- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01754545
Prophylactic Plasma Infusion Therapy for Congenital Thrombotic Thrombocytopenic Purpura
December 18, 2012 updated by: St. Olavs Hospital
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.
Study Overview
Status
Withdrawn
Conditions
Intervention / Treatment
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?
Study Type
Interventional
Phase
- Phase 4
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years to 65 years (ADULT, OLDER_ADULT)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: PREVENTION
- Allocation: RANDOMIZED
- Interventional Model: CROSSOVER
- Masking: QUADRUPLE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
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.
|
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:
|
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
|
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:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
A composite score of clinical and biochemical signs of disease activity
Time Frame: 4 hours and 24 hours after intervention
|
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 |
4 hours and 24 hours after intervention
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
ADAMTS13 activity half-life
Time Frame: 4 hours and 24 hours after intervention
|
Calculation of half-life of ADAMTS13 activity based on serial measurements of ADAMTS13 activity before and after plasma infusions
|
4 hours and 24 hours after intervention
|
ADAMTS13 allo-antibodies
Time Frame: At baseline
|
Presence of allo-antibodies towards ADAMTS13
|
At baseline
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Collaborators
Investigators
- Principal Investigator: Anne S von Krogh, MD, St. Olavs Hospital
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
September 1, 2012
Primary Completion (ACTUAL)
November 1, 2012
Study Completion (ACTUAL)
November 1, 2012
Study Registration Dates
First Submitted
December 12, 2012
First Submitted That Met QC Criteria
December 18, 2012
First Posted (ESTIMATE)
December 21, 2012
Study Record Updates
Last Update Posted (ESTIMATE)
December 21, 2012
Last Update Submitted That Met QC Criteria
December 18, 2012
Last Verified
December 1, 2012
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- 133995
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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