Prophylactic Plasma Infusion Therapy for Congenital Thrombotic Thrombocytopenic Purpura

This study has been withdrawn prior to enrollment.
(altered situation in study population; not enough patients meeting enrollment criteria)
Sponsor:
Collaborator:
Norwegian University of Science and Technology
Information provided by (Responsible Party):
St. Olavs Hospital
ClinicalTrials.gov Identifier:
NCT01754545
First received: December 12, 2012
Last updated: December 18, 2012
Last verified: December 2012
  Purpose

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.


Condition Intervention Phase
Purpura, Thrombotic Thrombocytopenic
Drug: Octaplas infusion and placebo (group 1)
Drug: Octaplas infusion and placebo (group 2)
Phase 4

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Prevention
Official Title: Prophylactic Plasma Infusion Therapy for Congenital Thrombotic Thrombocytopenic Purpura

Resource links provided by NLM:


Further study details as provided by St. Olavs Hospital:

Primary Outcome Measures:
  • A composite score of clinical and biochemical signs of disease activity [ Time Frame: 4 hours and 24 hours after intervention ] [ Designated as safety issue: No ]

    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 [ Time Frame: 4 hours and 24 hours after intervention ] [ Designated as safety issue: No ]
    Calculation of half-life of ADAMTS13 activity based on serial measurements of ADAMTS13 activity before and after plasma infusions

  • ADAMTS13 allo-antibodies [ Time Frame: At baseline ] [ Designated as safety issue: No ]
    Presence of allo-antibodies towards ADAMTS13


Enrollment: 0
Study Start Date: September 2012
Study Completion Date: November 2012
Primary Completion Date: November 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Octaplas infusion and placebo (group 1)
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.
Drug: Octaplas infusion and placebo (group 1)
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.
Other Names:
  • ATC B05A A02
  • Solvent/detergent plasma
  • Human plasma
Experimental: Octaplas infusion and placebo (group 2)
Active treatment with randomly assigned 400 ml octaplas intravenously once and 400 ml placebo (for octaplas)intravenously twice in two separate intervention weeks
Drug: Octaplas infusion and placebo (group 2)
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.
Other Names:
  • ATC B05A A02
  • Solvent/detergent plasma
  • Human plasma

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?

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01754545

Sponsors and Collaborators
St. Olavs Hospital
Norwegian University of Science and Technology
Investigators
Principal Investigator: Anne S von Krogh, MD St Olavs Hospital Trondheim University Hospital
  More Information

No publications provided

Responsible Party: St. Olavs Hospital
ClinicalTrials.gov Identifier: NCT01754545     History of Changes
Other Study ID Numbers: 133995
Study First Received: December 12, 2012
Last Updated: December 18, 2012
Health Authority: Norway: Norwegian Medicines Agency

Keywords provided by St. Olavs Hospital:
Infusions, Intravenous
Plasma
ADAMTS-13 protein, human

Additional relevant MeSH terms:
Purpura
Purpura, Thrombocytopenic
Purpura, Thrombotic Thrombocytopenic
Blood Coagulation Disorders
Hematologic Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations
Signs and Symptoms
Thrombotic Microangiopathies
Thrombocytopenia
Blood Platelet Disorders
Immune System Diseases
Thrombophilia

ClinicalTrials.gov processed this record on April 15, 2014