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Displaying 1 - 75 of 75

Cognitive Training

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Davangere Devanand, MD
Status: Currently Recruiting
People with mild cognitive impairment (MCI) are at increased risk of dementia, including Alzheimer's disease. Cognitive training may be a useful treatment strategy in MCI. The COGIT-2 study, which is funded by the National Institutes of Health, will compare the effects on cognition and function and brain MRI changes of high dose crosswords (4 puzzles…
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Immune Responses in Alzheimer's Disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Julian Agin-Liebes, MD
Status: Currently Recruiting
We are looking for participants for a study on the role of immune response in Parkinson's disease (PD) and Alzheimer's disease (AD). We are inviting people WITH PD, WITH AD, WITH amnestic Mild Cognitive Impairment, or WITHOUT PD or AD to come and participate. We will be doing several questionnaires, a motor exam, and a short cognitive assessment,…
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Alzheimer's Variant Imaging Study (ADVISe)

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Patrick Lao, PhD
Status: Currently Recruiting
The purpose of this study is to determine how inflammation is related to changes in the brain that occur during the progression of Alzheimer's disease. If you are eligible for this study, we will ask you to complete 3-4 study visits over the course of about 5 months. Participation includes memory testing, a blood draw, MRI, PET scans, and a lumbar…
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Early Age-Related Hearing Loss Investigation (EARHLI)

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Justin Golub, MD, MS
Status: Currently Recruiting
Early Age-Related Hearing Loss Investigation (EARHLI) is a study to obtain information on how a hearing aid-based intervention can prevent cognitive decline in those at risk for Alzheimer's Disease and Alzheimer's Disease Related Dementias (AD/ADRD). Age-related hearing loss (ARHL) is the third most common chronic condition in older adults. The…
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A Study to Identify Genes Underlying Alzheimer's Disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Richard Mayeux, MD, MSc
Status: Currently Recruiting
The goal of this study is to focus on the genetic influences on Alzheimer's disease (AD) risk. We are looking for families and/or individuals (affected or unaffected) of any ethnic background with a family history of Alzheimer's disease and willing to participate.
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Study of ABBV-383 in Patients with Amyloidosis

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…
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The Dawn Study - Genetics of Alzheimer's Disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Giuseppe Tosto, MD PhD
Status: Currently Recruiting
This study aims to find genes that cause Alzheimer's Disease in African Americans and Hispanics/Latinos. Participants are eligible if African American or Hispanic/Latino and 60 years of age or older. Study participation would entail completing a memory assessment, having their blood drawn, and a brief physical exam with a questionnaire. Compensation of…
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Understanding the Causes of Apathy in Alzheimer's Disease (Male subjects)

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Catherine Clelland, PhD
Status: Currently Recruiting
Apathy is a very common symptom experienced by people with Alzheimer's disease, and includes a lack of motivation and loss of interest in many activities. Patients and their caregivers often find apathy to be particularly distressing. This study aims to obtain a greater understanding of the causes of apathy in Alzheimer's disease. We are currently…
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Study of Birtamimab in Patients with Mayo Stage IV (AL) Amyloidosis

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…
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Genetic Variants of Microglial Activation in AD

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Philip De Jager, PhD
Status: Closed
The purpose of this study is to determine whether different genes are related to the amount of immune cell activity that occurs in the brain in patients with Alzheimer's disease. Participation in this study involves memory testing, a blood draw, MRI, and three PET scans. The second PET scan will involve putting a catheter in your wrist to draw blood…
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Understanding the Causes of Apathy in Alzheimer's Disease (Female subjects)

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Catherine Clelland, PhD
Status: Closed
Apathy is a very common symptom experienced by people with Alzheimer's disease, and includes a lack of motivation and loss of interest in many activities. Patients and their caregivers often find apathy to be particularly distressing. This study aims to obtain a greater understanding of the causes of apathy in Alzheimer's disease. We are currently…
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Trial Studying Metformin vs. Placebo in People with Mild Cognitive Impairment (55-90 Years of Age without Diabetes)

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Jose Luchsinger, MD
Status: Closed
MAP is a research study looking at whether metformin can help prevent memory decline for people with mild memory concerns. You may be able to join if: you are 55-90 years old, you or your loved ones have noticed some changes in your memory, and you are not already being treated for diabetes or dementia. Potential benefits: You will receive some of your…
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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 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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Study of BAN2401 in Subjects with Preclinical Alzheimer's Disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Karen Bell, MD
Status: Closed
We are seeking men & women, ages 55 to 80, who have preclinical Alzheimer's disease for a 216-week treatment, multi-center, double-blind, placebo-controlled, parallel-treatment arm study to determine whether the BAN2401 study drug will reduce the risk of developing Alzheimer's disease. The study participant will receive MRI, and PET scans and…
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Longitudinal Early-Onset Alzheimers Disease Study (LEADS) (Alzheimer's Subjects)

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
The Longitudinal Early-onset Alzheimer's Disease Study (LEADS) is a natural history, non-treatment study designed to look at disease progression in individuals with early onset cognitive impairment . Clinical/cognitive, imaging, biomarker, and genetic characteristics will be assessed across two cohorts: early onset Alzheimer's Disease (EOAD)…
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New IDEAS: Brain Imaging Study for Memory Loss

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
This is a research study for persons with memory problems that examines the utility of performing positron emission tomography (PET) scans for the brain amyloid protein found in Alzheimer's disease.
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Dominantly Inherited Alzheimer's Disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: James Noble, MD, MS
Status: Closed
Funded by a multi-year research grant from the National Institute on Aging, the DIAN Observational Study enrolls family members who have parents with a mutated gene known to cause dominantly inherited Alzheimer's (DIAD). Through the study, researchers follow individuals and family members to see if insights gained could lead to better understanding of…
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A Study to Evaluate the Effectiveness and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis

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.
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Study of AL002 for Patients with Early Alzheimers Disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
This is a phase 2 study evaluating a new drug, AL002, in persons with early Alzheimer's Disease. Participants will be randomized to receive either AL002 or placebo, intravenously every four weeks, for a total period of about two years. The purpose is to evaluate the efficacy and safety of this drug, and this study involves pen and paper testing, and…
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A Study to Evaluate the Effectiveness and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis

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…
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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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Non-Invasive Blood-Brain Barrier Opening in Alzheimer's Disease Patients Using a Focused Ultrasound Device

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Elisa Konofagou, PhD
Status: Closed
A clinical trial that uses focused ultrasound to non-invasively open the blood-brain barrier in Alzheimer's disease patients, in order to help get medication into their brain. In this study, we will focus on the safety and efficacy of this technique, without the use of an anti-Alzheimer's drug. We will also test whether treatment with focused…
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Dementia and Psychiatric Symptoms (Ages 40-90) (Alzheimer's Disease)

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Edward Huey, MD
Status: Closed
The purpose of this study is to carefully examine how dementia can affect mood and behavior. In this study, we will use Magnetic Resonance Imaging (MRI) to better understand the relationship between thinking, mood, behavior and the brain. We are currently seeking participants who have a diagnosis of dementia, such as Alzheimer's Disease, who are…
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Study of Posiphen in Subjects with Early Alzheimer's Disease (AD) DISCOVER

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
This study is an early phase study of a medication for Alzheimer's Disease, with examination of cerebrospinal fluid.
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Anti-Viral therapy in Alzheimer's disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Davangere Devanand, MD
Status: Closed
In the U.S., 82% of older adults test positive for antibodies to the herpes viruses. Basic and clinical evidence point to the herpes viruses as a possible cause or contributor to Alzheimer's disease. The long-standing theory that viruses may cause Alzheimer's disease has never been tested in a clinical trial. Anti-viral therapy in Alzheimer's…
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Study of STI-6129 in Patients with Relapsed or Refractory Systemic AL Amyloidosis

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.
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Study of CRISPR-CAS9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCS) in Subjects with Beta-Thalassemia

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 (…
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Study For Previously Treated Patients Of Amyloidosis Using Bendamustine and Dexamethasone

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…
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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 severe Sickle Cell Disease using study drug

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…
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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 at risk for memory loss using study drug solanezumab

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Karen Bell, MD
Status: Closed
The purpose of this study is to test whether an investigational drug called solanezumab can slow the progression of memory problems associated with brain amyloid. The study will also test 1) whether the anti-amyloid treatment can slow evidence of AD-related brain injury on imaging tests to look at brain structure and function and 2) the effect of…
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Study of Gantenerumab in Subjects with Early Alzheimer's Disease (Prodromal to Mild)

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
This is a randomized, double-blind, placebo-controlled, parallel-group study that will evaluate the efficacy and safety of gantenerumab versus placebo in participants with prodromal (very early form) to mild AD. Participants will have a chance of receiving either injections of gantenerumab or placebo as per the visit schedule. The primary efficacy…
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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 for patients with Alzheimer's Disease using study drug azeliragon (TTP488)

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
This study is for patients with Alzheimer's Disease. Alzheimers disease is a disorder characterized by progressive loss of memory, thinking, and behavior. An earlier study in patients having mild to moderate Alzheimers disease has demonstrated more pronounced benefit of treatment with azeliragon among participants with mild disease than among…
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S1702 Isatuximab in Treating Patients With Relapsed or Refractory Primary Amyloidosis

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…
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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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A study for patients with AL amyloidosis using study drug NEOD001

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…
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A study for patients with mild to moderate Alzheimer's Disease using study drug T-817MA

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Karen Bell, MD
Status: Closed
The purpose of this study is to determine whether an investigational drug, T-817MA, is safe and beneficial in delaying or altering the decline in memory and daily functioning when given to people with Alzheimer's disease. Participants will be given either an active study drug (T-817) or placebo (no active medication).
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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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Phase 3 study of safety and efficacy of Daratumumab in combination with other drugs in treating AL Amyloidosis

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…
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A study for the treatment of agitation in patients with Alzheimer's Disease using study drug AVP-786

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Davangere Devanand, MD
Status: Closed
The objectives of the study are to evaluate the effectiveness, safety, and tolerability of study drug AVP-786 compared to placebo (contains no active medication), for the treatment of agitation in patients with dementia of the Alzheimers type. Eligible patients will be randomly assigned at the Baseline visit to receive AVP-786 or matching placebo. Study…
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Study of CTX001 in Patients with Severe Sickle Cell Disease (SCD)

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…
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Efficacy and Safety Study of Drug (MTAU9937A) in Patients with Alzheimer's Disease Preclinical-to-Mild or Moderate Alzheimer's Disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
This is a Phase II, randomized, double-blind, placebo-controlled, parallel-group study that will evaluate the efficacy, safety and tolerability, pharmacokinetics, and pharmacodynamics of the study drug (an antibody to the tau protein called MTAU9937A) in patients with prodromal or mild Alzheimer's disease. The study consists of a screening period, a…
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A study for patients with myelofibrosis using study drug PF-04449913

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…
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Study of Effects of Itacitinib in Combination with Corticosteroids for of Graft-Versus-Host Disease (GVHD) in Pregnant Partner

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…
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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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A study for patients with Progressive Supranuclear Palsy (PCP) using study drug C2N-8E12

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
The purpose of this Phase I study is to see whether a single dose of an experimental drug (meaning a drugthat is not approved by the FDA) called C2N-8E12 given into your vein (called intravenous or IV) is safeand well-tolerated in subjects with Progressive Supranuclear Palsy (PSP). C2N-8E12 is an immunotherapy (antibody) drug designed to bind to and to…
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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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Study of New Drug in Patients with Cognitive Impairment or Mild Alzheimer's Disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
You have been selected as a possible participant in this study because you have memory or thinking problems (cognitive impairment) or mild Alzheimer's disease. Alzheimer's disease is a slow progressive disease of the brain that affects memory and brain function. Currently available medications for Alzheimer's disease provide limited…
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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 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 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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Anemia due to Myelodysplastic syndromes (MDS) using study drug luspatercept

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.
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A study for patients with early Alzheimer's Disease using study drug AZD3293

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
The purpose of this study is to see if the study drug AZD3293 is effective and safe in treating early Alzheimer's disease compared with a placebo. A placebo is a tablet containing no drug. This study will also measure the levels of AZD39293 and its metabolite in the blood and evaluate the relationship between drug levels, efficacy (how well it works)…
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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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MissionAD1: A 24-month Efficacy and Safety Study in Subjects with Early Alzheimer's Disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
The name of this trial is MissionAD. This is a 24-month treatment, multicenter, double-blind, placebo-controlled, parallel group, Phase 3 study in participants with Early Alzheimer's Disease (AD), includingmild cognitive impairment (MCI) due to AD, known as Prodromal AD, and the early stages of mild AD.The study will be conducted to evaluate the…
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A study for patients with dementia using study drug RVT-101

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
The purpose of this study is to investigate how well a drug called RVT-101 works to improve cognitiveand overall function as well as to investigate the safety of RVT-101. RVT-101 is an investigational drug.Investigational means that the drug tested in this study has not been approved by the U.S. Food and Drug Administration (FDA) or any other Health…
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Study For Previously Treated Patients Of Light-Chain Amyloidosis Using study drugs Dexamethasone and MLN9708

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…
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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 Syndrome (MDS) using study drug Vadastuximab Talirine

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…
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A study assessing the safety and effectiveness of study drug Crenezumab in patients with mild to moderate Alzheimer's Disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
The purpose of this study is to better understand why certain patients are more likely to respond totreatment than others, to facilitate the development of personalized medicinesto get the rightmedicine to the right patient. To achieve this goal, samples of blood will be collected from patients with Alzheimer's for future research. The sample will be…
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Study of Effectiveness of Ruxolitinib in combination with Decitabine in Patients with Myeloproliferative Neoplasms (MPN)

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…
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Magnetic Resonance Spectroscopy (MRS) to assess progression of Alzheimer Disease (AD)

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
THE PURPOSE OF THIS STUDY is to find out whether a type of brain imaging (called "Magnetic Resonance Spectroscopy Imaging" of "MRS") might be useful to help assess the presence of Alzheimer's disease (AD) and assess progression of the disease. MRS uses magnetic resonance imaging (MRI) methods to image the chemicals in the brain.…
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A study for patients with Alzheimer's disease using study drug crenezumab

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Lawrence Honig, MD, PhD
Status: Closed
The purpose of this study is to test the effects of an investigational drug called crenezumab for the treatment of Alzheimers disease. This study will use MRI scans to look at the effects of this treatment on the brain. This study will be conducted in many centers around the world, and it is important that the MRI scans are always performed in a similar…
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Study For Previously Treated Patients Of Light-Chain Amyloidosis Using Study Drug Carfilzomib

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…
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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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Study of BHV-4157 in Alzheimer's Disease (T2 Protect AD)

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Karen Bell, MD
Status: Closed
T2 Protect AD is a clinical trial testing the investigational drug troriluzole in people with mild to moderate Alzheimer's disease (AD). The study is designed to determine whether this new drug can protect against, slow down, or potentially improve memory and thinking problems that increase as Alzheimer's disease progresses.
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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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A study for patients with myeloproliferative disorders using study drug momelotinib

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…
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Testing the benefits of the vitamin supplement Benfotiamine in patients with Alzheimer's Disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Yaakov Stern, PhD
Status: Closed
The goal of this study is to investigate whether treatment with the vitamin supplement benfotiamine for one year will slow the memory problems in patients who meet the definition of Alzheimer's disease or amnestic mild cognitive impairment, an early manifestation of Alzheimer's disease.
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Cognitive Training

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Davangere Devanand, MD
Status: Closed
The purpose of this study is to evaluate if systematic cognitive training can improve cognitive performance in participants (ages 55-95 years) with memory loss. This study will evaluate the effects of Computerized Cognitive Training (CCT) for improvement in everyday cognitive and function status, in addition to long-term changes in brain networks over an 18…
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Treatment of Agitation in Alzheimer's Disease

Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Davangere Devanand, MD
Status: Closed
This NIH funded research study involves a 12-week double blind treatment trial in which Alzheimer's disease (ages 55-95) who have agitation/aggression with or without psychosis (hallucinations or delusions) will receive either low-dose lithium or placebo. Patients are allowed to receive other specific psychiatric medications if needed during the study…
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