This trial is active, not recruiting.

Condition thrombotic thrombocytopenic purpura
Treatments cyclosporine, prednisone
Phase phase 3
Sponsor Ohio State University
Start date November 2007
End date August 2017
Trial size 62 participants
Trial identifier NCT00713193, 2007H0194, R01FD003932


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

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking open label
Primary purpose treatment
Patients in this arm will receive cyclosporine (Neoral) at a dose of 2-3 mg/kg orally as an adjunct to plasma exchange.
cyclosporine Neoral
2-3 mg/kg orally in a twice day divided dose for 6 months
(Active Comparator)
Patients in this arm will receive prednisone at a dose of 1 mg/kg as an adjunct to plasma exchange.
1 mg/kg orally, daily for at least 30 days, then tapered over 30 days after achieving remission.

Primary Outcomes

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 Outcomes

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

Eligibility Criteria

Male or female participants at least 18 years old.

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.

Additional Information

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)
Principal investigator Spero R Cataland, MD
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.
Trial information was received from ClinicalTrials.gov and was last updated in April 2015.
Information provided to ClinicalTrials.gov by Ohio State University.