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…
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…
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 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…
This is a Phase 1 study of a drug called uproleselan. We are testing new experimental drugs such as uproleselan in the hopes of finding a treatment that may be effective against acute myeloid leukemia, myelodysplastic syndrome or mixed phenotype acute leukemia that has come back or that has not responded to standard therapy. This study looks at how well…
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…
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…
The purpose of this clinical study is to evaluate the safety and efficacy of two doses of GT005 administered as a single subretinal injection in individuals with geographic atrophy secondary to dry age-related macular degeneration (AMD) who qualify, based on genetic testing.
The abicipar is an injectable medication stored as a liquid in a single-use glass vial for delivery into the back of the eye. The active component is a protein drug that was made using bioengineering technology. It was designed to bind to and inactivate a protein called Vascular Endothelial GrowthFactor (VEGF) that plays an important role in causing AMD.…
Macular degeneration is a progressive eye disease that damages the macula. The macula is the part of the eye that is responsible for central vision, which is needed to perform straight-ahead activities, such as reading, driving, or watching TV. This research is being conducted to learn about the safety and efficacy (how effectively the study drug works) of…
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…
This study is for patients with Glaucoma. The purpose of this study is to investigate the safety and effectiveness of a study drug called Bimatoprost Sustained Release (SR) compared to treatment with eye drug Timolol. The Bimatoprost SR is an implant that is very small, is biodegradable (dissolves naturally in the body), and contains the bimatoprost drug.
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…
Stargardt macular degeneration isa genetic condition, which in mostcases causes vision loss early in lifeand leads to legal blindness. Thereis currently no FDA-approvedtreatment.This trial will evaluate the safetyand effects of ALK-001, a newpotential drug treatment, on theprogression of Stargardt disease.ALK-001 is a capsule administereddaily by mouth.This…
The purpose of this study is to find the best dose of pevonedistat that can be given safely with chemotherapy in children 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 Phase 1 multicenter study of Decitabine (DEC) and Vorinostat (VOR) followed by the standard chemotherapy drugs (Fludarabine, Cytarabine and G-CSF (FLAG)). The study treatment is considered experimental because it is not approved by the United States (US) Food and Drug Administration (FDA) for treating children with acute myeloid leukemia (AML)…
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…
Glaucoma is the leading cause of irreversible blindness worldwide. The most important test to detect progression is visual field testing. However, this test is very subjective, often unreliable, and variable. One of the main causes of unreliable tests is the lack of attentiveness or concentration during the test. Previous studies have shown that listening…
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…
Glaucoma is a given name of a group of diseases that affect the retinal ganglion cells in the eye and the optic nerve. Glaucoma cause degeneration of the retinal ganglion cells in the retina and their projections (axons) that form the optic nerve. Retinal ganglion cells and their axons transmit all of our vision information from the eye to the brain through…