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Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome

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ClinicalTrials.gov Identifier: NCT00001596
Recruitment Status : Completed
First Posted : November 4, 1999
Results First Posted : January 26, 2012
Last Update Posted : October 16, 2017
Sponsor:
Collaborator:
National Human Genome Research Institute (NHGRI)
Information provided by (Responsible Party):
William Gahl, M.D., National Human Genome Research Institute (NHGRI)

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Albinism
Inborn Errors of Metabolism
Oculocutaneous Albinism
Platelet Storage Pool Deficiency
Pulmonary Fibrosis
Interventions Drug: Pirfenidone
Drug: Placebo
Enrollment 35
Recruitment Details Patients were enrolled at the NIH Clinical Center between September 2005 to March 2009.
Pre-assignment Details  
Arm/Group Title Pirfenidone Placebo
Hide Arm/Group Description Subjects received pirfenidone 801 mg (3 pills of 267 mg each), three times daily. Subjects received placebo (3 pills), three times daily.
Period Title: Overall Study
Started 23 12
Completed 6 2
Not Completed 17 10
Reason Not Completed
Death             1             0
Withdrawal by Subject             3             1
Study Termination             12             9
Adverse Event             1             0
Arm/Group Title Pirfenidone Placebo Total
Hide Arm/Group Description Subjects received pirfenidone 801 mg (3 pills of 267 mg each), three times daily. Subjects received placebo (3 pills), three times daily. Total of all reporting groups
Overall Number of Baseline Participants 23 12 35
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 23 participants 12 participants 35 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
22
  95.7%
12
 100.0%
34
  97.1%
>=65 years
1
   4.3%
0
   0.0%
1
   2.9%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 23 participants 12 participants 35 participants
39.53  (11.00) 43.97  (8.02) 41.05  (10.18)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 23 participants 12 participants 35 participants
Female
15
  65.2%
6
  50.0%
21
  60.0%
Male
8
  34.8%
6
  50.0%
14
  40.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 23 participants 12 participants 35 participants
23 12 35
Forced Vital Capacity   [1] 
Mean (Standard Deviation)
Unit of measure:  % of predicted
Number Analyzed 23 participants 12 participants 35 participants
72.77  (8.12) 73.53  (10.09) 73.03  (8.70)
[1]
Measure Description: Forced Vital Capacity (FVC) is the volume of air that can be forcibly blown out from the lungs after full inspiration. It is recorded as the percentage of predicted volume (predicted FVC volume is calculated based on subject's height, age, sex, and weight). FVC normal range is 80-120% of predicted.
Total Lung Capacity   [1] 
Mean (Standard Deviation)
Unit of measure:  % of predicted volume
Number Analyzed 23 participants 12 participants 35 participants
73.84  (12.07) 73.28  (10.09) 73.65  (11.28)
[1]
Measure Description: Total Lung Capacity (TLC) is the volume in the lungs at maximal inflation. TLC is recorded as the percentage of predicted volume based on subject's height, age, sex, and weight. TLC normal range is 80-120% of predicted.
Adjusted Diffusing Capacity of the Lung for Carbon Monoxide   [1] 
Mean (Standard Deviation)
Unit of measure:  % of predicted volume
Number Analyzed 23 participants 12 participants 35 participants
67.82  (15.94) 66.83  (16.60) 67.48  (15.93)
[1]
Measure Description: Adjusted Diffusing Capacity of the lung for carbon monoxide (DLCOa) measures gas uptake during a single inspiration in a standard time, adjusted for subject's hemoglobin levels. DLCOa is recorded as a percentage of predicted volume, and normal range is 75-125% of predicted.
6 Minute Walk Test   [1] 
Mean (Standard Deviation)
Unit of measure:  Meters
Number Analyzed 23 participants 12 participants 35 participants
517.39  (112.79) 526.87  (103.00) 520.65  (107.94)
[1]
Measure Description: 6 minute walk test (6MWT) measures the distance that a patient can quickly walk on a flat hard surface in a period of six minutes.
1.Primary Outcome
Title Change in Forced Vital Capacity (36 Months)
Hide Description Change from baseline in the Forced Vital Capacity (FVC) measurement at 36 months. FVC is the volume of air that can be forcibly blown out from the lungs after full inspiration. FVC is recorded as the percentage of predicted volume (predicted FVC volume is calculated based on subject's height, age, sex, and weight).
Time Frame Measured at baseline and 36 months
Hide Outcome Measure Data
Hide Analysis Population Description
Participants from the Intent to Treat population for whom data was available at baseline and 36 months.
Arm/Group Title Pirfenidone Placebo
Hide Arm/Group Description:
Subjects received pirfenidone 801 mg (3 pills of 267 mg each), three times daily.
Subjects received placebo (3 pills), three times daily.
Overall Number of Participants Analyzed 11 3
Mean (Standard Deviation)
Unit of Measure: % of predicted volume
-23.52  (24.32) -20.93  (25.59)
2.Secondary Outcome
Title Change in Forced Vital Capacity (12 Months)
Hide Description Change from baseline in the Forced Vital Capacity (FVC) measurement at 12 months. FVC is the volume of air that can be forcibly blown out from the lungs after full inspiration. FVC is recorded as the percentage of predicted volume (predicted FVC volume is calculated based on subject's height, age, sex, and weight).
Time Frame Measured at baseline and 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
Participants from the Intent to Treat population for whom data was available at baseline and 12 months.
Arm/Group Title Pirfenidone Placebo
Hide Arm/Group Description:
Subjects received pirfenidone 801 mg (3 pills of 267 mg each), three times daily.
Subjects received placebo (3 pills), three times daily.
Overall Number of Participants Analyzed 18 12
Mean (Standard Deviation)
Unit of Measure: % of predicted volume
-7.27  (20.48) -3.55  (21.33)
3.Secondary Outcome
Title Change in Total Lung Capacity (36 Months)
Hide Description Change from baseline in Total Lung Capacity (TLC) measured at 36 months. TLC is the volume in the lungs at maximal inflation. TLC is recorded as the percentage of predicted volume based on subject's height, age, sex, and weight.
Time Frame Measured at baseline and 36 months
Hide Outcome Measure Data
Hide Analysis Population Description
Participants from the Intent to Treat population for whom data is available at baseline and 36 months.
Arm/Group Title Pirfenidone Placebo
Hide Arm/Group Description:
Subjects received pirfenidone 801 mg (3 pills of 267 mg each), three times daily.
Subjects received placebo (3 pills), three times daily.
Overall Number of Participants Analyzed 11 3
Mean (Standard Deviation)
Unit of Measure: % of predicted volume
-25.26  (24.42) -22.9  (23.69)
4.Secondary Outcome
Title Change in Total Lung Capacity (12 Months)
Hide Description Change from baseline in Total Lung Capacity (TLC) measured at 12 months. TLC is the volume in the lungs at maximal inflation. TLC is recorded as the percentage of predicted volume based on subject's height, age, sex, and weight.
Time Frame Measured at baseline and 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
Participants from the Intent to Treat population for whom data was available at baseline and 12 months.
Arm/Group Title Pirfenidone Placebo
Hide Arm/Group Description:
Subjects received pirfenidone 801 mg (3 pills of 267 mg each), three times daily.
Subjects received placebo (3 pills), three times daily.
Overall Number of Participants Analyzed 18 12
Mean (Standard Deviation)
Unit of Measure: % of predicted volume
-8.96  (21.03) 0.53  (21.27)
5.Secondary Outcome
Title Change in Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (36 Months)
Hide Description Change from baseline in adjusted Diffusing Capacity of the lung for carbon monoxide (DLCOa) measured at 36 months. DLCOa measures gas uptake during a single inspiration in a standard time, adjusted for subject's hemoglobin levels.
Time Frame Measured at baseline and 36 months
Hide Outcome Measure Data
Hide Analysis Population Description
Participants from the Intent to Treat population for whom data was available at baseline and 36 months.
Arm/Group Title Pirfenidone Placebo
Hide Arm/Group Description:
Subjects received pirfenidone 801 mg (3 pills of 267 mg each), three times daily.
Subjects received placebo (3 pills), three times daily.
Overall Number of Participants Analyzed 11 3
Mean (Standard Deviation)
Unit of Measure: % of predicted volume
-15.25  (29.89) -14.93  (29.74)
6.Secondary Outcome
Title Change in Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (12 Months)
Hide Description Change from baseline in adjusted Diffusing Capacity of the lung for carbon monoxide (DLCOa) measured at 12 months. DLCOa measures gas uptake during a single inspiration in a standard time, adjusted for subject's hemoglobin levels.
Time Frame Measured at baseline and 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
Participants from the Intent to Treat population for whom data is available at baseline and 12 months.
Arm/Group Title Pirfenidone Placebo
Hide Arm/Group Description:
Subjects received pirfenidone 801 mg (3 pills of 267 mg each), three times daily.
Subjects received placebo (3 pills), three times daily.
Overall Number of Participants Analyzed 18 12
Mean (Standard Deviation)
Unit of Measure: % of predicted volume
-3.11  (26.47) -4.74  (25.97)
7.Secondary Outcome
Title Change in 6 Minute Walk Test (36 Months)
Hide Description Change from baseline of the 6 minute walk test (6MWT) at 36 months. The 6MWT measures the distance that a patient can quickly walk on a flat hard surface in a period of six minutes.
Time Frame Measured at baseline and 36 months
Hide Outcome Measure Data
Hide Analysis Population Description
Participants from the Intent to Treat population for whom data is available at baseline and 36 months.
Arm/Group Title Pirfenidone Placebo
Hide Arm/Group Description:
Subjects received pirfenidone 801 mg (3 pills of 267 mg each), three times daily.
Subjects received placebo (3 pills), three times daily.
Overall Number of Participants Analyzed 10 2
Mean (Standard Deviation)
Unit of Measure: meters
-232.86  (284.26) -306.75  (314.69)
8.Secondary Outcome
Title Change in 6 Minute Walk Test (12 Months)
Hide Description Change from baseline of the 6 minute walk test (6MWT) at 12 months. The 6MWT measures the distance that a patient can quickly walk on a flat hard surface in a period of six minutes.
Time Frame Measured at baseline and 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
Participants from the Intent to Treat population for whom data is available at baseline and 12 months.
Arm/Group Title Pirfenidone Placebo
Hide Arm/Group Description:
Subjects received pirfenidone 801 mg (3 pills of 267 mg each), three times daily.
Subjects received placebo (3 pills), three times daily.
Overall Number of Participants Analyzed 16 12
Mean (Standard Deviation)
Unit of Measure: meters
-40.56  (192.66) -17.21  (201.21)
Time Frame Adverse event data were collected over the entire period of the double-blind portion of the study, 4 years.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Pirfenidone Placebo
Hide Arm/Group Description Subjects received pirfenidone 801 mg (3 pills of 267 mg each), three times daily. Subjects received placebo (3 pills), three times daily.
All-Cause Mortality
Pirfenidone Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Pirfenidone Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   6/23 (26.09%)      2/12 (16.67%)    
Cardiac disorders     
Chest pain * 1  2/23 (8.70%)  2 0/12 (0.00%)  0
Gastrointestinal disorders     
Haematochezia * 1  1/23 (4.35%)  1 0/12 (0.00%)  0
Investigations     
Blood creatinine phosphokinase increased * 1  1/23 (4.35%)  1 1/12 (8.33%)  1
Respiratory, thoracic and mediastinal disorders     
Respiratory failure * 1  2/23 (8.70%)  2 1/12 (8.33%)  1
Vascular disorders     
Deep vein thrombosis * 1  1/23 (4.35%)  1 0/12 (0.00%)  0
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA (11.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Pirfenidone Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   18/23 (78.26%)      9/12 (75.00%)    
Cardiac disorders     
Chest pain * 1  3/23 (13.04%)  3 1/12 (8.33%)  1
Palpitations * 1  3/23 (13.04%)  3 0/12 (0.00%)  0
Ear and labyrinth disorders     
Vertigo * 1  1/23 (4.35%)  1 0/12 (0.00%)  0
Gastrointestinal disorders     
Abdominal pain  1  0/23 (0.00%)  0 2/12 (16.67%)  2
Dyspepsia  1  12/23 (52.17%)  12 6/12 (50.00%)  6
Nausea  1  4/23 (17.39%)  4 1/12 (8.33%)  1
Vomiting  1  0/23 (0.00%)  0 2/12 (16.67%)  2
General disorders     
Fatigue * 1  2/23 (8.70%)  2 0/12 (0.00%)  0
Immune system disorders     
Drug eruption * 1  2/23 (8.70%)  2 0/12 (0.00%)  0
Urticaria * 1  0/23 (0.00%)  0 1/12 (8.33%)  1
Investigations     
Transaminases increased  1  1/23 (4.35%)  1 0/12 (0.00%)  0
Musculoskeletal and connective tissue disorders     
Arthalgia * 1  1/23 (4.35%)  1 0/12 (0.00%)  0
Myalgia * 1  0/23 (0.00%)  0 1/12 (8.33%)  1
Nervous system disorders     
Dizziness * 1  4/23 (17.39%)  4 0/12 (0.00%)  0
Somnolence * 1  1/23 (4.35%)  1 0/12 (0.00%)  0
Skin and subcutaneous tissue disorders     
Photosensitivity reaction  1  2/23 (8.70%)  2 0/12 (0.00%)  0
Rash  1  2/23 (8.70%)  2 2/12 (16.67%)  2
Indicates events were collected by systematic assessment
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA (11.0)
Study enrollment progressed more slowly than anticipated (spanning >3 years instead of one). The Data Safety and Monitoring Board performed an interim analysis one year after 30 subjects enrolled and directed the study to stop due to futility.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Dr. William A. Gahl
Organization: National Human Genome Research Institute
Phone: 301-402-2739
Responsible Party: William Gahl, M.D., National Human Genome Research Institute (NHGRI)
ClinicalTrials.gov Identifier: NCT00001596     History of Changes
Other Study ID Numbers: 970085
97-HG-0085
First Submitted: November 3, 1999
First Posted: November 4, 1999
Results First Submitted: December 12, 2011
Results First Posted: January 26, 2012
Last Update Posted: October 16, 2017