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Displaying 1 - 94 of 94
Condition: Cancer / Multiple Myeloma
Investigator: Rajshekhar Chakraborty, MD
Status: Currently Recruiting
The purpose of the study is to determine the proportion of patients with lowest minimal residual disease (MRD) response obtainable after receiving 6 cycles of study treatment. Minimal residual disease is multiple myeloma cells below the level of 1 cancer cell out of 100,000 in the bone marrow. For patients who are MRD positive at the end of 6 cycles of…
Study of Daratumumab in Addition to Chemotherapy for Patients with Smoldering Multiple Myeloma (SMM)
Condition: Cancer / Multiple Myeloma
Investigator: Rajshekhar Chakraborty, MD
Status: Currently Recruiting
The purpose of this study is to determine whether patients with high-risk smoldering multiple myeloma when treated with daratumumab in addition to lenalidomide and dexamethasone live longer when compared to patients with high-risk smoldering multiple myeloma patients treated with lenalidomide and dexamethasone. We would also like to know whether the period…
Condition: Cancer / Multiple Myeloma
Investigator: Divaya Bhutani, MD
Status: Currently Recruiting
The purpose of this study is to see if Isatuximab can alter the immune system favorably in patients with multiple myeloma or lymphoma upon recovery from the autologous stem cell transplantation. This study will have two arms. On one arm (control arm), Participants will receive standard transplant procedures and on the other arm (experimental arm),…
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 / Multiple Myeloma
Investigator: Rajshekhar Chakraborty, MD
Status: Currently Recruiting
This study is looking at people who have recently been newly diagnosed with a type of cancer called multiple myeloma (NDMM). The purpose of this study is to learn about the safety, tolerability (how your body reacts to the study drug), and effectiveness (tumor shrinkage) of linvoseltamab (called study drug) in participants with NDMM as a first step in…
Condition: Cancer / Multiple Myeloma
Investigator: Divaya Bhutani, MD
Status: Currently Recruiting
The purpose of this study is to test whether BMS-986393 in combination with other anti-cancer drugs is safe and effective at treating people with multiple myeloma (MM) that has come back after previous treatment (relapsed) or is not responding to current treatment (refractory). The researchers think that the combinations of alnuctamab, mezigdomide, or…
Condition: Cancer / Multiple Myeloma
Investigator: Rajshekhar Chakraborty, MD
Status: Currently Recruiting
This study is the first time REGN5458 will be combined with other cancer therapies. The main goal is to understand if REGN5458 can be given safely with other cancer study treatments, and if so, what dose of REGN5458 should be used for each combination. The investigational drug, REGN5458, is not approved by the Food and Drug Administration (FDA).
Condition: Cancer / Multiple Myeloma
Investigator: Divaya Bhutani, MD
Status: Currently Recruiting
The purpose of this study is to see how teclistamab by itself compares to study treatment combinations that are commonly used to treat multiple myeloma. These study treatment combinations are either pomalidomide, bortezomib and dexamethasone (called PVd) or carfilzomib and dexamethasone (called Kd). The patients will be randomly assigned to on of the study…
Condition: Cancer / Multiple Myeloma
Investigator: Rajshekhar Chakraborty, MD
Status: Currently Recruiting
The purpose of this study is assess the safety, safest dose, and effectiveness of venetoclax in combination with dexamethasone in subjects with t(11;14) positive relapsed (comes back) or refractory (did not get better) light chain amyloidosis. Furthermore, the purpose of phase 2 part of this study is to compare the effectiveness of venetoclax and…
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: Cancer / Multiple Myeloma
Investigator: Ran Reshef, MD
Status: Closed
The purpose of this study is to determine if the investigational therapy called bb2121 is effective in treating multiple myeloma. bb2121 is a type of therapy known as chimeric antigen receptor (CAR) T-cell therapy. T cells are a kind of white blood cell which form part of your body's immune system. Your T cells will be separated from the rest of the…
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: Cancer / Multiple Myeloma
Investigator: Ran Reshef, MD
Status: Closed
The purpose of this study is to see if Bortezomib, Lenalidomide and Dexamethasone (VRd) followed by cilta-cel is safe and useful for treating patients with newly diagnosed Multiple Myeloma, as compared with the use of VRd followed by Lenalidomide and Dexamethasone (Rd) therapy. Cilta-cel is a CAR-T cell therapy. In this type of treatment, your white blood…
Condition: Cancer / Multiple Myeloma
Investigator: Divaya Bhutani, MD
Status: Closed
This study will look at the safety of a drug (REGN5458) in patients with multiple myeloma. It will also try to find out what the best dose of REGN5458 is and look for any signs that REGN5458 can help treat your cancer. REGN5458 is investigational, which means it has not been approved by the US Food and Drug Administration (FDA) for any condition.
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 / Multiple Myeloma
Investigator: Ran Reshef, MD
Status: Closed
The purpose of this study is to determine if the investigational therapy, bb2121, in combination with other treatments, is safe and effective in treating relapsed and/or refractory multiple myeloma.
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 / Multiple Myeloma
Investigator: Rajshekhar Chakraborty, MD
Status: Closed
You are being asked to participate in this study because you have multiple myeloma that has either returned after your most recent treatment regimen or has not responded to your most recent treatment regimen. Multiple myeloma is a cancer that forms in a type of white blood cell called a plasma cell. Healthy plasma cells help you fight infections by making…
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 / Multiple Myeloma
Investigator: Suzanne Lentzsch, MD
Status: Closed
You are being asked to take part in this study because you have Multiple Myeloma. This study is being done to learn more about AMG 701 in people with Multiple Myeloma. It will see if AMG 701 is safe and tolerable and whether it causes any side effects. This study will also look at what doses of AMG 701 are safe for people to take and what the effect on…
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…
Condition: Cancer / Multiple Myeloma
Investigator: Suzanne Lentzsch, MD
Status: Closed
This is a research study. We invite you to participate in this research study because you have been diagnosed with Multiple Myeloma (MM). Your previous treatment(s) has/have not been successful and your cancer is progressing. The purpose of this research study is to see if selinexor (also known as KPT-330) has any effects on your cancer. Selinexor is…
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 / Multiple Myeloma
Investigator: Ran Reshef, MD
Status: Closed
The purpose of this research study is to obtain information on the safety and effectiveness of PBCAR269A to treat certain types of cancers. PBCAR269A is a new investigational treatment for certain types of cancer of the blood, such as multiple myeloma. It is made from a type of blood cells known as T cells. The T cells in PBCAR269A came from people who have…
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 / 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 / Multiple Myeloma
Investigator: Suzanne Lentzsch, MD
Status: Closed
We are doing this study to learn if the MMprofiler test can help your doctor in making decisions about your treatment. The MMprofiler genomic test will look at a large number of normal genes. The test will predict risk of your multiple myeloma (MM) by assessing whether these genes are turned on or off in your cancer cells. The purpose of this research is to…
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 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: Cancer / Multiple Myeloma
Investigator: Suzanne Lentzsch, MD
Status: Closed
This is research study for patients who have been newly diagnosed with Multiple Myeloma (MM). Their previous treatments have not been successful and their cancer is progressing. The purpose of this research study is to see if the study drug selinexor (KPT-330) has any effects against their cancer. This study combines the use of two drugs, selinexor (KPT-…
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 / Multiple Myeloma
Investigator: Divaya Bhutani, MD
Status: Closed
The main goals of this study are to evaluate the safety of the combination of the study drug radium-223 dichloride plus bortezomib and dexamethasone and to determine the optimal dose of radium-223 dichloride to be used in combination with bortezomib and dexamethasone. Radium-223 dichloride has been approved in some countries for treatment of patients with…
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 / 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…
Condition: Cancer / Multiple Myeloma
Investigator: Divaya Bhutani, MD
Status: Closed
This study will look to obtain information on the best dose of the research drug, TAK 079, when administered in combination with other approved multiple myeloma treatments for patients with newly diagnosed multiple myeloma. If you participate you will receive either Arm A (TAK-079 in combination with lenalidomide and dexamethasone) or Arm B (TAK-079 in…
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: Cancer / Multiple Myeloma
Investigator: Suzanne Lentzsch, MD
Status: Closed
The purpose of this research study is to explore the drug combination, lenalidomide, bortezomib, and dexamethasone alone or when combined with autologous stem cell transplantation to see what side effects it may have and how well it works for treatment of newly diagnosed multiple myeloma.
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: 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: 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: Cancer / Multiple Myeloma
Investigator: Divaya Bhutani, MD
Status: Closed
The purpose of this study is to find out whether treatment with daratumumab and lenalidomide will make your Multiple Myeloma better. There are 2 treatment groups in this study. You will either receive lenalidomide alone or both lenalidomide and daratumumab along with medications to take before and after the daratumumab injection.
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: 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 / Multiple Myeloma
Investigator: Suzanne Lentzsch, MD
Status: Closed
The purpose of this study is to look at what happens (both good and bad) when daratumumab is given with two other drugs called VELCADE and dexamethasone compared to just giving those two drugs, VELCADE and dexamethasone, alone. VELCADE is also known by the name bortezomib. Giving VELCADE and dexamethasone together is standard treatment for advanced multiple…
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 / Multiple Myeloma
Investigator: Siyang Leng, MD
Status: Closed
The purpose of this study is to test the safety, tolerability and anti-tumor activity of the research study drug, pembrolizumab (MK-3475) with or without pomalidomide and low dose dexamethasone in subjects with refractory or relapsed and refractory Multiple Myeloma (rrMM) measured by the rates of response to treatment, disease progression (disease getting…
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: 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 / 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 / Multiple Myeloma
Investigator: Suzanne Lentzsch, MD
Status: Closed
This is a study of a study drug, Carfilzomib in combination with bendamustine and dexamethasone for subjects with newly diagnosed multiple myeloma. The purpose of this study is to find the highest tolerable dose of carfilzomib with the combination of bendamustine and dexamethasone. The response rate and the safety of this drug combination will also be…
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: 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: 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 / 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 / Multiple Myeloma
Investigator: Suzanne Lentzsch, MD
Status: Closed
This research study is being done to compare the combination of lenalidomide and dexamethasone followed by autologous peripheral blood stem cell transplant (PBSCT) and lenalidomide and dexamethasone without PBSCT in subjects with untreated multiple myeloma. This comparison will include how many subjects respond to each study treatment combination, how long…
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: Cancer / Multiple Myeloma
Investigator: Divaya Bhutani, MD
Status: Closed
The purpose of this study is to compare the effectiveness of venetoclax and dexamethasone versus pomalidomide and dexamethasone in subjects with t(11;14) positive relapsed (comes back) or refractory (did not get better) multiple myeloma.
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 / 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 / Multiple Myeloma
Investigator: Divaya Bhutani, MD
Status: Closed
The purpose of this study is to find out how well Daratumumab works when it is given along with two other drugs called Lenalidomide and Dexamethasone compared to the drug combination of Lenalidomide and Dexamethasone to treat multiple myeloma.
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: Cancer / Multiple Myeloma
Investigator: Suzanne Lentzsch, MD
Status: Closed
This is a research study for subjects who have been diagnosed with multiple myeloma, a cancer of plasmacells (a type of immune cell that produce antibodies) in the bone marrow, that has returned (relapsed) or has not responded (refractory) to your most recent treatment regimen.The subject's disease has also progressed after prior treatment with…
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: Cancer / Multiple Myeloma
Investigator: Divaya Bhutani, MD
Status: Closed
Belantamab Mafodotin is a new therapy for patients with Multiple Myeloma. The drug works by combining chemotherapy and immunotherapy approach to treat Multiple Myeloma. The drug is a combination of an antibody against a target on plasma cells called BCMA and a chemotherapeutic agent called MMAF. The drug has shown activity in patients with late relapses of…
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: 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: Cancer / Multiple Myeloma
Investigator: Yvonne Saenger, MD
Status: Closed
The purpose of this study is to test two imaging techniques, one called whole body diffusion weighted magnetic resonance imaging (WB-DWI MRI), and another called F-18 3-deoxy-3-fluorothymidine PET (F-18-FLT PET). The goal is to see whether these imaging techniques would allow the study doctors to see changes in the size of a tumor earlier for patients with…
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