This study will determine the safety and effectiveness of sirolimus (Rapamune® (Registered
Trademark)) in treating focal segmental glomerulosclerosis (FSGS), a disease involving kidney
scarring and increased protein in the urine. About one-half of patients with FSGS go on to
develop end-stage kidney disease within 6 years, requiring dialysis or kidney transplant.
Therapies to reduce urine protein are likely to stop the progression of renal scarring and
reduce the chance of developing kidney failure. However, current treatments for FSGS, such as
prednisone, cyclophosphamide, and cyclosporine, are not effective in many patients and can
cause serious side effects. This study will see if sirolimus, a drug with both anti-scarring
and immune suppressing properties, can lower the amount of protein in the urine and slow or
stop the kidney disease.
Patients 13 years of age and older with FSGS who have had at least one standard treatment for
FSGS may be eligible for this 24-month study. Pregnant and nursing women may not participate.
Candidates will be screened with a medical history and physical examination, review of
medical records and kidney biopsy, 24-hour urine collection, and blood tests.
Participants will take sirolimus tablets once a day for 1 year. Three 24-hour urine
collections will be done before starting treatment. Blood will be drawn to measure drug
levels every week for the first month after starting treatment, then every other week for 1
month, and then every 2 months until treatment stops. Patients who do not have a complete
response to the drug at low levels will have their dose increased. Patients will be seen at
the NIH clinic in Bethesda, Md., for the screening visit and then at 1, 4, 8, 12, and 15
months for blood and urine tests. Additional urine collections and blood tests will be done
periodically throughout the 24-month study period by the patient's local physician.
Patients whose urine protein decreases on therapy will be asked to wait 3 months before
starting another treatment and will monitored during that time to determine if the response
is sustained. Patients whose urine protein levels do not decrease with sirolimus will not be
asked to wait 3 months before starting another therapy. Follow-up with the local physician
will continue at 18 and 24 months after starting the study.
Patients whose urine protein levels increase with sirolimus treatment will be taken off the
study and may seek other treatment at any time.
Phase:
Phase 2
Details
Lead Sponsor:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)