Pilot Study of a Multi-Drug Regimen for Severe Pulmonary Fibrosis in Hermansky-Pudlak Syndrome
Status:
Terminated
Trial end date:
2012-11-01
Target enrollment:
Participant gender:
Summary
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.