Seeking participants for a study about how individuals prefer to receive nutrition recommendations to support health management and preferences for visualizing health data. Participants may be invited to complete a 60-minute session to answer questionnaires and evaluate different styles of nutritional recommendations. In addition, they may be invited to…
We are conducting a research study on the effect of high insulin levels on the buildup of excessive liver fat ("fatty liver disease") in people at risk for type 2 diabetes. We are seeking adult volunteers, ages 18-65, who have obesity with a body mass index of 30-45, and have been diagnosed with, or are at risk for, prediabetes and metabolic…
The purpose of this study is to gather data about the safety and effectiveness of a new, investigational device (not FDA approved), the Thor laser atherectomy system (Thor system), for the treatment of peripheral artery disease (PAD). When there is a blockage (a deposit on an artery wall, made of fat or calcium, and/or blood clots) in one or more of your…
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 objective of this study is to learn more about how safe and effective the Bare Temporary Spur Stent System is when used in patients who have blocked arteries in their lower limbs. The selection of subjects will be those with Critical Limb Ischemia (CLI), a form of Peripheral Arterial Disease (PAD) in which an artery (blood vessel) in the lower leg (…
We are conducting an academic study to understand how people solve problems related to health. In the study, participants will be presented with a hypothetical problem related to diabetes self-management and asked to use an online health application to explore information and propose a solution. We invite all participants between 18 and 65 years old, who…
The purpose of this research study is to see whether the presence or absence of certain genes is associated with the development of AL amyloidosis in subjects 60 years of age or older with the blood disorders smoldering multiple myeloma (SMM) and monoclonal gammopathy of undetermined significance (MGUS). Each person's disease has different genetic…
The purpose of this study is to learn about whether the study drug (PF-07209326) is safe and effective in people with sickle cell disease and learn about what the right dose is. We will look at drug levels in the blood, see how fast drug levels rise and fall, and what effects the drug has, all of which are known as the pharmacokinetics (PK) and…
The objective of the LIFE-BTK RCT is to evaluate the safety and efficacy of the ESPRIT BTK device (which is a bioresorbable polymeric scaffold with the everolimus drug and a bioresorbable polymeric coating mounted on a balloon dilatation catheter) compared to Percutaneous Transluminal Angioplasty (PTA), which is is a procedure that can open up a blocked…
A research team at Columbia University Irving Medical Center is collecting information for a registry studying how safely and effectively the ClotTriever device works in treating patients at least 18 years old with Deep Vein Thrombosis (DVT), or blood clots in the vessels of the leg. The sponsor of this study, Inari Medical, Inc., is paying the research…
All vascular repair and reconstruction surgery requires patching material. PhotoFix, made of cow heart membrane, is one of the FDA-approved patches on the market for use in vascular repair and reconstruction surgery. Other vascular patches on the market are also available and used by other surgeons for procedures like the one you need. This trial will study…
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…
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 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…
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 the study drugs are absorbed in your body at different times), and efficacy (how well the study drugs work) of venetoclax in combination with azacitidine compared to azacitidine alone in subjects with previously untreated higher-risk MDS.
The purpose of this 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.
We will enroll and randomly assign 30 sedentary, healthy overweight men and women to two groups. Participants will either start by consuming peanuts for 4 weeks, and then go on to exercise at high intensity intervals (HIIT) for 4 weeks, or the reverse order. We will test and compare the effect of peanuts and exercise on inflammation and heart rate…
The purpose of this research is to learn how to help parent youth pairs work together to help youth with sickle cell disease take hydroxyurea every day as a daily habit. This study is for children and teens ages 10-18 with Sickle Cell Anemia taking hydroxyurea, and their parents (or guardians). Many children and teens do not use hydroxyurea every day. Daily…
The purpose of this 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.
Are you scheduled for an interventional vascular procedure, such as a Trans-catheter Aortic Valve Replacement (TAVR), Endovascular Aneurysm Repair (EVAR), Thoracic Endovascular Aortic Repair (TEVAR), or Balloon Aortic Valvuloplasty (BAV)? If yes, you may be eligible for this trial. This research trial studies the safety and efficacy of an Investigational…
HELIOS-A is a global Phase 3 randomized, open-label study designed to evaluate the safety and efficacy of ALN-TTRSC02 in adult patients with hATTR amyloidosis experiencing neurologic symptoms brought on by the disease. The study will also evaluate any changes in quality of life experienced by study participants. ALN-TTRSC02 utilizes the mechanism of RNA…
The purpose of this study is to evaluate the safety and tolerability of study drug CCX168 when it is given to patients who are receiving the standard therapy for a certain type of vasculitis called ANCA-associated vasculitis (AAV). The study will also assess the effectiveness of the study drug, CCX168, in combination with the standard treatment of AAV. This…
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…
Has your doctor determined that you have Type B Aortic Dissection and you would like to learn more about an alternative to open surgery? Type B Aortic Dissection patients are being invited to take part in a research study of an investigational device. Bolton Medical, Inc., the company sponsoring this clinical trial, is studying an investigational device…
The purpose of this study is to collect information on whether idasanutlin is effective in treating polycythemia vera (a slow-growing blood cancer in which your bone marrow makes too many red blood cells) and what effects, good or bad, idasanutlin has on you. Idasanutlin is an experimental drug, which means Health Authorities (such as the US FDA) have not…
The purpose of this study is see if an experimental gene product, the Hepatocyte Growth Factor (HGF) Plasmid (AMG0001) which will be injected into the affected leg's muscles is effective in treating the blockages in your leg without causing adverse effects. Affected leg means the leg that has the severest disease relating to the blockage or narrowing…
We are currently looking for overweight (body mass index 25 kg/m2) men and women, age 18-65 y, to participate in a sleep study. To participate you must have been diagnosed with Obstructive Sleep Apnea within the past year, and have been prescribed continuous positive airway pressure (CPAP) but not have not yet started treatment. This is a 2-phase,…
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.
Has your doctor determined that you have Peripheral Arterial Disease (PAD), and that an artery in your upper leg is blocked as a result? Is blood flow to your upper leg(s) blocked?This study will test the safety and effectiveness of the PQ Bypass System (two catheters called the DETOUR Crossing Device and the DETOUR Snare) in improving blood flow to your…
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.…