Pulmonary Fibrosis Clinical Trial
Official title:
Therapeutic Clinical Trial of Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome
Verified date | September 2017 |
Source | National Institutes of Health Clinical Center (CC) |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 35 |
Est. completion date | May 9, 2016 |
Est. primary completion date | September 2009 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility |
- INCLUSION CRITERIA For the portion of the protocol involving continuations of pirfenidone treatment, the criteria are simply previous enrollment in 97-HG-0085. For enrollment in the new clinical trial, the inclusion criteria involve enrollment in protocol 95-HG-0193, "Clinical and Basic Investigations into Hermansky-Pudlak Syndrome". This itself requires a diagnosis of HPS based upon molecular grounds or the electron microscopic demonstration of deficiency of platelet dense bodies. In addition, for protocol 97-HG-0085, patients must: - Be over 18 years of age. - Have an FVC greater than 50 percent and less than or equal to 85 percent of predicted OR a hemoglobin-corrected DL(co) greater than 35 percent and less than or equal to 80 percent of predicted, with no evidence of a pulmonary embolism. - Have evidence of reduced exercise tolerance lasting longer than one week on either the St. George's Hospital Respiratory Questionnaire or the Dyspnea Perception Scale. - FEV(1)/FVC greater than 80 percent of predicted after bronchodilators. - No evidence of improvement in pulmonary fibrosis within the past year defined as an FVC increased by 10 percent or a DL(co) increased by 15 percent. - Distance walked greater than or equal to 150 meters (492 feet) with oxygen saturation greater than or equal to 83 percent on less than or equal to 6 L/min. of oxygen during the 6-Minute Walk Test (6MWT). - Be available, willing, and able to come to the NIH Clinical Center for admission every 4 months for three years. EXCLUSION CRITERIA - History of clinically significant environmental exposure known to cause pulmonary fibrosis (including but not limited to drugs, asbestos, beryllium, radiation, domestic birds). - An explanation for interstitial lung disease other than HPS, including but not limited to radiation, sarcoidosis, hypersensitivity pneumonitis, bronchiolitis obliterans organizing pneumonia, cancer. - Diagnosis of any connective tissue disease including but not limited to scleroderma, systemic lupus erythematosus, rheumatoid arthritis. - Listing on a lung transplantation waiting list. - Pregnancy or lactation - Cigarette smoking in the past 6 months - History of ethanol abuse or recreational drug use in the past two years - History of human immunodeficiency virus (HIV) or chronic viral hepatitis infection - Chronic use of high-dose steroids (greater than 10 mg prednisone/day) - Prior use of pirfenidone - Use of any of the following within 28 days of enrollment: investigational therapy, cytotoxic/immunosuppressive agents other than corticosteroids (including but not limited to azathioprine, cyclosphosphamide, methotrexate, cyclosporine); cytokine modulators (including but not limited to etanercept and infliximab); therapies targeted to treat pulmonary fibrosis (including but not limited to D-penicillamine, colchicine, interferon gamma-1b, bosentan, N-acetylcysteine - Any severe medical complication including but not be limited to uncontrolled seizures, repeated transient ischemic attacks, abnormal mental status, severe ataxia, uncontrolled migraine headaches, diplopia, repeated episodes of syncope, untreated clinical depression, recent myocardial infarction (past 6 months), unstable angina, clinically relevant arrhythmias, uncontrolled hypotension or hypertension (systolic blood pressure less than 80 or greater than 180 mm Hg), myocarditis, hepatomegaly (liver greater than 3 cm below the right costal margin), renal glomerular impairment (creatinine clearance less than 35 ml/min/1.73 m2, pancreatitis, toxic thyroiditis, malignancy (except basal cell carcinoma) - Medications with a high frequency of life threatening side effects - Significant laboratory abnormalities, including but not limited to serum potassium less than 3.0 or greater than 5.4 mEq/L, SGPT greater than 100 U/L, CK greater than 700 U/L, hemoglobin less than 9.0 g/dL, platelets less than 70 k/mm3, leucocyte count less than 2.0 k/microliter, or cholesterol greater than 400 mg/dL. - For women of child bearing age, failure to have an effective method of birth control. |
Country | Name | City | State |
---|---|---|---|
United States | National Institutes of Health Clinical Center, 9000 Rockville Pike | Bethesda | Maryland |
Lead Sponsor | Collaborator |
---|---|
William Gahl, M.D. | National Human Genome Research Institute (NHGRI) |
United States,
Anikster Y, Huizing M, White J, Shevchenko YO, Fitzpatrick DL, Touchman JW, Compton JG, Bale SJ, Swank RT, Gahl WA, Toro JR. Mutation of a new gene causes a unique form of Hermansky-Pudlak syndrome in a genetic isolate of central Puerto Rico. Nat Genet. 2001 Aug;28(4):376-80. — View Citation
Gahl WA, Brantly M, Kaiser-Kupfer MI, Iwata F, Hazelwood S, Shotelersuk V, Duffy LF, Kuehl EM, Troendle J, Bernardini I. Genetic defects and clinical characteristics of patients with a form of oculocutaneous albinism (Hermansky-Pudlak syndrome). N Engl J Med. 1998 Apr 30;338(18):1258-64. — View Citation
Gahl WA, Brantly M, Troendle J, Avila NA, Padua A, Montalvo C, Cardona H, Calis KA, Gochuico B. Effect of pirfenidone on the pulmonary fibrosis of Hermansky-Pudlak syndrome. Mol Genet Metab. 2002 Jul;76(3):234-42. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Change in Forced Vital Capacity (36 Months) | Change from baseline in the Forced Vital Capacity (FVC) measurement at 36 months. FVC is the volume of air that can be forcibly blown out from the lungs after full inspiration. FVC is recorded as the percentage of predicted volume (predicted FVC volume is calculated based on subject's height, age, sex, and weight). | Measured at baseline and 36 months | |
Secondary | Change in Forced Vital Capacity (12 Months) | Change from baseline in the Forced Vital Capacity (FVC) measurement at 12 months. FVC is the volume of air that can be forcibly blown out from the lungs after full inspiration. FVC is recorded as the percentage of predicted volume (predicted FVC volume is calculated based on subject's height, age, sex, and weight). | Measured at baseline and 12 months | |
Secondary | Change in Total Lung Capacity (36 Months) | Change from baseline in Total Lung Capacity (TLC) measured at 36 months. TLC is the volume in the lungs at maximal inflation. TLC is recorded as the percentage of predicted volume based on subject's height, age, sex, and weight. | Measured at baseline and 36 months | |
Secondary | Change in Total Lung Capacity (12 Months) | Change from baseline in Total Lung Capacity (TLC) measured at 12 months. TLC is the volume in the lungs at maximal inflation. TLC is recorded as the percentage of predicted volume based on subject's height, age, sex, and weight. | Measured at baseline and 12 months | |
Secondary | Change in Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (36 Months) | Change from baseline in adjusted Diffusing Capacity of the lung for carbon monoxide (DLCOa) measured at 36 months. DLCOa measures gas uptake during a single inspiration in a standard time, adjusted for subject's hemoglobin levels. | Measured at baseline and 36 months | |
Secondary | Change in Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (12 Months) | Change from baseline in adjusted Diffusing Capacity of the lung for carbon monoxide (DLCOa) measured at 12 months. DLCOa measures gas uptake during a single inspiration in a standard time, adjusted for subject's hemoglobin levels. | Measured at baseline and 12 months | |
Secondary | Change in 6 Minute Walk Test (36 Months) | Change from baseline of the 6 minute walk test (6MWT) at 36 months. The 6MWT measures the distance that a patient can quickly walk on a flat hard surface in a period of six minutes. | Measured at baseline and 36 months | |
Secondary | Change in 6 Minute Walk Test (12 Months) | Change from baseline of the 6 minute walk test (6MWT) at 12 months. The 6MWT measures the distance that a patient can quickly walk on a flat hard surface in a period of six minutes. | Measured at baseline and 12 months |
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT04638517 -
The TELO-SCOPE Study: Attenuating Telomere Attrition With Danazol. Is There Scope to Dramatically Improve Health Outcomes for Adults and Children With Pulmonary Fibrosis
|
Phase 2 | |
Recruiting |
NCT05299333 -
Comparison of Pulmonary Telerehabilitation and Physical Activity Recommendations in Patients With Post Covid Fibrosis
|
N/A | |
Terminated |
NCT04119115 -
Integrated Radiographic and Metabolomics Risk Assessment in Patients With Interstitial Lung Diseases
|
||
Completed |
NCT01615484 -
Ex-vivo Perfusion and Ventilation of Lungs Recovered From Non-Heart-Beating Donors to Assess Transplant Suitability
|
N/A | |
Completed |
NCT01417156 -
Safety and PK Study of BIBF 1120 in Japanese Patients With IPF: Follow up Study From 1199.31(NCT01136174)
|
Phase 2 | |
Terminated |
NCT00981747 -
Targeting Vascular Reactivity in Idiopathic Pulmonary Fibrosis
|
Phase 2/Phase 3 | |
Completed |
NCT00052052 -
An Open-Label Study of the Safety and Efficacy of Subcutaneous Recombinant Interferon-Gamma 1b (IFN-Gamma 1b) in Patients With Idiopathic Pulmonary Fibrosis (IPF)
|
Phase 2 | |
Completed |
NCT01442779 -
Clinical Trial of Low Dose Oral Interferon Alpha in Idiopathic Pulmonary Fibrosis
|
Phase 2 | |
Completed |
NCT00366509 -
Role of Helicobacter Pylori and Its Toxins in Lung and Digestive System Diseases
|
||
Recruiting |
NCT00001532 -
Role of Genetic Factors in the Development of Lung Disease
|
||
Recruiting |
NCT04767074 -
A Non-pharmacological Cough Control Therapy
|
N/A | |
Recruiting |
NCT04864990 -
Dyspnea and Idiopathic Pulmonary Fibrosis
|
||
Completed |
NCT01961362 -
Supplemental Oxygen in Pulmonary Fibrosis
|
||
Completed |
NCT01271842 -
Long-term Outcome and Lung Capacity in Survivors of ARDS Due to Influenza A (H1N1) v2009 The RESPIFLU Study
|
N/A | |
Completed |
NCT00650091 -
Evaluating the Effectiveness of Prednisone, Azathioprine, and N-acetylcysteine in Patients With IPF
|
Phase 3 | |
Active, not recruiting |
NCT00258544 -
Microarray Analysis of Gene Expression in Idiopathic Pulmonary Fibrosis (IPF)
|
||
Enrolling by invitation |
NCT04930289 -
Global Utilization And Registry Database for Improved preservAtion of doNor LUNGs
|
||
Completed |
NCT02055222 -
Clinical Outcomes and Molecular Phenotypes in Smokers With Parenchymal Lung Disease
|
||
Enrolling by invitation |
NCT06327360 -
Illness Expectations in Pulmonary Fibrosis
|
||
Completed |
NCT06457269 -
Evaluating the Potential of Large Language Models for Respiratory Disease Consultations
|
N/A |