This is a phase 1 study for participants with relapsed/refractory acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or juvenile myelomonocytic leukemia (JMML) diagnosis. This study will observe the safety, side effects, and best dose of Imetelstat as an investigational drug, or drug that is not approved by the Food and Drug Administration (FDA…
This study will enroll with children, adolescents and young adults that are less than 22 years old that have returned or 'relapsed' acute myeloid leukemia (AML) diagnoses. Within this group, the study is also targeting those are also unable to receive additional chemotherapy treatments containing anthracycline, which is a type of antibiotic that…
We are conducting a research study on the effect of high insulin levels on the buildup of excessive liver fat ("fatty liver disease") in people at risk for type 2 diabetes. We are seeking adult volunteers, ages 18-65, who have obesity with a body mass index of 30-45, and have been diagnosed with, or are at risk for, prediabetes and metabolic…
This study is called a screening study and the purpose of this study to find better ways to diagnose and treat leukemia that has come back after treatment or difficult to treat. Bone marrow, blood, and medical information about cancer and treatment will be collected. The results from this screening study may give other information about leukemia that is…
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,…
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 that makes one predisposed to Multiple Sclerosis and various…
We are conducting a research study on how insulin controls blood sugar in both healthy people and those at risk of type 2 diabetes. We are seeking adult volunteers, aged 18-65, who have body mass indices either in the lean range (18-25 kg/m2) or in the obesity range (30-45 kg/m2) but who do not have prediabetes or diabetes. Participants will undergo 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…
The purpose of this study is to determine if measuring blood levels of proteins called POMC, AgRP, and chromogrannin A (CGA) can be used to diagnose and track tumor growth and recurrence in patients that have ACTH-dependent Cushing's syndrome, non-secreting pituitary tumors, and other neuroendocrine tumors. We invite you to participate if you have: a…
Polycystic ovary syndrome (PCOS) is one of the most common endocrine disorders in women ofreproductive age, affecting about 7% of this population when using the strictest diagnostic criteria and frequently presents in adolescence and young adulthood. Diagnostic features of PCOS includehyperandrogenism and ovulatory dysfunction. Additionally, both obese and…
This study is for individuals who have Cushings disease and want to join voluntarily in this study to find out if a study drug called osilodrostat (LCI699) is safe and has beneficial effects in people who have Cushings disease. Cushings disease is a rare but debilitating disease. Cushings disease is caused by a benign tumor of the pituitary gland. The tumor…
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…
The purpose of this study is to find the best dose of Quizartinib that can be given safely with chemotherapy in children and young adults with acute myeloid leukemia (AML) which has come back or has not responded to the standard therapy. Quizartinib will be given by mouth once a day from Day 6 through Day 28. It will be given in combination with…
We are currently recruiting individuals with Multiple Sclerosis (MS), first-degree family members of persons with MS, and healthy individuals to take part in our research on the effects of probiotics. Our goal is to better understand how the health of the gut microbiome influences the immune system.
The purpose of this study is to find out if the drugs called Nivolumab and 5-Azacytidine can be given safely in children and young adults with Acute Myeloid Leukemia (AML) which has come back after treatment or has not responded to standard therapy. These drugs are thought to work by turning on genes that limit the growth of cancer cells. The study drugs…
The purpose of these studies is to gain an understanding of the mechanisms that promote weight loss and improved glucose control. The ultimate goal is to identify therapeutic pathways for obesity and diabetes as a way to optimize surgical procedures and discover novel non-surgical targets for safe and effective drug therapy. Participants with obesity will…
We are recruiting healthy volunteers, aged 18-65, for a research study on the buildup of excessive liver fat ("fatty liver disease") in people with or at risk for type 2 diabetes because their bodies are resistant to the actions of the blood sugar-lowering hormone, insulin. To do this, we seek to induce temporary insulin resistance in 10-12…
This study will be used to evaluate the immunologic effects of Prebiotics (soluble fiber required by beneficial bacteria), in comparison to Probiotics (beneficial bacteria), in individuals with Multiple Sclerosis (MS) and Clinically Isolated Syndrome (CIS).
ASPIRE is a clinical trial to investigate aspirin as a treatment to improve exercise performance in people with multiple sclerosis (MS) who experience overheating during exercise. Persons with multiple sclerosis benefit from exercise, but many avoid it because of exhaustion and overheating. This randomized controlled trial (RCT) tests aspirin as a method to…
The purpose of this study is to provide samples for biomarker testing for the different MPD-RC treatment studies. As part of the treatment study you currently participating in, this is an additional research study in which you we will ask for you to donate blood and/or bone marrow samples, two nail clippings, and buccal (cheek) swab. In order to…
The purpose of this study is to investigate how well weekly injections of the growth hormone drug NNC0195-0092 works in adults with growth hormone deficiency. Growth hormone deficiency is a disorder that involves the pituitary gland (a small gland located at the base of the brain). The gland produces growth hormone and other hormones (chemical messengers of…
The purpose of this study is to compare two different combinations of treatment plans to a standard transplant procedure in order to see whether one or both of them are better at reducing the occurrence and severity of chronic GVHD. The research portion of this study involves manipulation of your stem cell product by removing T-cells, which cause chronic…
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, low HDL cholesterol (good…
This study is for children and teens who have been diagnosed with Acute Myeloid Leukemia (AML). AML is a cancer of the bone marrow, the spongy tissue inside the large bones of the body where blood cells are made. In AML, the bone marrow makes large numbers of immature white blood cells called blasts. These blast cells crowd out the normal cells of the bone…
The purpose of this study is to confirm the effectiveness and safety of LCI699 in treating patients with Cushing's disease. Patients will be treated with the invesitigational drug LCI699 at doses up to 60 mg a day, and possibly for a short period of time with placebo. A placebo is a dummy drug ("pill") with no active medicine inside.
This study is for patients who have been diagnosed with acromegaly and have been treated with the injectable medications ocreotide or lanreotide for at least 6 months. You should have had the same dose for at least 4 months, and be adequately controlled. The purpose of this study is to compare the safety and effectiveness of an investigational new drug…
The purpose of this study is to find the best dose of pevonedistat that can be given safely with chemotherapy in young adults with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS) that has either come back or has not responded to the standard therapy. Pevonedistat works by blocking some of the enzymes that are needed for cell growth.
This is a research study for subjects who have had gastric bypass surgery at least one year ago, and currently have type 2 diabetes. This study is looking at the treatment of diabetes after gastric bypass surgery. About 40% of patients do not experience diabetes remission after gastric bypass surgery. Of the patients who initially go into remission, about…
The purpose of this study is to see if ocrelizumab (study drug) will stabilize, or potentiallyimprove the signs and symptoms of your MS. Ocrelizumab is a type of drug called amonoclonal antibody. Monoclonal antibodies act like your bodys immune system andattach to certain cells in order to attack germs and other illnesses in your body.Ocrelizumab attaches…