Octaplas Pediatric Plasma Replacement Trial

Study Overview
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Brief Summary
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The purpose of this study is to investigate the safety, tolerability and efficacy of octaplas in pediatric patients who require replacement of multiple coagulation factors. Replacement of multiple coagulation factors in pediatric patients with acquired deficiencies due to liver disease and/or in pediatric patients requiring cardiac surgery or liver surgery.

Official Title
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An Open-label, Multicenter, Post-Marketing Requirement (PMR) Study to Investigate the Safety, Tolerability and Efficacy of Octaplas in the Management of Pediatric Patients Who Require Replacement of Multiple Coagulation Factors.

Conditions
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Liver Surgery, Liver Dysfunction, Coagulopathy, Cardiac Surgery

Intervention / Treatment
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* Biological: octaplas


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patient requiring liver or cardiac surgery and/or patient with liver dysfunction associated with coagulopathy in whom replacement of multiple coagulation factors is required. Voluntarily given, written and signed informed consent by the patient's legal representative(s) or guardian(s). Children deemed old enough by the Investigator/institution to understand the risks and benefits of the study should also be made aware of the risks/benefits of the study and provide written assent. Male or female patient ≤ 16 years of age. Exclusion Criteria: Patient with known homozygous congenital deficiency of protein S. Patient has a history of hypersensitivity reaction to blood or plasma-derived products or to any excipient of the investigational product. Patient has an already known IgA (Immunoglobulin A) deficiency with documented antibodies against IgA. Patient has a congenital factor deficiency or platelet disorder requiring plasma treatment. Patient is currently participating in another study investigating a new drug product or another interventional clinical study that may impact coagulation factors or has participated during the last three (3) months. Patient received FFP (Fresh Frozen Plasma), FP24 (Plasma frozen within 24 hours of collection) or any other plasma product other than Octaplas within the last 72 hours (cryoprecipitate and albumin are not exclusionary) prior to first Octaplas infusion. Patient is on ECMO (Extracorporeal Membrane Oxygenation) when plasma is ordered by the treating physician for the first infusion episode. Patient is pregnant. Patient is predicted to require massive blood transfusion defined as more than 40 mL per kilogram of all blood products in a 24-hour period Patient is receiving plasma exchange, therapeutic plasma exchange (TPE) or plasmapheresis. Patient is a premature neonate defined as less than 37 weeks gestation. Cardiac surgery patients who develop the need for plasma replacement greater than 72 hours after the end of the associated cardiac surgery and do not have coagulopathy due to hepatic dysfunction.

Ages Eligible for Study
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Maximum Age: 16 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Octaplas<br>Qualified patients will receive Octaplas as per protocol. | Biological: octaplas<br>* Octaplas S/D Plasma<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Adverse Drug Reactions (e.g., Allergic Reactions, TEs, TEEs (Thromboembolic Events) and Hyperfibrinolytic Events) |  | up to 6 days | 
| Monitoring of Clinically Significant Changes in White Blood Cells | Assesses Pre- and Post-infusion for Infusion Episode 1 | up to 6 days | 
| Monitoring of Clinically Significant Changes in Red Blood Cells | Assesses Pre- and Post-infusion for Infusion Episode 1 | up to 6 days | 
| Monitoring of Clinically Significant Changes in Hemoglobin | Assesses Pre- and Post-infusion for Infusion Episode 1 | up to 6 days | 
| Monitoring of Clinically Significant Changes in Hematocrit | Assesses Pre- and Post-infusion for Infusion Episode 1 | up to 6 days | 
| Monitoring of Clinically Significant Changes in Mean Corpuscular Volume (MCV) | Assesses Pre- and Post-infusion for Infusion Episode 1 | up to 6 days | 
| Monitoring of Clinically Significant Changes in Mean Corpuscular Hemoglobin (MCH) | Assesses Pre- and Post-infusion for Infusion Episode 1 | up to 6 days | 
| Monitoring of Clinically Significant Changes in Mean Corpuscular Hemoglobin Concentration (MCHC) | Assesses Pre- and Post-infusion for Infusion Episode 1 | up to 6 days | 
| Monitoring of Clinically Significant Changes in Red Cell Distribution Width (RDW) | Assesses Pre- and Post-infusion for Infusion Episode 1 | up to 6 days | 
| Monitoring of Clinically Significant Changes in Platelets | Assesses Pre- and Post-infusion for Infusion Episode 1 | up to 6 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Clinically Significant Changes in Hemostatic Parameters as Measured by the Following: International Normalized Ratio (INR) | This hemostatic parameter is figured out in the lab and helps to diagnose a bleeding disorder or excessive clotting disorder. The change of INR before and after 1st Octaplas infusion was scrutinized by analyzing the shifts between the classifications given below. | up to 6 days | 
| Number of Participants With Clinically Significant Changes in Hemostatic Parameters as Measured by the Following: Prothrombin Time (PT) | This hemostatic parameter is figured out in the lab and measures the time it takes for your blood to clot (the higher the PT the longer it takes your blood to clot). The change of PT before and after 1st Octaplas infusion was scrutinized by analyzing the shifts between the classifications given below. | up to 6 days | 
| Number of Participants With Clinically Significant Changes in Hemostatic Parameters as Measured by the Following: Thromboelastography (TEG) or Thromboelastometry (ROTEM). | TEG and ROTEM are methods of testing the efficiency of blood coagulation. The results were compared by looking at potential trends from TEG and ROTEM between pre-infusion vs post-infusion time points. | up to 6 days | 
| Number of Participants With Clinically Significant Changes in Hemostatic Parameters as Measured by the Following: Activated Partial Thromboplastin Time (aPTT) | aPTT measures the length of time (in seconds) that it takes for clotting to occur in a test cube. The higher the number of seconds the longer it takes the blood to clot. The changes between pre - and post infusion were analyzed | up to 6 days | 
| Volume (Dose in mL/kg) of Octaplas Used Per Infusion Episode for Each Patient. | Normal infusion: Replacement of multiple clotting factors Bypass priming: Limit hemodilution and reduce transfusion requirements Bypass warming up: Rewarm patients suffering from hypothermia during the surgery process | up to 6 days | 
| Medically Significant Changes in Blood Pressure |  | up to 6 days | 
| Medically Significant Changes in Heart Rate |  | up to 6 days | 
| Medically Significant Changes in Respiratory Rate |  | up to 6 days | 
| Medically Significant Changes in Oxygen Saturation |  | up to 6 days | 
| Medically Significant Changes in Body Temperature |  | up to 6 days | 
| Count of Investigator's Assessment of Overall Safety Observed for Patients by Category (Assessed to Have Overall Safety of 'Excellent', Assessed to Have Overall Safety of 'Moderate', Assessed to Have Overall Safety of 'Poor') |  | up to 6 days | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Pediatric, patients, replacement, coagulation factors, plasma, s/d plasma, Octaplas, plasma replacement