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Displaying 1 - 63 of 63
Condition: Blood Disorders / Amyloidosis
Investigator: Rajshekhar Chakraborty, MD
Status: Currently Recruiting
The purpose of this study is to see how safe and effective is the study drug, ABBV-383. ABBV-383 works by binding to a specific protein called B-cell maturation antigen, which is found on the surface of cells affected by amyloidosis. By engaging T-cells, a type of white blood cell, ABBV-383 helps activate the immune response to target and remove the…
Condition: Cancer / Sarcoma
Investigator: Brian Henick, MD
Status: Closed
This research study mainly aims to test the safety and tolerability of AGEN1181 and balstilimab and to determine the most appropriate dose of the study drug AGEN1181 alone and in combination with balstilimab. AGEN1181 targets cells that are a part of the body's immune system to block a protein called cytotoxic T-lymphocyte-associated protein-4 (CTLA-4…
Condition: Blood Disorders / Amyloidosis
Investigator: Rajshekhar Chakraborty, MD
Status: Closed
The purpose of this study is to evaluate whether birtamimab plus standard of care will improve survival in subjects with Mayo Stage IV AL amyloidosis. Birtamimab has not yet been approved by the Food and Drug Administration (FDA). During the first phase of the study, the purpose of this study is to evaluate whether birtamimab plus standard of care will…
Condition: Cancer / Sarcoma
Investigator: Prabhjot Mundi, MD
Status: Closed
You are being asked to participate in this study because you have been diagnosed with a progressive desmoid tumor. The purpose of this study is to see if the study drug, called AL102 (referred to from now on as the study drug), is safe and effective in people with progressive desmoid tumors. The study will also look at how your body responds to the study…
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 non-hematologic control for each…
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: 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: Cancer / Sarcoma
Investigator: Shaheer Khan, DO
Status: Closed
The purpose of this study is to test the good and bad effects of the drugs called belinostat and SGI-110 (guadecitabine). These drugs could shrink your cancer, but they could also cause side effects. The study doctors hope to learn if the study drugs will shrink the cancer more often than observed with other standard treatment options. We dont know if…
Condition: Cancer / Sarcoma
Investigator: Prabhjot Mundi, MD
Status: Closed
The purpose of this study is to evaluate the effectiveness and safety of an investigational drug, unesbulin. This investigational drug is being studied in unresectable or metastatic, relapsed, or refractory leiomyosarcoma along with Dacarbazine. Dacarbazine is a drug that is FDA-approved for the treatment of other types of cancer and recommended by the…
Condition: Cancer / Sarcoma
Investigator: Shaheer Khan, DO
Status: Closed
The study drug, CFT8634, is an investigational therapy that has been shown in animal studies to help break down the BRD9 protein, an important protein in the body that helps SMARCB-null tumors, such as your own, to grow. CFT8634 is able to do so by using the body's own protein-recycling equipment. (In short, your cells are capable of breaking down…
Condition: Cancer / Sarcoma
Investigator: Gulam Manji, MD, PhD
Status: Closed
The purpose of this study is to determine if treatment with PLX3397 and Sirolimus will be tolerated and result in shrinking of the cancer or stopping the cancer from growing. In the phase I portion, the maximum tolerated dose of the study drug will be determined. In the phase II portion, progression-free survival will be assessed at the dose level found in…
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: Cancer / Sarcoma
Investigator: Prabhjot Mundi, MD
Status: Closed
The purpose of this research study is to determine the effectiveness and safety of envafolimab, an experimental drug that is not approved for use, when given alone or in combination with ipilimumab to stimulate your immune system to attack cancer cells. Envafolimab is an experimental (investigational) cancer drug that overrides the PD-1/PD-L1 checkpoint,…
Condition: Cancer / Sarcoma
Investigator: Prabhjot Mundi, MD
Status: Closed
The experimental drug called INBRX 109 has been developed as a treatment for unresectable or metastatic conventional chondrosarcoma. The purpose of this study is to learn about the effects of the study drug, INBRX-109 on you, your immune system, and your cancer. This study is being done to understand if INBRX-109 is effective against your chondrosarcoma and…
Condition: Blood Disorders
Investigator: Suzanne Lentzsch, MD
Status: Closed
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…
Condition: Cancer / Sarcoma
Investigator: Benjamin Izar, MD
Status: Closed
The purpose of this study is to evaluate the effectiveness of a new investigational drug, ATX-101, for the treatment of dedifferentiated liposarcoma and leiomyosarcoma. ATX-101 is an intravenous (IV) drug which blocks the interaction of a protein called PCNA with a number of stress response proteins. These interactions are thought to be important for cancer…
Condition: Blood Disorders / Sickle Cell Disease
Investigator: Andrew Eisenberger, MD
Status: Closed
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…
Condition: Cancer / Sarcoma
Investigator: Prabhjot Mundi, MD
Status: Closed
The purpose of this study is to learn about the safety, tolerability, and effectiveness of an investigational drug called milademetan as compared to the use of trabectedin. In this study, subjects will be randomly assigned (like flipping a coin) to receive one of two study drugs: milademetan or trabectedin. This is an open-label study, meaning you will know…
Condition: Cancer / Sarcoma
Investigator: Mark Stein, MD
Status: Closed
The purpose of this study is to: 1. Compare treatment with chemotherapy (paclitaxel) alone to using a combination of nivolumab plus chemotherapy (paclitaxel) treatment in patients with angiosarcoma who have not been treated with paclitaxel chemotherapy alone. 2. Evaluate the effect of nivolumab in combination with cabozantinib on angiosarcoma (cancer) in…
Condition: Cancer / Sarcoma
Investigator: Gary Schwartz, MD
Status: Closed
The purpose of this study is to test the study drug, BI 907828 for safety, effectiveness, and side effects when compared to receiving the standard treatment, doxorubicin, in subjects with dedifferentiated liposarcoma (DDLPS). BI 907828 has proven to minimize a critical protein interaction between MDM2 and p53, two proteins in your body, which when their…
A Study of PTC596 in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)
Condition: Cancer / Sarcoma
Investigator: Matthew Ingham, MD
Status: Closed
The purpose of this study is to evaluate the effectiveness of a new investigational drug, PTC596, used in combination with dacarbazine, for the treatment of locally recurrent, unresectable, or metastatic relapsed/refractory leiomyosarcoma, a type of soft tissue sarcoma. The purpose of this study is to evaluate whether adding PTC596 to dacarbazine is safe…
Condition: Cancer / Sarcoma
Investigator: Gary Schwartz, MD
Status: Closed
The purpose of this study is to find out if an investigational drug, called ADP-A2M4, is safe and how well it works in attacking Synovial Sarcoma or Myxoid/Round Cell Liposarcoma (MRCLS) cancer cells. The study involves taking certain types of immune cells from the blood, called T cells, and changing them in a laboratory before putting them back into the…
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: Cancer / Sarcoma
Investigator: Gary Schwartz, MD
Status: Closed
The purpose of this study is to test the safety of a study drug called AMG 232 given with radiation in patients with soft tissue sarcoma (STS) prior to surgery. The AMG 232 is investigational. It has been tested in other cancers but not yet in sarcoma. AMG 232 has not been tested using the dosing schedule in this study and it has not been tested with…
Condition: Cancer / Sarcoma
Investigator: Gary Schwartz, MD
Status: Closed
You are being asked to participate in this study because you have been diagnosed with a progressive desmoid tumor. The purpose of this study is to see if the study drug, called AL102 (referred to from now on as the study drug), is safe and effective in people with progressive desmoid tumors. The study will also look at how your body responds to the study…
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 (…
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
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: 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: Cancer / Sarcoma
Investigator: Matthew Ingham, MD
Status: Closed
The purpose of this study is to evaluate the effectiveness of preoperative radiation therapy used in combination with the immunostimulatory agent talimogene laherparepvec (T-VEC) to improve outcomes for patients with resectable soft tissue sarcomas, including soft tissue sarcoma (liposarcoma (excluding myxoid liposarcoma), leiomyosarcoma and…
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 (such as the US FDA) have not…
Condition: Blood Disorders / Sickle Cell Disease
Investigator: Nancy Green, MD
Status: Closed
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…
Condition: Cancer / Sarcoma
Investigator: Gary Schwartz, MD
Status: Closed
The purpose of this research study is to learn more about adding the drug ADI-PEG 20 to a standard treatment regimen called gem-tax (the chemotherapy drugs gemcitabine and docetaxel) for subjects with bone and soft tissue sarcoma, and how the addition of ADI-PEG 20 affects the tumors response to treatment and the side effects you experience.
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
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: Joseph Jurcic, MD
Status: Closed
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.
Condition: Cancer / Sarcoma
Investigator: Matthew Ingham, MD
Status: Closed
The purpose of this study is to evaluate the safety and effectiveness of a new combination treatment involving two drugs: selinexor and ixazomib, for the treatment of dedifferentiated liposarcoma, malignant peripheral nerve sheath tumor, alveolar soft part sarcoma and Ewing sarcoma. The primary objective of the study is to determine the maximum tolerated…
Condition: Blood Disorders
Investigator: David Diuguid, MD
Status: Closed
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.…
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: Cancer / Sarcoma
Investigator: Gary Schwartz, MD
Status: Closed
The purpose of this study is to determine the safety and effectiveness of the study drug nirogacestat for the treatment of desmoid tumors/aggressive fibromatosis (DT/AF). One group will receive the study medication, the other group will receive a placebo. After the initial phase of the study, participants may elect to participate in another phase of the…
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
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: Mark Heaney, MD, PhD
Status: Closed
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…
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: Cancer / Sarcoma
Investigator: Matthew Ingham, MD
Status: Closed
The purpose of this study is to learn about the effects, safety, and proper dosage of tazemetostat in combination with doxorubicin for the treatment of soft tissue sarcoma. Doxorubicin is a common therapy for soft tissue sarcoma, but is not FDA approved in combination with tazemetostat. This study treatment combination may suppress or prevent tumor growth…
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: 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: 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; which are serious bone…
Condition: Cancer / Sarcoma
Investigator: Matthew Ingham, MD
Status: Closed
The researchers hope to study the effectiveness of an investigational drug called AL3818 in people who have metastatic (meaning their disease has spread) or advanced ), specifically alveolar soft partsarcoma (ASPS), leiomyosarcoma (LMS) and synovial sarcoma (SS). The main purpose of the study isto learn the percentage of patients that respond to treatment…
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: 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…
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. Genes, which we inherit from our parents, are pieces of information which provide the…
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: 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 cell disease. Patients will receive busulfan…
Condition: Cancer / Sarcoma
Investigator: Brian Henick, MD
Status: Closed
The purpose of this study is to establish the safety profile of the study drug HuMax-AXL-ADC in a mixed population of patients with specified solid tumors. HuMax-AXL-ADC binds to a specific antigen called Axl, which is expressed on the tumor cells in the cancer types treated in this trial. When the trial drug binds to the antigen on the tumor cell, the…
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
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 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…
Condition: Blood Disorders / Amyloidosis
Investigator: Thomas Brannagan, MD
Status: Closed
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…
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 with these three drugs compared to…
Condition: Cancer / Sarcoma
Investigator: Gary Schwartz, MD
Status: Closed
The main purpose of this research study is to test different doses of INCB059872 and see which doses are safe and tolerable in participants that have Ewing sarcoma. This study will research the effect that INCB059872 has on your cancer. Researchers will study the way INCB059872 come into and leave your body over time (pharmacokinetics). INCB059872 is a…
Condition: Cancer / Sarcoma
Investigator: Gary Schwartz, MD
Status: Closed
The purpose of this study is to determine if the study drug toripalimab is safe, to determine future doses to give to patients with cancer, and to measure the amount of toripalimab in the blood. Up to approximately 240 subjects will be enrolled in Part B. The dose given in Part B will be 240 mg. The dose was chosen based upon safety data obtained from…
Condition: Blood Disorders / Sickle Cell Disease
Investigator: Markus Mapara, MD
Status: Closed
This study is being done to learn more about the safety and effects of CTX001 (the Study Product) in patients with severe Sickle Cell Disease (SCD). The Study Product is considered investigational; investigational means the Study Product is not approved by the United States Food and Drug Administration (FDA). The goal of this study is to see if a single…
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