8 Clinical Trials for Various Conditions
This is a data collection study with the purpose of capturing dosing and treatment outcome data in participants with severe congenital protein C deficiency who were treated with protein C concentrate under an Emergency Use Investigational New Drug Application (IND).
The purpose of this study is to show that Protein C Concentrate is a safe and effective treatment for subjects with congenital protein C deficiency. Depending on the type of treatment required, patients are assigned to one of 3 study parts: Part 1 is for the treatment of acute episodes, Part 2 is for short-term prophylaxis, and Part 3 is for long-term prophylaxis in infants aged less than 6 months only.
The overall objective is to collect and assess data on the treatment, safety, and treatment outcomes of subjects prescribed, receiving and participating in the Ceprotin treatment registry.
Inherited deficiencies in any one of 3 genes (surfactant protein B, surfactant protein C, and ATP-binding cassette transporter A3) can cause neonatal respiratory distress syndrome by disrupting metabolism of the pulmonary surfactant. The investigators will use state of the art methods to link specific changes in the genetic code of each of these genes with disruption of discrete steps in the metabolism of the pulmonary surfactant in human newborn infants. These studies will lead to improved diagnostic capabilities and suggest novel strategies to correct surfactant deficiency in newborn infants.
The purpose of this study is to test the hypothesis that excess, rare, functionally disruptive single nucleotide polymorphisms (SNPs) characterize genes (e.g., the surfactant protein-B gene)(SFTPB) and gene networks (e.g., the pulmonary surfactant metabolic network or other gene networks that regulate alveolar type 2 cell function) associated with increased risk of neonatal respiratory distress syndrome (RDS).
The purpose of this study is to observe changes in iron status and vitamin D status during a 12-24 week supervised training program, specifically males and females between the ages of 30 and 50 years old.
The purpose of this study is to determine if treatment with vitamin D3 of 10,000 International Units (IU) daily in vitamin D deficient patients for 6 months will improve B type natriuretic peptide (BNP), a marker of heart function, compared to placebo. The investigators also aim to determine if vitamin D helps cardiopulmonary function as evaluated by cardiopulmonary exercise test (CPX), laboratory values, strength, and quality of life in patients with stable congestive heart failure (Class II or III).
The purpose of this study is to examine whether oral vitamin D supplementation in people with inadequate vitamin D concentrations will lower LDL-cholesterol and total cholesterol concentrations.