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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: January 13, 2015
Last verified: January 2015
Results First Received: October 7, 2014  
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
Condition: Autosomal Dominant Polycystic Kidney Disease
Interventions: Drug: Placebo
Drug: Everolimus

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
Everolimus Patients in the everolimus group initially received 5 mg/day everolimus divided in 2 equal doses (i.e. 2.5 mg b.i.d.). Dose adjustments were performed to achieve a blood trough level of 3-8 ng/mL (maximum daily dose: 10 mg/day [5 mg b.i.d.]).
Placebo Placebo tablets equivalent to the dosage of everolimus 5 mg/day, divided in 2 equal doses.

Participant Flow for 2 periods

Period 1:   Core Period
    Everolimus   Placebo
STARTED   214 [1]   217 [1] 
COMPLETED   144 [1]   185 [1] 
NOT COMPLETED   70   32 
Withdrawal by Subject                70                32 
[1] Core period

Period 2:   Extension Period
    Everolimus   Placebo
STARTED   214 [1]   217 [1] 
COMPLETED   64 [1]   77 [1] 
NOT COMPLETED   150   140 
Lost to Follow-up                44                52 
Did not consent to continue follow up                61                54 
Other                1                1 
Administrative                16                16 
Death                1                2 
Withdrawal by Subject                9                7 
Not included in follow up period                18                8 
[1] Extension



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups
  Description
Everolimus Patients in the everolimus group initially received 5 mg/day everolimus divided in 2 equal doses (i.e. 2.5 mg b.i.d.). Dose adjustments were performed to achieve a blood trough level of 3-8 ng/mL (maximum daily dose: 10 mg/day [5 mg b.i.d.]).
Placebo Placebo tablets equivalent to the dosage of everolimus 5 mg/day, divided in 2 equal doses.
Total Total of all reporting groups

Baseline Measures
   Everolimus   Placebo   Total 
Overall Participants Analyzed 
[Units: Participants]
 214   217   431 
Age [1] 
[Units: Years]
Mean (Standard Deviation)
 44.6  (10.1)   44.5  (10.4)   44.5  (10.3) 
[1] Average age of participants
Gender 
[Units: Participants]
     
Female   105   117   222 
Male   109   100   209 
Race/Ethnicity, Customized 
[Units: Participants]
     
Asian Oriental   4   0   4 
Black or African American   1   0   1 
White   208   217   425 
Other, Unknown or Not Reported   1   0   1 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Primary Efficacy Analysis of Total Kidney Volume (mITT Set, Multiple Imputation)   [ Time Frame: Baseline, Month 24 ]

2.  Secondary:   Course of Calculated GFR (mL/Min/1.73 m^2) From Month 24 to Month 60   [ Time Frame: Months 24, 36, 48 and 60 ]

3.  Secondary:   Calculated GFR, Change From Baseline at Month 60 by Baseline cGFR   [ Time Frame: Months 24, 36, 48 and 60 ]

4.  Secondary:   Changes in Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP)   [ Time Frame: Baseline, Months 12 and 24 ]

5.  Secondary:   Calculated GFR (mL/Min/1.73 m^2), Change From Baseline by Visit   [ Time Frame: Months 3, 6, 9, 12, 18 and 24 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Study Director
Organization: Novartis
phone: +1 (862) 778-8300


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
Results First Received: October 7, 2014
Last Updated: January 13, 2015
Health Authority: United States: Food and Drug Administration
Germany: Bundesinstitut für Arzneimittel und Medizinproduke