Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome

This study is ongoing, but not recruiting participants.

Study NCT00001596 Information provided by National Institutes of Health Clinical Center (CC)

First Received on November 3, 1999. Last Updated on January 24, 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

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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
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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

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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.

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

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1. Primary:

Change in Forced Vital Capacity (36 Months) [ Time Frame: Measured at baseline and 36 months ]

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2. Secondary:

Change in Forced Vital Capacity (12 Months) [ Time Frame: Measured at baseline and 12 months ]

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3. Secondary:

Change in Total Lung Capacity (36 Months) [ Time Frame: Measured at baseline and 36 months ]

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4. Secondary:

Change in Total Lung Capacity (12 Months) [ Time Frame: Measured at baseline and 12 months ]

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5. Secondary:

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

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6. Secondary:

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

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7. Secondary:

Change in 6 Minute Walk Test (36 Months) [ Time Frame: Measured at baseline and 36 months ]

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8. Secondary:

Change in 6 Minute Walk Test (12 Months) [ Time Frame: Measured at baseline and 12 months ]

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Serious Adverse Events

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Other Adverse Events

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More Information

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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:

Anikster Y, Huizing M, White J, Shevchenko YO, Fitzpatrick DL, Touchman JW, Compton JG, Bale SJ, Swank RT, Gahl WA, Toro JR. Mutation of a new gene causes a unique form of Hermansky-Pudlak syndrome in a genetic isolate of central Puerto Rico. Nat Genet. 2001 Aug;28(4):376-80.

Gahl WA, Brantly M, Kaiser-Kupfer MI, Iwata F, Hazelwood S, Shotelersuk V, Duffy LF, Kuehl EM, Troendle J, Bernardini I. Genetic defects and clinical characteristics of patients with a form of oculocutaneous albinism (Hermansky-Pudlak syndrome). N Engl J Med. 1998 Apr 30;338(18):1258-64.

Gahl WA, Brantly M, Troendle J, Avila NA, Padua A, Montalvo C, Cardona H, Calis KA, Gochuico B. Effect of pirfenidone on the pulmonary fibrosis of Hermansky-Pudlak syndrome. Mol Genet Metab. 2002 Jul;76(3):234-42.

Responsible Party:	William Gahl, NIH Clinical Center
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:	January 24, 2012
Health Authority:	United States: Federal Government; United States: Food and Drug Administration