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Displaying 1 - 26 of 26
Condition: Blood Disorders / Sickle Cell Disease
Investigator: Monica Bhatia, MD
Status: Closed
This study is being done to see if giving your child a lower dose of chemotherapy (drug therapy), than has been used historically in bone marrow transplant, to kill his/her own diseased cells before he/she receives a bone marrow transplant, will possibly result in the permanent control of your child's sickle…
Condition: Blood Disorders
Investigator: Monica Bhatia, MD
Status: Closed
This study is being done to learn more about the safety and effects of CTX001 in patients with severe SCD. CTX001 is created by changing (editing) the DNA in your own blood stem cells near a gene called BCL11A which is stored in a solution called dimethyl sulfoxide (DMSO) to try to prevent…
Condition: Neurological Disorders / Brain Tumors
Investigator: Fabio Iwamoto, MD
Status: Currently Recruiting
The purpose of this study is to learn the effects of the investigational new drug, tirabrutinib, when given for the treatment of PCNSL. Investigational new drug means a drug that has not been approved as a marketed product (that is, available to be prescribed or sold) by the United States (US) Food and Drug Administration (FDA). Tirabrutinib acts by…
Condition: Blood Disorders
Investigator: Markus Mapara, MD
Status: Closed
We are doing a research study to find out whether it is safe to use a virus to change the genes of bone marrow cells that become red blood cells. We hope that this type of therapy may be helpful in people with sickle cell disease, but it has not been tested before.…
Condition: Blood Disorders / Sickle Cell Disease
Investigator: Monica Bhatia, MD
Status: Closed
The purpose of this study is to find out if the study medicine called EDIT-301 will help in the treatment of Severe Sickle Cell Disease (SCD) and how safe it is to be used in people. EDIT-301 (study medicine) is a new investigational therapy which uses the patient's own stem…
Condition: Blood Disorders
Investigator: Markus Mapara, MD
Status: Closed
This study is being done to learn about the safety and efficacy of CTX001 (the "Study Product") to treat -Thalassemia. CTX001 is "investigational", which means that the Study Product is considered experimental and is not approved by the Food and Drug Administration (FDA), European Medicines Agency (EMA) or any other regulatory (government) authority and is…
Condition: Blood Disorders
Investigator: Diane George, MD
Status: Closed
The purpose of this study is to determine the incidence and severity of acute GVHD following CD34+ selection in children, adolescents and young adults receiving an allogeneic peripheral blood stem cell transplant from a family member or unrelated adult donor for a non-malignant condition.
Condition: Neurological Disorders
Investigator: Thomas Brannagan, MD
Status: Currently Recruiting
The purpose of this research is to see if an experimental drug called REGN7544 is effective to treat individuals with postural orthostatic tachycardia syndrome (POTS). REGN7544 is an antibody. An antibody is a kind of protein that your immune (defense) system normally makes to fight bacteria and viruses in your body.…
Condition: Neurological Disorders
Investigator: William Kreisl, MD
Status: Closed
This study is for patients who have been diagnosed with Multiple System Atrophy (MSA). The main goals of this study are to determine how safe and well tolerated AZD3241 is in patients who have MSA, and whether AZD3241 has certain effects in the brain of patients with MSA. These effects in the brain are measured with positron…
Condition: Blood Disorders / Amyloidosis
Investigator: Rajshekhar Chakraborty, MD
Status: Currently Recruiting
This study is being done to answer the following questions: Do people with amyloid light chain (AL) amyloidosis respond better to a consolidation treatment that uses either one high dose of the drug melphalan plus stem cell transplant or more chemotherapy alone? Which treatment plan gives…
Condition: Neurological Disorders / Brain Tumors
Investigator: Fabio Iwamoto, MD
Status: Currently Recruiting
The purpose of this study is to compare the effects of pembrolizumab simultaneously with the Optune device and maintenance temozolomide(TMZ) versus placebo simultaneously with Optune and maintenance TMZ for the study treatment of newly diagnosed GBM. In this study, a placebo is an inactive substance that looks the same as and is given the same way as…
Condition: Neurological Disorders / Brain Tumors
Investigator: Andrew Lassman, MD
Status: Closed
The purpose of this research study is to learn about the effects of the study drug, PF-06840003,and to find the best dose for treating research subjects/participants with malignant glioblastoma or anaplastic glioma. PF-068400003 is a new investigational drug. A new investigational drug is one that is currently not approved for sale in this country. PF-…
Condition: Blood Disorders / Amyloidosis
Investigator: Suzanne Lentzsch, MD
Status: Closed
This is a research study for subjects that have been previously treated for systemic light chain (AL)amyloidosis (a disease in which abnormal protein deposits can damage organs and tissues in your body) and nowrequire further treatment. The main purpose of this study is to evaluate the safety and determine the maximum…
Condition: Neurological Disorders / Brain Tumors
Investigator: Andrew Lassman, MD
Status: Closed
The purpose of this study is to evaluate and determine the efficacy of pemigatinib in participants with recurrent glioblastoma or other CNS tumors with changes in the FGFR1, FGFR2 or FGFR3 genes. Pemigatinib (also referred as INCB054828) is an investigational drug that is being studied by Incyte Corporation (Sponsor) for use in the treatment of previously…
Condition: Neurological Disorders / Multiple Sclerosis
Investigator: Claire Riley, MD
Status: Closed
The purpose of this study is to see if ocrelizumab (study drug) will stabilize, or potentiallyimprove the signs and symptoms of your MS. Ocrelizumab is a type of drug called amonoclonal antibody. Monoclonal antibodies act like your bodys immune system andattach to certain cells in order to attack…
Condition: Neurological Disorders / Neuromuscular
Investigator: Thomas Brannagan, MD
Status: Closed
This study is designed to test how well an intravenous human immunoglobulin concentrate, IGIV-C (compared with placebo) improves the ability to taper participant's dose of corticosteroids without causing significant increase in your muscle weakness and worsening MG symptoms. A placebo is a medication that looks like the study medication but has no active…
Condition: Neurological Disorders / Neuromuscular
Investigator: Thomas Brannagan, MD
Status: Closed
This study is designed to test how well an intravenous human immunoglobulin concentrate, IGIV-C (compared with placebo) improves your muscle weakness associated with MG symptoms. A placebo is a medication that looks like the study medication but has no active ingredient. Immunoglobulin is an antibody produced by white blood cell…
Condition: Neurological Disorders / Brain Tumors
Investigator: Mary Welch, MD
Status: Closed
The purpose of this study is to see if the newly discovered vaccine drug, DSP-7888, will help your immune system to fight against cancer cells that have a protein called WT1. The study will also compare the effectiveness of treatment with DSP-7888 in combination with Bevacizumab versus…
Condition: Neurological Disorders
Investigator: Lawrence Honig, MD, PhD
Status: Closed
The purpose of this study is to see how safe and well tolerated the study drug BMS-986168 is at different drug amounts and to measure the amount of study drug in your blood and cerebrospinal fluid (CSF) after taking the drug. This study will also measure the effect of the study drug on the amount of extracellular Tau (eTau) in…
Condition: Neurological Disorders / Brain Tumors
Investigator: Teri Kreisl, MD
Status: Closed
The purpose of this study is to find out what effects, good or bad, the experimental device, NovoTTF-100A has on patients with meningioma. This study is being done because currently there are no proveneffective medical treatments for a progressive meningioma that has failed surgery and/or radiation. The NovoTTF-100A System is…
Condition: Blood Disorders
Investigator: Mark Heaney, MD, PhD
Status: Closed
This is a Phase 2 study for patients with myelofibrosis previously treated with one or more Janus Kinase(JAK) Inhibitor. A JAK inhibitor targets a certain pathway that may be causing cancer to grow, bystopping or inhibiting the cycle of development of the pathway. There is one JAK inhibitor that has beenapproved for the treatment of patients with…
Condition: Neurological Disorders / Brain Tumors
Investigator: Mary Welch, MD
Status: Closed
The purpose of this clinical study is to evaluate safety of the study treatment, its effects on the immune system and on GBM. The study treatment includes several experimental therapies: INO-5401 + INO-9012 delivered by an electroporation device called CELLECTRA 2000 used for delivery of INO-5401…
Condition: Neurological Disorders / Brain Tumors
Investigator: Fabio Iwamoto, MD
Status: Closed
The purpose of this study is to test how well the study drug works in recurrent or isocitratedehydrogenase (IDH) mutant high grade gliomas. The study drug is called Nivolumab. Nivolumab is an antibody (a type of human protein) that is being tested to see if it will allow your own body's immune system to work against tumor…
Condition: Neurological Disorders / Brain Tumors
Investigator: Fabio Iwamoto, MD
Status: Closed
The purpose of this study is to test the effectiveness (how well the drug works), safety, and tolerability of an investigational drug called nivolumab (also known as BMS-936558). Nivolumab is an antibody (a type of human protein) that is being tested to see if it will allow the body's immune system to work against tumor…
Condition: Neurological Disorders / Brain Tumors
Investigator: Fabio Iwamoto, MD
Status: Closed
The purpose of this study is to test the effectiveness (how well the drug works), safety, and tolerability of an investigational drug called nivolumab (also known as BMS-936558). Nivolumab is an antibody (a type of human protein) that is being tested to see if it will allow the bodys immune system to work against tumor…
Condition: Neurological Disorders / Brain Tumors
Investigator: Fabio Iwamoto, MD
Status: Closed
The purpose of this study is to find out if SL-701 is safe and tolerable for patients with recurrent glioblastoma multiforme (GBM). The study will also look at what effect SL-701 has on patient's brain tumor. SL-701 is a type of anti-cancer vaccine made up of parts 3 different short proteins called "peptides" which are frequently found on this type of brain…
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