Efficacy, Safety and Tolerability of Everolimus in Preventing End-stage Renal Disease in Patients With Autosomal Dominant Polycystic Kidney Disease

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT00414440
First received: December 20, 2006
Last updated: March 6, 2014
Last verified: March 2014
  Purpose

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.


Condition Intervention Phase
Autosomal Dominant Polycystic Kidney Disease
Drug: RAD001 placebo
Drug: RAD001
Phase 4

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Multicenter, Randomized, Placebo-controlled, Double-blind Study on the Efficacy, Safety and Tolerability of Everolimus in Preventing End-stage Renal Disease (ESRD) in Patients With Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • Mean total kidney volume assessed by magnetic resonance imaging at 3 timepoints from the first day to the last day on study medication

Secondary Outcome Measures:
  • changes in mean cyst and parenchyma volumes assessed by magnetic resonance imaging between the first and last day on study medication.
  • changes in renal function assessed by urinalysis at 8 timepoints from the first day to last day on study medication.
  • incidence of newly developing end-stage renal disease (ESRD) assessed by number of patients who require renal replacement therapy during the conduct of the trial
  • safety and tolerability of the study drug assessed by monitoring and recording of adverse events, serious adverse events, and all infections
  • changes in blood pressure between the first and last day on study medication assessed by blood pressure measurements at 10 timepoints

Enrollment: 431
Study Start Date: December 2006
Study Completion Date: October 2013
Primary Completion Date: October 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1 Drug: RAD001
experimental
Other Name: certican
Placebo Comparator: 2 Drug: RAD001 placebo
placebo comparator

  Eligibility

Ages Eligible for Study:   18 Years to 50 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria

  1. Clinical diagnosis of autosomal dominant polycystic kidney disease ADPKD
  2. Chronic kidney disease (CKD) stage II / III
  3. 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

  1. ADPKD patients with normal renal function
  2. ADPKD patients with CKD stage IV
  3. Patients with a history of subarachnoid bleeding
  4. Patients with a history of severe infections
  5. Patients with life-threatening urinary tract or cyst infection in the past
  6. Patients who have received any investigational drug within four weeks prior to baseline
  7. 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

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00414440

Locations
Austria
Novartis Investigative Site
Innsbruck, Austria, INNSBRUCK
Novartis Investigative Site
Linz, Austria, A-4010
Novartis Investigative Site
Wien, Austria, 1090
France
Novartis Investigative Site
Brest, France, 29200
Novartis Investigative Site
Grenoble, France, 38043
Novartis Investigative Site
Nantes Cedex, France, 44035
Novartis Investigative Site
Paris cedex 15, France, 75015
Novartis Investigative Site
Toulouse Cedex 4, France, 31054
Germany
Novartis Investigative Site
Berlin, Germany, 13353
Novartis Investigative Site
Berlin, Germany, 10117
Novartis Investigative Site
Erlangen, Germany, 91054
Novartis Investigative Site
Essen, Germany, 45147
Novartis Investigative Site
Frankfurt am Main, Germany, 60596
Novartis Investigative Site
Freiburg, Germany, 79106
Novartis Investigative Site
Hamburg, Germany, 20246
Novartis Investigative Site
Heidelberg, Germany, 69120
Novartis Investigative Site
Homburg, Germany, 66421
Novartis Investigative Site
Kiel, Germany, 24105
Novartis Investigative Site
Koeln, Germany, 51109
Novartis Investigative Site
Leipzig, Germany, 04103
Novartis Investigative Site
Lübeck, Germany, 23538
Novartis Investigative Site
Muenster, Germany, 48149
Novartis Investigative Site
Regensburg, Germany, 93053
Novartis Investigative Site
Würzburg, Germany, 97080
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Additional Information:
No publications provided by Novartis

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT00414440     History of Changes
Other Study ID Numbers: CRAD001ADE12, 2006-001485-16
Study First Received: December 20, 2006
Last Updated: March 6, 2014
Health Authority: United States: Food and Drug Administration
Germany: Bundesinstitut für Arzneimittel und Medizinproduke

Keywords provided by Novartis:
end-stage renal disease, ESRD, autosomal dominant polycystic kidney disease, ADPKD, everolimus

Additional relevant MeSH terms:
Polycystic Kidney, Autosomal Dominant
Polycystic Kidney Diseases
Kidney Diseases
Kidney Failure, Chronic
Multicystic Dysplastic Kidney
Urologic Diseases
Renal Insufficiency, Chronic
Renal Insufficiency
Kidney Diseases, Cystic
Urogenital Abnormalities
Congenital Abnormalities
Sirolimus
Everolimus
Anti-Bacterial Agents
Anti-Infective Agents
Therapeutic Uses
Pharmacologic Actions
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on September 30, 2014