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Displaying 1 - 19 of 19

The Molecular Basis of Cardiopulmonary Disorders (Healthy Participants and Children Over 7 Welcome)

Condition: Healthy Volunteers
Investigator: Christine Kim Garcia, MD
Status: Currently Recruiting
The purpose of this study is to identify genetic causes of lung diseases and to develop a research repository so that we can better understand, prevent and treat these conditions. You have been asked to join this study as a healthy control. Even though you do not have lung disease, your sample is valuable to us. For example, you may be related to someone…
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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…
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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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Effects of Growth Hormone and Liraglutide

Condition: Healthy Volunteers
Investigator: Pamela Freda, MD
Status: Currently Recruiting
Growth hormone and insulin-like growth-factor 1 (IGF-1) are two hormones that are very important to human growth and metabolism. In disorders of GH and IGF-1 excess and deficiency, significant growth and metabolic abnormalities can develop. This study investigates the effects of GH on the appetite-stimulating hormone AgRP (…
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Mapping of Auricular Acupoints in Pediatric Patients with Functional Abdominal Pain Disorders

Condition: Healthy Volunteers
Investigator: Melanie Gold, MD
Status: Closed
Auriculotherapy is a type of Eastern medicine that has been used for thousands of years involving the ear. The ear has nerve connections to the whole body, and therefore using non-invasive techniques can be used to treat a wide variety of problems. This study is looking to see if auriculotherapy might be used to treat abdominal pain and find areas on the…
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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…
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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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Pediatric Anxiety Treatment Study

Condition: Healthy Volunteers
Investigator: Amir Levine, MD
Status: Closed
The Columbia University Medical Center/New York State Psychiatric Institute is currently recruiting healthy volunteers with no previous diagnosis of any psychiatric disorders to provide samples and answer questionnaires. If your child is eligible, he or she will be compensated up to $110.
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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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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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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 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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MRI Study Comparing Medications for Children Ages 6-17 with ADHD (Healthy Volunteers)

Condition: Healthy Volunteers
Investigator: Jonathan Posner, MD
Status: Closed
Children with Attention-Deficit/Hyperactivity Disorder (ADHD) are typically treated with two types of medications: stimulants and non-stimulants. These medications work in different ways to treat ADHD symptoms, but we do not fully understand the brain mechanisms underlying the medications' effects. This study will use MRI scanning to look at changes in…
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This study is examining the cognitive control and reward processing in unmedicated adults with and without OCD.

Condition: Healthy Volunteers
Investigator: Helen Simpson, MD, PhD
Status: Closed
Obssesive Compulsive Disorder (OCD) and related disorders characterized by repetitive thoughts and behaviors (e.g., Tourette Syndrome and Eating Disorders) are severe conditions that lead to significant health-related disability. Dysfunction in the brain circuits…
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Exploratory Evaluation of 11C MPC6827 Pharmokinetics (Healthy Volunteers)

Condition: Healthy Volunteers
Investigator: Akiva Mintz, MD, PhD
Status: Closed
Neurodegenerative diseases are conditions where the brain cells break down, causing mental and/or physical impairment. Alzheimer's Disease is a neurodegenerative disorder that affects millions of individuals and causes irreversible memory loss and cognitive impairment. Amyotrophic lateral sclerosis is also and incurable disorder that causes patients…
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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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Family Nurture Intervention: Behavior and Development Effects of Mother-Child Interaction in Preschool-Aged Children

Condition: Healthy Volunteers
Investigator: Martha Welch, MD
Status: Closed
The purpose of this current study is to investigate the efficacy of a group model of Family NurtureIntervention in ameliorating behavioral problems in preschool-aged children. The behavioral, neurobiological and clinical insights gained from this project may eventually lead to better treatment of emotional, behavioral and developmental…
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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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