This study aims to use new technology involving molecular and imaging tests to help detect liver cancer early in patients. Participants enrolled will participate in it for approximately 5 years. The purpose of this study is to determine if an experimental drug, INT-787, is safe and effective in patients who are hospitalized due to severe alcohol-associated…
This is a global, multicenter, placebo-controlled, phase 3 study evaluating the efficacy and safety of riliprubart in adult participants with CIDP who are refractory or had an insufficient response to standard of care (SOC) therapies, defined as Ig administered IV (IVIg) or SC (SCIg), or corticosteroids. Treatment duration will be 48 weeks. Participant must…
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…
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 compare the effects, good or bad, of tiragolumab plus atezolizumab plus bevacizumab versus placebo plus atezolizumab plus bevacizumab on patients with HCC. In this study, participants will be randomized you will get either tiragolumab plus atezolizumab plus bevacizumab or placebo plus atezolizumab plus bevacizumab. A placebo…
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 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…
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 blood sugar,…
The purpose of this study is to evaluate the efficacy and safety of study drug, fingolimod taken daily compared with a placebo (does not contain active medication) on delaying disability progression in patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). The study will consist of 3 periods: a Screening Period (lasting for up to 45…
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 product that is…
The purpose of this study is to learn more about TPST-1120. The aims of the study are to find out if TPST-1120 can be given safely to people, to determine a safe dose of the study drug, if it is safe when given in combination with other anti-cancer drugs, what effects the drug may have, and to learn more about how the body processes the study drug. This is…
The purpose of this study is to evaluate the efficacy and safety of ISIS TTR Rx given for 65 weeks in patients with Familial Amyloid Polyneuropathy (FAP). The purpose of this study is to determine if ISIS 420915 can help people with mild or moderate FAP. To be in this study you must have FAP and be able to walk without help or walk with using only one cane.
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…
Patients will be asked to participate in this research study because they have been diagnosed with hepatocellular carcinoma (HCC) which is a type of liver cancer. By doing this study, researchers hope to: Find out more about patients that have HCC including their age, race/ethnicity, and other medical conditions they may have; learn more about how…
Charcot-Marie-Tooth type 1A (CMT1A) disease is a rare, slowly progressing and debilitating hereditary condition commonly causing weakness of muscles in the feet and lower parts of the legs. There is currently no approved drug to cure CMT1A disease, and the available treatments commonly target the pain and inflammation associated with the disease. In order…
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.
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:…
Peripheral Neuropathy is a result of nerve damage, which may cause weakness, numbness, tingling, and pain in your hands, feet, and other areas of your body. Over 20 million Americans are affected by peripheral neuropathy, most commonly caused by diabetes. In approximately 25-50% of all people with peripheral neuropathy, the cause is unknown. These cases…
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)…
This study is looking at using topiramate as a potential therapy for patients with cryptogenic sensory peripheral neuropathy. Patients who are randomized into the study will return to the clinic for follow up every 16 weeks. The total length of the study is 96 weeks.
The purpose of this study is to see how effective two doses of an investigational new drug called IgPro20 is when given subcutaneously (a shot given into the fat layer between the skin and the muscle) to treat CIDP in patients that still require IVIG and also to look at how safe the IgPro20 is for patients.