Safety and Efficacy Study to Compare Uniplas With Cryosupernatant Plasma in Thrombotic Thrombocytopenic Purpura (TTP)

A Blinded Non-inferiority Study to Compare Uniplas With Cryosupernatant Plasma in Thrombotic Thrombocytopenic Purpura (TTP)

Prior to the use of plasma products, thrombotic thrombocytopenic purpura (TTP) was usually a fatal condition. During plasma exchange therapy, patients need transfusion plasma that is blood group specific. Transfusing a patient with an incorrect blood group may have fatal consequences. Uniplas is a universally applicable human plasma, which can be administered irrespective of the patient's blood group. This study will test the safety and efficacy of Uniplas in comparison to cryosupernatant plasma in treatment of patients with TTP.

Study Overview

• Status: Terminated
• Conditions: Thrombotic Thrombocytopenic Purpura (TTP)
• Intervention / Treatment: Biological: Uniplas; Biological: Cryosupernatant plasma

• Study Type: Interventional
• Enrollment (Actual): 8
• Phase: Phase 3

Contacts and Locations

Study Locations

• United States
  • Virginia
    • Centreville, Virginia, United States, 20120
      • Contact Octapharma for Facility details

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• 18 years of age and above.
• Definite diagnosis of acute thrombotic thrombocytopenic purpura (TTP).
• Thrombocytopenia.
• Diagnostic signs of microangiopathic hemolytic anemia.

Exclusion Criteria:

• Congenital thrombotic microangiopathies.
• Alternative secondary cause for microangiopathy.
• Co-morbid illness limiting life expectancy to less than 3 months independent of TTP.
• Patients known to be HIV positive.
• Patients known to have lupus.
• Refusal to accept blood products.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Uniplas; Participants will receive Uniplas intravenously in 4 cycles of 7 to 9 days each for 1 month. The first cycle will consist of 1.5 plasma volume exchanges (= 75 mL/kg) for 3 consecutive days, followed by a minimum of 4 and a maximum of 6 daily single volume plasma exchanges (= 50 mL/kg). Subsequent treatment will depend upon the response of the participant to the first cycle, as assessed by a blinded assessor.	Biological: Uniplas; Uniplas will be provided frozen in sterile plastic bags.; Other Names: Blood group independent, universally applicable, prion-depleted, solvent/detergent treated, human pooled plasma
Active Comparator: Cryosupernatant plasma; Participants will receive cryosupernatant plasma intravenously in 4 cycles of 7 to 9 days each for 1 month. The first cycle will consist of 1.5 plasma volume exchanges (= 75 mL/kg) for 3 consecutive days, followed by a minimum of 4 and a maximum of 6 daily single volume plasma exchanges (= 50 mL/kg). Subsequent treatment will depend upon the response of the participant to the first cycle, as assessed by a blinded assessor.	Biological: Cryosupernatant plasma; Cryosupernatant plasma will be provided frozen in sterile plastic bags.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in (log) platelet count 1 month after treatment initiation	Log platelet count was reported in units, where 1 unit = 10^9/L platelets.	Baseline to Month 1

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of participants who died at 1 and 3 months after treatment initiation		Baseline to Month 3
Percentage of participants with a complete response (CR), a partial response (PR), a non-response (NR), or a transient response (TR) after the first treatment cycle and at 1 month	A CR was defined as a platelet count > 150 x 10^9/L on 2 consecutive days, and a decrease of lactate dehydrogenase (LDH) to within 1.25 times the upper limit of the normal range, and a resolution of previous neurological symptoms, and no new neurological symptoms. A PR was defined as at least a 2-fold increase in platelet count from baseline which is > 50 x 10^9/L. A NR was defined as a < 2-fold increase in platelet count, or a platelet count < 50 x 10^9/L, or severe red blood cell (RBC) fragmentation, or the development of new neurological symptoms, or no improvement in neurological status as defined by the level of consciousness. A TR was defined as achievement of a complete or partial response which then deteriorated, defined by a 50% decrease in peak platelet count, or neurological deterioration, or a 100% increase in nadir LDH level, or severe RBC fragmentation.	Baseline to Month 1
Total volume of plasma exchange fluid administered during treatment cycles up to 1 month		Baseline to Month 1
Time to reach maximum platelet count	Platelet count was reported in units, where 1 unit = 10^9/L platelets.	Baseline to the end of the study (up to 7 months)
Best clinical response (complete response [CR], partial response [PR], non-response [NR], transient response [TR]) during the study	The percentage of participants with a CR, PR, NR, or TR, as their best clinical response during the study, is reported. A CR was defined as a platelet count > 150 x 10^9/L on 2 consecutive days, and a decrease of lactate dehydrogenase (LDH) to within 1.25 times the upper limit of the normal range, and a resolution of previous neurological symptoms, and no new neurological symptoms. A PR was defined as at least a 2-fold increase in platelet count from baseline which is > 50 x 10^9/L. A NR was defined as a < 2-fold increase in platelet count, or a platelet count < 50 x 10^9/L, or severe red blood cell (RBC) fragmentation, or the development of new neurological symptoms, or no improvement in neurological status as defined by the level of consciousness. A TR was defined as achievement of a complete or partial response which then deteriorated, defined by a 50% decrease in peak platelet count, or neurological deterioration, or a 100% increase in nadir LDH level, or severe RBC fragmentation.	Baseline to the end of the study (up to 7 months)

Collaborators and Investigators

• Sponsor: Octapharma
• Investigators: Study Director: Wolfgang Frenzel, M.D., Octapharma

Study record dates

Study Major Dates

• Study Start: May 1, 2007
• Primary Completion (Actual): February 1, 2008
• Study Completion (Actual): February 1, 2008

Study Registration Dates

• First Submitted: December 13, 2006
• First Submitted That Met QC Criteria: December 14, 2006
• First Posted (Estimate): December 15, 2006

Study Record Updates

• Last Update Posted (Actual): June 21, 2017
• Last Update Submitted That Met QC Criteria: June 19, 2017
• Last Verified: June 1, 2017

More Information

Terms related to this study

• Keywords: plasma; bleeding disorder; plasma exchange; universal plasma
• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Hematologic Diseases; Hemorrhage; Blood Coagulation Disorders; Skin Manifestations; Thrombocytopenia; Blood Platelet Disorders; Thrombophilia; Thrombotic Microangiopathies; Purpura; Purpura, Thrombocytopenic; Purpura, Thrombotic Thrombocytopenic
• Other Study ID Numbers: UNI-108