Prophylactic Plasma Infusion Therapy for Congenital Thrombotic Thrombocytopenic Purpura
Primary Purpose
Purpura, Thrombotic Thrombocytopenic
Status
Withdrawn
Phase
Phase 4
Locations
Study Type
Interventional
Intervention
Octaplas infusion and placebo (group 1)
Octaplas infusion and placebo (group 2)
Sponsored by
About this trial
This is an interventional prevention trial for Purpura, Thrombotic Thrombocytopenic focused on measuring Infusions, Intravenous, Plasma, ADAMTS-13 protein, human
Eligibility Criteria
Inclusion Criteria:
- Confirmed diagnosis of congenital TTP
- On regular prophylactic plasma treatment: > Once weekly (group 1) or >1 <3 times monthly (group 2)
- Between 18 and 65 years
- Capable of understanding and complying with the protocol
Exclusion Criteria:
- Pregnancy
- Acute bout of TTP requiring daily plasma infusions or -exchange for more than 3 days, within the last 2 weeks before intervention period
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
Octaplas infusion and placebo (group 1)
Octaplas infusion and placebo (group 2)
Arm Description
Active treatment with randomly assigned 400 ml octaplas intravenously 2-3 times a week and 400 ml placebo (for octaplas)intravenously 2-3 times a week over two weeks.
Active treatment with randomly assigned 400 ml octaplas intravenously once and 400 ml placebo (for octaplas)intravenously twice in two separate intervention weeks
Outcomes
Primary Outcome Measures
A composite score of clinical and biochemical signs of disease activity
Biochemical signs of disease activity are scored by measurements of lactate dehydrogenase, hemoglobin, platelet counts and ADAMTS13 activity.
Clinical signs of disease activity are measured by an evaluator's score and subjects symptom screening
Secondary Outcome Measures
ADAMTS13 activity half-life
Calculation of half-life of ADAMTS13 activity based on serial measurements of ADAMTS13 activity before and after plasma infusions
ADAMTS13 allo-antibodies
Presence of allo-antibodies towards ADAMTS13
Full Information
NCT ID
NCT01754545
First Posted
December 12, 2012
Last Updated
December 18, 2012
Sponsor
St. Olavs Hospital
Collaborators
Norwegian University of Science and Technology
1. Study Identification
Unique Protocol Identification Number
NCT01754545
Brief Title
Prophylactic Plasma Infusion Therapy for Congenital Thrombotic Thrombocytopenic Purpura
Official Title
Prophylactic Plasma Infusion Therapy for Congenital Thrombotic Thrombocytopenic Purpura
Study Type
Interventional
2. Study Status
Record Verification Date
December 2012
Overall Recruitment Status
Withdrawn
Why Stopped
altered situation in study population; not enough patients meeting enrollment criteria
Study Start Date
September 2012 (undefined)
Primary Completion Date
November 2012 (Actual)
Study Completion Date
November 2012 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
St. Olavs Hospital
Collaborators
Norwegian University of Science and Technology
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
Congenital thrombotic thrombocytopenic purpura (TTP), also called Upshaw-Schulman Syndrome or hereditary or familial TTP is a rare, but severe disease. The purpose of this study is to determine how infusions of plasma to patients with congenital TTP correlate with symptoms and signs of activity of the disease, and to determine why some patients need more frequent infusions of plasma than others to prevent acute attacks of the disease.
Detailed Description
Patients with congenital TTP have an inherited lack of function or amount of a protein in plasma called ADAMTS13, that otherwise is responsible for cleaving large von Willebrand-molecules into smaller parts. The patients suffer recurrent attacks of clotting of small blood vessels, that can cause damage to major organs, including the central nervous system. Acute attacks can be treated successfully with infusions of human plasma, and some patients also receive regular plasma therapy for prevention of acute attacks. A small group of patients receive preventive plasma infusions twice or more weekly, indicating a much higher need for plasma than what is otherwise recommended for preventive therapy. Do these patients have an ongoing activity of their disease despite a stimulus? Or a higher turn-over of transfused ADAMTS13? Have these patients developed antibodies against transfused ADAMTS13? Are any symptoms correlated with signs of disease activity?
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Purpura, Thrombotic Thrombocytopenic
Keywords
Infusions, Intravenous, Plasma, ADAMTS-13 protein, human
7. Study Design
Primary Purpose
Prevention
Study Phase
Phase 4
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Octaplas infusion and placebo (group 1)
Arm Type
Experimental
Arm Description
Active treatment with randomly assigned 400 ml octaplas intravenously 2-3 times a week and 400 ml placebo (for octaplas)intravenously 2-3 times a week over two weeks.
Arm Title
Octaplas infusion and placebo (group 2)
Arm Type
Experimental
Arm Description
Active treatment with randomly assigned 400 ml octaplas intravenously once and 400 ml placebo (for octaplas)intravenously twice in two separate intervention weeks
Intervention Type
Drug
Intervention Name(s)
Octaplas infusion and placebo (group 1)
Other Intervention Name(s)
ATC B05A A02, Solvent/detergent plasma, Human plasma
Intervention Description
Intervention period is Monday-Friday in two following weeks. Active treatment with Octaplas is given 2-3 times each week and placebo is given the other 2-3 days of intervention each week. Route of administration is intravenously.
Intervention Type
Drug
Intervention Name(s)
Octaplas infusion and placebo (group 2)
Other Intervention Name(s)
ATC B05A A02, Solvent/detergent plasma, Human plasma
Intervention Description
Intervention period is Monday-Wednesday in two separate weeks (minimum of three weeks between intervention weeks). Active treatment with Octaplas is given once and placebo is given twice each week. Route of administration is intravenously.
Primary Outcome Measure Information:
Title
A composite score of clinical and biochemical signs of disease activity
Description
Biochemical signs of disease activity are scored by measurements of lactate dehydrogenase, hemoglobin, platelet counts and ADAMTS13 activity.
Clinical signs of disease activity are measured by an evaluator's score and subjects symptom screening
Time Frame
4 hours and 24 hours after intervention
Secondary Outcome Measure Information:
Title
ADAMTS13 activity half-life
Description
Calculation of half-life of ADAMTS13 activity based on serial measurements of ADAMTS13 activity before and after plasma infusions
Time Frame
4 hours and 24 hours after intervention
Title
ADAMTS13 allo-antibodies
Description
Presence of allo-antibodies towards ADAMTS13
Time Frame
At baseline
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Confirmed diagnosis of congenital TTP
On regular prophylactic plasma treatment: > Once weekly (group 1) or >1 <3 times monthly (group 2)
Between 18 and 65 years
Capable of understanding and complying with the protocol
Exclusion Criteria:
Pregnancy
Acute bout of TTP requiring daily plasma infusions or -exchange for more than 3 days, within the last 2 weeks before intervention period
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Anne S von Krogh, MD
Organizational Affiliation
St. Olavs Hospital
Official's Role
Principal Investigator
12. IPD Sharing Statement
Learn more about this trial
Prophylactic Plasma Infusion Therapy for Congenital Thrombotic Thrombocytopenic Purpura
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