Giselle Sholler
An Intermediate Expanded Use Trial of DFMO
Conditions: Neuroblastoma, Medulloblastoma, Typical Teratoid Rhabdoid Tumor, Embryonal Tumor With Abundant Neuropil and True Rosettes, Ependymoblastoma, Medulloepithelioma
To provide DFMO in an expanded use setting to subjects with relapsed rare tumors withincreased LIN28 expression or MYCN amplification or up regulation of ornithinedecarboxylase.
Cook MyoSite
Expanded Access Program for Autologous Muscle Derived Cells (AMDCs)
Conditions:
This is an expanded access program (EAP) for eligible participants designed to provideaccess to autologous muscle derived cells (AMDCs). Treating physicians may submitrequests for expanded access to Cook MyoSite's investigational product by email toMYO-ExpandedAccess@CookMyoSite.com.
Chimerix
Intermediate-size Expanded Access to ONC201 for Patients With H3 K27M-mutant and/or Midline Gliomas
Conditions: Glioma, H3 K27M
This is an intermediate-size expanded access protocol to provide ONC201 (dordaviprone) topatients with H3 K27M-mutant and/or midline gliomas who cannot access ONC201(dordaviprone) through clinical trials.
Vanda Pharmaceuticals
Single-Patient Expanded Access Protocol for Tradipitant In A Single Patient With Gastroparesis
Conditions: Gastroparesis, Diabetic Gastroparesis
Primary Objective: To treat a single patient with gastroparesis who has requestedexpanded access with tradipitant
Leonard-Meron Biosciences, Inc.
Expanded Access: Mino-Lok Therapy (MLT) for the Treatment of CRBSI/CLABSI
Conditions: Catheter-Related Infections
This is an Intermediate-Size Expanded Access, Open-Label Study for Use of Mino-LokTherapy (MLT) in Combination with Systemic Antibiotics in the Treatment of Central LineAssociated Bloodstream Infection. Mino-Lok may be made available for patients whootherwise do not qualify for the phase 3 clinical trial (NCT02901717 )
Cancer Commons
Expanded Access to Ulixertinib (BVD-523) in Patients with Advanced MAPK Pathway-Altered Malignancies
Conditions: Pancreatic Cancer, Small Bowel Cancer, Colorectal Cancer, Melanoma, Non Small Cell Lung Cancer, Thyroid Cancer, Bladder Cancer, Head and Neck Cancer, Gastric Cancer, Esophageal Cancer, Cholangiocarcinoma, Ovarian Cancer, HepatoCellular Carcinoma, Glioblastoma, MAPK Gene Mutation, KRAS Activating Mutation, BRAF Gene Mutation, NRAS Gene Mutation, HRAS Gene Mutation, MEK Mutation, ERK Mutation
The objective of this expanded access program is to provide ulixertinib (BVD-523) forcompassionate use in advanced cancer patients with MAPK pathway-altered solid tumor(s),including but not limited to KRAS, NRAS, HRAS, BRAF, MEK, and ERK mutations who haveincomplete response to or have exhausted available therapies.Ulixertinib is available for treatment as monotherapy or in combination with otherclinically tolerable agent(s), conditionally approved by the drug manufacturer.
AIM ImmunoTech Inc.
Ampligen in Chronic Fatigue Syndrome
Conditions: Chronic Fatigue Syndrome
This is an open label study of Ampligen in patients with chronic fatigue syndrome.
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.
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.
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.