Pilot Study of a Multi-Drug Regimen for Severe Pulmonary Fibrosis in Hermansky-Pudlak Syndrome
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|ClinicalTrials.gov Identifier: NCT00467831|
Recruitment Status : Terminated (insufficient enrollment)
First Posted : May 1, 2007
Results First Posted : August 2, 2013
Last Update Posted : August 2, 2013
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.
|Condition or disease||Intervention/treatment||Phase|
|Hermansky-Pudlak Syndrome (HPS) Pulmonary Fibrosis Oculocutaneous Albinism Platelet Storage Pool Deficiency Metabolic Disease||Drug: Losartan Drug: Zileuton Drug: N-Acetylcysteine Drug: Pravastatin Drug: Erythromycin||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||3 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Pilot Study of a Multi-Drug Regimen for Severe Pulmonary Fibrosis in Hermansky-Pudlak Syndrome|
|Study Start Date :||April 2007|
|Actual Primary Completion Date :||November 2012|
|Actual Study Completion Date :||November 2012|
Experimental: Multi-Drug Regimen
Losartan, 25 mg by mouth every night at bedtime; Zileuton, 1200 mg by mouth twice daily; N-acetylcysteine, 600 mg by mouth three times daily; Pravastatin, 20 mg by mouth every night at bedtime; Erythromycin, 333 mg by mouth three times daily.
Losartan potassium tablet, 25 mg by mouth every night at bedtime.
Other Name: CozaarDrug: Zileuton
Zileuton tablet, 1200 mg by mouth twice daily.
Other Name: ZyfloDrug: N-Acetylcysteine
N-acetylcysteine solution, 600 mg by mouth three times daily.
Other Name: acetylcysteineDrug: Pravastatin
Pravastatin sodium tablet, 20 mg by mouth every night at bedtime.
Other Name: PravacholDrug: Erythromycin
Erythromycin tablet, 333 mg by mouth three times daily.
- Survival at 2 Years [ Time Frame: 24 months ]The number of subjects surviving after 24 months on study.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00467831
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike|
|Bethesda, Maryland, United States, 20892|
|Principal Investigator:||Thomas Markello, M.D.||National Human Genome Research Institute (NHGRI)|