This study aims to collect information about the symptoms and treatments of patients who have severely clogged arteries and are treated with commercially available Boston Scientific Corporation drug-eluting devices marketed for the treatment of lesions located in the peripheral vasculature. Participation in the study is expected to last for up to two (2)…
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 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…
Capnography-Assisted, Learned, Monitored (CALM) Breathing is a mind-body intervention for COPD that uses breathing exercises and biofeedback with the goal of reducing shortness of breath, anxiety symptoms and improving overall quality of life. The intervention is a 4-week (1 hour, twice weekly) program taking place in-person at New York Presbyterian…
This is a prospective, multi-center, observational, post-market study that will evaluate the safety and procedural success of performing trans-radial access for treatment of atherosclerotic lesions in the lower extremities. This research study will collect outcome data of patients undergoing percutaneous endovascular treatment of Peripheral Artery Disease (…
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.
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 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 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.…
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 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.
The purpose of this study is to deterimine if the Drug-Coated Chocolate Touch Balloon Catheters is safe and effective compared to a drug-coated balloon that is already approved in patients with a blockage in the blood vessels of their leg.
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…
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 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…
This study is for patients who suffer from a condition where one of the blood vessels that deliver blood to your leg has a significant narrowing. This is called peripheral artery disease (PAD). The narrowing may be suitable for a treatment technique called balloon angioplasty. One of the therapies that has been used to treat PAD is balloon angioplasty. It…
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 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).
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…
This study is to collect information about the symptoms and treatments of patient who have severely clogged arteries and to learn about their outcomes at one year. If you choose to be part of this research, we will ask you to be involved in five interviews with study staff. Each interview will take about 30 minutes of your time and we will contact you…
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…
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 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.
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 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…
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…
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…
This study is to test if a combination drug/device therapy using TEMSIROLIMUS (Temsirolimus Injection), with or without the generic steroid DEXAMETHASONE (Dexamethasone Sodium Phosphate Injection), can be delivered safely into the tissue around the blood vessel wall at the time of an angioplasty ( which is a a procedure to open blocked arteries and restore…
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 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…
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 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…