Joanne Kurtzberg, MD
Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants
Conditions: Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies
The objective of this study is to make T-cell depleted stem cells from a family memberwho is a half match (haplo-identical) available on an expanded access basis to patientsreceiving one or two unrelated cord blood transplants who are at a higher risk of notengrafting in a safe amount of time. The purpose of the related stem cells is the givethe bone marrow a "jump start" towards recovery. Ultimately, the cord blood cells willgrow and permanently rescue the bone marrow.
BioMarin Pharmaceutical
BMN 110 US Expanded Access Program
Conditions: Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
The Expanded Access Program (EAP) is an open-label, multicenter program to: 1. Provide patients who have been diagnosed with Mucopolysaccharidosis IVA (MPS IVA) access to BMN 110 until commercial product is available 2. Collect additional information on the safety and tolerability of BMN 110 administration in patients with MPS IVAPatients enrolled in the EAP will receive 2.0 mg/kg intravenous infusions of BMN 110every week during the program.
Pharmacyclics LLC.
An Open Label Treatment Use Protocol for Ibrutinib in Subjects With Relapsed or Refractory Mantle Cell Lymphoma
Conditions: Relapsed or Refractory Mantle Cell Lymphoma
The purpose of this study is to provide patients who have relapsed or refractory MantleCell Lymphoma (MCL) with early access to an investigational medication called ibrutinib(PCI-32765) and to collect safety information about the drug.
Celgene
Pomalidomide (POM) in Combination With Low Dose Dexamethasone (LD-Dex) in Patients With Relapsed or Refractory Multiple Myeloma
Conditions: Multiple Myeloma
To provide pomalidomide access to relapsed/refractory multiple myeloma subjects with alikelihood of benefit from the pomalidomide treatment while the medication is notcommercially available
Ariad Pharmaceuticals
Expanded Access Program of Ponatinib
Conditions: Chronic Myeloid Leukemia (CML), Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia (Ph+ ALL)
This protocol will allow expanded access of ponatinib to patients ≥18 years with chronicmyeloid leukemia (CML) any phase or Philadelphia chromosome positive acute lymphoblasticleukemia (Ph+ALL) who have failed all available treatment options.
Jubilant DraxImage Inc.
Expanded Access Protocol Using 131I-MIBG
Conditions: Neuroblastoma, Pheochromocytoma, Paraganglioma
Protocol JDI2007-01 is an Expanded Access Protocol with therapeutic 131I-MIBG forpatients with neuroblastoma or pheochromocytoma / paraganglioma, who otherwise do notqualify for available treatments, or where approved treatment is not commerciallyavailable.
Eyetech Pharmaceuticals
A Single-Center Trial of High Frequency Pegaptanib for Rapid Restoration of VEGF Levels in Diabetic Retinal Edema
Conditions: Diabetic Macular Edema
Establish the efficacy of initial high frequency loading of intravitreal pegaptanibbi-weekly during the initial treatment period when the VEGF levels are the greatest andthen gradually extending the administration frequency to monthly as homeostasis ensuesfor the treatment of DME, as measured by best-corrected visual acuity.
Amicus Therapeutics
Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
Conditions: Fabry Disease
This program allows physicians to request permission from Amicus Therapeutics (Amicus)for treatment access to migalastat hydrochloride (HCl) for specific adult patients withFabry disease. Treatment is open label for 6 months with renewal every 6 months.
Bolton Medical
Phase II Clinical Study of the Safety and Efficacy of the Relay Thoracic Stent-Graft
Conditions: Thoracic Aortic Aneurysm, Penetrating Ulcers
This study is a continuation of the pivotal trial studying the safety and efficacy of theRelay thoracic stent-graft system to treat thoracic aortic aneurysms. Efficacy is beingevaluated by the device-related adverse event rate of endovascular repair (via the Relaythoracic stent-graft) through 1 year. Safety is being evaluated by comparing majoradverse events through 1 year in subjects treated with the Relay thoracic stent-graft tothose who underwent surgical repair.
Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Study of Abiraterone Acetate in Patients With Advanced Prostate Cancer
Conditions: Prostate Neoplasms, Genital Neoplasms, Male, Urogenital Neoplasms, Genital Diseases, Male
The purpose of this study is to collect additional safety information on abirateroneacetate administered with prednisone to patients with metastatic castration-resistantprostate cancer (CRPC).