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Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD): The Role of Biomarkers in Predicting a Response to Therapy

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ClinicalTrials.gov Identifier: NCT00920309
Recruitment Status : Terminated (This study was stopped after larger studies published in the NEJM failed to show a benefit in treating ADPKD)
First Posted : June 15, 2009
Results First Posted : April 14, 2014
Last Update Posted : April 14, 2014
Sponsor:
Information provided by (Responsible Party):
Yale University

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Autosomal Dominant Polycystic Kidney Disease
Interventions Drug: Rapamycin
Other: Standard of Care-Placebo
Enrollment 21
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Rapamycin Standard of Care-Placebo
Hide Arm/Group Description

Drug: Rapamycin

Other Names:

sirolimus The starting dose of rapamycin will be 1 mg daily. The dose will be increased as needed to achieve a 24 hour trough level of 4-6 ng/ml.

[Not Specified]
Period Title: Overall Study
Started 14 7
Completed 14 7
Not Completed 0 0
Arm/Group Title Rapamycin Standard of Care-Placebo Total
Hide Arm/Group Description

Drug: Rapamycin

Other Names:

sirolimus The starting dose of rapamycin will be 1 mg daily. The dose will be increased as needed to achieve a 24 hour trough level of 4-6 ng/ml.

[Not Specified] Total of all reporting groups
Overall Number of Baseline Participants 14 7 21
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 14 participants 7 participants 21 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
13
  92.9%
7
 100.0%
20
  95.2%
>=65 years
1
   7.1%
0
   0.0%
1
   4.8%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 14 participants 7 participants 21 participants
Female
5
  35.7%
2
  28.6%
7
  33.3%
Male
9
  64.3%
5
  71.4%
14
  66.7%
1.Primary Outcome
Title Total Kidney Volume (mL)
Hide Description [Not Specified]
Time Frame 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Outcome Measures were not analyzed due to study termination
Arm/Group Title Rapamycin Standard of Care-Placebo
Hide Arm/Group Description:
The study was terminated before any enrolled patients were treated for 2 years.
[Not Specified]
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
2.Secondary Outcome
Title Glomerular Filtration Rate (Kidney Function)
Hide Description [Not Specified]
Time Frame 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Outcome measures were not analyzed due to study termination
Arm/Group Title Rapamycin Standard of Care-Placebo
Hide Arm/Group Description:

Drug: Rapamycin

Other Names:

sirolimus The starting dose of rapamycin will be 1 mg daily. The dose will be increased as needed to achieve a 24 hour trough level of 4-6 ng/ml.

[Not Specified]
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame The study occurred over a 1 year time period which was on adverse events were collected. This study was terminated early.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Rapamycin Standard of Care-Placebo
Hide Arm/Group Description

Drug: Rapamycin

Other Names:

sirolimus The starting dose of rapamycin will be 1 mg daily. The dose will be increased as needed to achieve a 24 hour trough level of 4-6 ng/ml.

[Not Specified]
All-Cause Mortality
Rapamycin Standard of Care-Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Rapamycin Standard of Care-Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   3/14 (21.43%)   0/7 (0.00%) 
Endocrine disorders     
Hyperglycemia *  1/14 (7.14%)  0/7 (0.00%) 
General disorders     
Flank Pain *  1/14 (7.14%)  0/7 (0.00%) 
Renal and urinary disorders     
Proteinuria *  1/14 (7.14%)  0/7 (0.00%) 
*
Indicates events were collected by non-systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Rapamycin Standard of Care-Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   14/14 (100.00%)   0/7 (0.00%) 
Blood and lymphatic system disorders     
Thrombocytopenia *  1/14 (7.14%)  0/7 (0.00%) 
Neutropenia *  1/14 (7.14%)  0/7 (0.00%) 
Anemia *  1/14 (7.14%)  0/7 (0.00%) 
Cardiac disorders     
Heart Palipitations with Hypertension *  1/14 (7.14%)  0/7 (0.00%) 
Endocrine disorders     
Hyperglycemia *  1/14 (7.14%)  0/7 (0.00%) 
Dysuria *  1/14 (7.14%)  0/7 (0.00%) 
Gastrointestinal disorders     
Emesis/Diarrhea *  2/14 (14.29%)  0/7 (0.00%) 
Abdominal Pain *  1/14 (7.14%)  0/7 (0.00%) 
Nausea *  1/14 (7.14%)  0/7 (0.00%) 
Bloating/Flatulence *  1/14 (7.14%)  0/7 (0.00%) 
General disorders     
Insomnia *  1/14 (7.14%)  0/7 (0.00%) 
Sinus Headache *  1/14 (7.14%)  0/7 (0.00%) 
Allergic Rhinitis *  1/14 (7.14%)  0/7 (0.00%) 
Fatigue *  1/14 (7.14%)  0/7 (0.00%) 
Epistaxis *  1/14 (7.14%)  0/7 (0.00%) 
Weakness *  1/14 (7.14%)  0/7 (0.00%) 
Ear/Neck Pain *  1/14 (7.14%)  0/7 (0.00%) 
Facial Sensitivity *  1/14 (7.14%)  0/7 (0.00%) 
Hepatobiliary disorders     
Increased Liver Function Test *  2/14 (14.29%)  0/7 (0.00%) 
Infections and infestations     
Cellulitis of the ear *  1/14 (7.14%)  0/7 (0.00%) 
Upper Respiratory Infesion *  1/14 (7.14%)  0/7 (0.00%) 
Apothous Ulcer *  2/14 (14.29%)  0/7 (0.00%) 
Musculoskeletal and connective tissue disorders     
Back Pain *  4/14 (28.57%)  0/7 (0.00%) 
Arthralgias *  1/14 (7.14%)  0/7 (0.00%) 
Left Hip Stiffness *  1/14 (7.14%)  0/7 (0.00%) 
Myalgia *  1/14 (7.14%)  0/7 (0.00%) 
Nervous system disorders     
Headache *  1/14 (7.14%)  0/7 (0.00%) 
Impaired Depth Perception *  1/14 (7.14%)  0/7 (0.00%) 
Renal and urinary disorders     
Hematuria *  1/14 (7.14%)  0/7 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Dyspnea *  1/14 (7.14%)  0/7 (0.00%) 
Skin and subcutaneous tissue disorders     
Acne *  1/14 (7.14%)  0/7 (0.00%) 
Skin Abscess *  2/14 (14.29%)  0/7 (0.00%) 
Hives *  1/14 (7.14%)  0/7 (0.00%) 
Vascular disorders     
Edema/Weight gain *  3/14 (21.43%)  0/7 (0.00%) 
Pedal Edema *  1/14 (7.14%)  0/7 (0.00%) 
*
Indicates events were collected by non-systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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.
Results Point of Contact
Name/Title: Dr. Neera Dahl
Organization: Yale University School of Medicine
Phone: 203-785-4184
Responsible Party: Yale University
ClinicalTrials.gov Identifier: NCT00920309     History of Changes
Other Study ID Numbers: HIC#0903004934
First Submitted: June 12, 2009
First Posted: June 15, 2009
Results First Submitted: August 29, 2013
Results First Posted: April 14, 2014
Last Update Posted: April 14, 2014