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  • Closed (24)

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Displaying 1 - 24 of 24

Tissue Repository of Samples from Patients with Blood Disorders

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…
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Study of EDIT-301 in Patients with Severe Sickle Cell Disease (SCD)

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…
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Adipose Inflammation in Rheumatoid Arthritis - AIR ART (RA Treatment Arm)

Condition: Autoimmune Disorders / Rheumatoid Arthritis
Investigator: Jon Giles, MD
Status: Closed
We are inviting patients with active Rheumatoid Arthritis (RA) to help us better understand how treatment options affect inflammation in soft tissues. With a combination of blood work, a full-body FDG PET-CT scan, questionnaires, and a soft tissue biopsy (from an area around the belly button), we hope to see how RA interacts with these multiple areas of…
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Screening to Improve Survival in AL Amyloidosis

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…
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Study of PF-07209326 in Patients with Sickle Cell Disease

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…
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Study of Brain Activity and Decision-Making in Marijuana Smokers

Condition: Addiction / Marijuana
Investigator: Suzette Evans, PhD
Status: Closed
RESEARCH VOLUNTEERS: Healthy male and female (age 21-60) MARIJUANA SMOKERS needed to study effects of marijuana on brain activity and decision-making. The study involves 2-3 outpatient visits and a 5-day/5-night stay at the NYS Psychiatric Institute and includes Magnetic Resonance Imaging (MRI) scans. Compensation for time could be between $870-$1230 (…
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Effects of Abatacept on Heart Inflammation in Rheumatoid Arthritis (AMiRA)

Condition: Autoimmune Disorders / Rheumatoid Arthritis
Investigator: Laura Geraldino-Pardilla, MD, MPH
Status: Closed
This study aims to understand how two commonly prescribed drugs impact myocardial inflammation in rheumatoid arthritis.
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Study of ION-682884 (SC) in patients with TTR Amyloidosis Polyneuropathy

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…
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A study for patients with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS) using study drug SY-1425

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).
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Study of stem cell transplant effects in patients with sickle cell disease

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…
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A Study to Evaluate the Safety of a Dose of Autologous CRISPR-Cas9 Human Stem Cells in Subjects With Severe Sickle Cell Disease

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…
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A study for patients with Sickle Cell Disease using study drug Rivipansel

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…
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A study for patients with Myelodysplastic Syndromes (MDS) using study drug Venetoclax

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.
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Treatments Against RA and Effect on FDG PET-CT: The TARGET Trial

Condition: Autoimmune Disorders / Rheumatoid Arthritis
Investigator: Joan Bathon, MD
Status: Closed
The study aims to compare the effect two different RA treatments on cardiovascular health in patients who are considering a treatment change. The study is recruiting patients who are only taking methotrexate and are switching treatment due to a lack of improvement. The trial will randomize participants to take a TNF inhibitor (Enbrel or Humira) in addition…
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Study of Patients Receiving a Stem Cell Transplant for Non-Malignant Disease

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.
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A study for children with sickle cell using study drug Hydroxyurea

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…
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A study for patients with myelodysplastic syndrome (MDS) using study drug venetoclax

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.
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Rheumatoid Arthritis Study - RHYTHM Renewal

Condition: Autoimmune Disorders / Rheumatoid Arthritis
Investigator: Joan Bathon, MD
Status: Closed
We are doing a research study to learn more about why people with rheumatoid arthritis (RA) are prone to heart disease. We are recruiting patients diagnosed with Rheumatoid Arthritis without a personal history of heart disease. This study involves one initial full visit as described below, and a brief follow up visit that will take place approximately 2 or…
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HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

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…
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A study for patients with Iron Refractory Iron-Deficiency Anemia (IRIDA) using study drug Triferic

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…
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Study of Experimental Drug Therapy in Patients with Polycythemia Vera (PV)

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…
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A study for patients with myelofibrosis (bone marrow disorder) using study drug INCB050465

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.
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Medication treatment of marijuana use and Attention-Deficit/Hyperactivity disorder

Condition: Addiction / Marijuana
Investigator: Frances Levin, MD
Status: Closed
The study is an outpatient treatment study of the safety and benefit of extended-release mixed amphetamine salt (Adderall-XR) in the treatment of individuals with cannabis use disorder and Attention Deficit/Hyperactivity Disorder (ADHD). The study is a 13-week trial and requires participants to come twice per week. The primary objective of the study is to…
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Study of Drug for Patients with Sickle Cell Disease

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.…
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