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Displaying 1 - 53 of 53
Condition: Blood Disorders
Investigator: Aaron Viny, MD
Status: Closed
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…
Condition: Blood Disorders
Investigator: Mark Heaney, MD, PhD
Status: Closed
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…
Condition: Blood Disorders
Investigator: Mark Heaney, MD, PhD
Status: Closed
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.
Condition: Blood Disorders / Amyloidosis
Investigator: Suzanne Lentzsch, MD
Status: Closed
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…
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 tolerated dose of aninvestigational new…
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: Blood Disorders / Amyloidosis
Investigator: Suzanne Lentzsch, MD
Status: Closed
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…
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 + INO-9012 into your muscle. You will also receive another…
Condition: Blood Disorders / Amyloidosis
Investigator: Suzanne Lentzsch, MD
Status: Closed
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…
Condition: Blood Disorders / Amyloidosis
Investigator: Divaya Bhutani, MD
Status: Closed
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…
Condition: Blood Disorders
Investigator: Mark Heaney, MD, PhD
Status: Closed
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…
Condition: Blood Disorders
Investigator: Margaret Lee, MD
Status: Closed
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…
Condition: Neurological Disorders / Brain Tumors
Investigator: Andrew Lassman, MD
Status: Closed
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.…
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 cells from dying while they are frozen. BCL11A is the area in the…
Condition: Blood Disorders
Investigator: Joseph Jurcic, MD
Status: Closed
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…
Condition: Blood Disorders
Investigator: Joseph Jurcic, MD
Status: Closed
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).
Condition: Blood Disorders
Investigator: Joseph Jurcic, MD
Status: Closed
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.
Condition: Blood Disorders
Investigator: Joseph Jurcic, MD
Status: Closed
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.
Condition: Blood Disorders
Investigator: Ran Reshef, MD
Status: Closed
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…
Condition: Neurological Disorders / Brain Tumors
Investigator: Andrew Lassman, MD
Status: Closed
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.
Condition: Neurological Disorders / Brain Tumors
Investigator: Andrew Lassman, MD
Status: Closed
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…
Condition: Neurological Disorders / Brain Tumors
Investigator: Fabio Iwamoto, MD
Status: Closed
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…
Condition: Neurological Disorders / Brain Tumors
Investigator: Stergios Zacharoulis, MD
Status: Closed
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…
Condition: Neurological Disorders / Brain Tumors
Investigator: Andrew Lassman, MD
Status: Closed
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…
Condition: Neurological Disorders / Brain Tumors
Investigator: Tony J. Wang, MD
Status: Closed
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…
Condition: Neurological Disorders / Brain Tumors
Investigator: Gurcharanjeet Kaur, MD
Status: Closed
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…
Condition: Blood Disorders / Amyloidosis
Investigator: Divaya Bhutani, MD
Status: Closed
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.
Condition: Blood Disorders / Amyloidosis
Investigator: Divaya Bhutani, MD
Status: Closed
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…
Condition: Neurological Disorders / Brain Tumors
Investigator: Fabio Iwamoto, MD
Status: Closed
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…
Condition: Neurological Disorders / Brain Tumors
Investigator: Teri Kreisl, MD
Status: Closed
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…
Condition: Neurological Disorders / Brain Tumors
Investigator: Mikhail Doubrovin, MD
Status: Closed
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…
Condition: Neurological Disorders / Brain Tumors
Investigator: Mary Welch, MD
Status: Closed
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.…
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 cells, modifies them by CRISPR technology, and infuses them back to the…
Condition: Neurological Disorders / Brain Tumors
Investigator: James Hinkley Garvin Jr, MD
Status: Closed
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…
Condition: Neurological Disorders / Brain Tumors
Investigator: Andrew Lassman, MD
Status: Closed
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…
Condition: Neurological Disorders / Brain Tumors
Investigator: Andrew Lassman, MD
Status: Closed
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.
Condition: Neurological Disorders / Brain Tumors
Investigator: Andrew Lassman, MD
Status: Closed
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…
Condition: Neurological Disorders / Brain Tumors
Investigator: Andrew Lassman, MD
Status: Closed
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…
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 Bevacizumab alone in patients with glioblastoma (GBM). Bevacizumab has…
Condition: Neurological Disorders / Brain Tumors
Investigator: Fabio Iwamoto, MD
Status: Closed
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…
A study for patients with recurrent glioblastoma or anaplastic astrocytoma using study drug Toca 511
Condition: Neurological Disorders / Brain Tumors
Investigator: Fabio Iwamoto, MD
Status: Closed
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…
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…
Condition: Neurological Disorders / Brain Tumors
Investigator: Fabio Iwamoto, MD
Status: Closed
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.
Condition: Neurological Disorders / Brain Tumors
Investigator: Brian Gill, MD
Status: Closed
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…
Condition: Neurological Disorders / Brain Tumors
Investigator: Andrew Lassman, MD
Status: Closed
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…
Condition: Blood Disorders / Amyloidosis
Investigator: Suzanne Lentzsch, MD
Status: Closed
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.
Condition: Neurological Disorders / Brain Tumors
Investigator: Mary Welch, MD
Status: Closed
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.
Condition: Neurological Disorders / Brain Tumors
Investigator: Angela Lignelli-Dipple, MD
Status: Closed
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…
Condition: Blood Disorders / Anemia
Investigator: Katherine Ender, MD
Status: Closed
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…
Condition: Neurological Disorders / Brain Tumors
Investigator: Mary Welch, MD
Status: Closed
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…
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 a portable medical device. It…
Condition: Neurological Disorders / Brain Tumors
Investigator: Yazmin Odia, MD
Status: Closed
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.
Condition: Blood Disorders / Amyloidosis
Investigator: Thomas Brannagan, MD
Status: Closed
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…