Study on the Safety and Efficacy of Cryopreserved Platelets in Hypoproliferative Thrombocytopenic Patients
Condition: Hypoproliferative Thrombocytopenia
MeSH terms: Thrombocytopenia
Study type: Interventional
Phase: Phase 1/Phase 2

Summary: 
      The purpose of this study is to study the safety and efficacy of pooled buffy-coat derived
      platelets which had been frozen with dimethyl sulphoxide (DMSO), in the prevention of
      bleeding for patients with hypoproliferaitve thrombocytopenia. These platelets are hereafter
      referred to as cryopreserved platelets. Patients who have severely low platelet count due to
      impaired bone marrow function from chemotherapy or certain haematological conditions may need
      platelet transfusion to prevent spontaneous bleeding. Currently, platelets are stored in
      liquid form, and must be used within five to seven days of collection. In this study, DMSO is
      used to preserve platelets during freezing so that they can be stored for longer than five to
      seven days. Investigators hope to learn if thawed cryopreserved platelets are functional and
      safe for transfusion in humans.
    

Detailed description: 
      Platelets are currently stored in liquid form for a maximum of five to seven days. To extend
      the shelf-life of platelets, DMSO is added to freeze platelets for long-term storage. In
      vitro studies have shown that such cryopreserved platelets can be kept for at least two years
      at -80oC. This study is a clinical trial that aims to primarily assess the safety of
      cryopreserved pooled buffy coat-derived platelets in patients with hypoproliferative
      thrombocytopenia and no platelet refractoriness.

      Subjects will be randomised into two arms either a liquid platelet (control) or frozen
      platelet arm (treatment) and may receive four or more platelet transfusions per
      thrombocytopenic cycle. Each subject may participate in the study for up to two
      thrombocytopenic period, assuming a wash-out period of at least five days (during which the
      subject receives no platelet transfusions) between the two thrombocytopenic periods. If
      subjects participate in the study for more than one thrombocytopenic period, they will
      automatically be enrolled in the opposing arm for their second thrombocytopenic period.
    

Intervention name(s): Control arm receiving normal (never before frozen) platelets as per current clinical practice; Treatment arm receiving cryopreserved platelets
Intervention type(s): Biological; Biological
Intervention description(s): Liquid platelets arm (control) and may receive multiple platelet transfusions per thrombocytopenic cycle.; Cryopreserved platelets arm (treatment) and may receive multiple platelet transfusions per thrombocytopenic cycle.
Allocation: Randomized
Intervention model: Crossover Assignment
Intervention model description: This is a phase 1b/2a single centre clinical trial. Eligible hypoproliferative thrombocytopenic adult patients enrolled in the study will be randomised into two arms. One arm is the "treatment" arm using cryopreserved platelets and the second is the "control" arm using liquid platelets. A recruitment period of two years is expected for 17 subjects in each arm. Subjects will automatically be crossed over to the other treatment arm once they have completed the first treatment arm and there is at least a 5-days interval without platelet transfusion. Subjects can be both inpatient and outpatient. During the thrombocytopenic period of 28 days, subjects may be required to have multiple platelet transfusions during this period.
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Hypoproliferative thrombocytopenia; Cryopreserved; Transfusion; Platelets; Safety; Efficacy