Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT00001596
• Date of registration: 03/11/1999
• Prospective Registration: Yes
• Primary sponsor: William Gahl, M.D.
• Public title: Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome
• Scientific title: Therapeutic Clinical Trial of Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome
• Date of first enrolment: September 2005
• Target sample size: 35
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00001596
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Puerto Rico, United States

Key inclusion & exclusion criteria

- INCLUSION CRITERIA

For the portion of the protocol involving continuations of pirfenidone treatment, the
criteria are simply previous enrollment in 97-HG-0085.

For enrollment in the new clinical trial, the inclusion criteria involve enrollment in
protocol 95-HG-0193, "Clinical and Basic Investigations into Hermansky-Pudlak Syndrome".
This itself requires a diagnosis of HPS based upon molecular grounds or the electron
microscopic demonstration of deficiency of platelet dense bodies. In addition, for protocol
97-HG-0085, patients must:

- Be over 18 years of age.

- Have an FVC greater than 50 percent and less than or equal to 85 percent of predicted
OR a hemoglobin-corrected DL(co) greater than 35 percent and less than or equal to 80
percent of predicted, with no evidence of a pulmonary embolism.

- Have evidence of reduced exercise tolerance lasting longer than one week on either the
St. George's Hospital Respiratory Questionnaire or the Dyspnea Perception Scale.

- FEV(1)/FVC greater than 80 percent of predicted after bronchodilators.

- No evidence of improvement in pulmonary fibrosis within the past year defined as an
FVC increased by 10 percent or a DL(co) increased by 15 percent.

- Distance walked greater than or equal to 150 meters (492 feet) with oxygen saturation
greater than or equal to 83 percent on less than or equal to 6 L/min. of oxygen during
the 6-Minute Walk Test (6MWT).

- Be available, willing, and able to come to the NIH Clinical Center for admission every
4 months for three years.

EXCLUSION CRITERIA

- History of clinically significant environmental exposure known to cause pulmonary
fibrosis (including but not limited to drugs, asbestos, beryllium, radiation, domestic
birds).

- An explanation for interstitial lung disease other than HPS, including but not limited
to radiation, sarcoidosis, hypersensitivity pneumonitis, bronchiolitis obliterans
organizing pneumonia, cancer.

- Diagnosis of any connective tissue disease including but not limited to scleroderma,
systemic lupus erythematosus, rheumatoid arthritis.

- Listing on a lung transplantation waiting list.

- Pregnancy or lactation

- Cigarette smoking in the past 6 months

- History of ethanol abuse or recreational drug use in the past two years

- History of human immunodeficiency virus (HIV) or chronic viral hepatitis infection

- Chronic use of high-dose steroids (greater than 10 mg prednisone/day)

- Prior use of pirfenidone

- Use of any of the following within 28 days of enrollment: investigational therapy,
cytotoxic/immunosuppressive agents other than corticosteroids (including but not
limited to azathioprine, cyclosphosphamide, methotrexate, cyclosporine); cytokine
modulators (including but not limited to etanercept and infliximab); therapies
targeted to treat pulmonary fibrosis (including but not limited to D-penicillamine,
colchicine, interferon gamma-1b, bosentan, N-acetylcysteine

- Any severe medical complication including but not be limited to uncontrolled seizures,
repeated transient ischemic attacks, abnormal mental status, severe ataxia,
uncontrolled migraine headaches, diplopia, repeated episodes of syncope, untreated
clinical depression, recent myocardial infarction (past 6 months), unstable angina,
clinically relevant arrhythmias, uncontrolled hypotension or hypertension (systolic
blood pressure less than 80 or greater than 180 mm Hg), myocarditis, hepatomegaly
(liver greater than 3 cm below the right costal margin), renal glomerular impairment
(creatinine clearance less than 35 ml/min/1.73 m2, pancreatitis, toxic thyroiditis,
malignancy (except basal cell carcinoma)

- Medications with a high frequency of life threatening side effects

- Significant laboratory abnormalities, including but not limited to serum potassium
less than 3.0 or greater than 5.4 mEq/L, SGPT greater than 100 U/L, CK greater than
700 U/L, hemoglobin less than 9.0 g/dL, platelets less than 70 k/mm3, leucocyte count
less than 2.0 k/microliter, or cholesterol greater than 400 mg/dL.

- For women of child bearing age, failure to have an effective method of birth control.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Inborn Errors of Metabolism

Oculocutaneous Albinism

Platelet Storage Pool Deficiency

Albinism

Pulmonary Fibrosis

Intervention(s)

Drug: Placebo

Drug: Pirfenidone

Primary Outcome(s)

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

Secondary Outcome(s)

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

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

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

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

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

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

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

Secondary ID(s)

970085

97-HG-0085

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

National Human Genome Research Institute (NHGRI)

Results

• Results available: Yes
• Date Posted: 26/01/2012
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00001596