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Home > "O" Clinical Trials Conditions > Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome  Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome
Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome
For Condition: Albinism,Inborn Errors of Metabolism,Oculocutaneous Albinism,Platelet Storage Pool Deficiency,Pulmonary Fibrosis
Status: Recruiting
Sponsor(s): National Human Genome Research Institute (NHGRI) ,
Synopsis: Hermansky-Pudlak Syndrome (HPS) is an inherited disease which results in decreased pigmentation (oculocutaneous albinism), bleeding problems due to a platelet abnormality (platelet storage pool defect), and storage of an abnormal fat-protein compound (lysosomal accumulation of ceroid lipofuscin). The disease can cause poor functioning of the lungs, intestine, kidneys, or heart. The most serious complication of the disease is pulmonary fibrosis and typically causes death in patients ages 40 - 50 years old. The disorder is common in Puerto Rico, where many of the clinical research studies on the disease have been conducted. Neither the full extent of the disease nor the basic cause of the disease is known. There is no known treatment for HPS. The drug Pirfenidone blocks the biochemical process of inflammation and has been reported to slow or reverse pulmonary fibrosis in animal systems. In this study researchers will select 40 patients diagnosed with pulmonary fibrosis 20 who have not received steroid therapy in the last 3 months and 20 currently taking steroids. The patients will be randomly divided into 4 groups. The patients will not know if they are taking pirfenidone or a placebo "sugar pill". 1. Group one will be patients not taking steroids who will receive pirfenidone. 2. Group two will be patients not taking steroids who will receive a placebo "sugar pill" 3. Group three will be patients taking steroids who will receive pirfenidone. 4. Group four will be patients taking steroids who will receive a placebo "sugar pill". The major outcome measurement of the therapy will be a change in the lung function (forced vital capacity). The study will be stopped if one therapy proves to be more effective than the others.
Details: Hermansky-Pudlak Syndrome (HPS) is a rare autosomal recessive disease consisting of the triad of oculocutaneous albinism, a platelet storage pool defect, and lysosomal accumulation of ceroid lipofuscin. The most serious complication of this disorder, which is common in Puerto Rico, is pulmonary fibrosis, generally fatal in the fourth or fifth decade. There is no treatment for the pulmonary disease of HPS, which resembles idiopathic pulmonary fibrosis. However, a drug called pirfenidone has antifibrotic effects in animal models of lung fibrosis. Pirfenidone is an IND drug provided by Marnac, Inc. and licensed to InterMune, Inc.. Pirfenidone inhibits cytokine-induced inflammation. Reported side effects are limited to drowsiness, different types of gastrointestinal upset, a photosensitivity rash, and palpitations. Between 1997 and 2001, we performed a randomized, placebo-controlled trial under this protocol that found pirfenidone to be safe and efficacious when the data were analyzed using a repeated measures model. Using a random coefficients model, however, the data were definitive only in the restricted group of subjects whose initial forced vital capacity was greater than 50% of predicted. The repeated measures analysis was chosen a priori as the optimal model and, based upon this analysis, the DSMB stopped the study and directed that all patients receive perfenidone. Of the 23 original patients, 4 are still receiving pirfenidone. We know wish to continue the study in two ways. First as promised in the original protocol, we will provide pirfenidone treatment to all interested subjects enrolled in the original protocol, regardless of whether they were receiving pirfenidone or placebo. These patients will be admitted to the NIH Clinical Center for 2-3 days every 6 months. Second, we will determine to a statistician's satisfaction whether or not pirfenidone is efficacious in slowing lung fibrosis in HPS patients. Specifically we will initiate a stratified, block randomized, placebo-controlled, double-blind trial involving up to 50 HPS patients whose forced vital capacity is 51-85% of predicted. Since patients with this amount of residual lung function are selected a priori, they no longer constitute a restricted group, and any demonstrated efficacy will be accepted. For every patient randomly assigned to the placebo group, two will receive pirfenidone. Patients will largely be drawn from the Puerto Rican population and will be previously or simultaneously enrolled in clinical protocol 95-HG-193. They will be admitted to the NIH Clinical Center for 2-3 day admissions every 4 months. The primary efficacy variable will be rate of change in forced vital capacity, determined on every admission and analyzed by the random coefficients method. Secondary efficacy variables will also be examined. A CT scan of the chest, bone density, and arterial blood gases will be performed yearly.
Eligibility:
Study Type: Interventional, Treatment, Safety/Efficacy
Minimum Age/Maximum Age: /
Genders: Both
Protocol Entry Criteria: INCLUSION CRITERIA: For the portion of the protocol involving continuation 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-193, (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 16-70 years of age and have an FVC greater than 50% and less than or equal to 85% of predicted. Finally, they must be available and willing to come to the NIH Clinical Center for admission every 4 months for three years. EXCLUSION CRITERIA: Exclusion criteria include pregnancy, cigarette smoking, the chronic use of high-dose steroids or other drugs for treating pulmonary disease, and any severe medical complication. This would include, but not 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 arrythmias, 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 (creatine clearance less than 35 ml/min/1.73 m(2)), or other significant conditions such as pancreatitis, toxic thyroiditis, malignancy (Except basal cell carcinoma), and medications with a high frequency of life-threatening side effects. In addition, patients will be excluded if the serum potassium is less than 3.0 or greater than 5.4 mEq/L, the SGPT is greater than 100 U/L, the CK is greater than 700 U/L, the hemoglobin is less than 9.0 g/dL, the platelets are less than 70 k/mm(3), the leucocyte count is less than 2.0 k/microL, or the cholesterol is greater than 400 mg/dL.
Total Enrollment: 73
Location and Contact Information:
National Human Genome Research Institute (NHGRI) *Recruiting*
Bethesda, Maryland, 20892
United States
Recruiting Patient and Public Liaison Office 1-800-411-1222
Additional Information:
Study ID Numbers: 970085; 97-HG-0085
Study Start Date: March 7, 1997
Record last reviewed: February 13, 2004
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00001596
Other Oculocutaneous Albinism Studies:
1. Clinical and Basic Investigations into Hermansky-Pudlak Syndrome
2. Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome
Related Studies:
Other Oculocutaneous Albinism Clinical Trials
Other Maryland Clinical Trials
Other Bethesda Clinical Trials
Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome
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