Pulsed Oral Sirolimus in Autosomal Dominant Polycystic Kidney Disease (RAP)
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|ClinicalTrials.gov Identifier: NCT02055079|
Recruitment Status : Recruiting
First Posted : February 4, 2014
Last Update Posted : October 31, 2018
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.
|Condition or disease||Intervention/treatment||Phase|
|Polycystic Kidney, Type 1 Autosomal Dominant Disease Polycystic Kidney, Type 2 Autosomal Dominant Disease||Drug: Sirolimus Drug: Placebo||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||68 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||Pulsed Oral Sirolimus in Autosomal Dominant Polycystic Kidney Disease - The Vienna RAP Study|
|Study Start Date :||April 2014|
|Estimated Primary Completion Date :||April 2019|
|Estimated Study Completion Date :||December 2019|
Fixed oral dose of 3 mg Sirolimus (blinded) once weekly for 24 months.
Fixed oral dose of 3 mg sirolimus (blinded) once weekly for 24 months.
Other Name: Rapamune
Placebo Comparator: Placebo
Fixed oral dose of placebo (blinded) once weekly for 24 months.
Fixed oral dose of 3 mg placebo (blinded) once weekly for 24 months.
- Change in kidney function from baseline to month 24 [ Time Frame: Baseline, 24 months ]Fifty percent reduction in doubling of serum creatinine, or initiation of dialysis over a period of two years. Less or equal than 1.5 fold increase in serum creatinine without initiation of dialysis over two years is considered a beneficial outcome, increases in serum creatinine greater than 1.5 over two years or initiation of dialysis are considered a non-beneficial outcome.
- Change of safety parameters from baseline to month 24 [ Time Frame: Baseline, 24 months ]Safety, change in proteinuria, as indicated by albumin/creatinine- and protein/creatinine ratio, respectively.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02055079
|Contact: Markus Riegersperger, MD||0043140400 ext email@example.com|
|Contact: Gere Sunder-Plassmann, MD||0043140400 ext firstname.lastname@example.org|
|Division of Nephrology and Dialysis, Department of Medicine III, Medical University of Vienna||Recruiting|
|Vienna, Austria, 1090|
|Contact: Markus Riegersperger, MD 0043140400 ext 4391 email@example.com|
|Contact: Gere Sunder-Plassmann, MD 0043140400 ext 4391 firstname.lastname@example.org|
|Principal Investigator: Markus Riegersperger, MD|
|Sub-Investigator: Gere Sunder-Plassmann, MD|
|Principal Investigator:||Markus Riegersperger, MD||Medical University of Vienna|
|Principal Investigator:||Gere Sunder-Plassmann, MD||Medical University of Vienna|