The purpose of this study is to examine the causes and pathology of alopecia areata and other hair and skin disorders. Skin biopsies and blood and/or clinical information collected in the study will be used for research aiming at developing new options for the treatment of alopecia areata and…
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…
The study uses brain imaging and cortisol to understand risk and protective factors for mental health among adolescents on social media. We are currently recruiting adolescents ages 14-17 who struggle with mental health. To participate in our study, we would ask your child to: 1. Complete a mental health interview (over Zoom), 2. Complete a Magnetic…
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…
We are studying the natural course of autoimmune hemolytic anemia (AIHA). If you are an adult between 18 and 120 years old, receive care at Columbia University Irving Medical Center-New York Presbyterian Hospital, are willing to provide a blood sample every 6 months for research, and have one of the following diagnoses: AIHA,…
This research study aims to obtain a greater understanding of the causes of cognitive decline in Alzheimer's disease. We are currently enrolling male and female participants (ages 55 years and older) with Alzheimer's disease, mild cognitive impairment, or healthy controls. Enrolled participants with undergo a small blood draw…
This research is being done to determine how variations in genes may explain the different risk to develop scleroderma (or CREST) seen in African American patients compared to other populations. People who are African American with scleroderma (or CREST) may join. This research involves a blood draw and physical exams.
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 should consider joining this study if you have been diagnosed with a lung disease that has a genetic cause or if other family members have been diagnosed with a similar lung…
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…
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…
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.
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 blooddisorders smoldering multiple myeloma (SMM) and monoclonal gammopathy of undetermined significance…
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…
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…
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…
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…
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…
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,…
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.
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.
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…
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.
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…
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 aim of this study is to develop better biomarkers for the diagnosis and treatment of Lewy Body disorders. In particular, there is a need to understand how the contribution of coexisting Alzheimer's disease may affect the diagnosis and clinical course of Lewy Body disorders. This study will…
The purpose of this research is to see if an experimental drug called REGN7544 is effective to treat individuals with postural orthostatic tachycardia syndrome (POTS). REGN7544 is an antibody. An antibody is a kind of protein that your immune (defense) system normally makes to fight bacteria and viruses in your body. Scientists can now make antibodies in…
We are currently enrolling individuals with Major Depressive Disorder (MDD) to participate in this research study. This is a one-year open-label study to evaluate the long-term safety and tolerability of an investigational drug called REL-1017 (esmethadone) when taken alone (monotherapy) or when taken with other antidepressants (adjunctive therapy). Study…
MS Genetics aims to better understand the genetic component of demyelinating diseases by doing genetic testing on blood samples of participants. This study involves a one-time blood draw and completion of questionnaires spread out over a couple of years in hopes to better understand the genes…
We are recruiting individuals and/or families with members under the age of 65 who have dementia or are experiencing memory problems. Our initial screening can be completed in person or over the phone. The evaluation will take about 1 to 1.5 hours per person and includes a: 1. Blood sample (5 tablespoons) or saliva (1 teaspoon…
For over 30 years, The Columbia University Alzheimer's Disease Research Center (ADRC) has worked with thousands of participants who have helped provide important insights into how the brain ages and why Alzheimer's disease may develop. We are actively recruiting adults 65 years of age and older who share our commitment to be part of this nationwide study.…
Lymphangioleiomyomatosis (LAM) is a rare lung disease primarily affecting young women. Currently, LAM is often diagnosed in patients with radiology imaging (CTs) and/or lung biopsies; however, these methods can be imprecise, invasive, and/or can carry significant risks. The current study aims to devise a less invasive tool for diagnosis of this rare…
We are conducting a research study on how psoriasis might be caused or worsened by high levels of the blood sugar-lowering hormone insulin. We are seeking people with plaque psoriasis, ages 18-65, who are overweight or obese with a body mass index of 25-35. Participants will have blood drawn and…
The Center for Behavioral Cardiovascular Health (CBCH) is conducting a research study to examine the relationship between sleep and nighttime blood pressure levels and whether this relationship has something to do with the way our nervous system functions. Participants must speak English or Spanish and be between 25 and 60…
Celiac disease is associated with an increased risk of osteoporosis and fractures. Men with celiac disease were found to be at higher risk for metabolic bone disease compared to women with celiac disease. The mechanisms underlying metabolic bone disease in celiac disease remain incompletely understood. The goal of this study is to assess skeletal…
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…
We will evaluate the D-cycloserine (DCS) and lurasidone with MRI measurement of brain responses to DCS. DCS is a drug that works on the same brain receptor as ketamine. DCS is not approved by the FDA for bipolar depression. Study participants will receive a dose of lurasidone or placebo in one MRI and a dose of DCS in another, followed by an optional follow…
We are looking for patients who have been diagnosed with Psoriatic Arthritis (PsA) to help us better understand the involvement of inflammation on the body. With a combination of bloodwork, a bone density scan, questionnaires, and a one-time soft tissue biopsy (from an area around the belly button), we hope to see how PsA…
You are being asked to participate in this study because you have a condition known as symptomatic neurogenic orthostatic hypotension (NOH) associated with Primary Autonomic Failure Parkinsons Disease (PD), Multiple System Atrophy (MSA) or Pure Autonomic Failure (PAF), or Non-Diabetic Autonomic Neuropathy (NDAN) or Dopamine Beta Hydroxylase (DBH) deficiency…
The purpose of this research study is to test whether the advanced perfusion MRI done prior to starting Bevacizumab and a few weeks after starting Bevacizumab can determine your response to the treatment better than the standard MRI performed at typical time points. The researchers want to see if this type of advanced MRI is better or the same as what is…
Patients with Charcot-Marie-Tooth (CMT) disease have a degenerative nerve disease that can cause muscle weakness and decreased muscle size in different muscles in their body. This study drug is designed to evaluate ACE-083 for its ability to increase the size of your muscles, which could improve your muscle strength. The purpose of this research study is…
We are currently looking for men and women, age 18-65 y, to participate in a sleep study. To participate you must have experienced insomnia (difficulty falling asleep, staying asleep, or experiencing sleep that is unrefreshing) for at least 1 month. This is a 2-phase outpatient and inpatient study. We will test 2 different non-drug based methods to improve…
The purpose of this study is to investigate the safety and effectiveness of tirasemtiv as a potential new therapy for the improvement of breathing, muscle weakness and muscle fatigue in patients with Amyotrophic Lateral Sclerosis (ALS). The effects of tirasemtiv on the performance of your elbow, wrist, knee, ankle, and hand muscles and how long you can…
The purpose of this study is to see how safe and well tolerated the study drug BMS-986168 is at different drug amounts and to measure the amount of study drug in your blood and cerebrospinal fluid (CSF) after taking the drug. This study will also measure the effect of the study drug on the amount of extracellular Tau (eTau) in…
We will enroll children with severe early onset obesity (BMI greater than 99th percentile) diagnosed prior to 6 years of age or other endocrine disorders. We will ask questions about their health, eating behavior and perform a brief physical examination. We will collect saliva or blood to perform…
Genes and Environment in MS (GEMS) is a longitudinal cohort study investigating how genetics and environment can lead to the onset of Multiple Sclerosis. We are recruiting first-degree relatives (siblings, parents, and children) of people who have been diagnosed with MS. This study involves the collection of magnetic resonance imaging (MRI) of the brain,…
We are looking for participants for a study on the role of immune response in Parkinsons 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…
We are looking for participants for a study on the role of immune response in Parkinsons 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…
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,…
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 (…
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…
The Snapshot study aims to understand brain and spinal cord aging in healthy individuals and individuals with multiple sclerosis (MS). We aim to identify new tools that will help us to achieve successful brain aging in the general population and in people with an underlying autoimmune disease. A critical aspect of this study is that we are looking for…
You are being considered to participate in this research study because you have type I or II diabetes with current treatment control and, you are experiencing painful diabetic peripheral neuropathy (DPN) in both lower extremities legs.The specific events that result in painful diabetic peripheral neuropathy are not well understood, but high…
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…
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.…
Children and young adults with diffuse midline glioma will be treated with Focused Ultrasound (FUS) with DEFINITY microbubbles and neuro-navigator-controlled sonication. The overall goal of this study is to see if the study treatment plan will improve the outcome of participants with diffuse midline glioma, for which no known cure exists, and determine…
Polycystic ovary syndrome (PCOS) is a common disorder. Young women with PCOS have irregular or absence of menstruation and excessive acne and or hair on their bodies. They may have multiple cysts on their ovaries. Adolescents and women affected with PCOS are at risk of developing abnormalities in their blood including high fat…
This research study is being done to understand how CPAP for sleep apnea may affect hypertensive disorders of pregnancy and other complications of pregnancy such as gestational diabetes. The purpose of the home sleep test is to find women with sleep apnea. Sleep apnea is when you have one or more pauses in breathing or shallow…
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…
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…
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,…
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…
We are studying the relationship between mitochondrial function and the experience of fatigue. Mitochondria are the powerhouses of the cell. Cells within different organs contain hundreds to thousands of mitochondria, which sustain life and fuel energy-dependent processes that we do every day including muscle contraction and cognitive activity. They act…
International GBS Outcome Study (IGOS) is a study of the Inflammatory Neuropathy Consortium (INC) and Peripheral Nerve Society (PNS) on disease course and outcome in Guillain-Barr syndrome (GBS). Guillian Barre syndrome is an autoimmune disorder affecting the peripheral nervous system, leading to paralysis and potential fatality if it progresses to involve…
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…
Tricuspid Regurgitation is a disorder in which the Tricuspid valve, the valve in between the two right chambers that does not close properly. As a result, blood flows backward through the Tricuspid valve. The Edwards Tricuspid Transcatheter Repair System includes an artificial device, made to serve as a spacer to treat the…
The purpose of the study is to evaluate the safety of Lutathera alone in patients with recurrent glioblastoma and the combination of Lutathera with radiotherapy with temozolomide (TMZ) in patients with newly diagnosed glioblastoma (GB) and to establish the appropriate dose of this combination. Lutathera is a radioligand therapy, which is a targeted…
The purpose of this study is to evaluate the differences between immune profiles of individuals remaining on Ocrelizumab/Rituximab versus those transitioning to diroximel fumarate at two years. Baseline visits for subjects transitioning to diroximel fumarate (DRF) will take place 6 months after last Ocrelizumab (OCR) or Rituximab (RTX) dose, prior to…
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…
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…
This study is of a drug called CT1812, that is being evaluated as a possible treatment for Dementia with Lewy Bodies. In this study, which is carried out at multiple sites in the country, participants receive either CT1812 at one of two doses or placebo. The duration of the study is up to about 35 weeks. Participants receive blood…
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…
The purpose of this study is to see how safe the study medicine, IGV-001 plus standard of care (SOC), is and to compare IGV-001 with placebo see if it works in slowing glioblastoma multiforme (GBM) tumor regrowth. SOC refers to the usual medicine(s) or procedures used to treat your cancer. A placebo looks like the study medicine but does not contain any…
We are inviting patients without autoimmune disorders to help us compare to rheumatoid arthritis patients with 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…
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…
FoxBioNet ECV-004 is an observational study sponsored by the Michael J. Fox Foundation for Parkinson's Research. The purpose of this study is to investigate a gene called LRRK2. Mutations in the LRRK2 gene are associated with Parkinson's disease (PD), and mutation carriers have greater LRRK2 protein activity. The goal of this study is to identify reliable…
The purpose of this study is to investigate the safety and effectiveness of eltrombopag (investigational drug) in treating children and adolescents with newly-diagnosed immune thrombocytopenia (ITP), which is a rare blood disorder.
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.
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…
The purpose of this study is to find the best dose of a drug called MTX110 and a contrast agent called gadolinium that can be given safely in children with newly diagnosed diffuse midline gliomas, which is a kind of brain tumor. All patients enrolled in the study will receive infusion of MTX110 and Gadolinium delivered with a pump directly into the tumor…
Columbia Medical Center is recruiting for a study to test whether the drug oxytocin can improve symptoms of emotion and behavior (e.g. apathy) in people with frontotemporal dementia. The study will last for up to 24 weeks (6 months). This is a crossover study, which means that each participant will receive both oxytocin and placebo (medically inactive…
This research study uses non-invasive brain imaging called functional near-infrared spectroscopy (fNIRS). For fNIRS, a small infrared bulb shines light through the skull, and a special light detector is able to measure oxygen use in the brain. Our goal is to understand oxygen use by brain mitochondria cells in major depression and how this is affected by…
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.
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…
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…
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…
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…
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…
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…
This study is designed to test how well an intravenous human immunoglobulin concentrate, IGIV-C (compared with placebo) improves the ability to taper participant's dose of corticosteroids without causing significant increase in your muscle weakness and worsening MG symptoms. A placebo is a medication that looks like the study medication but has no active…
This is a research study to test the effectiveness of Toca 511/Toca FC, an experimental drug, for treatment of recurring brain tumors compared to current standard of care. The study will enroll 370 subjects age 18-85 years who choose to undergo removal of the tumor a second time. At the time of surgery patients will be randomly assigned in a 1:1 ratio to…
The purpose of this study is to look for abnormal genes and gene expression profiles that help determine why a person develops amyotrophic lateral sclerosis (ALS) and related motor neuron diseases (MND) and why their symptoms present and progress with a particular pattern. For up to three years, subjects will be followed every 3 months at the Columbia ALS…
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,…
This study is designed to test how well an intravenous human immunoglobulin concentrate, IGIV-C (compared with placebo) improves your muscle weakness associated with MG symptoms. A placebo is a medication that looks like the study medication but has no active ingredient. Immunoglobulin is an antibody produced by white blood…
We are doing this study to find out if the research drug known as Rozanolixizumab can have additional benefit for people who are already receiving IVIG( Intravenous Immune Globulin) treatment for CIDP, and to have further safety evaluation of the research drug as well. If you choose to be part of this research study, the following procedures will occur:…
The reason for this study is to find out if the experimental study drug, tesevatinib, is effective as a possible treatment for recurrent glioblastoma. Certain genetic information about your tumor will also be collected, by drawing blood, to help better understand why tesevatinib did or did not work in treating your recurrent…
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…
Patients with hypoplastic left heart syndrome and other single right ventricle lesions who have undergone the Fontan procedure have a high risk of neurodevelopmental disorders that affect quality of life and adulthood employment. This study will leverage the ongoing National Heart, Lung, and Blood…
The main purpose of this research study is to investigate if a drug called Vedolizumab is safe and effective in preventing acute graft-versus-host disease (attack of donor cells on recipient healthy organs after transplant) of the gut in comparison to placebo (dummy drug). This will be studied in patients undergoing Allogeneic Hematopoietic Stem Cell…
Kidney disease patients have a variety of severe bone disorders that result in rapid bone loss and fractures. We are doing this research study to determine relationships between bone quality determined from bone imaging and bone biopsy and by non-invasive measures from blood and skin. We are…
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…
Subjects with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) will be enrolled in the study and Intravenous Immunoglobulin (IVIg) will be given to eligible subjects on the study. CIDP is a chronic inflammatory condition that causes the immune system to attack the nerves. IVIg is one common treatment for CIDP. IVIG is a blood…
The purpose of this study is to collect information about how patients with FAP respond to the study drug ALN-TTR02 over 18 months. This includes studying any potential improvements in FAP and any side effects that participants may have from the study drug. In addition, the study will examine how the body handles (distributes, break down, eliminates) the…
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…
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 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…
This is a research study to test the effectiveness of ICT-107, an experimental drug, with the standard of care (SOC) for treatment of glioblastoma compared to enriched blood cells (PBMC) with the SOC. The study will enroll 400 subjects age 18 years and older with newly diagnosed glioblastoma. Following an initial SOC radiation…
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…