Ependymoma is a type of rare childhood cancer that occurs in the brain and spinal cord. Survival statistics are generally disappointing with a 5-year survival of 50-64%. The standard of care for ependymoma is maximal surgical resection followed by radiation therapy directed at the primary site of disease.
Radiation therapy is associated with immediate and long-term toxicities in children, especially young children. For this reason, it has been the practice of some doctors not to give radiation therapy to children with ependymoma when the tumor has been completely surgically removed. The investigators who designed this study have created strict measures to choose those who will not receive additional treatment after surgery and careful follow-up to minimize the risks to those who are assigned to observation only.
The primary objective of Enroll-HD is to develop a comprehensive repository of prospective and systematically collected clinical research data (demography, clinical features, family history, genetic characteristics) and biological specimens (blood) from individuals with manifest HD, unaffected individuals known to carry the HD mutation or at risk of carrying the HD mutation, and control research participants (e.g., spouses, siblings or offspring of HD mutation carriers known not to carry the HD mutation). Enroll-HD is conceived as a broad-based and long-term project to maximize the efficiencies of non-clinical research and participation in clinical research while ensuring privacy and protections for consenting research participants.
This study is for patients that have been diagnosed with acute myelogenous leukemia (AML) . The investigational drug in this study is AG-120. The purpose of this study is to identify and test the highest dose of AG-120 that can be given safely. Participants can expect to be in the study for as long as the study doctor feels it is in the participants best interest.
The purpose of this study is to use neuroimaging to understand how networks in the brain change over time. Although the single most significant risk factor for developing Alzheimer's disease (AD) is age, the neurobiological processes underlying the transition from normal aging to AD are not well understood. Our group of researchers has developed ways to use MRI to detect small changes in certain parts of the brain. We will use neuroimaging to understand how the connections in the brain change over time in healthy aging. The goal is to discover which brain changes are present in healthy aging.
Participants will have two study visits (about 2 years apart) where they will undergo tests to assess mental function, fill out questionnaires, and undergo a blood draw, brain MRI and PET scan and provide a saliva sample. At the second visit, participants will not repeat PET scan. Participants will continue to be followed longitudinally every two years as long as the study is funded.
Participants are required to have a Co-Participant accompany them for the first portion of each visit. This individual must be a reliable informant that has contact with the participant at least once per week.
This study is for patients that have been diagnosed with multisystem Langerhans cell histiocytosis (LCH). The purpose of this study is to find out if prolonging the treatment and adding a drug called 6-mercaptopurine will be beneficial. Other goals include fining out if these changes will result in fewer patients having relapse of their LCH, and fewer patients having long term disease related problems. Participants can expect to be in this study for up to 24 months and will be followed on this study fir at least 5 years.
This study is for patients that have been diagnosed with advanced prostate cancer with bone metastases.The purpose of this research study is to compare any good and bad effects of using radium-223 along with docetaxel chemotherapy treatment versus using docetaxel alone. The addition of radium-223 to docetaxel could have an overall survival benefit, but it could also cause side effects. The study drugs, radium-223 and docetaxel, are considered experimental drugs that are being evaluated together as a combination therapy for participants with certain solid tumors (cancer). Patients will be seen at MUSC for about 8 months. After they finish the treatment, the doctor will continue to watch the patient for side effects and follow their condition for the rest of their life, or for as long as they wish to remain on the study.
Babies that are born extremely prematurely are at higher risk of developing chronic (long term) lung disease (CLD) and other complications (problems). The purpose of this study is to test the safety and effectiveness of an investigational drug called mecasermin rinfabate (rhIGF-1/rhIGTBP-3) or OHB-607. The researchers want to find out if OHB-607 can help reduce the risk of chronic lung disease in babies born prematurely and if it can help reduce the risk of other complications.
Stroke is a leading cause of disability in the U.S. and many Veteran stroke survivors live with severe disability. Despite recent advances in rehabilitation treatments many stroke survivors have persistent physical and mental difficulties such as reduced arm and leg function, difficulty thinking, and depression.
Developing treatments that address these problems is necessary to improve long-term recovery for stroke survivors. Aerobic exercise (AEx) can improve physical and mental function, and reduce depression. Additionally, AEx may enhance physical rehabilitation by making the brain more receptive to, and consequently improving the response to a rehabilitation treatment. Therefore, combining AEx with physical rehabilitation has the potential to improve multiple parts of stroke recovery. This study will examine the effect of combining AEx with physical rehabilitation on physical and mental function in stroke survivors. By gaining a better understanding of the effects of this combined intervention we aim to advance the rehabilitative care of Veteran stroke survivors.
This is a study looking at the effects of Belimumab, a medication approved by the FDA to treat lupus, in people who have been recently diagnosed with lupus. It proposes that the early use of Belimumab may prevent long-term tissue damage from the disease. The study will last 2 years with clinic visits every 4 weeks.
Underserved, racial and ethnic minority communities are experiencing higher rates of COVID-19 cases and associated mortality compared to whites due to long standing social and structural inequities that also drive disparities in chronic diseases such as stroke, cardiovascular disease, diabetes, and hypertension. Patients with underlying chronic diseases who are recovering from COVID-19 depend on the support of family and friends (informal caregivers/care partners) who are being exposed to the same pandemic and racial stressors, exposure that can affect the health and quality of life of both partners. The primary goal of this study is to test the efficacy of an adapted, telehealth-enhanced intervention that targets barriers impacting family illness management behaviors of Black/African American (AA) adult COVID-19 survivors and carepartner dyads for improved quality of life and COVID/chronic illness health related outcomes.