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Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome

NCT00001596 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 35

Last updated 2017-10-16

Study results available
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Summary

Hermansky-Pudlak Syndrome (HPS) is an inherited disease that 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 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 up to 40 HPS patients diagnosed with pulmonary fibrosis. The patients will be randomly divided into 2 groups. The patients will not know if they are taking pirfenidone or a placebo "sugar pill".

1. Group one will be patients who will receive pirfenidone.
2. Group two will be patients 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 other.

Conditions

  • Albinism
  • Inborn Errors of Metabolism
  • Oculocutaneous Albinism
  • Platelet Storage Pool Deficiency
  • Pulmonary Fibrosis

Interventions

DRUG

Pirfenidone

Treatment with pirfenidone 801 mg (3 pills of 267 mg each), three times daily.

DRUG

Placebo

Placebo (3 pills), three times daily.

Sponsors & Collaborators

  • National Human Genome Research Institute (NHGRI)

    collaborator NIH

  • William Gahl, M.D.

    lead NIH

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2005-09-30
Primary Completion
2009-09-30
Completion
2016-05-09

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00001596 on ClinicalTrials.gov