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Study of Cyclosporine or Corticosteroids as an Adjunct to Plasma Exchange in Thrombotic Thrombocytopenic Purpura (TTP)

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ClinicalTrials.gov Identifier: NCT00713193
Recruitment Status : Completed
First Posted : July 11, 2008
Last Update Posted : September 25, 2017
Food and Drug Administration (FDA)
Information provided by (Responsible Party):
Spero Cataland, Ohio State University

Brief Summary:
This research involves the use of immune base therapy as an adjunct to plasma exchange, the present standard of care for thrombotic thrombocytopenic purpura (TTP). Funding source -FDA OOPD

Condition or disease Intervention/treatment Phase
Thrombotic Thrombocytopenic Purpura Drug: Cyclosporine Drug: Prednisone Phase 3

Detailed Description:

TTP is a rare blood disorder that causes blood clots to form in the small blood vessels throughout the body, including the kidneys, brain, abdomen, and the heart. Plasma exchange is the standard treatment for TTP. Plasma exchange is a treatment that removes the plasma (the liquid portion of the blood without any cells) from a patient and replaces it with plasma from a donor. With plasma exchange, 90% of patients achieve a remission of the disease. Unfortunately, up to one half of patient will relapse after the plasma exchange has stopped, leading to significant complications and added risks to the patient.

This study randomizes patients to receive either prednisone or cyclosporine as an adjunct to plasma exchange, with the cyclosporine arm being the experimental arm of the study. All patients will undergo plasma exchange but will be randomized to receive either prednisone or cyclosporine as an adjunct to plasma exchange. Previous studies suggested that cyclosporine was superior to prednisone as an adjunct to plasma exchange, and therefore this randomized study attempts to confirm the findings of two previous single institution studies.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 16 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multi-Center, Randomized Study of Cyclosporine or Corticosteroids as an Adjunct to Plasma Exchange in the Initial Therapy of Thrombotic Thrombocytopenic Purpura (TTP)
Study Start Date : November 2007
Actual Primary Completion Date : September 20, 2017
Actual Study Completion Date : September 20, 2017

Arm Intervention/treatment
Experimental: 1
Patients in this arm will receive cyclosporine (Neoral) at a dose of 2-3 mg/kg orally as an adjunct to plasma exchange.
Drug: Cyclosporine
2-3 mg/kg orally in a twice day divided dose for 6 months
Other Name: Neoral

Active Comparator: 2
Patients in this arm will receive prednisone at a dose of 1 mg/kg as an adjunct to plasma exchange.
Drug: Prednisone
1 mg/kg orally, daily for at least 30 days, then tapered over 30 days after achieving remission.

Primary Outcome Measures :
  1. To determine if CSA given individually as an adjunct to PE decreases the frequency of exacerbation in patients with TTP compared to those patients given corticosteroids as an adjunct to PE. [ Time Frame: 30 days ]

Secondary Outcome Measures :
  1. To compare the CSA and PE-treated patients to the corticosteroid and PE-treated patients in terms of the number of exchange procedures to achieve clinical remission and the relapse rate after tapering the adjuvant immune-based therapy. [ Time Frame: 2-5 years ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Patients with a clinical diagnosis of idiopathic TTP as defined by a microangiopathic hemolytic anemia and thrombocytopenia (<100 x 103)
  • Additional components of the pentad (fever, renal and neurologic abnormalities) need not be present.
  • Additional explanations for the microangiopathic changes including DIC and malignancy should be excluded.
  • Patients with pregnancy associated TTP will be permitted on this therapeutic trial if the child is delivered prior to the initiation of therapy for TTP. However, female patients that are breastfeeding and are unwilling to discontinue breastfeeding at the time of enrollment will be excluded from this study
  • Patients with a previous diagnosis of TTP are eligible to be enrolled provided they meet eligibility criteria and have not been treated for an TTP in the past 30 days
  • Given the potential for nephrotoxicity with CSA, all patients must have a serum creatinine of < 2.5 mg/dl prior to enrollment

Exclusion Criteria:

  • In light of concern for the prompt initiation of PE, all patients with suspected TTP may be enrolled on this trial. If it is subsequently found that the patient does not meet enrollment criteria, they will be removed and their spot replaced for study purposes. Patients removed from the study after enrollment will continue to be followed longitudinally for 6 months to be monitored for safety and will be included in the safety database.
  • Patients with TTP clinically categorized as secondary to stem cell transplant and solid organ, bloody diarrhea associated, malignancy associated, and drug associated will not be enrolled on this therapeutic study.
  • Incarcerated patients will be excluded from the study due to the inherent difficulties in maintaining close follow-up for study purposes in patients who are incarcerated.
  • Any patients already being treated chronically with corticosteroids or cyclosporine and taking these at the time of their presentation will be excluded from this study.
  • Female patients that are breastfeeding and are unwilling to discontinue breastfeeding at the time of enrollment will be excluded from this study
  • Patients taking any medications contraindicated in combination with CSA that cannot be safely discontinued will be excluded from this study.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00713193

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United States, Ohio
Ohio State University
Columbus, Ohio, United States, 43210
Sponsors and Collaborators
Ohio State University
Food and Drug Administration (FDA)
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Principal Investigator: Spero R Cataland, MD Ohio State University
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Spero Cataland, Professor of Internal Medicine, Ohio State University
ClinicalTrials.gov Identifier: NCT00713193    
Other Study ID Numbers: 2007H0194
R01FD003932 ( U.S. FDA Grant/Contract )
First Posted: July 11, 2008    Key Record Dates
Last Update Posted: September 25, 2017
Last Verified: September 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: data being evaluated and reviewed, manuscript in preparation for final report to FDA
Keywords provided by Spero Cataland, Ohio State University:
Idiopathic TTP
Plasma Exchange
Additional relevant MeSH terms:
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Purpura, Thrombocytopenic
Purpura, Thrombotic Thrombocytopenic
Blood Coagulation Disorders
Hematologic Diseases
Pathologic Processes
Skin Manifestations
Thrombotic Microangiopathies
Blood Platelet Disorders
Immune System Diseases
Anti-Inflammatory Agents
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Immunosuppressive Agents
Immunologic Factors
Antifungal Agents
Anti-Infective Agents
Dermatologic Agents