Pilot Study of a Multi-Drug Regimen for Severe Pulmonary Fibrosis in Hermansky-Pudlak Syndrome

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.

Conditions

• Hermansky-Pudlak Syndrome (HPS)
• Pulmonary Fibrosis
• Oculocutaneous Albinism
• Platelet Storage Pool Deficiency
• Metabolic Disease

Interventions

DRUG

Losartan

Losartan potassium tablet, 25 mg by mouth every night at bedtime.

DRUG

Zileuton

Zileuton tablet, 1200 mg by mouth twice daily.

DRUG

N-Acetylcysteine

N-acetylcysteine solution, 600 mg by mouth three times daily.

DRUG

Pravastatin

Pravastatin sodium tablet, 20 mg by mouth every night at bedtime.

DRUG

Erythromycin

Erythromycin tablet, 333 mg by mouth three times daily.

Sponsors & Collaborators

• National Human Genome Research Institute (NHGRI)

  lead NIH

Principal Investigators

• Thomas Markello, M.D. · National Human Genome Research Institute (NHGRI)

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

18 Years

Max Age

70 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2007-04-30

Primary Completion

2012-11-30

Completion

2012-11-30

Countries

• United States

Study Locations