheresis Platelets in Patients With Hypoproliferative Thrombocytopenia (MIPLATE)

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ClinicalTrials.gov Identifier: NCT02964325
Recruitment Status : Terminated (Based interim analysis results, Data Monitoring Committee did not believe the primary efficacy endpoint would be met. No patient safety concerns.)
First Posted : November 16, 2016
Results First Posted : July 16, 2021
Last Update Posted : August 19, 2021
Sponsor:
Collaborator:
Biomedical Advanced Research and Development Authority
Information provided by (Responsible Party):
Terumo BCTbio

Tracking Information
First Submitted Date  ICMJE November 9, 2016
First Posted Date  ICMJE November 16, 2016
Results First Submitted Date  ICMJE June 25, 2021
Results First Posted Date  ICMJE July 16, 2021
Last Update Posted Date August 19, 2021
Actual Study Start Date  ICMJE May 5, 2017
Actual Primary Completion Date June 25, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 25, 2021)
Days of ≥ Grade 2 Bleeding [ Time Frame: From the first post-randomization platelet transfusion through 28 days following the first transfusion. ]
Number of days of Grade 2 or higher bleeding recorded from treatment start date through 28 days following the first transfusion, until transfusion independence (10 days without PLT transfusion) prior to Day 28, or study termination, whichever occurred first. Subjects who obtained transfusion independence prior to Day 28 were assumed to have zero bleeding events between the date of transfusion independence and Day 28. Observed and simulated data for off-protocol transfusion intervals were included.
Original Primary Outcome Measures  ICMJE
 (submitted: November 10, 2016)
Number of days of grade 2 or higher bleeding [ Time Frame: From the first post-randomization platelet transfusion through 28 days following the first transfusion OR until transfusion independence (10 days without platelet transfusion) prior to day 28 ]
Number of days of grade 2 or higher bleeding recorded from treatment start date through 28 days following the first transfusion, until transfusion independence (10 days without PLT transfusion) prior to Day 28, or study termination, whichever occurs first. Subjects who obtain transfusion independence prior to Day 28 will be assumed to have zero bleeding events between the date of transfusion independence and Day 28.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: July 28, 2021)
  • Number and Percentage of Subjects With Human Leukocyte Antigen (HLA) Alloimmunization [ Time Frame: HLA antibodies were measured at Baseline and Days 14, 28, and 56. ]
    The outcome was the development of a new HLA Class I antibodies among subjects negative at baseline within each treatment group. Positivity for Class I HLA antibodies was determined by the 5 SD normalized background ratio cutoffs assay threshold (>59.2, LABScreen Mixed LSM12, One Lambda).
  • Number and Percentage of Subjects With ≥ Grade 2 Bleeding [ Time Frame: From the first post-randomization platelet transfusion through 28 days following the first transfusion. ]
    The number and percentage of subjects with at least 1 day of ≥ Grade 2 bleeding from Day 0 through Day 27 (or until transfusion independence was achieved) by treatment group
  • Number and Percentage of Subjects at the First Timepoint of ≥ Grade 2 Bleeding [ Time Frame: From the first post-randomization platelet transfusion through 28 days following the first transfusion. ]
    The time to first ≥ Grade 2 bleeding was analyzed using a log-rank test comparing survival curves stratified by treatment group.
  • Number and Percentage of Subjects With ≥ Grade 3 Bleeding [ Time Frame: From the first post-randomization platelet transfusion through 28 days following the first transfusion. ]
    The number and percentage of subjects with at least 1 day of ≥ Grade 3 bleeding from Day 0 through Day 27 (or until transfusion independence was achieved).
  • Number and Percentage of Subjects With PLT Refractoriness [ Time Frame: From the first post-randomization platelet transfusion through 28 days following the first transfusion. ]
    The number and percentage of subjects with PLT refractoriness defined as 2 sequential transfusions, each with corrected count increments (CCIs) < 5000 measured 1 hour post-transfusion.
  • Number and Percentage of Subjects With Immune Platelet Refractoriness [ Time Frame: Initial post-randomization platelet transfusion through high Class I HLA development. ]
    The number and percentage of subjects with PLT refractoriness for each treatment group. Subjects were defined as immune PLT refractoriness based on 2 sequential transfusion episodes, each with CCIs < 5000 measured 1 hour post transfusion, and who also had a positive antibody test within 14 days before or after the onset of PLT refractoriness.
Original Secondary Outcome Measures  ICMJE
 (submitted: November 10, 2016)
  • Human leukocyte antigen (HLA) alloimmunization [ Time Frame: Prior to the first study transfusion and weekly for 28 days and on Days 42 and 56 ]
    be based on a comparison of the proportion of subjects with HLA alloimmunization between test versus control populations.
  • Proportion of subjects with ≥ grade 2 bleeding [ Time Frame: From treatment start date through 28 days following the first transfusion, until transfusion independence (10 days without PLT transfusion) prior to Day 28, or study termination, whichever occurs first. ]
  • Time to first ≥ grade 2 bleed [ Time Frame: From treatment start date through 28 days following the first transfusion, until transfusion independence (10 days without PLT transfusion) prior to Day 28, or study termination, whichever occurs first. ]
  • Proportion of subjects with ≥ grade 3 bleeding [ Time Frame: From treatment start date through 28 days following the first transfusion, until transfusion independence (10 days without PLT transfusion) prior to Day 28, or study termination, whichever occurs first. ]
  • Proportion of subjects with PLT refractoriness [ Time Frame: From treatment start date through 28 days following the first transfusion, until transfusion independence (10 days without PLT transfusion) prior to Day 28, or study termination, whichever occurs first. ]
    Proportion of subjects with PLT refractoriness defined as 2 sequential transfusions, each with corrected count increments (CCIs) < 5000 measured 1 hour post-transfusion
  • Immune platelet refractoriness [ Time Frame: Within 14 days before or after onset of platelet refractoriness ]
    Immune platelet refractoriness will be subjects with platelet refractoriness as defined above and who also have a positive antibody test within 14 days before or after the onset f platelet refractoriness
Current Other Pre-specified Outcome Measures
 (submitted: July 28, 2021)
Number and Percentage of Subjects With Unanticipated Adverse Device Effects (UADEs) [ Time Frame: From initial post-randomization PLT transfusion through 72 hours following the last per protocol PLT transfusion. ]
UADEs are identified as treatment emergent adverse events reported by the investigator as serious, unanticipated, at least possibly related to study device or at least possibly related to treatment. UADEs were coded using Medical Dictionary for Regulatory Activities (MedDRA) Version 19.1
Original Other Pre-specified Outcome Measures
 (submitted: November 10, 2016)
  • Treatment-emergent adverse events (TEAEs) [ Time Frame: from the initial post-randomization PLT transfusion through 72 hours following the last per protocol PLT transfusion ]
  • Serious adverse events (SAEs) [ Time Frame: from the initial post-randomization PLT transfusion through 72 hours following the last per protocol PLT transfusion ]
  • Unanticipated adverse device effects (UADEs) [ Time Frame: from the initial post-randomization PLT transfusion through 72 hours following the last per protocol PLT transfusion ]
 
Descriptive Information
Brief Title  ICMJE Efficacy of Mirasol-treated Apheresis Platelets in Patients With Hypoproliferative Thrombocytopenia
Official Title  ICMJE Clinical Effectiveness of Conventional Versus Mirasol-treated Apheresis Platelets in Patients With Hypoproliferative Thrombocytopenia
Brief Summary This is a prospective, multi-center, controlled, randomized, non-inferiority study to evaluate the clinical effectiveness of Conventional versus Mirasol-treated apheresis platelets in subjects with hypoproliferative thrombocytopenia who are expected to have platelet count(s) ≤ 10,000/μL requiring ≥ 2 platelet transfusions.
Detailed Description

Patients will be randomized 1:1 to Mirasol-treated platelets (test platelets) or to conventional, untreated platelets (control platelets). The blood centers will collect the apheresis donor platelets and supply the test platelets to the hospital sites for transfusion into patients. Hospital sites will order control platelets as per their normal process, from their standard vendor.

The target population for the MIPLATE study are patients with hematologic malignancies with hypoproliferative thrombocytopenia who are expected to have platelet (PLT) count(s) of ≤ 10,000/μL requiring ≥ 2 PLT transfusions.

The primary objective of MIPLATE is to determine if the hemostatic efficacy of Mirasol-treated plasma stored Trima Accel® Aph PLTs are non-inferior to Conventional plasma stored Aph PLTs in subjects with hypoproliferative thrombocytopenia requiring PLT transfusions. The secondary objectives include comparing other efficacy and safety endpoints between the treatment groups.

Subjects with hematologic malignancies with hypoproliferative thrombocytopenia are anticipated to experience a "transfusion episode" where they will require PLT transfusion support until bone marrow recovery. During this period all PLT transfusions required for a study subject will be given according to the subject's treatment allocation for 28 days after the initial PLT transfusion OR until transfusion independence (10 days without PLT transfusion) prior to Day 28.

Additionally, serum samples for HLA antibody testing will be collected on Days 14, 28 and 56.

At a minimum, the initial post-randomization prophylactic PLT transfusion will be initiated for a PLT count ≤ 10,000/µL. Thereafter, indications for PLT transfusions may be PLT count-related prophylaxis, intervention-related prophylaxis, or therapeutic (treatment of active bleeding) as determined by the treating physician(s). The indication(s) for the transfusion(s) will be captured.

Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Investigator, Outcomes Assessor)
Masking Description:
Though this is not a blinded study, treatment assignment is obtained through electronic system and should not be shared those performing the primary outcome assessment or assessors of adverse events.
Primary Purpose: Supportive Care
Condition  ICMJE
  • Hematologic Malignancies
  • Hypoproliferative Thrombocytopenia
Intervention  ICMJE
  • Device: Mirasol platelets (MIR PLTs)
    The final product to be transfused to the subject will be leukoreduced (LR), apheresis (Aph) single-donor platelets (PLTs) at the standard therapeutic dose of 1 unit of Aph PLTs containing ≥ 3.0 × 1.0E11 PLTs. MIR PLTs will be treated with the Mirasol pathogen reduction technology system.
  • Device: Reference platelets (REF PLTs)
    The final product to be transfused to the subject will be LR-Aph single-donor PLTs at the standard therapeutic dose of 1 unit of Aph PLTs containing ≥ 3.0 × 1.0E11 PLTs.
Study Arms  ICMJE
  • Experimental: Mirasol platelets (MIR PLTs)
    Leukoreduced, Trima Accel® apheresis platelets stored in 100% plasma, pathogen reduced with the Mirasol® Pathogen Reduction Technology (PRT) System
    Intervention: Device: Mirasol platelets (MIR PLTs)
  • Active Comparator: Reference platelets (REF PLTs)
    Leukoreduced, apheresis platelets stored in 100% plasma
    Intervention: Device: Reference platelets (REF PLTs)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: June 25, 2021)
422
Original Estimated Enrollment  ICMJE
 (submitted: November 10, 2016)
556
Actual Study Completion Date  ICMJE June 25, 2020
Actual Primary Completion Date June 25, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Weight > 10 kg (22 lbs)
  2. Subject has a hematologic malignancy with hypoproliferative thrombocytopenia and is expected to have PLT count(s) ≤ 10,000/µL requiring ≥ 2 PLT transfusions
  3. Laboratory results within 5 days prior to anticipated initiation of the first post randomization PLT transfusion:

    1. Prothrombin time (PT) and/or international normalized ratio (INR) ≤ 1.3 × the upper limit of normal (ULN)
    2. Activated partial thromboplastin time (aPTT) ≤ 1.3 × ULN
    3. Fibrinogen ≥ 100 mg/dL
  4. Women of childbearing potential must have a negative pregnancy test and agree to practice a medically acceptable contraception regimen for the study duration. Women who are postmenopausal for at least 1 year (> 12 months since last menses) or are surgically sterilized do not require this test
  5. IC from the subject or assent from the subject and consent from a parent or guardian, if the subject is < 18 years of age

Exclusion Criteria:

  1. Treatment with pathogen-reduced blood products within previous 6 months
  2. Subject has been previously enrolled in this study and received at least 1 per protocol PLT transfusion
  3. a.) Subject is receiving therapeutic doses of antiplatelet agents, antifibrinolytics, and/or PLT specific growth factors within 10 days prior to randomization or b.) Subject is receiving therapeutic doses of anticoagulant, pro-coagulant or antithrombotic agents within 10 days prior to randomization. Subjects can be included if receiving the following: prophylactic dosing of anticoagulants (heparin, any low molecular weight heparin, enoxaparin, or fondaparinux), anticoagulants/thrombolytic agents used to maintain or re-establish the patency of catheters (heparin flushes or tissue-plasminogen activase [TPA], therapeutic doses of anticoagulants with a half-life of < 24 hours if it will be discontinued at least 24 hours prior to the first study transfusion, single periprocedural doses of anticoagulants with a half-life of < 24 hours or low dose aspirin (81 mg per day)
  4. Subject has ≥ grade 2 bleeding at the time of randomization
  5. Planned administration of bedside LR PLT transfusion(s)
  6. Presently with or a history of acute promyelocytic leukemia (APML), idiopathic thrombocytopenic purpura (ITP), thrombotic thrombocytopenic purpura (TTP), or hemolytic uremic syndrome (HUS)
  7. HLA and/or HPA-alloimmunization and/or platelet refractory as determined by the investigator
  8. Hypersplenism as evidenced by splenomegaly based on investigator assessment at baseline
  9. History or diagnosis of a disease affecting hemostasis
  10. Currently taking, or participating in a clinical study involving PLT substitutes, PLT growth factors, or pharmacologic agents intended to enhance (i.e, antifibrinolytic agents) or decrease PLT hemostatic function
  11. Acute or chronic medical disorder that, in the opinion of the investigator, would impair the ability of the subject to receive protocol treatment
  12. Subject is pregnant or lactating
  13. Inability of the subject to comply with study procedures and/or follow-up
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02964325
Other Study ID Numbers  ICMJE CTS-5030
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: Yes
Device Product Not Approved or Cleared by U.S. FDA: Yes
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Terumo BCTbio
Study Sponsor  ICMJE Terumo BCTbio
Collaborators  ICMJE Biomedical Advanced Research and Development Authority
Investigators  ICMJE
Study Director: Robert Cortes, MD Terumo BCT
Principal Investigator: Sherrill Slichter, MD Bloodworks Northwest
PRS Account Terumo BCTbio
Verification Date July 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP