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

NCT02964325 | Terminated Results DMC FDA Device

• 1 other identifier: CTS-5030
• Study Type: interventional
• Target: 422
• Locations: 1 country
• Sites: 10

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.

Conditions

Hematologic Malignancies; Hypoproliferative Thrombocytopenia

Keywords

hypoproliferative thrombocytopenia; hematologic malignancies; thrombocytopenia; platelet therapy; apheresis; pathogen reduction therapy

Outcome Measures

Primary Outcomes (1)

• Days of ≥ Grade 2 Bleeding

  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.

  From the first post-randomization platelet transfusion through 28 days following the first transfusion.

Secondary Outcomes (6)

• Number and Percentage of Subjects With Human Leukocyte Antigen (HLA) Alloimmunization

  HLA antibodies were measured at Baseline and Days 14, 28, and 56.

• Number and Percentage of Subjects With ≥ Grade 2 Bleeding

  From the first post-randomization platelet transfusion through 28 days following the first transfusion.

• Number and Percentage of Subjects at the First Timepoint of ≥ Grade 2 Bleeding

  From the first post-randomization platelet transfusion through 28 days following the first transfusion.

• Number and Percentage of Subjects With ≥ Grade 3 Bleeding

  From the first post-randomization platelet transfusion through 28 days following the first transfusion.

• Number and Percentage of Subjects With PLT Refractoriness

  From the first post-randomization platelet transfusion through 28 days following the first transfusion.

Other Outcomes (1)

• Number and Percentage of Subjects With Unanticipated Adverse Device Effects (UADEs)

  From initial post-randomization PLT transfusion through 72 hours following the last per protocol PLT transfusion.

Study Arms (2)

Mirasol platelets (MIR PLTs)

EXPERIMENTAL

Leukoreduced, Trima Accel® apheresis platelets stored in 100% plasma, pathogen reduced with the Mirasol® Pathogen Reduction Technology (PRT) System

Device: Mirasol platelets (MIR PLTs)

Reference platelets (REF PLTs)

ACTIVE COMPARATOR

Leukoreduced, apheresis platelets stored in 100% plasma

Device: Reference platelets (REF PLTs)

Interventions

Mirasol platelets (MIR PLTs) DEVICE

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.

Mirasol platelets (MIR PLTs)

Reference platelets (REF PLTs) DEVICE

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.

Reference platelets (REF PLTs)

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Weight \> 10 kg (22 lbs)
• Subject has a hematologic malignancy with hypoproliferative thrombocytopenia and is expected to have PLT count(s) ≤ 10,000/µL requiring ≥ 2 PLT transfusions
• Laboratory results within 5 days prior to anticipated initiation of the first post randomization PLT transfusion:
• Prothrombin time (PT) and/or international normalized ratio (INR) ≤ 1.3 × the upper limit of normal (ULN)
• Activated partial thromboplastin time (aPTT) ≤ 1.3 × ULN
• Fibrinogen ≥ 100 mg/dL
• 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
• IC from the subject or assent from the subject and consent from a parent or guardian, if the subject is \< 18 years of age

You may not qualify if:

• Treatment with pathogen-reduced blood products within previous 6 months
• Subject has been previously enrolled in this study and received at least 1 per protocol PLT transfusion
• 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)
• Subject has ≥ grade 2 bleeding at the time of randomization
• Planned administration of bedside LR PLT transfusion(s)
• Presently with or a history of acute promyelocytic leukemia (APML), idiopathic thrombocytopenic purpura (ITP), thrombotic thrombocytopenic purpura (TTP), or hemolytic uremic syndrome (HUS)
• HLA and/or HPA-alloimmunization and/or platelet refractory as determined by the investigator
• Hypersplenism as evidenced by splenomegaly based on investigator assessment at baseline
• History or diagnosis of a disease affecting hemostasis
• 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
• Acute or chronic medical disorder that, in the opinion of the investigator, would impair the ability of the subject to receive protocol treatment
• Subject is pregnant or lactating
• Inability of the subject to comply with study procedures and/or follow-up

Sponsors & Collaborators

• Terumo BCTbio: lead
• Biomedical Advanced Research and Development Authority: collaborator

Study Sites (10)

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

University of Florida Health Shands Hospital

Gainesville, Florida, 32608, United States

Location

Emory University/Children's Hospital of Atlanta

Atlanta, Georgia, 30322, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

John Hopkins University School of Medicine/Johns Hopkins Kimmel Cancer Center

Baltimore, Maryland, 21231, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Washington University in St. Louis

St Louis, Missouri, 63110, United States

Location

University of Nebraska Medical Center

Omaha, Nebraska, 68198, United States

Location

Robert Wood Johnson Medical School/RWJ University Hospital

New Brunswick, New Jersey, 08903, United States

Location

University of Washington Medical Center

Seattle, Washington, 98109, United States

Location

MeSH Terms

Conditions

Hematologic Neoplasms; Thrombocytopenia

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Blood Platelet Disorders; Cytopenia

Limitations and Caveats

The clinical trial was terminated early on the recommendation of the Data Monitoring Committee and in agreement with the Clinical Trial Steering Committee and the Sponsor.

Results Point of Contact

• Title: Robert Cortes, Jr. MD
• Organization: Terumo Blood and Cell Technologies

Robert.Cortes@terumobct.com

+1.303.231.4353

Study Officials

• Robert Cortes, MD

  Terumo BCT

  STUDY DIRECTOR

• Sherrill Slichter, MD

  Bloodworks Northwest

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: 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.
• Purpose: SUPPORTIVE CARE
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 9, 2016
• First Posted: November 16, 2016
• Study Start: May 5, 2017
• Primary Completion: June 25, 2020
• Study Completion: June 25, 2020
• Last Updated: August 19, 2021
• Results First Posted: July 16, 2021

Record last verified: 2021-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (10)