Clinical trial NCT02050841: Octaplas Pediatric Plasma Replacement Trial
Official title: 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.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Liver Surgery
Liver Dysfunction
Coagulopathy
Cardiac Surgery
Keywords: Pediatric
patients
replacement
coagulation factors
plasma
s/d plasma
Octaplas
plasma replacement
Lead sponsor: Octapharma
Sponsor class: INDUSTRY
First posted: 2014-01-31
Enrollment: 50
Sex: ALL
Maximum age (years): 16
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: Inclusion Criteria:

1. Patient requiring liver or cardiac surgery and/or patient with liver dysfunction associated with coagulopathy in whom replacement of multiple coagulation factors is required.
2. 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.
3. Male or female patient ≤ 16 years of age.

Exclusion Criteria:

1. Patient with known homozygous congenital deficiency of protein S.
2. Patient has a history of hypersensitivity reaction to blood or plasma-derived products or to any excipient of the investigational product.
3. Patient has an already known IgA (Immunoglobulin A) deficiency with documented antibodies against IgA.
4. Patient has a congenital factor deficiency or platelet disorder requiring plasma treatment.
5. 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.
6. 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.
7. Patient is on ECMO (Extracorporeal Membrane Oxygenation) when plasma is ordered by the treating physician for the first infusion episode.
8. Patient is pregnant.
9. 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
10. Patient is receiving plasma exchange, therapeutic plasma exchange (TPE) or plasmapheresis.
11. Patient is a premature neonate defined as less than 37 weeks gestation.
12. 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.

Primary outcomes:
measure: Number of Participants With Adverse Drug Reactions (e.g., Allergic Reactions, TEs, TEEs (Thromboembolic Events) and Hyperfibrinolytic Events)
timeFrame: up to 6 days
measure: Monitoring of Clinically Significant Changes in White Blood Cells
timeFrame: up to 6 days
measure: Monitoring of Clinically Significant Changes in Red Blood Cells
timeFrame: up to 6 days
measure: Monitoring of Clinically Significant Changes in Hemoglobin
timeFrame: up to 6 days
measure: Monitoring of Clinically Significant Changes in Hematocrit
timeFrame: up to 6 days
measure: Monitoring of Clinically Significant Changes in Mean Corpuscular Volume (MCV)
timeFrame: up to 6 days
measure: Monitoring of Clinically Significant Changes in Mean Corpuscular Hemoglobin (MCH)
timeFrame: up to 6 days
measure: Monitoring of Clinically Significant Changes in Mean Corpuscular Hemoglobin Concentration (MCHC)
timeFrame: up to 6 days
measure: Monitoring of Clinically Significant Changes in Red Cell Distribution Width (RDW)
timeFrame: up to 6 days
measure: Monitoring of Clinically Significant Changes in Platelets
timeFrame: up to 6 days
Secondary outcomes:
measure: Number of Participants With Clinically Significant Changes in Hemostatic Parameters as Measured by the Following: International Normalized Ratio (INR)
timeFrame: up to 6 days
measure: Number of Participants With Clinically Significant Changes in Hemostatic Parameters as Measured by the Following: Prothrombin Time (PT)
timeFrame: up to 6 days
measure: Number of Participants With Clinically Significant Changes in Hemostatic Parameters as Measured by the Following: Thromboelastography (TEG) or Thromboelastometry (ROTEM).
timeFrame: up to 6 days
measure: Number of Participants With Clinically Significant Changes in Hemostatic Parameters as Measured by the Following: Activated Partial Thromboplastin Time (aPTT)
timeFrame: up to 6 days
measure: Volume (Dose in mL/kg) of Octaplas Used Per Infusion Episode for Each Patient.
timeFrame: up to 6 days
measure: Medically Significant Changes in Blood Pressure
timeFrame: up to 6 days
measure: Medically Significant Changes in Heart Rate
timeFrame: up to 6 days
measure: Medically Significant Changes in Respiratory Rate
timeFrame: up to 6 days
measure: Medically Significant Changes in Oxygen Saturation
timeFrame: up to 6 days
measure: Medically Significant Changes in Body Temperature
timeFrame: up to 6 days
measure: 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')
timeFrame: up to 6 days
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Octapharma Research Site
state: Alabama
city: Atlanta
country: United States
facility: Octapharma Research Site
state: Georgia
city: Minneapolis
country: United States
facility: Octapharma Research Site
state: Minnesota
city: Saint Louis
country: United States
facility: Octapharma Research Site
state: Missouri
Study officials:
affiliation: International Medical Director
name: Wolfgang Frenzel
role: STUDY_DIRECTOR
References:
citation: Spinella PC, Borasino S, Alten J. Solvent/Detergent-Treated Plasma in the Management of Pediatric Patients Who Require Replacement of Multiple Coagulation Factors: An Open-Label, Multicenter, Post-marketing Study. Front Pediatr. 2020 Sep 17;8:572. doi: 10.3389/fped.2020.00572. eCollection 2020.