This study is being done to test the orally administered study drug, CA-4948 alone, in combination with ibrutinib, or ibrutinib alone in adult patients ages 18 and over diagnosed with relapsed or refractory (RR) Primary Central Nervous System Lymphoma (PCNSL). This study will take place at multiple centers globally. The study drug, CA-4948, is thought to…
The purpose of this study is to find out if performing Magnetic Resonance Image (MRI) scans of your brain with adaptive planning during your radiation treatment of your high-grade glioma will help determine how sensitive the images collected of the brain are at detecting the response of the tumor to the cancer treatment. In this study, four or seven MRI…
This research study seeks to better understand the biological and behavioral aspects of specific phobia and evaluate how the "Self-Distancing" technique might help youth confront their fears. The study will be conducted at Columbia University in Manhattan. Participation involves completing questionnaires and attending a single visit for a…
The purpose of the study is to evaluate the safety of Lutathera alone in patients with recurrent glioblastoma and the combination of Lutathera with radiotherapy with temozolomide (TMZ) in patients with newly diagnosed glioblastoma (GB) and to establish the appropriate dose of this combination. Lutathera is a radioligand therapy, which is a targeted…
Children and young adults with diffuse midline glioma will be treated with Focused Ultrasound (FUS) with DEFINITY microbubbles and neuro-navigator-controlled sonication. The overall goal of this study is to see if the study treatment plan will improve the outcome of participants with diffuse midline glioma, for which no known cure exists, and determine…
The purpose of this study is to test good and bad effects of these two different drugs against meningioma tumors with altered genes. Today, therapy for meningioma is the same for all patients and is not based on tumor genetic testing. This trial is trying to see if tumor genetic testing would be helpful at guiding treatment in patients with meningioma.…
The purpose of this study is to find out if REC-2282, an investigational new drug, is an effective and safe treatment in patients with progressive NF2 mutated meningiomas who have either NF2 disease-related meningioma or recurrent sporadic meningiomas that have NF2 mutation. Investigational new drug means a drug that has not been approved as a marketed…
The purpose of this study is to see how safe and effective Safusidenib erbumine, the study drug, is in treating brain glioma and to find the recommended dose of the study drug for clinical studies. Safusidenib is not Food and Drug Administration (FDA) approved.
This is a Tissue Repository to collect and store tissue samples from normal donors and patients with hematological disorders for future study research studies. The purpose of the storage bank is to obtain a large number of the bone marrow aspirates, bone marrow biopsies, and/or blood as well as buccal smears (to be used as a non-hematologic control for each…
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 cells, modifies them by CRISPR technology, and infuses them back to the…
The purpose of this study is to evaluate how safe the investigational drug, DSP-0390, is at different doses and to identify a recommended dose that is safe for further study. Investigational means that the study drug is currently being tested and has not been approved for use alone or in combination with any drug by the U.S. Food and Drug Administration (…
The purpose of this study is to see how safe the study medicine, IGV-001 plus standard of care (SOC), is and to compare IGV-001 with placebo see if it works in slowing glioblastoma multiforme (GBM) tumor regrowth. SOC refers to the usual medicine(s) or procedures used to treat your cancer. A placebo looks like the study medicine but does not contain any…
The purpose of the study is to determine if the drug, CAEL-101 in combination with plasma cell treatment improves overall survival for patients who are diagnosed with stage 3b of AL amyloidosis. Patients who participate in this study will receive CAEL-101 as well as plasma cell treatment or Placebo as well as plasma cell treatment to compare the effects.
The purpose of the study is to determine if the drug, CAEL-101 in combination with plasma cell treatment improves overall survival for patients who are diagnosed with stage 3a of amyloid light chain (AL) amyloidosis. Patients who participate in this study will receive CAEL-101 as well as plasma cell treatment or Placebo as well as plasma cell treatment to…
The purpose of this research study is to see whether the presence or absence of certain genes is associated with the development of AL amyloidosis in subjects 60 years of age or older with the blood disorders smoldering multiple myeloma (SMM) and monoclonal gammopathy of undetermined significance (MGUS). Each person's disease has different genetic…
The purpose of this study is to learn about whether the study drug (PF-07209326) is safe and effective in people with sickle cell disease and learn about what the right dose is. We will look at drug levels in the blood, see how fast drug levels rise and fall, and what effects the drug has, all of which are known as the pharmacokinetics (PK) and…
The purpose of this study is to find the best dose of a drug called MTX110 and a contrast agent called gadolinium that can be given safely in children with newly diagnosed diffuse midline gliomas, which is a kind of brain tumor. All patients enrolled in the study will receive infusion of MTX110 and Gadolinium delivered with a pump directly into the tumor…
The purpose of this research study is to see if the combination of medications approved for the treatment of glioblastoma, together with the study drug, selinexor (also known as XPOVIO), has any effects on your cancer. Selinexor works by trapping tumor suppressor proteins within the cell nucleus. This is expected to cause the cancer cells to die or stop…
This study is being done to evaluate the safety and usefulness of the study drug, AG-881, as compared to placebo (a medically inactive substance) in residual or recurrent non-enhancing Grade 2 glioma (a type of brain cancer) that has a mutation in the IDH1 or IDH2 metabolic enzyme (a substance produced by the body to assist in breaking down chemicals).…
The purpose of this research study is to test how safe and how well an investigational drug known as ONC201 works in treating high grade glioma (a type of brain cancer). The FDA (the U.S. Food and Drug Administration) has not approved ONC201 as a treatment for this condition.
Sertraline (also known as Zoloft) is an FDA approved medication that is used to treat anxiety in children and adolescents. We aim to better understand how children and adolescents with anxiety respond to sertraline and whether we can predict medication response. If your child is eligible, he or she can participate in a research study and will receive both a…
The purpose of this study is to measure how well and how safe eflornithine is in combination with lomustine, compared to lomustine taken alone, in treating patients whose anaplastic astrocytoma has come back after radiation and chemotherapy. Safety and how well you can tolerate the drug will be determined on the basis of physical exams, laboratory tests,…
The purpose of this study is to evaluate the safety and effectiveness of an antibody drug (STI-6129) in patients with AL amyloidosis that has returned or has not responded to treatment (relapsed/refractory). The study drug (STI-6129) will be given in 3 injections through a needle, into a vein as an intravenous (IV), with 21 days between each injection.
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…
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.
The purpose of this study is to: See if taking the study agent, armodafinil, at a dose of 150mg or 250mg, will improve problems with fatigue in patients who have been diagnosed with cancer and are experiencing fatigue. See the effects (good and bad) of taking Armodafinil compared to placebo (an inactive agent) on cancer-related fatigue
The purpose of this study is to find out whether an experimental drug, bevacizumab with temozolomide is safe when given to elderly patients with brain tumors and investigate the side effects of the drug combination and how often they occur. This study will also explore whether bevacizumab and temozolomide can be effective in shrinking tumors when given to…
The purpose of this research study is to determine the effects of INCB050465 in combination with ruxolitinib treatment on spleen size and/or symptoms and to learn about any of the side effects that might occur during or following dosing with this combination of molecules in patients diagnosed with myelofibrosis.
This research study is being done to determine if an investigational drug, SY-1425 (tamibarotene) works in treating AML (acute myeloid leukemia) and MDS (myelodysplastic syndrome) in patients with a certain biomarker in their blood (a blood test that may indicate the drug target is present in your type of AML or MDS).
In this research study, we are looking for Information on how well nivolumab with or without ipilimumab works in combination with TTFields in adult patients with glioblastoma. You will receive the Nivolumab and/or Ipilimumab study treatment until you decide to stop study treatment or your study doctor decides to remove you from the study. This means that…
This is a research study to test the effects of VB-111 combined with Bevacizumab versus Bevacizumab alone on glioblastoma . This study will enroll 252 subjects age 18 years and older who have recurrent glioblastoma. All subjects will be randomly assigned in a 1:1 ratio to receive either VB-111 combined with Bevacizumab or Bevacizumab alone. Survival, tumor…
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 cell disease. Patients will receive busulfan…
The purpose of this study is to determine the safety of directly injecting a tumor chemotherapy drug called Topotecan and a contrast agent called Gadolinium into malignant brain tumors.
The purpose of this research is to learn how to help parent youth pairs work together to help youth with sickle cell disease take hydroxyurea every day as a daily habit. This study is for children and teens ages 10-18 with Sickle Cell Anemia taking hydroxyurea, and their parents (or guardians). Many children and teens do not use hydroxyurea every day. Daily…
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-…
HELIOS-A is a global Phase 3 randomized, open-label study designed to evaluate the safety and efficacy of ALN-TTRSC02 in adult patients with hATTR amyloidosis experiencing neurologic symptoms brought on by the disease. The study will also evaluate any changes in quality of life experienced by study participants. ALN-TTRSC02 utilizes the mechanism of RNA…
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 cells. Nivolumab is not…
RESEARCH STUDY:Brief Intervention for Public Speaking Fears (IRB#7293).- Are you between the ages of 18 - 60?- Do you have bothersome fears of public speaking?- Are you interested in trying a novel intervention?This research study tests whether a brief intervention using a medication called propranolol is effective at reducing public speaking fears. This…
The main purpose of this study is to see if people with certain types of anemia due to MDS will experience a decreased need for regular blood transfusions if they take luspatercept plus best supportive care. The safety of luspatercept will be also evaluated in this study.
This is a two-part research study of an investigational drug called ABBV-321 in subjects with glioblastoma (brain tumor). This study will be conducted in two-parts: 1) Dose Escalation Phase: The purpose of the first part, called a Dose Escalation Phase, is where we will increase the dose of ABBV-321 until we find the highest dose before unmanageable side…
This is a Phase 2b/3 study to compare the effectiveness of the study drug ABT-414 against a placebo. Patients will be randomized to either receive the standard-of-care therapy which consists of radiation therapy/TMZ plus adjuvant TMZ which will be followed by eiter the study drug ABT-414 or a placebo monotherapy.
The purpose of this study is to see if daratumumab when given with three other drugs cyclophosphamide, bortezomib (VELCADE) and dexamethasone is useful for treating patients with Amyloid light chain Amyloidosis (also known as AL Amyloidosis). The study will look at what happens (both good and bad) when daratumumab is given with these three drugs compared to…
The purpose of this study is to compare any good and bad effects of avoiding the hippocampus during whole-brain radiation plus memantine to using the usual whole-brain radiation plus memantine. The hippocampus is a brain structure that is important for memory. The addition of the hippocampal avoidance technique to the usual whole-brain radiation plus…
The purpose of this study is to find out if MLN9708 plus dexamethasone improves against systemic light chain amyloidosis better than the physicians choice chemotherapy treatment. Physicians choice chemotherapy treatment is a treatment regimen that is commonly used to treat amyloidosis patients. The study will also determine if MLN9708 plus dexamethasone can…
The purpose of this study is to test for any good and bad effects of the study drug called isatuximab. Isatuximab may or may not improve your AL amyloidosis, but it could also cause side effects. Isatuximab is not approved by the Food and Drug Administration (FDA). Isatuximab has been shown to have good effects in some patients with recurring multiple…
The main purpose of this study is to improve treatment of ocular side effects that result from depatuxizumab mafodotin (Depatux-M) use in patients with glioblastoma (GBM). Most people treated with Depatux-M will experience eye side effects. When these side effects are not tolerated, Depatux-M is usually interrupted until symptoms improve and then restarted…
This study is for patients have been diagnosed with Intermediate or High-risk myelodysplastic syndrome (MDS). MDS is a condition where there is a low count of white blood cells, red blood cells and platelets because of a malfunction of bone marrow that is responsible for producing healthy mature blood cells. evaluate the combination of the investigational…
This is a research study to test the effectiveness of ICT-107, an experimental drug, with the standard of care (SOC) for treatment of glioblastoma compared to enriched blood cells (PBMC) with the SOC. The study will enroll 400 subjects age 18 years and older with newly diagnosed glioblastoma. Following an initial SOC radiation treatment, patients will be…
Itacitinib is an investigational drug that is being developed by Incyte Corporation for use in the treatment of acute graft-versus-host disease (GVHD). At this time, it is not known whether the study drug has an effect on sperm or eggs, whether they are secreted in the semen or whether they have an effect on a fetus. We would like to collect medical…
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 Bevacizumab alone in patients with glioblastoma (GBM). Bevacizumab has…
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 tolerated dose of aninvestigational new…
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 cells. The effectiveness of BMS-936558…
The purpose of this study is to collect information on whether idasanutlin is effective in treating polycythemia vera (a slow-growing blood cancer in which your bone marrow makes too many red blood cells) and what effects, good or bad, idasanutlin has on you. Idasanutlin is an experimental drug, which means Health Authorities (such as the US FDA) have not…
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 + INO-9012 into your muscle. You will also receive another…
This study will test an experimental drug, momelotinib, for the treatment of primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis. Primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis are all myeloproliferative disorders; which are serious bone…
The purpose of this research study is to test whether the advanced perfusion MRI done prior to starting Bevacizumab and a few weeks after starting Bevacizumab can determine your response to the treatment better than the standard MRI performed at typical time points. The researchers want to see if this type of advanced MRI is better or the same as what is…
The purpose of this study is to evaluate the efficacy and safety of ION-682884 given for 24 months in patients with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN) also known as Familial Amyloid Polyneuropathy (FAP), and determine if it can help people with mild or moderate polyneuropathy. To be in this study you must have FAP and be…
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 a portable medical device. It…
The purpose of this study is to evaluate the safety, pharmacokinetics (how much the study drugs are absorbed in your body at different times), and efficacy (how well the study drugs work) of venetoclax in combination with azacitidine compared to azacitidine alone in subjects with previously untreated higher-risk MDS.
The purpose of this research study is to see if Selinexor (KPT-330) has any effects against glioblastoma, a type of brain cancer. The study will be conducted at 3-5 sites in the United States and Europe and plan to enroll 30 patients with brain cancer. Patients will be screened prior to study participation. If their brain tumor is evaluated to require…
The study is being done to see if the combination of bendamustine and dexamethasone will help people with amyloidosis that has returned after standard treatment. Bendamustine is currently approved by the Food and Drug Administration (FDA) for treatment of chronic lymphocytic leukemia (CLL) and is under clinical development in the United States for the…
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 cells.
The main purpose of this research study is to determine the effect of the study drug compared to placebo in increasing red blood cell counts and reducing the severity of symptoms of Sickle Cell Disease (SCD). A placebo is a dummy drug: it looks like the study drug but contains no medicine. Subjects being asked to take part in this research study have SCD.…
The purpose of this study is to look at the effects of an investigational anti-cancer drug (BGB-290) in combination with radiation therapy and/or temozolomide in patients with newly diagnosed or recurring glioblastoma. BGB-290 is expected to work by preventing cancer cells from repairing their damaged genes, causing the progression of cancer to slow down.
We are currently enrolling individuals with social anxiety disorder to participate in this study. The purpose of this study is to assess whether a brief computer-based research treatment helps improve social anxiety symptoms. For eligible participants, this study will involve a brief, novel research treatment for social anxiety, delivered in 8 or 12…
This study is for patients with sickle cell disease (SCD). SCD is an inherited genetic disorder in which red blood cells become abnormally shaped (sickle cells). These sickle cells may block blood vessels and result in a vaso occlusive crisis (VOC). This leads to inflammation and tissue damage and causes pain in various parts of the body. The purpose of…
The purpose of this study is to investigate a novel treatment regimen for patients with recurrent Glioblastomas (GBM). In this phase 2 trial, the potentially synergistic action of PARP inhibition and anti-angiogenesis on DNA repair and biomarkers of angiogenesis and DNA repair will be evaluated.
The purpose of this study is to find out whether study drug bortezomib with radiation therapy and Temozolomide is safe when given to patients with brain tumors. This study will also evaluate the side effects and see whether the combination of bortezomib with radiation and Temozolomide can be effective in shrinking tumors. Bortezomib is the investigational…
The purpose of this study is to evaluate the safety, pharmacokinetics (how much of the study drugs is present in your body at different times), and efficacy (how well the study treatment works) of venetoclax alone and of venetoclax in combination with azacitidine in subjects with higher-risk MDS after HMA-failure.
This research study is a Phase II clinical trial. Phase II clinical trials test the safety and effectiveness ofan investigational drug to learn whether the study drug works in treating a specific disease.Investigational means that the drug is being studied. In this research study, we are looking to compare the effects, good and bad, of the standard of care…
This is a research study to test the effectiveness of Toca 511/Toca FC, an experimental drug, for treatment of recurring brain tumors compared to current standard of care. The study will enroll 370 subjects age 18-85 years who choose to undergo removal of the tumor a second time. At the time of surgery patients will be randomly assigned in a 1:1 ratio to…
The purpose of this phase I study is to test the safety and tolerability of ruxolitinib at different dose levels in combination with decitabine in patients with accelerated or blast phase Myeloproliferative Neoplasm (MPN), which is a group of diseases of the bone marrow in which excess cells are produced. You may qualify to take part in this research study…
New data from the study of ABT-414 and ABBV-221 in patients with recurrent glioblastoma multiforme (GBM) have shown several examples where it seems evidence of disease progression (cancer that continues to grow and spread) was seen in MRI images, but examination of surgically removed tissue under the microscope did not agree with the MRI.
The goal of this study is to advance our understanding of how the brain processes emotion in adolescents. Adolescents with and without symptoms of anxiety are invited to participate. This study takes place over two days and includes an evaluation and a brain imaging session. Participants will be compensated up to $250. This study does not provide treatment.
The purpose of this study is to evaluate whether study drug NEOD001 will improve survival in subjects with AL amyloidosis and/or increase the interval of time that they can go without requiring hospitalization for problems with their hearts. This study will also evaluate whether NEOD001 improves the function of subjects' organs that have been affected…
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 cells from dying while they are frozen. BCL11A is the area in the…
The purpose of the research study is to see if selinexor has any effects against your cancer and toevaluate the side-effects of selinexor or selinexor in combination with temozolomide (TMZ ). One specific way cancer cells continue to grow is by getting rid of certain proteins called tumor suppressor proteins that would normally cause cancer cells to die.…
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…
The main purpose of the study is to find out whether Triferic, when taken by mouth (orally) with Shohls solution, is safe and effective for the treatment of Iron Refractory Iron-Deficiency Anemia (IRIDA). The study drug, ferric pyrophosphate citrate (FPC), is also called Triferic. It is an iron salt that has been approved by the Food and Drug Administration…
The reason for this study is to find out if the experimental study drug, tesevatinib, is effective as a possible treatment for recurrent glioblastoma. Certain genetic information about your tumor will also be collected, by drawing blood, to help better understand why tesevatinib did or did not work in treating your recurrent glioblastoma. Although…
The purpose of this study is to test whether the new drug, ABT-414, given either alone or with a drug called temozolomide, which is commonly used to treat brain tumors like yours, gives a better outcome than the standard therapy and is safe. The standard therapy can be one of two different regimens, either a drug called lomustine or temozolomide.
Two different studies have been developed to treat people with anaplastic glioma brain tumors. Onetreatment research study requires a tumor that has the 1p/19q co-deletion tumor marker; the other does not. This treatment research study requires that the subject has a certain kind of tumor, one that is missing part of both chromosomes 1 and 19. These missing…