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

NCT ID: NCT02964325

Last Updated: 2021-08-19

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: NA
• Total Enrollment: 422 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-05-05
• Study Completion Date: 2020-06-25

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.

Conditions

Hematologic Malignancies; Hypoproliferative Thrombocytopenia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: SUPPORTIVE_CARE
• Blinding Strategy: DOUBLE

Study Groups

Mirasol platelets (MIR PLTs)

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

Group Type: EXPERIMENTAL

Mirasol platelets (MIR PLTs)

Intervention Type: 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.

Reference platelets (REF PLTs)

Leukoreduced, apheresis platelets stored in 100% plasma

Group Type: ACTIVE_COMPARATOR

Reference platelets (REF PLTs)

Intervention Type: 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.

Interventions

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.

Intervention Type: 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.

Intervention Type: DEVICE

Eligibility Criteria

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

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Biomedical Advanced Research and Development Authority

FED

Sponsor Role: collaborator

Terumo BCTbio

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Robert Cortes, MD

Role: STUDY_DIRECTOR

Terumo BCT

Sherrill Slichter, MD

Role: PRINCIPAL_INVESTIGATOR

Bloodworks Northwest

Locations

Children's National Medical Center

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

University of Florida Health Shands Hospital

Gainesville, Florida, United States

Emory University/Children's Hospital of Atlanta

Atlanta, Georgia, United States

University of Iowa

Iowa City, Iowa, United States

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

Baltimore, Maryland, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Washington University in St. Louis

St Louis, Missouri, United States

University of Nebraska Medical Center

Omaha, Nebraska, United States

Robert Wood Johnson Medical School/RWJ University Hospital

New Brunswick, New Jersey, United States

University of Washington Medical Center

Seattle, Washington, United States

Countries

United States

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

CTS-5030

Identifier Type: -

Identifier Source: org_study_id