Trial Outcomes & Findings for Pirfenidone to Treat Kidney Disease (Focal Segmental Glomerulosclerosis) (NCT NCT00001959)

NCT ID: NCT00001959

Last Updated: 2014-05-26

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 21 participants
• Primary outcome timeframe: 12 months from baseline
• Results posted on: 2014-05-26

Participant Flow

21 adults patients were enrolled between 2000-2004 at the clinical center of NIH

Participant milestones

Measure	Pirfenidone 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 Study STARTED	21
Overall Study COMPLETED	18
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Measure	Pirfenidone 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 Study newly diagnosed colon cancer	1
Overall Study disabling sedation	1
Overall Study non-compliance	1

Baseline Characteristics

Pirfenidone to Treat Kidney Disease (Focal Segmental Glomerulosclerosis)

Baseline characteristics by cohort

Measure	Pirfenidone n=18 Participants 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.
Age, Continuous	47 years STANDARD_DEVIATION 10 • n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants
Sex: Female, Male Male	11 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	2 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	8 Participants n=5 Participants
Race (NIH/OMB) White	8 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	18 participants n=5 Participants
Baseline GFR	26.0 ml/min/1.73 m^2 STANDARD_DEVIATION 9.4 • n=5 Participants
Baseline period proteinuria	3.2 g/d n=5 Participants
Decrease in GFR during baseline period	-0.61 ml/min/1.73 m^2 n=5 Participants
Duration of angiotensin antagonist therapy before treatment	15 month n=5 Participants

PRIMARY outcome

Timeframe: 12 months from baseline

Population: ITT

Outcome measures

Measure

Pirfenidone n=18 Participants 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.

Decrease in GFR During Treatment Period

-0.45 ml/min/1.73 m2 Interval -0.78 to -0.16

SECONDARY outcome

Timeframe: 12 months from baseline

Outcome measures

Measure

Pirfenidone n=18 Participants 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.

Proteinuria After Treatment

4.3 g/d Interval 1.2 to 6.7

SECONDARY outcome

Timeframe: 12 months from baseline

Outcome measures

Measure

Pirfenidone n=18 Participants 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.

Proportion of Patients With Positive Change in GFR

13 participants

Adverse Events

Pirfenidone

Serious events: 3 serious events

Other events: 16 other events

Deaths: 0 deaths

Serious adverse events

Measure	Pirfenidone n=21 participants at risk 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.
Injury, poisoning and procedural complications Death (unrelated to the trial)	4.8% 1/21 • Number of events 1 • 60 months Include all adverse events either judged to be related to the study or unrelated to the study
Gastrointestinal disorders Colon cancer (unrelated to the trial)	4.8% 1/21 • Number of events 1 • 60 months Include all adverse events either judged to be related to the study or unrelated to the study
Hepatobiliary disorders elevated liver enzymes (judged possibly related to the trial)	4.8% 1/21 • Number of events 1 • 60 months Include all adverse events either judged to be related to the study or unrelated to the study

Other adverse events

Measure	Pirfenidone n=21 participants at risk 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.
Gastrointestinal disorders Gastrointestinal discomfort	38.1% 8/21 • Number of events 8 • 60 months Include all adverse events either judged to be related to the study or unrelated to the study
General disorders Sedation or fatigue	28.6% 6/21 • Number of events 6 • 60 months Include all adverse events either judged to be related to the study or unrelated to the study
Skin and subcutaneous tissue disorders photosensitivity dermatitis	9.5% 2/21 • Number of events 2 • 60 months Include all adverse events either judged to be related to the study or unrelated to the study

Additional Information

Jeffrey B Kopp, MD

NIDDK, NIH

Phone: 3015943403

Email: jbkopp@nih.gov

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place