Text Size

Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
National Human Genome Research Institute (NHGRI)
Information provided by (Responsible Party):
William Gahl, National Human Genome Research Institute (NHGRI)
ClinicalTrials.gov Identifier:
NCT00001596
First received: November 3, 1999
Last updated: November 30, 2012
Last verified: November 2012
History of Changes
Results First Received: December 12, 2011  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, 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

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Patients were enrolled at the NIH Clinical Center between September 2005 to March 2009.

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
Pirfenidone Subjects received pirfenidone 801 mg (3 pills of 267 mg each), three times daily.
Placebo Subjects received placebo (3 pills), three times daily.

Participant Flow:   Overall Study
    Pirfenidone     Placebo  
STARTED     23     12  
COMPLETED     6     2  
NOT COMPLETED     17     10  
Death                 1                 0  
Withdrawal by Subject                 3                 1  
Study Termination                 12                 9  
Adverse Event                 1                 0  



  Baseline Characteristics
  Hide Baseline Characteristics

Reporting Groups
  Description
Pirfenidone Subjects received pirfenidone 801 mg (3 pills of 267 mg each), three times daily.
Placebo Subjects received placebo (3 pills), three times daily.
Total Total of all reporting groups

Baseline Measures
    Pirfenidone     Placebo     Total  
Number of Participants  
[units: participants]
 		  23     12     35  
Age  
[units: participants]
 		     
<=18 years     0     0     0  
Between 18 and 65 years     22     12     34  
>=65 years     1     0     1  
Age  
[units: years]
Mean ± Standard Deviation 		  39.53  ± 11.00     43.97  ± 8.02     41.05  ± 10.18  
Gender  
[units: participants]
 		     
Female     15     6     21  
Male     8     6     14  
Region of Enrollment  
[units: participants]
 		     
United States     23     12     35  
Forced Vital Capacity [1]
[units: % of predicted]
Mean ± Standard Deviation 		  72.77  ± 8.12     73.53  ± 10.09     73.03  ± 8.70  
Total Lung Capacity [2]
[units: % of predicted volume]
Mean ± Standard Deviation 		  73.84  ± 12.07     73.28  ± 10.09     73.65  ± 11.28  
Adjusted Diffusing Capacity of the Lung for Carbon Monoxide [3]
[units: % of predicted volume]
Mean ± Standard Deviation 		  67.82  ± 15.94     66.83  ± 16.60     67.48  ± 15.93  
6 Minute Walk Test [4]
[units: meters]
Mean ± Standard Deviation 		  517.39  ± 112.79     526.87  ± 103.00     520.65  ± 107.94  
[1] 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.
[2] 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.
[3] 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.
[4] 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.



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Change in Forced Vital Capacity (36 Months)   [ Time Frame: Measured at baseline and 36 months ]
  Show Outcome Measure 1

2.  Secondary:   Change in Forced Vital Capacity (12 Months)   [ Time Frame: Measured at baseline and 12 months ]
  Hide Outcome Measure 2

Measure Type Secondary
Measure Title Change in Forced Vital Capacity (12 Months)
Measure 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  
Safety Issue No  

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.
Participants from the Intent to Treat population for whom data was available at baseline and 12 months.

Reporting Groups
  Description
Pirfenidone Subjects received pirfenidone 801 mg (3 pills of 267 mg each), three times daily.
Placebo Subjects received placebo (3 pills), three times daily.

Measured Values
    Pirfenidone     Placebo  
Number of Participants Analyzed  
[units: participants]
 		  18     12  
Change in Forced Vital Capacity (12 Months)  
[units: % of predicted volume]
Mean ± Standard Deviation 		  -7.27  ± 20.48     -3.55  ± 21.33  

No statistical analysis provided for Change in Forced Vital Capacity (12 Months)



3.  Secondary:   Change in Total Lung Capacity (36 Months)   [ Time Frame: Measured at baseline and 36 months ]
  Show Outcome Measure 3

4.  Secondary:   Change in Total Lung Capacity (12 Months)   [ Time Frame: Measured at baseline and 12 months ]
  Show Outcome Measure 4

5.  Secondary:   Change in Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (36 Months)   [ Time Frame: Measured at baseline and 36 months ]
  Show Outcome Measure 5

6.  Secondary:   Change in Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (12 Months)   [ Time Frame: Measured at baseline and 12 months ]
  Show Outcome Measure 6

7.  Secondary:   Change in 6 Minute Walk Test (36 Months)   [ Time Frame: Measured at baseline and 36 months ]
  Show Outcome Measure 7

8.  Secondary:   Change in 6 Minute Walk Test (12 Months)   [ Time Frame: Measured at baseline and 12 months ]
  Show Outcome Measure 8


  Serious Adverse Events
  Show Serious Adverse Events


  Other Adverse Events
  Show Other Adverse Events


  More Information
  Hide More Information

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


Limitations and Caveats
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
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.  


Results Point of Contact:  
Name/Title: Dr. William A. Gahl
Organization: National Human Genome Research Institute
phone: 301-402-2739
e-mail: gahlw@mail.nih.gov


Publications:


Responsible Party: William Gahl, National Human Genome Research Institute (NHGRI)
ClinicalTrials.gov Identifier: NCT00001596     History of Changes
Other Study ID Numbers: 970085, 97-HG-0085
Study First Received: November 3, 1999
Results First Received: December 12, 2011
Last Updated: November 30, 2012
Health Authority: United States: Federal Government
United States: Food and Drug Administration