polycystic kidney disease MedDRA version: 8.1 Level: LLT Classification code 10011771 Term: Cystic kidney disease
Conditions
Interventions
Product Name: Certican Tabletten
Product Code: RAD001
Pharmaceutical Form: Tablet
INN or Proposed INN: Everolimus
Current Sponsor code: RAD001
Concentration unit: mg milligram(s)
Concentration type: e
Sponsors
Novartis Pharma GmbH
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Males or females, aged 18 – 65 years 2. Clinical diagnosis of autosomal dominant polycystic kidney disease (ADPKD), (Appendix 6) 3. CKD stage II/III, i.e. calculated GFR (MDRD formula) (ml/min/1.73 m²) has to be 30 - 89 or CKD I, i.e. calculated GFR (MDRD formula) (ml/min/1.73 m²) is = 90 and a kidney volume (estimated by ultrasound) > 1000 mL 4. 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 (see Section 8.2) 5. Patients who are willing and able to participate in the study and from whom written informed consent has been obtained 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) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. CKD I (a calculated GFR = 90 mL/min/1.73m2) and a kidney volume (estimated by ultrasound) of = 1000 ml 2. Patients with CKD stage IV (a calculated GFR 3 times UNL) 11. Patients who are HIV positive or Hepatitis B surface antigen positive or who are known to have chronic active Hepatitis C. Laboratory results obtained within 6 months prior to baseline are acceptable 12. Patients with symptoms of significant somatic or mental illness. Inability to cooperate or communicate with the investigator, who are unlikely to comply with the study requirements, or who are unable to give informed consent 13. Patients with unresolved history or evidence of drug or alcohol abuse 14. Presence of severe hypercholesterolemia (= 350 mg/dL, 9.1 mmoL/L), or hypertriglyceridemia (= 500 mg/dL, 5.6 mmoL/L), if hyperlipidemia is not sufficiently controlled by fluvastatines 15. Patients with white blood cell count count = 4,000/mm³, or platelet count = 100,000/mm³ 16. Patients with a history of malignancy during the last five years, except for successfully treated localized squamous or basal cell carcinoma of the skin 17. Patients with any history of coagulopathy or medical condition requiring long-term anticoagulation (low-dose aspirin treatment is allowed) 18. Patients with abnormal physical or laboratory findings of clinical significance within two weeks prior to randomization which at the investigators discretion would interfere with the objectives of the study 19. Females of childbearing potential who are planning to become pregnant, who are pregnant or breast feading, and/or who are unwilling to use effective means of contraception during the study period and until six weeks after discontinuation of study medication. 20. Patients with a contraindication against MRI
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of this trial is to demonstrate that everolimus (Certican®) has superior efficacy compared with placebo in reducing the mean total kidney volume from baseline to 24 months of treatment in patients with ADPKD.;Secondary Objective: Secondary objectives of this trial are: • To assess changes from baseline at Month 24 in mean cyst and parenchyma volumes • To assess changes in renal function between baseline and Month 24 as assessed by calculated GFR (MDRD formula), serum creatinine, and proteinuria • To assess incidence of end-stage renal disease (ESRD), defined by the requirement for renal replacement therapy • To assess changes from baseline at Month 24 in blood pressure • To assess the overall survival • To assess safety and tolerability of the study drug;Primary end point(s): The primary variable for assessment of total kidney volume measured by MRI is the change from baseline in total renal volume [ml/year] at Month 24. | — |
Countries
Austria, France, Germany
Outcome results
None listed