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

Phase 3

Terminated

Conditions: Thrombotic Thrombocytopenic Purpura (TTP)

Interventions: Biological: Uniplas
Biological: Cryosupernatant plasma

Registration Number: NCT00411801

Lead Sponsor: Octapharma

Brief Summary: 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.

Detailed Description: Not available

Recruitment & Eligibility

Status: TERMINATED

Sex: All

Target Recruitment: 8

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 & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Uniplas	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.
Cryosupernatant plasma	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.

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
Percentage of participants who died at 1 and 3 months after treatment initiation	Baseline to Month 3
Total volume of plasma exchange fluid administered during treatment cycles up to 1 month	Baseline to Month 1
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	Baseline to Month 1	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.
Time to reach maximum platelet count	Baseline to the end of the study (up to 7 months)	Platelet count was reported in units, where 1 unit = 10\^9/L platelets.
Best clinical response (complete response [CR], partial response [PR], non-response [NR], transient response [TR]) during the study	Baseline to the end of the study (up to 7 months)	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.