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Sponsored by: |
National Human Genome Research Institute (NHGRI) |
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Information provided by: | National Institutes of Health Clinical Center (CC) |
ClinicalTrials.gov Identifier: | NCT00467831 |
This study will examine whether five drugs (pravastatin, Losartan, Zileuton, N-acetylcysteine and erythromycin) used together can slow the course of pulmonary fibrosis (scarring of the lung tissue) in patients with Hermansky-Pudlak Syndrome (HPS). Patients with this disease have decreased skin color (albinism), bleeding problems, and sometimes colon problems. Two of the known types of Hermansky Pudlak syndrome, type 1 and type 4, are at high risk of pulmonary fibrosis between the ages of 30 and 50.
Patients 18 to 70 years of age who have Hermansky-Pudlak Syndrome with a serious loss of lung function due to pulmonary fibrosis may be eligible for this study.
Participants begin taking pravastatin on study day 2 and start a new drug every 3 days. Patients who experience no problems with the medicines return home and continue on the drugs for the next 2 years. They return to the NIH Clinical Center every 3 months for a medical history, physical examination, and blood, urine and lung function tests. CT and bone density scans are done every year. The study may continue for up to 3 years.
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Condition | Intervention | Phase |
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Hermansky-Pudlak Syndrome (HPS) Pulmonary Fibrosis Oculocutaneous Albinism Platelet Storage Pool Deficiency Metabolic Disease |
Drug: Losartan Drug: Zileuton Drug: N-Acetylcysteine Drug: Prevastatin Drug: Erythromycin |
Phase I Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Active Control, Single Group Assignment, Safety/Efficacy Study |
Official Title: | Pilot Study of a Multi-Drug Regimen for Severe Pulmonary Fibrosis in Hermansky-Pudlak Syndrome |
Estimated Enrollment: | 6 |
Study Start Date: | April 2007 |
Estimated Study Completion Date: | March 2010 |
Estimated Primary Completion Date: | March 2010 (Final data collection date for primary outcome measure) |
Hermansky-Pudlak Syndrome (HPS) is a rare autosomal recessive disease consisting of oculocutaneous albinism and a platelet storage pool defect. The most serious complication of this disorder, pulmonary fibrosis, occurs only in genetic subtypes HPS-1 and HPS-4 and is generally fatal in the fourth or fifth decade. HPS-1 is very common in northwest Puerto Rico. There is no effective treatment for the pulmonary disease of HPS (HPS-PF), which resembles idiopathic pulmonary fibrosis (IPF). A preliminary study of the antifibrotic drug, pirfenidone, gave promising results for mild to moderate HPS-PF, but not for severe pulmonary fibrosis. A second study is currently addressing only mild to moderate HPS-PF. Other drugs, studied in IPF as single agents, have some efficacy for mild to moderate disease, but none has had a major effect on mortality. Recently, a call has been made for consideration of multi-drug therapy (i.e., an oncologic approach) for severe pulmonary fibrosis. Based upon positive responses from companies producing relevant drugs, we propose a multi-drug trial using five agents: Losartan, Zileuton, a generic statin (Pravastatin), generic N-acetylcysteine, and generic Erythromycin.
Participants with severe pulmonary fibrosis will be drawn largely from the Puerto Rican population. Eligibility will require a molecular diagnosis of HPS-1 or HPS-4, radiographic evidence of interstitial lung disease, persistent pulmonary function testing less than or equal to 45% of predicted after bronchodilation, and absence of other causes of lung dysfunction. Participants will be admitted to the NIH Clinical Center for a 21-day admission to establish baseline function and to begin medication therapy. Follow-up admissions (3 days) will occur every 3 months. The primary outcome parameter will be survival at 2 years. The main secondary outcome parameter will be rate of decline of forced vital capacity (FVC), but serum markers of interstitial lung disease, change in findings on CT scan of the chest, 6-min walk test, and results of arterial blood gases will also be followed.
Ages Eligible for Study: | 18 Years to 70 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
To be eligible for this protocol, participants must:
Have evidence of severe pulmonary fibrosis, i.e.:
EXCLUSION CRITERIA:
Contact: Patient Recruitment and Public Liaison Office | (800) 411-1222 | prpl@mail.cc.nih.gov |
Contact: TTY | 1-866-411-1010 |
United States, Maryland | |
National Institutes of Health Clinical Center, 9000 Rockville Pike | Recruiting |
Bethesda, Maryland, United States, 20892 |
Responsible Party: | National Institutes of Health ( Thomas C. Markello, M.D./National Human Genome Research Institute ) |
Study ID Numbers: | 070132, 07-HG-0132 |
Study First Received: | April 28, 2007 |
Last Updated: | August 24, 2009 |
ClinicalTrials.gov Identifier: | NCT00467831 History of Changes |
Health Authority: | United States: Federal Government |
Restrictive Lung Disease Pulmonary Fibrosis Albinism Platelet Storage Pool Deficiency Metabolic Disease |
Hermansky-Pudlak Syndrome HPS Pulmonary Fibrosis Lung Disease |
Erythromycin stearate Anti-Infective Agents Antioxidants Erythromycin Ethylsuccinate Fibrosis Hemostatic Disorders Pulmonary Fibrosis Albinism Anti-Bacterial Agents Metabolism, Inborn Errors Hypopigmentation Hemorrhagic Disorders Respiratory Tract Diseases Acetylcysteine Eye Diseases, Hereditary |
Hypomelanotic Disorder Skin Diseases, Genetic Metabolic Disorder Losartan Lung Diseases, Interstitial Metabolic Diseases Skin Diseases Amino Acid Metabolism, Inborn Errors Hematologic Diseases Eye Diseases Pigmentation Disorders Blood Coagulation Disorders Blood Platelet Disorders Storage Pool Platelet Disease Erythromycin |
Anti-Infective Agents Respiratory System Agents Antioxidants Molecular Mechanisms of Pharmacological Action Fibrosis Physiological Effects of Drugs Pulmonary Fibrosis Albinism Anti-Bacterial Agents Metabolism, Inborn Errors Hypopigmentation Pathologic Processes Hemorrhagic Disorders Respiratory Tract Diseases Therapeutic Uses |
Syndrome Free Radical Scavengers Acetylcysteine Eye Diseases, Hereditary Skin Diseases, Genetic Antidotes Lung Diseases, Interstitial Disease Metabolic Diseases Skin Diseases Amino Acid Metabolism, Inborn Errors Hematologic Diseases Eye Diseases Pigmentation Disorders Blood Coagulation Disorders |