Prophylactic Plasma Infusion Therapy for Congenital Thrombotic Thrombocytopenic Purpura

NCT01754545 | Withdrawn Phase 4 DMC

• 1 other identifier: 133995
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Purpura, Thrombotic Thrombocytopenic

Keywords

Infusions, Intravenous; Plasma; ADAMTS-13 protein, human

Outcome Measures

Primary Outcomes (1)

• 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

  4 hours and 24 hours after intervention

Secondary Outcomes (2)

• ADAMTS13 activity half-life

  4 hours and 24 hours after intervention

• ADAMTS13 allo-antibodies

  At baseline

Study Arms (2)

Octaplas infusion and placebo (group 1)

EXPERIMENTAL

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)

Octaplas infusion and placebo (group 2)

EXPERIMENTAL

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)

Interventions

Octaplas infusion and placebo (group 1) DRUG

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.

Also known as: ATC B05A A02, Solvent/detergent plasma, Human plasma

Octaplas infusion and placebo (group 1)

Octaplas infusion and placebo (group 2) DRUG

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.

Also known as: ATC B05A A02, Solvent/detergent plasma, Human plasma

Octaplas infusion and placebo (group 2)

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• Pregnancy
• Acute bout of TTP requiring daily plasma infusions or -exchange for more than 3 days, within the last 2 weeks before intervention period

Sponsors & Collaborators

• St. Olavs Hospital: lead
• Norwegian University of Science and Technology: collaborator

MeSH Terms

Conditions

Purpura, Thrombotic Thrombocytopenic

Interventions

Solvents

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Thrombophilia; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Specialty Uses of Chemicals; Chemical Actions and Uses

Study Officials

• Anne S von Krogh, MD

  St. Olavs Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: PREVENTION
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 12, 2012
• First Posted: December 21, 2012
• Study Start: September 1, 2012
• Primary Completion: November 1, 2012
• Study Completion: November 1, 2012
• Last Updated: December 21, 2012

Record last verified: 2012-12