Sirolimus treatment in patients with autosomal dominant polycystic kidney disease renal efficacy and safety - ND

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2006-003427-37-IT

Enrollment

Registered

2006-12-27

Start date

2007-02-05

Completion date

Unknown

Last updated

2019-06-30

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Autosomal-Dominant Polycystic Kidney Disease ADPKD MedDRA version: 9.1 Level: LLT Classification code 10010428 Term: Congenital cystic kidney disease

Interventions

Trade Name: RAPAMUNE*30CPR RIV 1MG Pharmaceutical Form: Tablet INN or Proposed INN: Sirolimus Concentration unit: mg milligram(s) Concen

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: - Age 18 years - Clinical and ultrasound diagnosis of ADPKD - GFR 40 ml/min/1.73 m2 estimated by the 4 variable MDRD equation - Written informed consent Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: - Diabetes - Overt proteinuria urinary protein excretion rate 1g/24 hours or abnormal urinalysis suggestive of concomitant, clinically significant glomerular disease - Urinary tract lithiasis, infection or obstruction - Cancer - Psychiatric disorders and any condition that might prevent full comprehension of the purposes and risks of the study - Pregnancy, lactation or child bearing potential and ineffective contraception estrogen therapy in post menopausal women should not be stopped

Design outcomes

Primary

Measure	Time frame
Main Objective: The general aim of this randomized, open, cross-over study is to assess the efficacy and safety of 6 month treatment with sirolimus on the top of the best available therapy as compared to conventional therapy in adult patients with ADPKD and normal renal function or mild to moderate renal insufficiency. Specifically, the following aims will be pursued Main objective To compare the change over baseline of the total kidney volume estimated by computed tomography, CT in sirolimus and conventional treatment ADPKD groups during 6 month follow-up.;Secondary Objective: As secondary efficacy endpoints, the following parameters will be compared in the two ADPKD groups at baseline and at 6 months follow-up - Total kidney volume - Renal cyst volume - Renal parenchymal volume - Renal intermediate volume;Primary end point(s): To compare the change over baseline of the total kidney volume estimated by computed tomography, CT in sirolimus and conventional treatment ADPKD groups during 6 month follow-up.	—

Measure

Time frame

Main Objective: The general aim of this randomized, open, cross-over study is to assess the efficacy and safety of 6 month treatment with sirolimus on the top of the best available therapy as compared to conventional therapy in adult patients with ADPKD and normal renal function or mild to moderate renal insufficiency. Specifically, the following aims will be pursued Main objective To compare the change over baseline of the total kidney volume estimated by computed tomography, CT in sirolimus and conventional treatment ADPKD groups during 6 month follow-up.;Secondary Objective: As secondary efficacy endpoints, the following parameters will be compared in the two ADPKD groups at baseline and at 6 months follow-up - Total kidney volume - Renal cyst volume - Renal parenchymal volume - Renal intermediate volume;Primary end point(s): To compare the change over baseline of the total kidney volume estimated by computed tomography, CT in sirolimus and conventional treatment ADPKD groups during 6 month follow-up.

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Countries

Italy

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026