This study is for patients who have been diagnosed with relapsed (the disease has gotten worse after a period of improvement) or refractory (the disease does not respond to treatment) Epstein-Barr Virus-Positive (EBV+) lymphomas.
The investigational drug in this study is Nanatinostat. Investigational means it is not approved by the United States' Food and Drug Administration (US FDA). Nanatinostat is a tablet that will be taken by mouth once a day.
The purpose of this research study is to determine the effectiveness and safety of the combination therapy, nanatinostat and valganciclovir, on treating lymphoma cancers. Valganciclovir is an approved anti-viral drug (a drug that fights against virus in your body), which has been used in the prevention and treatment of infection caused by a virus called cytomegalovirus (CMV), which is similar to Epstein-Barr virus (EBV). The safety and effectiveness of nanatinostat on its own will also be reviewed in a small subset of patients.
Participants can expect to receive treatment until disease progression, unacceptable toxicity/ side effects, or participants withdraw. Participants can expect to be in follow up for up to 5 years.
This study is for participants that have been diagnosed with Medulloblastoma. The purpose of this study is to test the effectiveness of using an investigational agent called DFMO for Medulloblastoma. An investigational drug is one that has not been approved by the U.S. Food and Drug Administration (FDA). This study will look at the ability of this study drug to either keep participants tumor in remission or if they have active tumor, for their tumor to respond to the treatment and will also look at the safety and tolerability of DFMO. After this first day participants will be seen in clinic once every 30 days for the first 6 months of the study, after that they will be seen once every 90 days for the remainder of the study. These visits will last about 2 hours.
This is a research study to find out if a study drug called EDIT-301 is safe and effective in treatment of patients with severe Sickle Cell Disease (SCD). The EDIT-301 study medicine is a new investigational therapy, which in this case means this is first-in-human use of this study drug. This study medication uses patients' own stem cells, modifies the cells with genetic modification, and transplants them back to the patient (by infusion) to treat SCD.
Participation in this study is expected to last approximately 30 months, including time for screening, collection of cells, transplant, and a 24 month follow-up period after transplant. At the end of that 24 month follow-up, the participant will be asked to participate in an additional long-term follow-up study, totaling 15 years of post-transplant follow-up.
This study is for subjects that have been newly diagnosed with diffuse large B cell lymphoma (DLBCL). This study is testing an "investigational" (not yet approved by the Food and Drug Administration (FDA)) combination of the drug zanubrutinib (BRUKINSA™) and a standard chemotherapy treatment called R-CHOP. This study will test the how well the drug combination works and how safe it is. The subject will be given the study drug combination in 21 day cycles. At any given visit, the subject may undergo procedures, such as a physical exam, blood samples, bone marrow biopsies, and imaging scans. The subject may remain in the study for up to approximately 2 years.
Both withdrawal symptoms and external stressors can contribute to relapse in people that are trying to quit using marijuana. These negative feelings may be more severe in people that also have Major Depressive Disorder, making it especially hard for them to quit. The purpose of this study is to examine differences in withdrawal symptoms and stress response in people with Cannabis Use Disorder that do or do not also have major depression. Study participation will involve 3 on-site laboratory visits over 6 days. Participants will be asked to not use marijuana for 3 of those days. Participants will complete mobile phone surveys multiple times each day, wear a watch to assess sleep quality, and provide blood samples for testing at each laboratory visit. At the final laboratory visit, participants will complete a stress task.
This is a research study to find out if mental health symptoms in patients admitted to the Institute of Psychiatry are affected by a form of ear stimulation called transcutaneous auricular vagus nerve stimulation, or taVNS. Participants will receive ear stimulation during their inpatient treatment at the Institute of Psychiatry. Participants will be randomly assigned to either receive ear stimulation for 1 day (up to 9 treatments over the course of a single day) or 3 days (up to 3 treatments per day), and will have a 50:50 chance of being in either group (like the flip of a coin). Each treatment will last up to 30 minutes and there will be a break of at least 30 minutes in between treatments. The study team will ask participants to complete a group of questionnaires at the beginning and end of the study. In addition, there are several questionnaires that will be completed daily while participants are receiving ear stimulation. Participants in the 1 day stimulation group will also receive 2 days of follow up questionnaires after the initial day of stimulation. The questionnaires will ask questions about mental health symptoms that subjects may or may not be experiencing, including questions about mood, anxiety, and sleep. The ear stimulation treatments will not interfere with the care subjects are receiving from the medical team. In order to qualify for this study, participants must be actively receiving inpatient care at the MUSC Institute of Psychiatry.
The purpose of this study is to compare the effect of a combination of art therapy (AT) and physical therapy (PT) to PT only in children undergoing hematopoietic cell transplant (HCT). Each child will receive daily AT and PT or only PT for 5 days per week for 2 weeks. These sessions will begin approximately on day 15 following the transplant. Prior to starting the sessions and following 2-weeks of sessions, we will measure your self-care and mobility skills. During each session, heart rate variability (i.e., time between heart beats) using a small monitor on the chest (about the size of a quarter), walking distance using an accelerometer (similar to wearing a watch), and self-reported happiness and excitability will be measured. Although results cannot be guaranteed, it is expected each group will benefit and demonstrate improvements in emotional state, self-care, and mobility skills.
An experimental drug called HCW9218 is being tested in this study. HCW9218 is not approved by the Food and Drug Administration (FDA) or any other health authority for the treatment of cancer or any other disease. Since HCW9218 is experimental, it is only available to people taking part in this study. This study is for participants diagnosed with advanced pancreatic and the disease has spread or cannot be surgically corrected. This is a Phase 1b/2 study, meaning that it is a first in human research study and there is no data on HCW9218 in humans. This study tests different doses of HCW9218 to see which dose is safer in people, how well the treatment is tolerated and whether it has effects on the disease. The study doctor will tell participants which part of this study that they will take part in, the dose escalation phase or dose expansion phase. The dose escalation phase the part of a study that determines the best dose of a new drug or treatment. In a dose-escalation study, the dose of the test drug is increased a little at a time in different groups of people until the highest dose that does not cause harmful side effects is found. In the dose-expansion part of a study allows more participants to receive the study drug to further test the potential side effects of the drug. HCW9218 is given as an injection under the skin (subcutaneous injection). If participants continue in this study, he/she will begin receiving HCW9218 treatment every 28 days. HCW9218 is given as an injection under the skin (subcutaneous injection). For each day participants receive HCW9218, participants will be treated as an outpatient in a treatment center. Participants will be asked to stay in the treatment center for up to 6 hours after the first dose, 3 hours after the second dose, and 30 minutes after subsequent doses, so that the study team can carefully monitor the body's functions and closely watch for potential side effects of the study drug. This study will last about 4 years. Participants will be treated with HCW9218 until disease progression or until they can no longer tolerate the study drug. Participants could be in this study for up to 3 years. There may be risks associated with being treated with HCW9218. Some of the most common side effects that the study doctors know about based on drugs that are similar to HCW9218 are: injection site irritation/ reaction, fatigue/ tiredness, and flu-like symptoms.
This study aims to evaluate the safety and efficacy of isotretinoin ointment (TMB-001 0.05%) in treating subjects > or = to 6 years of age with lamellar ichthyosis, and aims to assess the bodily absorption of the cream across application frequencies. Subjects with ARCI/RXLI will be randomized 2:1, to either receive the TMB-001 0.05% isotretinoin ointment or a vehicle ointment - applied daily - for 3 weeks. Subsequently, dosing will be increased to twice daily for 9 weeks. If significant improvement is observed at the end of the 9 weeks, subjects will be randomized 1:1 to receive the TMB-001 0.05% ointment, either applied daily or twice-daily, for 12 weeks. Prior to the Phase III trial, subjects will have the option to participate in a 14 day treatment period with TMB-001 0.05% - twice daily - followed by continued treatment with TMB-001 0.05% twice daily for 10 weeks.
This is a prospective, multicenter open-label study in adults with CF who are colonized with NTM. Subjects will receive two 5-day infusion cycles of IV gallium. The study will evaluate the safety and antimycobacterial effect of two 5-day infusions of IV gallium. Subjects will be on study for up to 148 days, with a screening period of up to 7 days and active study period up to 20 days. About 40 people with CF who are 18 years of age or older will take part in this study at about 10 hospitals and clinics around the country.