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Pulsed Oral Sirolimus in Autosomal Dominant Polycystic Kidney Disease

NCT02055079 · Status: UNKNOWN · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 68

Last updated 2018-10-31

No results posted yet for this study

Summary

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.

Conditions

  • Polycystic Kidney, Type 1 Autosomal Dominant Disease
  • Polycystic Kidney, Type 2 Autosomal Dominant Disease

Interventions

DRUG

Sirolimus

Fixed oral dose of 3 mg sirolimus (blinded) once weekly for 24 months.

DRUG

Placebo

Fixed oral dose of 3 mg placebo (blinded) once weekly for 24 months.

Sponsors & Collaborators

  • Medical University of Vienna

    lead OTHER

Principal Investigators

  • Markus Riegersperger, MD · Medical University of Vienna

  • Gere Sunder-Plassmann, MD · Medical University of Vienna

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-04-30
Primary Completion
2019-04-30
Completion
2019-12-31

Countries

  • Austria

Study Locations

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Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02055079 on ClinicalTrials.gov