Children's Hospital Of Philadelphia

This study tests whether a special exercise program combined with a vitamin-like supplement (called NR) can help teens and young adults who had a bone marrow transplant rebuild lost muscle and improve their fitness. About 80 participants, ages 10 to 30, will be randomly assigned …

This study compares two different ventilator approaches for children with acute respiratory distress syndrome (ARDS), a serious lung condition. The goal is to see which method helps children recover breathing faster. About 160 children will take part, and results will help design…

This study tests whether adding specially prepared donor immune cells can help children recover from stem cell transplants faster and avoid dangerous infections. Participants are children under 25 with high-risk blood cancers who have already received a transplant. The goal is to…

This study tests whether adding a special type of immune cell (memory T cells) after a donor stem cell transplant can help prevent serious viral and fungal infections. It involves up to 100 children and young adults with blood cancers or immune disorders. The approach aims to spe…

This early-phase trial tests whether combining the drug cabozantinib with high-dose ifosfamide is safe and effective for children and adults with sarcomas that have returned or not responded to standard treatment. About 30 participants with bone or soft tissue sarcomas will recei…

This study tests whether a mobile health program that helps children sleep longer can lower their blood pressure. Ten children recently diagnosed with essential hypertension who sleep 7.5 hours or less on school nights will use a sleep tracker and receive coaching. The goal is to…

This study tests whether giving targeted drugs before radioactive iodine (RAI) can make thyroid cancer cells absorb more RAI, potentially improving treatment. It includes 32 children and young adults with advanced thyroid cancer that has spread to the lungs. The goal is to see if…

This program provides stem cell transplants for up to 100 children and young adults with life-threatening blood disorders, such as leukemia or immune deficiencies, who do not have a matched sibling donor. The approach uses a special technique to remove certain immune cells from t…

This study is testing a drug called lurbinectedin in people aged 10 and older whose solid tumors (like Ewing sarcoma) have come back or not responded to standard treatment. The goal is to see if the drug is safe and can shrink tumors or delay their growth. Participants must have …

This study tests a specific way of helping children breathe during CPR after a cardiac arrest. The goal is to see if this method helps more children survive and have good brain function. About 1,530 children who need CPR and a breathing tube will take part.

This study is for people with sickle cell disease who need regular blood transfusions. It tests whether giving blood that closely matches the patient's own blood type (called RH genotype matching) can prevent the body from making antibodies that attack donor blood. About 35 parti…

This study tests whether giving a drug called selpercatinib for 6 months before radioactive iodine therapy can improve treatment outcomes in children, teens, and young adults (ages 2-25) with a specific genetic form of thyroid cancer (RET fusion). The goal is to make the cancer a…

This study tests a new gene therapy for adults with transfusion-dependent beta-thalassemia, a severe blood disorder. Doctors take the patient's own blood stem cells, add a healthy gene to make normal hemoglobin, and return the cells. The goal is to reduce or stop the need for reg…

This study tests a new treatment called CART19 for teens and young adults (ages 12-29) with severe lupus that hasn't responded to standard therapies. The treatment uses a patient's own immune cells, modified to target and calm the overactive immune system causing lupus. The goal …

This study tests a program called PrTNER that helps young people aged 15-29 who are at risk for or living with HIV and also have substance use issues. The program provides support to start HIV prevention medicine (PrEP) or to lower the amount of virus in the body for those alread…

This study is testing whether a drug called larotrectinib can make radioactive iodine therapy more effective for people with advanced thyroid cancer that has spread to the lungs. The goal is to see if taking larotrectinib before standard radioactive iodine treatment helps shrink …

This study looks at whether a special ultrasound with contrast dye can find internal belly injuries in children after a hard hit, just as well as a CT scan. CT scans use radiation and can be stressful for kids. The goal is to see if this safer, bedside ultrasound can replace the …

This study is testing whether a special PET-MRI scan using 18F-Fluciclovine can help doctors tell the difference between actual tumor growth and harmless changes that happen after treatment in children with high-grade glioma. About 30 children aged 1 to 21 with this type of brain…

This study looks at whether a home sleep apnea test is as accurate as the standard in-lab sleep study for diagnosing obstructive sleep apnea in children aged 5 to 12. Researchers will also check if the home test helps doctors make the same treatment decisions and if families pref…

This study uses a special PET scan called 18F-DOPA to find tiny insulin-making spots in the pancreas of people with low blood sugar from conditions like congenital hyperinsulinism or insulinoma. The goal is to see if the scan can accurately locate these spots so doctors can remov…

This study explores whether a special ultrasound with contrast dye can safely and accurately diagnose and monitor hydrocephalus (fluid buildup in the brain) in infants under 1.5 years old. The goal is to replace or reduce the need for invasive pressure measurements and shunts. Re…

This study tests whether a 16-week home exercise program, guided by a smartwatch, can help children aged 8-18 with pulmonary hypertension become more active and improve their muscle strength and quality of life. Participants will wear activity trackers and follow a personalized e…

This study tests whether a 12-week physical activity program can help teens (ages 13-18) with chronic kidney disease (CKD) increase their daily moderate-to-vigorous exercise. Researchers will use Fitbits to track activity changes and gather feedback from participants. The goal is…

This study tests whether a home-based digital exercise program can help children aged 10-17 with Fontan circulation (a heart condition) become more physically active and fit. Participants will wear an activity tracker for 12 months and complete fitness tests. Half will also recei…

This study looks at whether massage therapy can improve sleep for children and teens (ages 12-21) who are in the hospital for cancer treatment or a stem cell transplant. Researchers will compare sleep minutes and quality between those who get standard care and those who also rece…

This study is testing a precise type of radiation called proton therapy for children and young adults with kidney tumors like Wilms tumor. The goal is to see if this approach causes fewer short-term side effects compared to standard radiation. About 28 participants under 30 years…

This study tests whether talk therapy can help autistic teens and young adults (ages 16-25) feel more motivated to do daily tasks like chores or hygiene. Researchers compare three approaches: focusing on personal values, using rewards, or a mix of both. Each participant brings a …

This study tests a medical food powder (Encala®) designed to help pancreatic cancer patients absorb fat more easily. Many patients have trouble digesting fat, leading to stomach issues, weight loss, and poor nutrition. The study will enroll 18 adults with pancreatic cancer to see…

This study is testing a group training program for parents of children aged 10-17 who have had chronic pain for at least 3 months. The program aims to help parents better support their child, with the goal of reducing the child's pain and improving their daily functioning. About …

This study looks at whether different types of driver training can help reduce crash risk for new teen drivers. Researchers will follow 1,300 teens aged 16-17 in Pennsylvania who have a learner's permit and plan to get their license. The study uses a smartphone app to track drivi…

This study looks at whether a virtual program can help new mothers with postpartum depression find and use mental health services. Researchers will interview 10 women to understand what makes it hard to get help, then test a tailored online navigation program with 30 women over t…

This study is testing a digital checklist to help doctors and nurses place breathing tubes more safely in children in intensive care units. The checklist uses information from the patient's electronic health record and is tailored to each child and care team. The goal is to reduc…

This study watches how doctors place breathing tubes in children in intensive care and emergency rooms. It aims to find ways to make the procedure safer and reduce complications. Up to 150,000 children will be observed across many hospitals to track problems and improve care.

This study looks at how exercise changes oxygen use in people with mitochondrial disease compared to healthy volunteers. Researchers will measure oxygen consumption and heart function during different types of exercise. The goal is to better understand the disease, not to test a …

This study looks at how sleep duration relates to blood sugar control in 90 adolescents (ages 12-20) with type 2 diabetes. Researchers will measure sleep with a wrist monitor for 14 days and check blood sugar levels. They will also test if offering rewards for meeting sleep goals…

This study looks at ways to help doctors use a preferred asthma treatment called SMART for children with moderate to severe asthma. Researchers will test tools like electronic reminders and education to see if they increase how often doctors prescribe SMART. About 18 clinics and …

This study follows 600 people of any age who have a genetic change in the STXBP1 or SYNGAP1 gene. Researchers will track development, seizures, and quality of life over time using standard tests. The goal is to better understand these rare disorders and prepare for future clinica…

This study looks at whether a special ultrasound can give doctors real-time information about blood flow to the hip joint in infants having surgery for hip dysplasia. About 125 babies aged 4 to 24 months will take part. The goal is to see if this technique can help predict and pr…

This study watches brain oxygen levels and electrical activity in 900 newborns having surgery for birth defects. The goal is to learn when and why brain oxygen drops during and after surgery. This knowledge may help doctors improve care for these fragile infants.

This study looks at how to help doctors prescribe a newer, more effective asthma treatment called SMART for children ages 5 to 18 with moderate to severe asthma. Researchers will test different support strategies, like community health workers and nurse care managers, to see whic…

This study looks at brain chemicals in people with primary mitochondrial disease, a genetic disorder that affects energy production and can damage the nervous system. Researchers will use MRI scans to measure levels of glutathione and other substances in the brain, comparing 30 p…

This study is looking at whether a protein called titin, found in urine, can be used as a marker for muscle injury in people with Duchenne or Becker muscular dystrophy. Researchers will measure titin levels before and after physical activity, and compare them to activity levels r…

This study tests whether a program called PRAISE, led by school staff with coaching from researchers, can reduce bullying and aggression in 3rd-5th grade students. About 1,000 students and their teachers from participating schools will take part. The program teaches kids how to h…

This study is testing whether a special PET-MRI scan using a tracer called 18F-Fluciclovine can better monitor low-grade gliomas (a type of brain tumor) in children and young adults. About 30 participants will get the scan before starting treatment, then again at 3 months and 1 y…

This study follows up to 1300 people with primary mitochondrial myopathy, a rare muscle disease, to learn how it changes over time. Researchers will measure muscle strength, balance, and daily function through exams and surveys. The goal is to create better tools for tracking the…

This study looks at children with irritable bowel syndrome (IBS) to understand how their diet and gut movement affect their symptoms. Researchers will use a wearable patch to track gut activity, collect stool and urine samples, and ask about food intake. The goal is to find patte…

This study tests whether personalized vancomycin dosing can better protect the kidneys of children with sepsis. Vancomycin is an antibiotic that can cause kidney injury if levels get too high. Researchers will use new models that account for each child's kidney function to find t…

This study looks at how to help hospital units reduce the use of vancomycin, an antibiotic, in preterm infants. Researchers will compare units that get extra support to those that do not, to see which approach works better. The goal is to improve antibiotic use and reduce risks f…

This study collects medical information and biological samples from up to 12,000 people with leukodystrophies—rare brain disorders affecting white matter. Researchers will use this data to find new genetic causes, develop biomarkers, and better understand how these diseases progr…

This study uses a special device placed on the belly to record stomach muscle activity in children aged 8 to 25 with digestive symptoms like nausea or pain. The goal is to find patterns that are linked to these symptoms, which could help doctors better understand and diagnose sto…

This study tests whether digital messages (texts, emails, or apps) can encourage teens and young adults who survived childhood cancer to schedule and attend their long-term follow-up appointments. About 530 survivors aged 15-29 who are overdue for care will receive different sequ…

This study looks at brain activity in girls with Rett syndrome to find measurable markers that change with the disease. Researchers will use EEG tests to compare brain signals between girls with Rett syndrome and typically developing girls. The goal is to identify stable markers …

This study is testing a new, tiny oxygen sensor placed in the forearm muscle to see how well it measures muscle function in people with mitochondrial myopathy (a muscle disease caused by energy problems in cells) compared to healthy volunteers. The goal is to find a reliable way …

This early-phase study is testing a new, minimally invasive nanosensor that measures oxygen levels in muscle tissue. Researchers want to see if it can tell the difference between people with mitochondrial myopathy and healthy volunteers. The goal is to develop a reliable tool to …

This study compares two strategies to help hospital staff use a supraglottic airway (a tube placed above the voice box) early during newborn resuscitation. Over 36,000 newborns and their healthcare providers will take part across many hospitals. The goal is to see which strategy …

This study tests a personalized planning tool (PINS Bundle) to see if it can reduce complications when placing breathing tubes in newborns. About 3,000 infants in the NICU will take part. The goal is to improve safety and outcomes during this common procedure.

This study follows 200 people with Alexander Disease, a rare brain disorder, to see how symptoms like movement, speech, and swallowing change over time. Researchers collect samples and test results to better understand the disease. The information gathered will help design future…

This study aims to see if a program that includes guidelines, feedback, and education can reduce the use of vancomycin (a strong antibiotic) in children with suspected sepsis in intensive care units. About 52,500 children will be involved across five hospitals. The goal is to low…

This study watches how doctors perform CPR on children whose hearts have stopped. Researchers will check if following specific guidelines for chest compressions leads to better survival. The goal is to find the best CPR techniques to improve outcomes for kids in cardiac arrest.