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Trial record 1 of 1 for:    7935686 [PUBMED-IDS]
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Pirfenidone to Treat Kidney Disease (Focal Segmental Glomerulosclerosis)

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ClinicalTrials.gov Identifier: NCT00001959
Recruitment Status : Completed
First Posted : January 19, 2000
Results First Posted : June 1, 2012
Last Update Posted : May 26, 2014
Sponsor:
Information provided by (Responsible Party):
Jeffrey Kopp, M.D., National Institutes of Health Clinical Center (CC)

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Fibrosis
Focal Glomerulosclerosis
Kidney Failure
Nephrotic Syndrome
Proteinuria
Intervention Drug: Pirfenidone
Enrollment 21
Recruitment Details 21 adults patients were enrolled between 2000-2004 at the clinical center of NIH
Pre-assignment Details  
Arm/Group Title Pirfenidone
Hide Arm/Group Description During the study drug period of 12 months, patients will receive oral pirfenidone daily. For patients whose initial renal function is 50-80 ml/min as assessed by the MDRD equation, the initial pirfenidone dosage will be calculated at 40 mg/kg/d, with a maximum dose of 800 mg TID. For patients whose initial renal function is 30-50 ml/min, the initial dose will be 30 mg/kg/d. For patients whose initial renal function is between 15 and 30 ml/min, the initial dose will be 20 mg/kg/d.
Period Title: Overall Study
Started 21
Completed 18
Not Completed 3
Reason Not Completed
newly diagnosed colon cancer             1
disabling sedation             1
non-compliance             1
Arm/Group Title Pirfenidone
Hide Arm/Group Description During the study drug period of 12 months, patients will receive oral pirfenidone daily. For patients whose initial renal function is 50-80 ml/min as assessed by the MDRD equation, the initial pirfenidone dosage will be calculated at 40 mg/kg/d, with a maximum dose of 800 mg TID. For patients whose initial renal function is 30-50 ml/min, the initial dose will be 30 mg/kg/d. For patients whose initial renal function is between 15 and 30 ml/min, the initial dose will be 20 mg/kg/d.
Overall Number of Baseline Participants 18
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 18 participants
47  (10)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants
Female
7
  38.9%
Male
11
  61.1%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants
American Indian or Alaska Native
0
   0.0%
Asian
2
  11.1%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
8
  44.4%
White
8
  44.4%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 18 participants
18
Baseline GFR  
Mean (Standard Deviation)
Unit of measure:  Ml/min/1.73 m^2
Number Analyzed 18 participants
26.0  (9.4)
Baseline period proteinuria  
Median (Inter-Quartile Range)
Unit of measure:  G/d
Number Analyzed 18 participants
3.2
(1.6 to 4.4)
Decrease in GFR during baseline period  
Median (Inter-Quartile Range)
Unit of measure:  Ml/min/1.73 m^2
Number Analyzed 18 participants
-0.61
(-1.31 to -0.41)
Duration of angiotensin antagonist therapy before treatment   [1] 
Median (Inter-Quartile Range)
Unit of measure:  Month
Number Analyzed 18 participants
15
(8.4 to 33)
[1]
Measure Description: 2 patients did not receive angiotensin antagonist therapy during the baseline periods because of angioedema and hypotension.
1.Primary Outcome
Title Decrease in GFR During Treatment Period
Hide Description [Not Specified]
Time Frame 12 months from baseline
Hide Outcome Measure Data
Hide Analysis Population Description
ITT
Arm/Group Title Pirfenidone
Hide Arm/Group Description:
During the study drug period of 12 months, patients will receive oral pirfenidone daily. For patients whose initial renal function is 50-80 ml/min as assessed by the MDRD equation, the initial pirfenidone dosage will be calculated at 40 mg/kg/d, with a maximum dose of 800 mg TID. For patients whose initial renal function is 30-50 ml/min, the initial dose will be 30 mg/kg/d. For patients whose initial renal function is between 15 and 30 ml/min, the initial dose will be 20 mg/kg/d.
Overall Number of Participants Analyzed 18
Mean (Inter-Quartile Range)
Unit of Measure: ml/min/1.73 m2
-0.45
(-0.78 to -0.16)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Pirfenidone
Comments H0: GFR Decrease during baseline period and treatment period are same
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.01
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
2.Secondary Outcome
Title Proteinuria After Treatment
Hide Description [Not Specified]
Time Frame 12 months from baseline
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Pirfenidone
Hide Arm/Group Description:
During the study drug period of 12 months, patients will receive oral pirfenidone daily. For patients whose initial renal function is 50-80 ml/min as assessed by the MDRD equation, the initial pirfenidone dosage will be calculated at 40 mg/kg/d, with a maximum dose of 800 mg TID. For patients whose initial renal function is 30-50 ml/min, the initial dose will be 30 mg/kg/d. For patients whose initial renal function is between 15 and 30 ml/min, the initial dose will be 20 mg/kg/d.
Overall Number of Participants Analyzed 18
Median (Inter-Quartile Range)
Unit of Measure: g/d
4.3
(1.2 to 6.7)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Pirfenidone
Comments H0: Proteinuria during baseline and treatment periods are same
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.16
Comments [Not Specified]
Method Kruskal-Wallis
Comments [Not Specified]
3.Secondary Outcome
Title Proportion of Patients With Positive Change in GFR
Hide Description [Not Specified]
Time Frame 12 months from baseline
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Pirfenidone
Hide Arm/Group Description:
During the study drug period of 12 months, patients will receive oral pirfenidone daily. For patients whose initial renal function is 50-80 ml/min as assessed by the MDRD equation, the initial pirfenidone dosage will be calculated at 40 mg/kg/d, with a maximum dose of 800 mg TID. For patients whose initial renal function is 30-50 ml/min, the initial dose will be 30 mg/kg/d. For patients whose initial renal function is between 15 and 30 ml/min, the initial dose will be 20 mg/kg/d.
Overall Number of Participants Analyzed 18
Measure Type: Number
Unit of Measure: participants
13
Time Frame 60 months
Adverse Event Reporting Description Include all adverse events either judged to be related to the study or unrelated to the study
 
Arm/Group Title Pirfenidone
Hide Arm/Group Description During the study drug period of 12 months, patients will receive oral pirfenidone daily. For patients whose initial renal function is 50-80 ml/min as assessed by the MDRD equation, the initial pirfenidone dosage will be calculated at 40 mg/kg/d, with a maximum dose of 800 mg TID. For patients whose initial renal function is 30-50 ml/min, the initial dose will be 30 mg/kg/d. For patients whose initial renal function is between 15 and 30 ml/min, the initial dose will be 20 mg/kg/d.
All-Cause Mortality
Pirfenidone
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Pirfenidone
Affected / at Risk (%) # Events
Total   3/21 (14.29%)    
Gastrointestinal disorders   
Colon cancer (unrelated to the trial)  1/21 (4.76%)  1
Hepatobiliary disorders   
elevated liver enzymes (judged possibly related to the trial)  1/21 (4.76%)  1
Injury, poisoning and procedural complications   
Death (unrelated to the trial) [1]  1/21 (4.76%)  1
[1]
traumatic subdural hematoma
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Pirfenidone
Affected / at Risk (%) # Events
Total   16/21 (76.19%)    
Gastrointestinal disorders   
Gastrointestinal discomfort  8/21 (38.10%)  8
General disorders   
Sedation or fatigue  6/21 (28.57%)  6
Skin and subcutaneous tissue disorders   
photosensitivity dermatitis  2/21 (9.52%)  2
No control group, measurement imprecision, results may not applicable to all patients with FSGS, needs histologic evidence to support our findings, Optimal dosage needs to be identified.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Jeffrey B Kopp, MD
Organization: NIDDK, NIH
Phone: 3015943403
Responsible Party: Jeffrey Kopp, M.D., National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier: NCT00001959     History of Changes
Other Study ID Numbers: 000042
00-DK-0042
First Submitted: January 18, 2000
First Posted: January 19, 2000
Results First Submitted: June 16, 2010
Results First Posted: June 1, 2012
Last Update Posted: May 26, 2014