TARGET-HCC is a 5-year, longitudinal, observational study of the natural history and management of patients with HCC. The study will address important clinical questions that remain unanswered in the management of HCC with a unique research registry of participants with HCC from academic and community real-world practices. TARGET-HCC is disease focused, not drug specific, which allows for continuous acquisition of real-world evidence regarding the natural history, management, and outcomes of treatment with current therapies and new treatments that may be utilized in usual clinical practice.
This study is for participants who have been diagnosed with cancers of the blood who do not have a related perfect tissue "match" for BMT. This research is being done to find out if a bone marrow transplant (BMT) from a relative who is not a perfect tissue match is safe and effective in people with cancer. The primary goal of this study is to use chemotherapy and/or radiation therapy to completely get rid of a person's existing bone marrow, give them new bone marrow from a relative who is not a perfect tissue match, and then give them two doses of additional chemotherapy shortly after receiving the new bone marrow. Participants can expect to be in this study for up to 2 years.
This research study is a Phase I clinical trial. Phase I clinical trials test the safety of an investigational drug. The participants in this study will be the first people to receive PLX7486-TsOH. PLX7486-TsOH works by targeting and inhibiting certain molecules that interact with tumor cells. These molecules assist with tumor growth through various functions, including promoting tumor invasion of organs and nerves. By interfering with these molecules, PLX7486-TsOH may help to prevent tumor invasion and kill tumor cells. PLX7486-TsOH has shown anti-cancer activity in laboratory studies; however, it is not yet known whether it will work in humans. Participants will have a screening procedures done to ensure they are eligble to particpate in the trial. If they are eligible and agree to participate, subjects will take the PLX7486-TsOH by mouth daily, come to the MUSC clinic when specified for study tests and procuedres and maintain a drug diary that will be provided to them. Subjects may continue pariticpating in this study as long as they are tolerating the study drug well and their tumor(s) is not growing.
The study is being done to test how well nifurtimox works against neuroblastoma and medulloblastoma in children. Early observation and testing suggest that nifurtimox has effect against these types of cancer. Nifurtimox is well tolerated by children and doesn't seem to have long term side effects when used to treat Chagas' disease. The goal of the study is to learn if nifurtimox in combination with other chemotherapy drugs is effective in shrinking/killing neuroblastoma and medulloblastoma cells.