
Long-term Study, Comparing Vest Therapy to Positive Expiratory Pressure (PEP) Therapy in the Treatment...
Cystic FibrosisThis study is a preliminary study designed to determine the safety and effectiveness of HFCWO using the InCourage vest system as an airway clearance technique for the treatment of CF. The study will compare HFCWO to the most commonly used airway clearance technique in Canada, namely the Positive Expiratory Pressure technique in patients with CF over a period of one year. As this is a preliminary study, it will not be able to determine if the vest is equivelant to PEP, but will provide the information needed to plan a larger study to answer that question.

Infant Study of Inhaled Saline in Cystic Fibrosis
Cystic FibrosisThe purpose of this study is to assess whether 7% hypertonic saline (HS) is an effective and safe therapy in infants and young children with CF.

Autologous Bone Marrow Stem Cells in Cirrhosis Patients
Stem Cell TransplantationCirrhosisLiver cirrhosis (LC) is the end stage of chronic liver disease. The liver transplantation is one of the only effective therapies available to such patients. However, lack of donors, surgical complications, rejection, and high cost are it's serious problems. The potential for stem cells in bone marrow (BM) to differentiate into hepatocytes cells was recently confirmed. Moreover, BMC transplantation has been performed to treat hematological diseases, and several clinical studies have applied BMC injection to induce regeneration of myocardium and blood vessels. In this study we will evaluate safety and feasibility of autologous bone marrow mono nuclear (BM-MNC) and enriched CD133+ hematopoietic stem cell transplantation through the portal vein in patients with decompensate cirrhosis.

Efficacy of Bucelipase Alfa (BSSL) in Patients With Cystic Fibrosis and Pancreatic Insufficiency...
Cystic FibrosisExocrine Pancreatic InsufficiencyThe purpose of this study is to evaluate the efficacy in patients with cystic fibrosis and pancreatic insufficiency following treatment with BSSL

Study Comparing Tetrathiomolybdate vs Standard Treatment in Primary Biliary Cirrhosis
Primary Biliary CirrhosisThe University of Michigan is conducting a study investigating a potential new treatment aimed at slowing/halting progression of primary biliary cirrhosis. This will be a 2 arm double blind study in which half of the patients will be randomly selected to receive a placebo (capsule with no active ingredient) and half will receive the new treatment drug, tetrathiomolybdate. Neither the patient nor the treating physician will know which arm the patient is in. The length of the study for each patient is 24 months of drug therapy. Lab draws will be necessary weekly for the first 6 weeks of the study, followed by every other week for 3 weeks, and then monthly for the remainder of the 2 year period. In addition, intermittent history and physicals and urine samples will also be necessary. There is no cost to you for any experimental treatment. All patients in both arms will continue on ursodiol and receive standard of care treatment

Use of Ultrase® MT12 in Young Cystic Fibrosis Children (CF)
Cystic FibrosisPancreatic InsufficiencyMulticenter, explorative, phase IIIb, open-label study to assess the efficacy and safety of Ultrase® MT12, in the control of steatorrhea and clinical signs and symptoms of malabsorption in CF children with pancreatic insufficiency (PI). This study is sponsored by Aptalis Pharma (formerly Axcan).

Study of VX-809 in Cystic Fibrosis Subjects With the ∆F508-CFTR Gene Mutation
Cystic FibrosisThe primary objective of the study was to evaluate the safety and tolerability of VX-809 in participants with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.

Macitentan Use in an Idiopathic Pulmonary Fibrosis Clinical Study
Idiopathic Pulmonary FibrosisThe AC-055B201/MUSIC study is a Phase II study, comparing one dose of ACT-064922 (macitentan) 10 mg with placebo in patients with idiopathic pulmonary fibrosis (IPF). The main study objective is to demonstrate that macitentan positively affects the forced vital capacity (FVC) in comparison with placebo in patients with idiopathic pulmonary fibrosis (IPF). The secondary objectives are to evaluate the effect of macitentan on the time to disease worsening or death in patients with IPF, and to evaluate the benefit/risk profile of macitentan in the treatment of patients with IPF.

Efficacy and Safety of Ultrase® MT20 in Improving the Coefficient of Fat Absorption (CFA) in Children...
Cystic FibrosisPancreatic InsufficiencyThis is a Phase III, multicenter, open-label study, that will evaluate the improvement of nutrient absorption when participants will receive Ultrase® MT20. This study is sponsored by Aptalis (formerly Axcan). This study is performed in children from 7 to 11 years old.

Study of INT 747 in Combination With URSO in Patients With Primay Biliary Cirrhosis (PBC)
Liver CirrhosisBiliaryThe primary hypothesis is that INT-747 will cause a reduction in alkaline phosphatase levels in Primary Biliary Cirrhosis patients, over a 12 week treatment period, as compared to placebo.