Genentech, Inc.
A Study of Vismodegib (GDC-0449) in Patients With Locally Advanced or Metastatic Basal Cell Carcinoma
Conditions: Basal Cell Carcinoma
This is an open-label, non-comparative, multicenter, expanded access study of Vismodegib(GDC-0449) in patients with locally advanced basal cell carcinoma (BCC) or metastatic BCC(mBCC) who are otherwise without satisfactory treatment options.
Shire
Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease
Conditions: Gaucher Disease, Type 1
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of theenzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB,glucocerebroside accumulates within macrophages leading to cellular engorgement,organomegaly, and organ system dysfunction. The purpose of this treatment protocol is toobserve the safety of velaglucerase alfa in patients with type 1 Gaucher disease who areeither treatment naive (newly diagnosed) or who are currently being treated with theEnzyme Replacement Therapy (ERT) imiglucerase.
Mentor Worldwide, LLC
Mentor Adjunct Study for Silicone Gel-Filled Mammary Prosthesis
Conditions: Breast Reconstruction
Mentor is undertaking a five-year prospective clinical study designed to collect safetydata associated with the implantation of its gel-filled mammary prostheses. This study isan "adjunct" study, which will encompass clinical reviews of reconstructive cases in allpatients who meet clinical and regulatory criteria for breast reconstruction withgel-filled mammary prostheses. This "adjunct" study will be accomplished under a limitedclinical protocol in which specific parameters will be required but with controlssomewhat less stringent than those normally required in Investigational Device Exemption(IDE) Trials.
Supernus Pharmaceuticals, Inc.
Long Term Study of the Safety of OXC XR as in Pediatric Epilepsy Subjects
Conditions: Epilepsy
Follow-on study to continue evaluation of the safety of OXC XR as adjunctive therapy inpediatric epilepsy
SynCardia Systems. LLC
SynCardia Freedom Driver System Study
Conditions: Biventricular Failure
The purposes of this study are to confirm that the Freedom Driver System is a suitablepneumatic driver for clinically stable TAH-t subjects, and that patients and laycaregivers can be trained to manage the Freedom Driver System safely outside thehospital.
GE Healthcare
Expanded Access Protocol for [123I]mIBG for Patients With Known or Suspected Neuroblastoma
Conditions: Neuroblastoma
GE Healthcare has recently submitted a New Drug Application (NDA) for Iobenguane I 123Injection ([123I]mIBG (AdreView)) as a diagnostic nuclear imaging agent for the detectionof primary or metastatic neuroblastoma and pheochromocytoma. The present protocolestablishes an Expanded Access program to provide AdreView to pediatric medical centersand hospitals that treat neuroblastoma patients. AdreView will be provided for use indiagnostic assessment of patients with known or suspected neuroblastoma for whom there isan appropriate clinical indication for [123I]mIBG imaging.
Genzyme, a Sanofi Company
Alglucosidase Alfa Temporary Access Program
Conditions: Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-Onset), Acid Maltase Deficiency Disease, Glycogenosis 2
Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiencyof a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA isused by the body's cells to break down glycogen (a stored form of sugar) withinspecialized structures called lysosomes. In patients with Pompe disease, an excessiveamount of glycogen accumulates and is stored in various tissues, especially heart andskeletal muscle, which prevents their normal function. The objective of this expandedaccess study is to provide patients with Pompe disease in the United States (US), accessto alglucosidase alfa produced from a scaled up manufacturing process for a limited timeuntil production at this scale is approved for commercial use by the Food and DrugAdministration.
Gilead Sciences
Aztreonam Lysine for Inhalation in Patients With Cystic Fibrosis and Pseudomonas Aeruginosa Airway Infection
Conditions: Cystic Fibrosis, Pseudomonas Aeruginosa Airway Infection
The primary objective of this program is to provide expanded access to aztreonam lysinefor inhalation (AZLI) 75 mg prior to its commercial availability to patients with cysticfibrosis (CF) and chronic P. aeruginosa airway infection who have limited treatmentoptions and are at risk for disease progression.
BioMarin Pharmaceutical
Sapropterin Expanded Access Program
Conditions: Phenylketonuria
The Purpose of this study is to provide patients with hyperphenylalaninemia (HPA) due toPhenylketonuria (PKU) access to sapropterin dihydrochloride and to collect moreinformation about the safety of the drug in an expanded access program (EAP) untilcommercial product is available.
Alexion Pharmaceuticals, Inc.
The Paroxysmal Nocturnal Hemoglobinuria Early Access Treatment Protocol
Conditions: Hemoglobinuria, Paroxysmal
The primary objective is to provide access to eculizumab for PNH patient pendingcommercial availability.