Pilot Study of Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD)

This study has been completed.
Sponsor:
Collaborator:
Wyeth is now a wholly owned subsidiary of Pfizer
Information provided by (Responsible Party):
The Cleveland Clinic
ClinicalTrials.gov Identifier:
NCT00286156
First received: February 1, 2006
Last updated: March 31, 2015
Last verified: March 2014
Results First Received: March 31, 2015  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Efficacy Study;   Intervention Model: Factorial Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Polycystic Kidney Diseases
Intervention: Drug: Rapamune

  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
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Standard Rapamycin Dose (STD)

Arm 1 Rapamune dose 2-6mg aimed at maintaining trough levels 5-8 ng/ml

Rapamycin: Group 1- doses of Rapamune aimed at maintaining trough levels 5-8ng/ml

Low Dose Rapamycin (LD)

Arm 2 Rapamune dose 2-6 mg aimed at maintaining trough levels of 2-5ng/ml

Rapamycin: Group 2 - doses of Rapamune aimed at maintaining trough levels 2-5ng/ml

Standard Care Standard Care: fluid intake primarily water of 2500-3000ml/24hrs, low sodium diet of 2300mg/24 hrs, caffeine avoidance, control of hypertension

Participant Flow:   Overall Study
    Standard Rapamycin Dose (STD)     Low Dose Rapamycin (LD)     Standard Care  
STARTED     10     10     10  
COMPLETED     8     9     9  
NOT COMPLETED     2     1     1  
Missed 12 month visit                 0                 0                 1  
AE: pulmonary embolus                 0                 1                 0  
AE: nephrotic-range proteinuruia                 1                 0                 0  
AE: pneumonia                 1                 0                 0  



  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.
No text entered.

Reporting Groups
  Description
Standard Rapamycin Dose (STD)

Arm 1 Rapamune dose 2-6mg aimed at maintaining trough levels 5-8 ng/ml

Rapamycin: Group 1- doses of Rapamune aimed at maintaining trough levels 5-8ng/ml

Low Dose Rapamycin (LD)

Arm 2 Rapamune dose 2-6 mg aimed at maintaining trough levels of 2-5ng/ml

Rapamycin: Group 2 - doses of Rapamune aimed at maintaining trough levels 2-5ng/ml

Standard Care Standard Care: fluid intake primarily water of 2500-3000ml/24hrs, low sodium diet of 2300mg/24 hrs, caffeine avoidance, control of hypertension
Total Total of all reporting groups

Baseline Measures
    Standard Rapamycin Dose (STD)     Low Dose Rapamycin (LD)     Standard Care     Total  
Number of Participants  
[units: participants]
  10     10     10     30  
Age  
[units: years]
Mean (Standard Deviation)
  53.2  (15.0)     44.9  (8.6)     49.4  (11.0)     49.3  (12.0)  
Gender  
[units: participants]
       
Female     5     5     3     13  
Male     5     5     7     17  
Race (NIH/OMB)  
[units: participants]
       
American Indian or Alaska Native     0     0     0     0  
Asian     0     0     0     0  
Native Hawaiian or Other Pacific Islander     0     0     0     0  
Black or African American     0     0     1     1  
White     10     10     9     29  
More than one race     0     0     0     0  
Unknown or Not Reported     0     0     0     0  
Region of Enrollment  
[units: participants]
       
United States     10     10     10     30  
Baseline characteristics and risk factors [1]
[units: participants]
       
Hypertension     3     3     5     11  
Family history of ESRD     4     8     7     19  
Initial iGFR 25-59 ml/min per 1.73 m^2     3     4     2     9  
Initial TKV>1500ml     6     7     6     19  
Initial height-adjusted TKV>=600ml/m     6     8     6     20  
[1] ADPKD, autosomal dominant polycystic kidney disease; iGFR, 125I-iothalamate GFR; TKV, total kidney volume; Hypertension is a BP >= 140/90 mmHg before 35 years old; family history of ESRD occurs from ADPKD before 56 years old



  Outcome Measures
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1.  Primary:   Change in GFR From Baseline to 12 Months   [ Time Frame: From baseline to 12 months ]

2.  Secondary:   Change in Total Kidney Volume as Measured by 3D-CT From Baseline to 12 Months   [ Time Frame: From baseline to 12 months ]


  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:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Dr William Braun Consultant Staff Nephrology, Cleveland Clinic
Organization: Cleveland Clinic
phone: 216/444/6995
e-mail: braunw@ccf.org


No publications provided


Responsible Party: The Cleveland Clinic
ClinicalTrials.gov Identifier: NCT00286156     History of Changes
Other Study ID Numbers: 7736
Study First Received: February 1, 2006
Results First Received: March 31, 2015
Last Updated: March 31, 2015
Health Authority: United States: Food and Drug Administration