Clinical trial NCT01164098: Rituximab to Prevent Recurrence of Proteinuria
Official title: The Use of Rituximab to Prevent Recurrence of Proteinuria in Patients Receiving Kidney Transplant for FSGS
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: FSGS
Proteinuria
Lead sponsor: University of Miami
Sponsor class: OTHER
Collaborators: name: Genentech, Inc.
name: National Institutes of Health (NIH)
name: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
First posted: 2010-07-16
Enrollment: 30
Sex: ALL
Minimum age (years): 7
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The investigators propose to study novel targets of rituximab in podocytes, with a particular focus on recurrent focal segmental glomerulosclerosis (FSGS). The proposed study has strong clinical implications, since it may extend the approved indications for rituximab treatment to recurrent FSGS as well as to other proteinuric diseases. Furthermore, it will offer new insights into the role of sphyngomyelin related enzymes in podocyte function in health and disease, thus allowing the identification of novel targets for antiproteinuric drug development. Finally, the proposed study offers the opportunity to identify a correlation between the patient's specific clinical outcome and the experimental results obtained after exposing podocytes to patient sera in the presence or absence of rituximab. Therefore, it may lead to the development of an assay for the pre-transplant identification of patients at high-risk for recurrent disease and, among them, may allow the identification of those patients that will respond to rituximab.

Detailed description: A total of 60 patients will be enrolled in the study.

Eligibility criteria: Inclusion:

1. Patient has been fully informed and has signed a dated IRB-approval informed consent form.
2. Age 7-65 years.
3. Male and Females diagnosed of FSGS by kidney biopsy. Kidney biopsy report is not required once the physician confirms the diagnosis. Transcribed reports from referring physicians are also valid.

Exclusion:

1. Recipient or donor is seropositive for human immunodeficiency virus (HIV), Hepatitis C viruses, or Hepatitis B virus antigenemia.
2. Patient has a current malignancy or a history of malignancy (within the past 5 years), except non-metastatic basal or squamous cell carcinoma of the skin that has been treated successfully or carcinoma in situ of the cervix that has been treated successfully.
3. Patient has uncontrolled concomitant infections and/or severe diarrhea, vomiting, active upper gastro-intestinal tract malabsorption or an active peptic ulcer or any other unstable medical condition that could interfere with study objectives.
4. Patient is pregnant or lactating.
5. Patient has any form of substance abuse, psychiatric disorder or a condition that, in opinion of the investigator, may invalidate communication with the investigator.
6. Patients with a defined genetic cause of FSGS.

Primary outcomes:
measure: Maximum of the Urine Protein/Creatinine Ratio Observed Between Post-Transplant Days 3-30.
timeFrame: between post-transplant day 3 and day 30
Secondary outcomes:
measure: Maximum of the Urine Protein/Creatinine Ratio Observed Between Post-Transplant Months 3-12
timeFrame: Post-Transplant Months 3-12
measure: Renal Function as Measured by eGFR (Estimated Glomerular Filtration Rate)
timeFrame: 12 months post-transplant
Other outcomes:
Locations:
city: Miami
country: United States
facility: University of Miami
state: Florida
Study officials:
affiliation: University of Miami
name: Alessia Fornoni, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Miami
name: George W. Burke, M.D.
role: STUDY_DIRECTOR
References: