Polycystic Kidney, Type 1 Autosomal Dominant Disease Clinical Trial
Official title:
Pulsed Oral Sirolimus in Autosomal Dominant Polycystic Kidney Disease - The Vienna RAP Study
Sirolimus (SIR) has lead to a reduction of overall kidney size, a decrease in cyst density
and general tubular cell proliferation in animal models, and to a reduction of the increase
in creatinine and blood urea nitrogen by 34 and 39 percent respectively, as well as a
reduction of cyst proliferation, expressed by a 30 percent reduction of overall kidney
enlargement, a reduction in general cyst volume, and a reduction of the cyst volume density
in the renal cortex in humans.
However, despite promising data from animal- and in vivo studies, most mammalian target of
rapamycin inhibitor (mTOR-I) studies in patients with autosomal-dominant polycystic kidney
disease (ADPKD) produced only subtle if any clinically relevant effects on cyst growth and
the preservation of renal function.
In this study we will investigate if pulsed administration of SIR in a fixed weekly oral dose
of 3 mg over 24 months compared to placebo significantly reduces cyst growth and preserves
excretory renal function in patients with ADPKD and an estimated glomerular filtration (eGFR)
rate below 60 mL/min per 1.73m2.
n/a
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT02948179 -
Using Preimplantation Genetic Diagnosis in Autosomal Dominant Polycystic Kidney Disease Patients: a Multicenter Clinical Trial
|
N/A |