Clinical trial NCT00251277: Use of Rituximab Treatment in Addition to Standard Care for Newly Presenting Thrombotic Thrombocytopenic Purpura
Official title: Use of Rituximab Treatment in Addition to Standard Care for Newly Presenting Thrombotic Thrombocytopenic Purpura
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE1
PHASE2
Conditions: Thrombotic Thrombocytopenic Purpura
Keywords: Thrombotic Thrombocytopenic Purpura
TTP
Hematology
Lead sponsor: Weill Medical College of Cornell University
Sponsor class: OTHER
Collaborators: name: Genentech, Inc.
First posted: 2005-11-09
Enrollment: 0
Sex: ALL
Minimum age (years): 17
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose is to evaluate safety and feasibility of the use of Rituximab as an adjunct to standard therapy (plasmapheresis + steroids) for patients with thrombotic thrombocytopenic purpura (TTP). This includes evaluating the rate and type of treatment failure.

Detailed description: With this study we hope to evaluate safety and feasibility of the use of Rituximab as an adjunct to standard therapy (plasmapheresis + steroids) for patients with thrombotic thrombocytopenic purpura (TTP). This includes evaluating the rate and type of treatment failure.

Eligibility criteria: Inclusion Criteria:

Patients will be included in the trial based on the following criteria:

* Patients must have TTP with platelet count \< 100,000/mL and microangiopathic hemolytic anemia which is defined as presence of at 3-10 fragmented red blood cells (schistocytes) per high power filed on the peripheral blood smear.
* Either gender, age 17 or older
* Men and women of reproductive potential must agree to use an acceptable method of birth control during treatment and for six months after completion of treatment.
* TTP not related to underlying cancer, treatment of cancer or transplantation
* New onset TTP, or previously diagnosed TTP with an unmaintained remission for \>12 months.
* LDH \>2X upper limit of normal
* Prothrombin time (PT), partial thromboplastin time (PTT) normal
* Direct antiglobulin test (DAT) negative
* Subject has provided written informed consent
* Patients who have received up to 3 plasmapheresis.

Exclusion Criteria:

Patients will be excluded from the trial based on the following criteria:

* A diagnosis of AIDS. Patients with HIV infection with absolute CD4 counts \>200/ul and no active, significant opportunistic infection are eligible
* Patients with a known hepatitis C infection (HCV) and/or with hepatitis B
* Patients receiving pheresis more than once a day
* Recent (within 1 year) bone marrow or hematopoietic stem cell transplant
* Patient is on calcineurin inhibitors, or is unable to come off them
* Acute or chronic disseminated intravascular coagulation (DIC), defined by D-dimers \>8mg/ml and fibrinogen \< 100 mg (0.1g)/dl
* A diagnosis of metastatic or non-metastatic malignancy other than basal cell carcinoma.
* Malignant hypertension (systolic blood pressure \[BP\] \> 200 mm Hg or a diastolic BP \> 130 mm Hg)
* Pregnancy (a negative serum pregnancy test should be performed for all women of childbearing potential within 7 days of treatment). Eligibility resumes 3 days after delivery
* Patients with family history of or a previous diagnosis of congenital TTP
* Patients with hemolytic uremic syndrome (HUS)
* Patients with sepsis

Primary outcomes:
measure: Failure to maintain the complete response until day 120; Non-protocol treatment for TTP, such as other immunosuppressive agents or splenectomy, reinstitution of plasma exchange within the first 90 days
timeFrame: Four months
Secondary outcomes:
Other outcomes:
Locations:
city: New York
country: United States
facility: Weill Medical College of Cornell University/New York Presbyterian Hospital
state: New York
Study officials:
affiliation: Weill Medical College of Cornell University
name: James B Bussel, M.D.
role: PRINCIPAL_INVESTIGATOR
References: