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Fibrosis clinical trials

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NCT ID: NCT01347190 Completed - Cystic Fibrosis Clinical Trials

Safety and Tolerability Study of Liquid Alpha1 Proteinase Inhibitor (API) in Subjects With Cystic Fibrosis

Start date: April 2011
Phase: Phase 1
Study type: Interventional

Study to assess safety and tolerability of a single dose of study-drug administered to Cystic Fibrosis (CF) patients.

NCT ID: NCT01343524 Completed - Cystic Fibrosis Clinical Trials

Changes in Lung Function Based on Differences in Spirometry Equipment Used in Children With Cystic Fibrosis

Start date: March 2010
Phase: N/A
Study type: Observational

The investigators' Upstate Cystic fibrosis (CF) Care Center is very active in research. For example, in 2009, 68 of our 135 pediatric patients have been participating in 16 clinical trials. Sponsors often provide their study sites with spirometers to use for PFTs. These machines are meant to provide uniform PFTs results for studies, which include measurement of FEV1 values. In 2009, among the 52 patients at our Center who are 6-12 years old, 20 (38%) have been in studies with sponsor-provided spirometers. Among the 32 patients ages 13-17 years, 13 (40%) have been in studies with sponsor-provided spirometers. Anecdotally, the investigators have noted that FEV1 results obtained during clinical trials for our patients appear to be lower than those of our patients' usual values, especially with younger children. The investigators hypothesize that the apparent difference in PFT results might be related to the different incentives (if any) provided by study PFT equipment. Further, if this difference exists, the investigators believe that this may account for the apparent average decline in the lung function results of our patients over the recent years, given the large proportion of our patients who participate in clinical trials. Finally, the investigators hypothesize that younger children may be more affected by the difference in incentives than older ones. The purpose of this study is to collect an additional set of Pulmonary Function Tests (PFTs) using our regular clinic equipment, on all study subjects following their study PFTs if these are done with sponsor-provided equipment. The investigators will compare the results from both types of machines and report regarding differences identified.

NCT ID: NCT01335477 Completed - Pulmonary Fibrosis Clinical Trials

Safety and Efficacy of BIBF 1120 at High Dose in Idiopathic Pulmonary Fibrosis Patients II

Start date: May 2011
Phase: Phase 3
Study type: Interventional

Idiopathic Pulmonary Fibrosis (IPF) is a chronic disease of unknown cause that results in scarring of the lung and there is a high unmet medical need for effective treatment to halt lung function decline, delay or avoid exacerbation (flare-ups), and ultimately to reduce the death rate. In a large Phase 2 trial (1199.30) (NCT00514683), investigating the effects of 52 weeks of treatment with BIBF 1120 in patients with IPF, a positive effect was seen on lung function of patients treated with high dose of BIBF 1120 compared to placebo. Hence it is the purpose of this trial to investigate and confirm the efficacy and safety of BIBF 1120 at a high dose in treating patients with IPF, compared with placebo. The trial will be conducted as a prospective, randomised design with the aim to collect safety and efficacy data. Respiratory function is globally accepted for assessment of treatment effects in IPF patients. The chosen endpoint (Forced Vital Capacity (FVC) decline) is easy to obtain and is part of the usual examinations done in IPF patients.

NCT ID: NCT01335464 Completed - Pulmonary Fibrosis Clinical Trials

Safety and Efficacy of BIBF 1120 at High Dose in Idiopathic Pulmonary Fibrosis Patients

Start date: April 2011
Phase: Phase 3
Study type: Interventional

Idiopathic Pulmonary Fibrosis (IPF) is a chronic disease of unknown cause that results in scarring of the lung and there is a high unmet medical need for effective treatment to halt lung function decline, delay or avoid exacerbation (flare-ups), and ultimately to reduce the death rate. In a large Phase 2 trial (1199.30) (NCT00514683), investigating the effects of 52 weeks of treatment with BIBF 1120 in patients with IPF, a positive effect was seen on lung function of patients treated with high dose of BIBF 1120 compared to placebo. Hence it is the purpose of this trial to investigate and confirm the efficacy and safety of BIBF 1120 at a high dose in treating patients with IPF, compared with placebo. The trial will be conducted as a prospective, randomised design with the aim to collect safety and efficacy data. Respiratory function is globally accepted for assessment of treatment effects in IPF patients. The chosen endpoint (Forced Vital Capacity (FVC) decline) is easy to obtain and is part of the usual examinations done in IPF patients.

NCT ID: NCT01331980 Completed - Cystic Fibrosis Clinical Trials

Bone Microarchitecture at the Radius: a Pilot Comparison Between Children With Cystic Fibrosis and Healthy Controls

Start date: January 2011
Phase: N/A
Study type: Observational

Cystic fibrosis (CF) affects an estimated 30,000 people in the United States and is caused by a mutation in the gene encoding a protein called CF transmembrane regulator (CFTR). The hallmarks of CF are recurrent pulmonary exacerbations and declining pulmonary function. However, there are other problems in CF that affect both health and quality of life. These include CF related diabetes, liver disease, and bone disease. The median age of survival for patients with CF has been increasing steadily and is currently more than 37 years. With this improvement in life expectancy, it has become increasingly important to address the long-term complications of CF. Currently, patients with CF are evaluated annually for bone disease with dual X-ray absorptiometry (DXA), and screening usually starts at age 12. However, this may not be sufficient to detect early bone changes that may impact fracture risk. Furthermore, bone disease in children may manifest earlier than adolescence, which would suggest that screening should start at an earlier age in these vulnerable patients. The following study is therefore proposed to examine the potential role of peripheral quantitative computed tomography (pQCT) as a screening approach for bone disease in children with CF. The investigators expect to find bone problems by pQCT but not DXA.

NCT ID: NCT01327703 Completed - Cystic Fibrosis Clinical Trials

Control of Steatorrhea in Participants With Cystic Fibrosis and Exocrine Pancreatic Insufficiency

Start date: April 2011
Phase: Phase 4
Study type: Interventional

This study by Aptalis (formerly Axcan) assesses the efficacy and safety of Panzytrat® 25,000 compared to Kreon® 25,000 in the control of steatorrhea in participants with cystic fibrosis (CF) and exocrine pancreatic insufficiency (EPI).

NCT ID: NCT01323101 Completed - Cystic Fibrosis Clinical Trials

Doxycycline Effects on Inflammation in Cystic Fibrosis

Start date: April 2008
Phase: Phase 4
Study type: Interventional

Doxycycline is known to exhibit immune modulatory activities beyond its antibacterial effects. In particular, doxycycline is a potent inhibitor of matrix metalloproteinase 9, which is a protease derived largely from neutrophils. Recent studies demonstrate a significant correlation between pulmonary disease severity and sputum concentrations of MMP-9 in patients with CF. In addition, sputum MMP-9 levels are associated with airway remodeling in CF. The goal of this study is to determine the therapeutic potential of doxycycline in modulating host airway inflammation in patients with CF. Specifically, the study will characterize the PK /PD of doxycycline, evaluate the safety of short term therapy, and explore the concentration effect relationship between doxycycline exposure and sputum biomarker levels.

NCT ID: NCT01316276 Completed - Cystic Fibrosis Clinical Trials

Extension Study of Liposomal Amikacin for Inhalation in Cystic Fibrosis (CF) Patients With Chronic Pseudomonas Aeruginosa (Pa) Infection

Start date: October 5, 2012
Phase: Phase 3
Study type: Interventional

The purpose of this study is to evaluate the long term safety and tolerability of Liposomal Amikacin for Inhalation (LAI) 590 mg once daily (QD) in Cystic Fibrosis patients with chronic infection due to pseudomonas aeruginosa. This long-term, open-label, multi-cycle extension study enrolled subjects who had successfully completed study TR02-108, were compliant with the study protocol, and did not meet any of the listed study discontinuation criteria. The safety and tolerability of LAI were evaluated for up to approximately 2 years.

NCT ID: NCT01315665 Completed - Cystic Fibrosis Clinical Trials

Effect of Sulforaphane in Broccoli Sprouts on Nrf2 Activation

Start date: April 2011
Phase: N/A
Study type: Interventional

The purpose of this study to investigate the effect of sulforaphane from macerated broccoli sprouts in humans and to evaluate less invasive methods of assessing potential anti-inflammatory drugs in CF.

NCT ID: NCT01306279 Completed - Cystic Fibrosis Clinical Trials

The Infective Pulmonary Exacerbations in Cystic Fibrosis - an Ecological Perspective

Start date: February 2011
Phase: N/A
Study type: Observational

Given the treatment burden and excess morbidity and mortality associated with acute infective exacerbations in cystic fibrosis, a clear understanding of the mechanisms involved in the origins of an infective exacerbation and the response to antibiotics is vital to improving long-term outcomes in CF. This study will examine 3 areas of interest in CF exacerbations. 1. Bacterial biodiversity and its clinical significance 2. The role of bacteria which are able to rapidly mutate (hypermutators) 3. Inter-bacterial communication and its role in infective exacerbations Study Hypothesis 1 Increased microbiological diversity represents a balanced community of bacteria. The presence of a diverse population of bacteria in CF infections therefore predicts a better outcome for treatment than when a population consists of a small number of more virulent organisms. Study Hypothesis 2 Pseudomonas aeruginosa hypermutators can mutate much more often than ordinary Pseudomonas aeruginosa bacteria. Hypermutators are likely to grow better when the bacteria are under stress, such as during antibiotic treatment or during an infection. They are, however, weaker organisms because of the multiple mutations they have undergone. Their presence does not relate to clinical outcome but may be associated with the emergence of antibiotic resistance. Study Hypothesis 3 Some Pseudomonas aeruginosa bacteria communicate with each other by secreting and responding to chemicals known as quorum sensing (QS)molecules. As well as affecting the behaviour of bacteria, these QS molecules can cause inflammation in the lung of CF patients. Selective growth of QS-producing organisms can trigger lung exacerbations in CF. If antibiotics kill this population of bacteria and QS molecule levels drop in the lung, patients recover from infection quickly. Failure to kill these bacteria with antibiotics allow QS molecule levels to remain elevated and patients to have prolonged infections.