Rituximab Treatment of Focal Segmental Glomerulosclerosis
Collaborators: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Principal Investigator: Mark D. Pescovitz, MD Indiana University
Contact: Mark Pescovitz, MD 317-274-1010 mpescov@iupui.edu
Sharon Henson, LPN 317-278-0040 shenson1@iupui.edu
We have recently had a case of immediate post transplant recurrence of FSGS in a child that failed
to respond to MMF, steroids, long-term plasmapheresis and conversion from tacrolimus to
cyclosporine. The FSGS associated proteinuria however completely resolved at about 6 months
after treatment of post transplant lymphoma with 6 doses of rituximab. A similar case that also
resolved after treatment of PTLD with rituximab was recently reported. While the mechanism of
action of both cyclosporine and MMF is unknown in FSGS, both drugs have been shown to have
activity against B cells. The response seen in these two recent cases following treatment with a B
cell specific agent leads to the hypothesis that at least some cases of FSGS have an autoimmune
mechanism in which B cells play a central role. We propose to test this hypothesis by the treatment
of patients with recurrent or primary persistent FSGS with rituximab. For more information...
Novel Therapies for Resistant FSGS (FONT II): Phase II Clinical Trial
Collaborators:
North Shore Long Island Jewish Health System,
University of North Carolina,
The Cleveland Clinic
Principal Investigators: Howard Trachtman, MD Schneider Children's Hospital of NS-LIJ System
Debbie Gipson, MD University of North Carolina
Jennifer Gassman, PhD The Cleveland Clinic
Contact: Howard Trachtman, MD 718-470-3423 trachtma@lij.edu
Debbie Gipson, MD 919-966-2561 ext 235 debbie_gipson@unc.med.edu
A significant percentage of patients with primary FSGS are resistant to corticosteroids and other
immunosuppressive medications. In view of the rising incidence of this disease and the grim
prognosis for patients with resistant disease, it is imperative that new therapeutic approaches be
evaluated in an efficient and systematic manner. This will enable accurate assessment of the risk-
benefit ratio of novel therapies and guide the design of future Phase III randomized clinical
trials. For more information...
Permeability Factor in Focal Segmental Glomerulosclerosis
Collaborators: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Contact: Patient Recruitment and Public Liaison Office (800) 411-1222 prpl@mail.cc.nih.gov
Focal segmental glomerulosclerosis (FSGS) is a renal syndrome characterized by proteinuria
(usually nephrotic range), limited response to conventional therapy, and a poor renal prognosis,
with progression to end stage renal failure in at least 50% of patients. As a syndrome, FSGS likely
has many specific etiologies, only a few of which are well-defined. Recently, it has been suggested
that some idiopathic FSGS patients have elevated circulating levels of a protein that induces
glomerular permeability in vitro and in vivo. While there has been no consistent term for this factor, i
It will be termed here FSGS permeability factor (FPF). For more information...
Genetic Markers for Focal Segmental Glomerulosclerosis
Collaborators: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Contact:
Patient Recruitment and Public Liaison Office (800) 411-1222; prpl@mail.cc.nih.gov
Researchers believe that environmental factors may interact with genetic mutations to cause
FSGS, at least in some patients. This study will attempt to identify genetic factors associated with
the development of FSGS. The study population will be made up of 600 total subjects divided into 3
groups. Group one will be 200 African-Americans with FSGS. Group two will be 200 African-
Americans with HIV but without FSGS. Group three will be 200 non-African-Americans with FSGS.
Study participation requires that researchers obtain 20 ml (2 tubes of blood). The genetic material
(DNA) will be prepared from the white blood cells and analyzed. The results of each group will be
compared with the results from the other groups to determine if one or more genes predisposes to
FSGS. In the long run, studies that demonstrate a genetic basis for FSGS may help us identify
patients earlier and may lead to improved therapies. For more information...
Cause of Focal Segmental Glomerulosclerosis
Collaborators: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Contact:
Patient Recruitment and Public Liaison Office (800) 411-1222; prpl@mail.cc.nih.gov
The present protocol seeks to advance our understanding of glomerular diseases characterized by
progressive glomerulosclerosis in the absence of established immune-mediated renal injury. To this
end, we will enroll patients with primary minimal change, focal segmental glomerulosclerosis,
collapsing glomerulopathy and diabetic nephropathy, as well as patients with the clinical diagnosis
(prior to renal biopsy) with one of these conditions. For more information...
Steroid Treatment for Kidney Disease
Collaborators: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Contact:
Patient Recruitment and Public Liaison Office (800) 411-1222; prpl@mail.cc.nih.gov
Focal segmental glomerulosclerosis (FSGS) and minimal change disease are kidney diseases that
are associated with increased excretion of protein in the urine. Approximately half of FSGS patients
will lose kidney function within 8 years of diagnosis and will require dialysis. The purpose of this
study is to determine whether intermittent oral steroid therapy can cause sustained remission of
FSGS and MCD. Approximately 70 participants, including adults and children older than age 2, will
be enrolled in this study. They will receive 48 doses of oral dexamethasone over a period of 48
weeks. One group will take two daily doses every 2 weeks; the other group will take four daily doses
every 4 weeks. Doctors will monitor participants before, during, and after the steroid treatment with
extensive exams and testing. At the completion of the study, researchers will evaluate the safety
and efficacy of the drug treatment. For more information...
Kidney Disease Biomarkers
Collaborators: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Contact:
Patient Recruitment and Public Liaison Office (800) 411-1222; prpl@mail.cc.nih.gov
This study will identify biomarkers (proteins and other molecules in the blood or urine) that may help
scientists predict what kidney disease a patient has and whether a given patient would respond to
particular therapies. The study will look for biomarkers in the blood and urine of patients with
various kidney diseases and study of the effects of angiotensin converting enzyme inhibitors (ACE
inhibitors) and angiotensin receptor blockers (ARB) on biomarkers. Blood and urine from healthy
volunteers will be studied for comparison. For more information...
Retinoids for Minimal Change Disease and Focal Segmental Glomerulosclerosis
Collaborators: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Contact:
Patient Recruitment and Public Liaison Office (800) 411-1222; prpl@mail.cc.nih.gov
Retinoids are analogues of vitamin A that regulate cellular differentiation, leading to therapeutic use
in skin disease and malignancy. In animal models of kidney disease, retinoids restore podocyte
phenotype (podocytes are specialized cells within the kidney involved in filtration) toward normal
and reduce proteinuria (the loss of protein in the urine). The objective of this trial is to evaluate
safety and develop preliminary evidence of efficacy of retinoid treatment in patients with podocyte
disease including 10 adult patients with biopsy-proven minimal change disease, FSGS, or
collapsing glomerulopathy. Inclusion criteria will include a prior trial of immunosuppressive therapy
and proteinuria greater than or equal to 3.5 g/d while on angiotensin antagonist therapy. For more
information...
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