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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Withdrawn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
St. Olavs Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

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

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

    First Posted
    December 12, 2012
    Last Updated
    December 18, 2012
    Sponsor
    St. Olavs Hospital
    Collaborators
    Norwegian University of Science and Technology
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    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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