This Phase III trial investigates if the incorporation of immunotherapy will improve progression-free survival (PFS) and maintain overall survival (OS) while simultaneously minimizing long-term morbidity and treatment-related mortality by reducing exposure to radiotherapy and high cumulative chemotherapy doses. The study will enroll patients ages 5 to 60…
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…
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…
The purpose of this study is to better understand the use of Valchlor in clinical practice. Other goals include describing the demographic and clinical characteristics of disease and health outcomes of patients treated with Valchlor. Participating in this study will not influence the usual care you receive from your doctor.
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…
This study is being done to determine if a study drug called lenalidomide can be combined with full doses of EPOCH chemotherapy for patients with Adult T-Cell Leukemia-Lymphoma (ATLL), and to define the highest dose and longest duration of lenalidomide that can be given safely in combination with EPOCH. EPOCH chemotherapy is a combination of five…
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…
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…
This is a phase 2 study to determine the efficacy and safety of Naloxone HCL Lotion 0.5% for the treatment of mycosis fungoides (MF) type of cutaneous T-cell lymphoma (CTCL). Selected subjects will have the study drug applied to determine its effect on reducing itchiness and any potential side effects. Naloxone has previously been approved for direct 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 purpose of this study is to see if study drug IMO-8400 is safe and effective in the treatment of patientswith relapsed or refractory Diffuse Large B-Cell Lymphoma following conventional treatment.Examples of conventional treatment are radiotherapy, immunotherapy, chemotherapy, orradioimmunotherapy. Patients are scheduled to participate in this study…
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…
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 phase 3 clinical trial to determine the efficacy of topical SGX301 (synthetic hypericin) in combination with visible light therapy on cutaneous T-cell lymphoma (CTCL). Selected subjects can participate in 3 cycles each consisting of a 6 week treatment period where a chosen lesion will be treated with the study ointment (twice a week) followed by…
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 determine a safe dose by testingthe investigational product, AGS67E at different dose levels. Secondly, to test what effects, good and/or bad, it has on subjects with refractory or relapsed non-Hodgkin lymphoma (NHL), chronic lymphocytic leukemia (CLL), prolymphocytic leukemia (PLL) or hairy cell leukemia (HCL). We will also…
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.
You are being asked to participate in a research study because you have Cutaneous T-Cell Lymphoma/Mycosis Fungoides, and one of your symptoms is itching. This study is being done to assess the efficacy and safety of Naloxone HCL Lotion 0.5% compared to Vehicle Lotion for the treatment of itching in patients with mycosis fungoides (MF) which is a type of…
The purpose of this study is to gather information about the effectiveness and safety of as drug called brentuximab vedotin when given in combination with another drug called bendamustine for Hodgkin Lymphoma. The study will assess the safety profile of the combination treatment and determine what proportion of patients achieve a complete remission.…
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 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…
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…
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…
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 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.
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…
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…
This is a 2-part, multicenter study of a study drug in pediatric and young adult patients with mature B-cell non-Hodgkin Lymphoma which has come back or has not responded to the standard therapy. The study treatment is considered an experimental because the study drug is not approved by the United States (US) Food and Drug Administration (FDA) for treating…