An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen)...
Lipid MetabolismInborn Errors18 moreTo evaluate the safety and efficacy of extended dosing with mipomersen (ISIS 301012) in participants with familial hypercholesterolemia or severe hypercholesterolemia on lipid-lowering therapy who had completed either the 301012-CS5 (NCT00607373), 301012-CS7 (NCT00706849), 301012-CS17 (NCT00477594) or MIPO3500108 (NCT00794664) clinical drug trials.
Phase I/II Thymus Transplantation With Immunosuppression #950
DiGeorge AnomalyComplete DiGeorge Anomaly3 moreThe study purpose is to determine if cultured thymus tissue implantation (CTTI) (previously described as transplantation) with tailored immunosuppression based on the recipient's pre-implantation T cell population is a safe and effective treatment for complete DiGeorge anomaly. This study will also evaluate whether cultured thymus tissue implantation and parathyroid transplantation with immunosuppression is a safe and effective treatment for complete DiGeorge anomaly and hypoparathyroidism.
Prevention of Photoparoxysmal Abnormalities by Mozart K448
Photoparoxysmal AbnormalitiesEpilepsy1 moreThe purpose of this clinical trial is to examine the effect of non-invasive cortical activation by specific patterned auditory stimulation (Mozart K448- Piano Sonata for Two Hands in D Major), in altering or eliminating photoparoxysmal abnormalities. Recent work has indicated that such evoked activity (i.e. by specific music) can result in significant reductions, or even complete termination of epileptiform activity and/or seizures across a broad range of individuals with epilepsy. A hypothesis is generated that specific music activation would beneficially effect photoparoxysmal based epileptiform abnormalities.
Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia
Lipid MetabolismInborn Errors18 moreThe purpose of this study is to evaluate the safety and efficacy of extended dosing of mipomersen in patients with familial hypercholesterolemia on lipid-lowering therapy who have completed either the 301012-CS8 (NCT00280995) or 301012-CS9 (NCT00281008) clinical drug trials.
Measure Liver Fat Content After ISIS 301012 (Mipomersen) Administration
Lipid MetabolismInborn Errors17 moreThis study will assess what, if any, effect that ISIS 301012 (mipomersen) has on liver triglyceride content in multiple groups of subjects with varying degrees of risk for hepatic steatosis. In order to enroll subject groups with varying degrees of risk, the study has included multiple cohorts (Cohorts A-G). Additions and removal of cohorts has been accomplished with protocol amendments.
Tetracycline-Derivatives for Treatment of Cerebral Arteriovenous Malformations and Aneurysms
AneurysmsArteriovenous MalformationsThe purpose of this pilot study is to investigate the use of minocycline and doxycycline as medical therapy for inoperable or partially treated arteriovenous malformations (AVMs) and giant aneurysms.
Comparison of Two Types of Shunts in Infants With Single Ventricle Defect Undergoing Staged Reconstruction--Pediatric...
Heart DefectsCongenitalThis trial will evaluate the efficacy and safety of the modified Blalock-Taussig shunt (MBTS) compared to the right ventricle to pulmonary artery (RV-to-PA) shunt; compare the effect of the MBTS to that of the RV-to-PA shunt on the incidence of death or cardiac transplantation at 12 months post randomization; and compare the effect of the two shunts on intensive care unit (ICU) morbidity, unintended cardiovascular interventional procedures, right ventricular function, tricuspid valve regurgitation, pulmonary artery growth, and neurodevelopmental outcome.
Outcome of Preserved Mitral Valve Apparatus During Mitral Valve Replacement
Diseases of Mitral ValvesAbnormality of Mitral Valve Annulus (Disorder)1 moreAre there differences in outcome of mitral valve replacement with preservation of mitral apparatus among rheumatic and/or ischemic mitral lesions?. Mitral valve replacement with preservation of leaflets, and added coronary artery-bypass surgery, when indicated, is it a feasible and reproducible procedure?. The study was designed to compare outcome after prosthetic mitral replacement with preservation of mitral apparatus for rheumatic valve disease with outcome of replacement for ischemic myocardium and mitral valve disease, The outcomes will be guided by clinical assessment. and echo-cardiograph.
Synthetic Cranioplasty PEEK Versus UHMWP
Cranium; DeformityThe purpose of this study is to report the investigators experience with synthetic reconstruction of cranial defects using computer guided milled UHMWP, in terms of benefits and limitations both clinically and radiographically in comparison to PEEK implants.
Safety and Performance of the COR-VG-001 Conduit in Pediatric Patients for Extracardiac Total Cavopulmonary...
Heart DefectsCongenitalThe extracardiac Fontan surgery/procedure involves diverting the venous blood from the inferior vena cava to the pulmonary arteries without passing through the morphologic right ventricle. In the extracardiac conduit type of Fontan, one end of a synthetic tube graft is connected to the inferior vena cava and the other end to the pulmonary artery confluence. Xeltis developed a biodegradable prosthesis, the Xeltis Vascular Graft Model COR-VG-001, to be used as an extracardiac conduit between right atrium and the pulmonary arteries. The prosthesis is immediately mechanically functional, while its physiochemical characteristics should enable cell infiltration and tissue formation. The Xeltis Vascular Graft Model COR-VG-OO is specifically designed to enhance the Fontan surgery outcome by reducing synthetic material related complications and improving hemodynamic characteristics.