Octaplas Pediatric Plasma Replacement Trial

• ClinicalTrials.gov Identifier: NCT02050841
• Recruitment Status: Completed
• First Posted: January 31, 2014
• Results First Posted: March 25, 2019
• Last Update Posted: June 23, 2020
• Sponsor: Octapharma
• Information provided by (Responsible Party): Octapharma

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Liver Surgery; Liver Dysfunction; Coagulopathy; Cardiac Surgery	Biological: octaplas	Phase 4

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 50 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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 Start Date: December 2014
• Actual Primary Completion Date: December 4, 2017
• Actual Study Completion Date: December 4, 2017

Arms and Interventions

Arm	Intervention/treatment
Experimental: Octaplas; Qualified patients will receive Octaplas as per protocol.	Biological: octaplas; Octaplas S/D Plasma

Outcome Measures

Primary Outcome Measures :

1. Number of Participants With Adverse Drug Reactions (e.g., Allergic Reactions, TEs, TEEs (Thromboembolic Events) and Hyperfibrinolytic Events) [ Time Frame: up to 6 days ]
2. Monitoring of Clinically Significant Changes in White Blood Cells [ Time Frame: up to 6 days ]
   Assesses Pre- and Post-infusion for Infusion Episode 1
3. Monitoring of Clinically Significant Changes in Red Blood Cells [ Time Frame: up to 6 days ]
   Assesses Pre- and Post-infusion for Infusion Episode 1
4. Monitoring of Clinically Significant Changes in Hemoglobin [ Time Frame: up to 6 days ]
   Assesses Pre- and Post-infusion for Infusion Episode 1
5. Monitoring of Clinically Significant Changes in Hematocrit [ Time Frame: up to 6 days ]
   Assesses Pre- and Post-infusion for Infusion Episode 1
6. Monitoring of Clinically Significant Changes in Mean Corpuscular Volume (MCV) [ Time Frame: up to 6 days ]
   Assesses Pre- and Post-infusion for Infusion Episode 1
7. Monitoring of Clinically Significant Changes in Mean Corpuscular Hemoglobin (MCH) [ Time Frame: up to 6 days ]
   Assesses Pre- and Post-infusion for Infusion Episode 1
8. Monitoring of Clinically Significant Changes in Mean Corpuscular Hemoglobin Concentration (MCHC) [ Time Frame: up to 6 days ]
   Assesses Pre- and Post-infusion for Infusion Episode 1
9. Monitoring of Clinically Significant Changes in Red Cell Distribution Width (RDW) [ Time Frame: up to 6 days ]
   Assesses Pre- and Post-infusion for Infusion Episode 1
10. Monitoring of Clinically Significant Changes in Platelets [ Time Frame: up to 6 days ]
    Assesses Pre- and Post-infusion for Infusion Episode 1

Secondary Outcome Measures :

1. Number of Participants With Clinically Significant Changes in Hemostatic Parameters as Measured by the Following: International Normalized Ratio (INR) [ Time Frame: up to 6 days ]
   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.
2. Number of Participants With Clinically Significant Changes in Hemostatic Parameters as Measured by the Following: Prothrombin Time (PT) [ Time Frame: up to 6 days ]
   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.
3. Number of Participants With Clinically Significant Changes in Hemostatic Parameters as Measured by the Following: Thromboelastography (TEG) or Thromboelastometry (ROTEM). [ Time Frame: up to 6 days ]
   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.
4. Number of Participants With Clinically Significant Changes in Hemostatic Parameters as Measured by the Following: Activated Partial Thromboplastin Time (aPTT) [ Time Frame: up to 6 days ]
   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
5. Volume (Dose in mL/kg) of Octaplas Used Per Infusion Episode for Each Patient. [ Time Frame: up to 6 days ]
   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
6. Medically Significant Changes in Blood Pressure [ Time Frame: up to 6 days ]
7. Medically Significant Changes in Heart Rate [ Time Frame: up to 6 days ]
8. Medically Significant Changes in Respiratory Rate [ Time Frame: up to 6 days ]
9. Medically Significant Changes in Oxygen Saturation [ Time Frame: up to 6 days ]
10. Medically Significant Changes in Body Temperature [ Time Frame: up to 6 days ]
11. 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') [ Time Frame: up to 6 days ]

Eligibility Criteria

• Ages Eligible for Study: up to 16 Years (Child)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

United States, Alabama

Octapharma Research Site

Birmingham, Alabama, United States, 35233

United States, Georgia

Octapharma Research Site

Atlanta, Georgia, United States, 30322

United States, Minnesota

Octapharma Research Site

Minneapolis, Minnesota, United States, 55455

United States, Missouri

Octapharma Research Site

Saint Louis, Missouri, United States, 63110

Sponsors and Collaborators

Octapharma

Investigators

• Study Director: Wolfgang Frenzel; International Medical Director

  Study Documents (Full-Text)

Documents provided by Octapharma:

Study Protocol  [PDF] November 19, 2016

Statistical Analysis Plan  [PDF] December 1, 2017

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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.

• Responsible Party: Octapharma
• ClinicalTrials.gov Identifier: NCT02050841
• Other Study ID Numbers: LAS-212
• First Posted: January 31, 2014
• Results First Posted: March 25, 2019
• Last Update Posted: June 23, 2020
• Last Verified: June 2020

• Studies a U.S. FDA-regulated Drug Product: Yes

Keywords provided by Octapharma:

Pediatric; patients; replacement; coagulation factors; plasma; s/d plasma; Octaplas; plasma replacement

Additional relevant MeSH terms:

Liver Diseases; Digestive System Diseases