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Displaying 1 - 31 of 31
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…
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…
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.
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…
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…
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…
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…
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,…
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.
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.
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.
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…
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, in addition…
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,…
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…
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 patient to…
Condition: Neurological Disorders / Alzheimer's Disease
Investigator: Jose Luchsinger, MD
Status: Closed
The BenfoTeam clinical research trial is based on evidence from decades of research on the role of thiamine in Alzheimer's disease (AD). BenfoTeam scientists have found that thiamine (vitamin B1) does not enter the brains of people with early Alzheimer's disease despite having a sufficient supply of thiamine in their blood.…
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…
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…
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…
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,…
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 enrolling…
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…
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) participants…
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…
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…
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…
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…
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,…
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…
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…