In current Dermatology practice, options for vitiligo remain limited. The purpose of this study is to determine if once daily dosed topical rapamycin is effective for the treatment of patients with vitiligo. Participants will apply either 0.1% topical rapamcyin or 0.001% topical rapamycin for six months to a lesion on one side of the body, and topical placebo to a corresponding lesion on the opposite side of the body. The study also aims to evaluate patient satisfaction and identify any adverse effects on these dosing regimens.

This study is for patients that have been diagnosed with non-germinomatous germ cell tumor (NGGCT). The goal of this study is to see if radiation therapy (RT) to the spine and a portion of the brain works just as well as the standard treatment, which includes RT to the whole brain and spine, for people with NGGCT who agree to take part in this study, and whose disease responds well to induction chemotherapy, or who have no signs of disease following surgery. Participants can expect to receive treatment on this study for about 6 to 11 months, depending on which therapy they receive. After study completion, participants will continue to be followed for up to 10 years.

This study is for newly diagnosed previously untreated neurofibromatosis type 1 (NF1) associated with low-grade glioma (LGG). The purpose of this study is to see if selumetinib works just as well as the standard treatment of carboplatin/vincristine (CV) for subjects with NF1-associated LGG, and to see if selumetinib is better than CV in improving vision in subjects with LGG of the optic pathway. Participants can expect to receive treatment for up to 24 months, until the cancer gets worse or until the side effects are too great. After study completion, participants will continue to be followed for up to 10 years.

A prospective parallel cohort study generating two groups of participants will be performed in NEPTUNE. The two groups are: (1) Cohort A which includes the FSGS/MCD Cohort; and the MN Cohort, both incipient and prevalent biopsied patients; and (2) Cohort B – a non-biopsy, treatment-naïve, pediatric cohort less than 19 years of age, cNEPTUNE. The sample size for the combined FSGS/MCD and MN Cohorts is a minimum of 800 participants, with a minimum of 375 new patients recruited under Protocol V5.0. The sample size for the second group, cNEPTUNE, will be a minimum of 200 participants. Participants will be recruited into each subgroup concurrently. All participants who meet the inclusion criteria at the participating centers will be enrolled if the participants or their legally authorized representative(s) provide comprehensive written informed consent. A recruit-to-replace strategy will be employed throughout the enrollment phase. Cohort A study visits including screening/eligibility, baseline, biopsy, and follow-up visits, and SMS texting. Study visits for Cohort B, cNEPTUNE, including screening/eligibility, baseline, follow-up visits, and SMS texting.

Subjects in the first group will be enrolled in the GM-CSF for Reversal of Immunoparalysis in Pediatric Sepsis-induced MODS (GRACE-2) trial, comparing GM-CSF versus placebo. Subjects in the second group will be an observational cohort with no intervention, because this group has very low mortality and morbidity. Subjects in the third group will be enrolled in the Targeted Reversal of Inflammation in Pediatric Sepsis-induced MODS (TRIPS) trial, comparing anakinra and placebo. The fourth group, with very severe inflammation,
will be an observational cohort because clinical management of the inflammation
is standard of care, and there is no equipoise about enrolling these children in a placebo controlled trial. The primary outcome of both trials will be duration and severity of organ dysfunction using the cumulative PELOD-2 score, and secondary outcomes will assess health related quality of life and family functioning at 3 and 12 months.

This study is for newly diagnosed or previously untreated low-grade glioma (LGG). The overall goal of this study is to see if selumetinib works just as well as the standard treatment using carboplatin and vincristine (called CV) for subjects with LGG. Selumetinib is a drug that works by blocking a protein (a basic building block of the human body) that lets cancer cells grow without stopping. Participants can expect to receive treatment on this study for about 1–2 years. After study completion, participants will continue to be followed for up to 10 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 study is for participants with tumors from pediatric cancers and genomic/molecular testing was done as part of standard of care treatment. This is an observational study; therefore, only information about the disease and medical treatment will be collected and participants will not receive any treatments or additional medications. The sponsor, Beat Childhood Cancer, will collect and store personal health information and molecular/genomic test results, tissue samples, and bodily fluids (examples: additional tube(s) of blood, urine, bone marrow or cerebral spinal fluid) that are left over after testing or treatment is completed in a data registry and a specimen bank, and make these available for future research. Database personnel will continue to collect and store participant information from future visits, as long as they do not withdraw from participation in this study.

The purpose of this research study is to develop a better understanding of the cause and natural history of vascular anomalies and related syndromes. This study is being done in order to develop a better understanding of the cause of vascular anomalies in order to to improve care for people who are affected by these anomalies and related syndromes.

This study is being done at the University of Wisconsin-Madison (UW-Madison) and other sites in North America and Europe. A total of about 1000 people will participate in this study. About 20 – 30 people will take part in the study here at the Medical University of South Carolina.

The purpose of this research study is to evaluate Parent Child Interaction Therapy (PCIT) delivered via tele-health for young children with a developmental diagnosis (ex. suspected or diagnosed autism, ADHD, global developmental delay, etc.) and disruptive behavior problems. Participants will go through a screening to determine eligibility. Once screening is complete, participants will complete a pre-therapy assessment in clinic, followed by 10 telehealth sessions one time per week, at no cost. Participants will then be asked to complete an in-person post-therapy assessment and follow-up questionnaires will be re-administered 3 months following the completion of therapy. Families will be compensated for their time.