Children with injury to one side of the brain (stroke, cerebral palsy, or traumatic brain injury) often have difficulty with many life activities due to problems with their ability to pay attention and also problems with keeping their balance. This project will help explain how a child's problems with paying attention impacts their ability to keep themselves safe while standing and moving during childhood life activities. This study will have children participate in a several different assessments to evaluate how they are paying attention, how well they keep their balance, how well they are able to move and play, and will ask parents to complete a couple of surveys to provide information about what the children are like at home. This study will consist of one visit to MUSC campus that will take about 2 hours to complete the testing.
This study is going to test a cold atmospheric plasma device (CAP), in particular a floating electrode-dielectric barrier device (FE-DBD), to treat warts and molluscum. The treatment device in this study generates cold atmospheric plasma (gaseous ionized molecules) to rid the virus from the body. Based on the successes of previous dermatologic studies, FE-DBD is being tested for this study to treat warts and molluscum. Patients will be enrolled to test the efficacy and safety of this device.
Patients aged between 4-21 years are eligible to participate. The duration of the study is 4-12 weeks depending on treatment clearance. The number of lesions will be chosen by the dermatologist. Patients will receive standard of care therapy and/or NTAP depending on the number of lesions. Safety profile and patient tolerability will be measured, as well as changes in size, pain and appearance. Photographs and clinical impression will be used to measure treatment response. Pain and tolerability will be assessed by a visual analogue scale and tolerability assessment scale.
The standard treatment for growth hormone deficiency is daily injections under the skin of recombinant human growth hormone (rhGH). LUM-201 is an investigational drug (not approved by the US Food and Drug Administration) thought to increase the body's ability to release growth hormone. This study seeks to determine if oral LUM-201 at various doses may achieve similar catch-up growth compared to rhGH and provide a safe and effective treatment alternative to daily injections. Participant enrollment for the study will be for 8 months with 7 study visits.
This is a randomized Double-blind Placebo-controlled Phase 3 Study to determine whether a study drug called maralixibat is safe and effective in treating itchy skin (pruritus) in children with Progressive Familial Intrahepatic Cholestasis (PFIC). There is currently no treatment approved for PFIC and available medical approaches have limited success.
Randomized means by chance, like the flip of a coin. Subjects will be randomly assigned to one of the two arms of the study. Subjects will have a 50:50 chance of receiving maralixibat as compared to placebo.
Double blinded means a study in which neither the investigators nor the subjects know which intervention the subject is receiving.
A Phase 3 study is large scale trial to confirm and expand information on safety and usefulness of a new drug.
Subjects who qualify to participate in this study will be randomly assigned to receive the study drug or a placebo. The dose of the study drug will be gradually increased during the study upto a tolerable dose . Subjects will continue to take the medication for about 5 months. Subjects will have approximately 9 study visits over 8 months in this study. Subjects will also be asked questions about health and complete questionnaires at the study visits and as well as at home. A Physical exam, Electrocardiogram, Liver ultrasound will be performed at some study visits. A blood and urine sample will also be collected at some of the study visits.
It is possible that the maralixibat may or may not improve symptoms from PFIC. Even if there is no benefit, other children may benefit from what is learned in this study.
In current practice, options for venous and lymphatic malformations remain limited. Recently an oral medication, sirolimus, has been found to benefit patients when taken once or twice a day for several months. Unfortunately there are many side effects associated with this medication, some of which can be severe including, neutropenia, oral ulcerations, and lab abnormalities. The purpose of this study is to determine if once weekly dosed sirolimus will be effective for the treatment of venous and lymphatic malformations. Additionally, the study will evaluate patient satisfaction and identify adverse effects. Participants will be on the medication for 6 months with an option to continue after this time period.
Babies that are born extremely prematurely are at higher risk of developing a serious inflammatory condition, called necrotizing enterocolitis (NEC), that damages parts of the intestines. It is the most common cause of illness of the intestinal tract in premature infants in the first few weeks of life. It is a very serious condition that can lead to death in up to half of affected infants.The main purpose of this study is to see if IBP-9414 ( a probiotic) can prevent NEC and if it is safe and tolerable.
IBP-9414 (Lactobacillus reuteri) is a live bacterial therapy given to infant's through a feeding tube or it is given by mouth once daily starting at or before 48 hours of life. IBP-9414 will be given daily until the infant reaches 34 weeks + 6 days Post-Menstrual Age (PMA) (the the baby's gestational age at birth plus the postnatal days of life), or until treatment is permanently discontinued if earlier.
This study is for patients that have been diagnosed with a hepatic malignancy. The purpose of this research is to reduce therapy associated toxicity for patients with non-metastatic hepatoblastoma (HB) and hepatocellular carcinoma (HCC) without adversely affecting long term outcomes. Participants can expect to receive treatment for up to 3 months. After treatment, participants will be followed for up to 10 years.
This study is for participants that have been diagnosed with a high-risk neuroblastoma that cannot be treated or did not improve with existing therapies, or the cancer came back after treatment with existing therapies. This study involves investigational drugs called 64Cu-SARTATE and 67Cu-SARTATE. The investigational drugs will be given as an IV injection. The study is divided into 2 parts: The Dose Escalation Phase and the Cohort Expansion Phase. The phase that participants will enroll to will depend on when they enter the study. The age range for participants is from 12 months to 25 years. Participants can expect to be in this study for approximately 14 months.
Growth hormone may increase the size of tonsils in the airway. Children who take growth hormone may be at an increased risk for sleep apnea. The goal of this study is to check for sleep problems in children before and after starting growth hormone. This research will also help us know if and when other children who start growth hormone need to be checked for sleep problems.
This study is for patients that have newly diagnosed High-Risk B-ALL, Risk-Adapted Post-Induction therapy for High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and Disseminated B-LLy. The treatment involves medicine called chemotherapy, which fights cancer. Some patients may also need radiation therapy depending on whether the cancer has spread to the brain and spinal fluid, or the testes for males. The investigational drug on this study is inotuzumab ozogamicin. Participants can expect to be on this study for a little over 2 years and followed for up to 10 years.