
Umbilical Cord Mesenchymal Stem Cell Transplantation for Decompensated Hepatitis B Cirrhosis
Liver CirrhosisThis study aims to evaluate the safety and clinical efficacy of umbilical cord mesenchymal stem cell transplantation for decompensated hepatitis B cirrhosis.

Testing Drug Efficacy in Cystic Fibrosis Through N-of-1 Trials
Cystic FibrosisThe purpose of this study is to validate and utilize a personalized medicine approach to identify potential treatments with current FDA approved CFTR modifiers for non-approved CF gene mutations. The study will perform ex vivo testing of CFTR function and current marketed CFTR modulating drugs on expanded nasal cells at Cincinnati Children's Human Nasal Epithelium (HNE) Core Laboratory. The results will be confirmed and translated into bedside care through an N of 1 trial to determine effectiveness of treatment.

Efficiency Study for Acute Radiation-induced and Chemotherapy-induced Pulmonary Fibrosis With Bevasizumab...
Pulmonary FibrosisDue to the radiosensitivity of the lung, radiation-induced and chemotherapy-induced pneumonitis and pulmonary fibrosis are frequent happened following cancer therapy. It not only compromise cancer treatment, but also influence patient's life qualities and even death. there are no specific treatment modalities for this treatment-induced complication. Bevasizumab (Avastin), a VEGF inhibitor, can attenuate serum high expression VEGF and pulmonary permeability, maybe effective in the control acute pulmonary fibrosis. Patients will be randomized to receive Bevasizumab (7mg/kg iv) on day one and 21, followed by Dexamethasone (10mg iv d 1-10, then 5mg iv d11-15, 2.5mg iv d16-20) or Dexamethasone alone. The thoracic CT, plasma TNF-a, IL-6, VEGF and C-reactive protein are accessed on before treatment, 24 hours after Bevasizumab, 7 days, 4 and 8 weeks. the target sample size is 30 cases.

Jin-shui Huan-xian Granule in the Treatment of IPF
Idiopathic Pulmonary FibrosisThis study is to evaluate the efficacy and safety of Jin-shui Huan-xian granule for idiopathic pulmonary fibrosis (IPF), establish the treatment scheme, and obtain the high quality clinical evidences.

Home Airway Clearance in CF Patients
Pulmonary Cystic FibrosisRegular bronchial clearance is essential in patients with cystic fibrosis for their bronchial health. SIMEOX® (Physio-Assist, Aix en Provence, France) is an innovative medical device for the drainage of the bronchial tree. By changing the rheological properties of mucus, SIMEOX® helps to mobilize secretions and assists their transport to the upper airways. This technology is based on fundamental research on bronchial mucus rheology. At the present time, SIMEOX® device is mainly used over a short period at the time or after an exacerbation in healthcare structures (hospitals, physiotherapy practices, post-care, and rehabilitation units, etc…). The clinical effects observed in the short term encourages long-term autonomous use by the patients themselves. The overall objective of this study is to evaluate the efficiency and acceptability of SIMEOX® used at home by the patient himself for bronchial clearance in patients with cystic fibrosis.

To Study Efficacy of Albumin in Cirrhosis With Spontaneous Bacterial Peritonitis at Low Risk for...
Liver CirrhosisAcute Kidney InjuryThe role of Albumin in prevention and Treatment of Acute Kidney Injury (AKI) in patients with Spontaneous Bacterial Peritonitis (SBP) who are at high risk of AKI development has been clearly defined , which decreases the morbidity and mortality. But the role of Albumin in patient with SBP who are at low risk of AKI development (Serum Bilirubin <4mg/dl, Creatinine <1mg/dl at the time of presentation) has been controversial and there are no placebo controlled trials. We propose that Albumin at the standard doses is beneficial in preventing development of AKI in patients with SBP who are at low risk of AKI development.

Saracatinib in the Treatment of Idiopathic Pulmonary Fibrosis
Idiopathic Pulmonary Fibrosis (IPF)Scarring of the lung, termed pulmonary fibrosis (PF), is a chronic, progressive, and usually fatal disorder. While two anti-fibrotic drugs have been approved for treating PF of unknown cause (idiopathic pulmonary fibrosis or IPF), neither drug is curative, and nearly 40% of patients stop taking the prescribed drug within a year because of side effects. The study includes the use of saracatinib, an investigational drug originally developed to treat certain types of cancers, in the treatment of IPF in a Phase 1b/2a clinical trial. The objectives of this study are to: i) evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics, and to explore the efficacy of saracatinib in IPF; ii) identify biomarkers of Src kinase activity and fibrogenesis linked to pulmonary fibrosis; and iii) explore the application of these biomarkers to assess the anti-fibrotic effect of saracatinib in IPF patients

Effects of Metformin on Airway Ion Channel Dysfunction in Cystic Fibrosis-related Diabetes
Cystic Fibrosis-related DiabetesCystic FibrosisThe purpose of this study is to assess the efficacy of metformin to improve airway ion channel function in those with CF-related diabetes (CFRD)

A Phase 1 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Immunogenicity of HuL001...
Idiopathic Pulmonary FibrosisThis is a first-in-human, two-part, Phase 1 study that will characterize the safety, tolerability, PK, and immunogenicity of HuL001.

A New Posaconazole Dosing Regimen for Paediatric Patients With Cystic Fibrosis and Aspergillus Infection...
Cystic FibrosisAspergillosisThis study will provide: (1) new insights in the prevalence of Aspergillus infection in children and adolescents with CF aged 8-17 yrs; (2) an in silico modelled dose of posaconazole for children and adolescents with CF and Aspergillus infection aged 8-17 yrs; (3) an intensive sampling PK study to define the optimal dose in a limited number of children and adolescents with CF and Aspergillus infection aged 8-17 yrs; (4) a prospective clinical validation to reduce the residual variability and to allow investigation into PK-PD; and (5) an efficacy evaluation of this dosing regimen to treat Aspergillus infection in children and adolescents with CF to inform future primary efficacy trials.