Phase 4
N=431
Efficacy, Safety and Tolerability of Everolimus in Preventing End-stage Renal Disease in Patients With Autosomal Dominant Polycystic Kidney Disease
Autosomal Dominant Polycystic Kidney Disease
Bottom Line
View on ClinicalTrials.gov: NCT00414440 ↗Enrolled (actual)
431
Serious AEs
30.4%
Results posted
Jan 2015
Primary outcome: Primary: Primary Efficacy Analysis of Total Kidney Volume (mITT Set, Multiple Imputation) — 230.1; 300.8 mL
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- Placebo (Drug); Everolimus (Drug)
- Age
- Adult · 18+ yrs
- Sex
- All
- Sponsor
- Novartis Pharmaceuticals
- Primary completion
- Oct 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Primary Efficacy Analysis of Total Kidney Volume (mITT Set, Multiple Imputation) |
230.1; 300.8 | — |
| SECONDARY Course of Calculated GFR (mL/Min/1.73 m^2) From Month 24 to Month 60 |
43.9; 48.8; 42.6; 44.9; 39.6; 42.7 | — |
| SECONDARY Calculated GFR, Change From Baseline at Month 60 by Baseline cGFR |
-14.8; -20.5; -16.1; -15.6; -17.0; -20.0 | — |
| SECONDARY Changes in Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP) |
136; 135; 134; 134; 134; 134 | — |
| SECONDARY Calculated GFR (mL/Min/1.73 m^2), Change From Baseline by Visit |
2.0; -0.9; 1.7; -0.9; 0.6; -1.2 | — |
Summary
This study will assess whether everolimus (RAD001) is effective in preventing cyst and kidney expansion as well as worsening of renal function in patients with ADPKD and whether the application of 5 mg/day everolimus as monotherapy is safe and well tolerated.
Eligibility Criteria
Inclusion Criteria
- Clinical diagnosis of autosomal dominant polycystic kidney disease ADPKD
- Chronic kidney disease (CKD) stage II / III
- Females capable of becoming pregnant must have a negative serum pregnancy test within 7 days prior to or at baseline, and are required to practice an approved method of birth control for the duration of the study and for a period of 6 weeks following discontinuation of study medication, even where there has been a history of infertility
Exclusion Criteria
- ADPKD patients with normal renal function
- ADPKD patients with CKD stage IV
- Patients with a history of subarachnoid bleeding
- Patients with a history of severe infections
- Patients with life-threatening urinary tract or cyst infection in the past
- Patients who have received any investigational drug within four weeks prior to baseline
- Patients who have been treated with any non-protocol immunosuppressive drug or treatment within one month prior to baseline
Other protocol-defined inclusion/exclusion criteria may apply
Data sourced from ClinicalTrials.gov (NCT00414440). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.