Prophylactic Plasma Infusion Therapy for Congenital Thrombotic Thrombocytopenic Purpura

Prophylactic Plasma Infusion Therapy for Congenital Thrombotic Thrombocytopenic Purpura

Sponsors

Lead Sponsor: St. Olavs Hospital

Collaborator: Norwegian University of Science and Technology

Source St. Olavs Hospital
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?

Overall Status Withdrawn
Start Date September 2012
Completion Date November 2012
Primary Completion Date November 2012
Phase Phase 4
Study Type Interventional
Primary Outcome
Measure Time Frame
A composite score of clinical and biochemical signs of disease activity 4 hours and 24 hours after intervention
Secondary Outcome
Measure Time Frame
ADAMTS13 activity half-life 4 hours and 24 hours after intervention
ADAMTS13 allo-antibodies At baseline
Condition
Intervention

Intervention Type: Drug

Intervention Name: Octaplas infusion and placebo (group 1)

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.

Arm Group Label: Octaplas infusion and placebo (group 1)

Intervention Type: Drug

Intervention Name: Octaplas infusion and placebo (group 2)

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.

Arm Group Label: Octaplas infusion and placebo (group 2)

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

Gender: All

Minimum Age: 18 Years

Maximum Age: 65 Years

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Anne S von Krogh, MD Principal Investigator St. Olavs Hospital
Verification Date

December 2012

Responsible Party

Type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 2
Arm Group

Label: Octaplas infusion and placebo (group 1)

Type: Experimental

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.

Label: Octaplas infusion and placebo (group 2)

Type: Experimental

Description: Active treatment with randomly assigned 400 ml octaplas intravenously once and 400 ml placebo (for octaplas)intravenously twice in two separate intervention weeks

Study Design Info

Allocation: Randomized

Intervention Model: Crossover Assignment

Primary Purpose: Prevention

Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Source: ClinicalTrials.gov