Children's Hospital Of Philadelphia

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 kids recover faster and breathe better. About 160 children will take part, and results will help d…

This study tests whether adding a specific type of immune cell (memory T cells) after a stem cell transplant can help prevent serious viral and fungal infections. About 100 children and young adults with blood cancers, immune disorders, or bone marrow failure will receive this ex…

This study tests whether the drug larotrectinib can make thyroid cancer cells more sensitive to radioactive iodine therapy. It includes children and adults with a specific genetic change (NTRK fusion) whose cancer has spread to the lungs. The goal is to see if this combination ca…

This study is for people with sickle cell disease who need regular blood transfusions. It tests whether matching donor blood by a specific genetic marker (RH genotype) can reduce the risk of the body forming antibodies against the transfused blood. About 35 participants will rece…

This study tests a new gene therapy for people with a severe blood disorder called beta-thalassemia, who currently need regular blood transfusions. The therapy involves taking the patient's own blood stem cells, adding a healthy gene to help produce normal red blood cells, and re…

This study tests a new cell therapy called CART19 for teens and young adults (ages 12-29) with severe lupus that hasn't responded to other treatments. The therapy uses the patient's own immune cells to target and reduce lupus activity. The goal is to control the disease and reduc…

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 and who sleep 7.5 hours or less on school nights will use a sleep tracker and receive coaching. The goal i…

This study tests whether a home-based digital exercise program can increase physical activity and fitness in children aged 10-17 with Fontan circulation, a heart condition from single-ventricle defects. Two hundred participants will wear activity trackers for 12 months and be ran…

This study is for children under 25 who have had a stem cell transplant for blood cancers like leukemia. After transplant, their immune system is weak, putting them at high risk for dangerous infections. Researchers are adding back a specific type of donor cell (CD45RA-depleted) …

This program provides stem cell transplants for up to 100 children and young adults with life-threatening blood or immune system diseases who do not have a perfectly matched sibling donor. The transplant uses a special technique to remove certain immune cells from the donated ste…

This early-phase trial is testing whether combining two drugs, cabozantinib and high-dose ifosfamide, is safe and effective for people with sarcomas (a type of cancer) that have returned or not improved after standard treatment. The study includes both children and adults. The ma…

This study tests whether giving targeted drugs before radioactive iodine (RAI) can make thyroid cancer tumors absorb more RAI, potentially improving treatment. About 32 children and teens with metastatic differentiated thyroid cancer and specific genetic mutations will receive ta…

This study tests a special breathing method (called the OPTI-VENT bundle) for children whose hearts stop beating in the hospital. The goal is to see if this method helps more children survive and have better brain function compared to standard care. About 1,530 children receiving…

This study tests whether a tailored exercise program combined with a vitamin-like supplement (NAD+ precursor) can improve muscle mass, strength, and fitness in teens and young adults who had a bone marrow transplant for leukemia or other blood disorders. Participants will be rand…

This study tests whether a program that includes guidelines, feedback, and education can help doctors use less vancomycin (a strong antibiotic) in children with suspected sepsis in intensive care units. The goal is to reduce side effects like kidney damage while still treating in…

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 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 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 looks at whether a home sleep apnea test can work as well as the standard overnight sleep study done in a lab for children ages 5 to 12. Researchers will check how accurate the home test is, whether it helps doctors make the right treatment decisions, and how kids and …

This study tests whether a special ultrasound with contrast dye can safely and effectively diagnose and monitor hydrocephalus (fluid buildup in the brain) in infants under 1.5 years old. Hydrocephalus affects up to 2 in 500 births and often requires a surgically placed shunt, but…

This study tests if a special ultrasound (contrast-enhanced) can detect organ injuries in children with blunt belly trauma as accurately as a CT scan, but without radiation and at the bedside. About 146 stable children who already need a CT will get this ultrasound to compare res…

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 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 tests whether a mobile health app can help teenagers (ages 13–18) who come to the emergency room with a concussion get faster access to a specialist. Participants report their symptoms daily through the app, which alerts doctors if they need follow-up care. The goal is…

This study is testing whether a group-based training program for parents can help children aged 10-17 who have chronic pain (lasting 3 months or more). The program aims to teach parents skills to support their child and reduce pain-related disability. About 48 parent-child pairs …

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 helps teens with chronic kidney disease (CKD) become more active. About 100 teens aged 13-18 with CKD stages II-V (not on dialysis or with a transplant) will wear a Fitbit to track their activity. The goal is to see if …

This study tests three types of talk therapy to help autistic teens and young adults (ages 16-25) feel more motivated to complete daily living skills, like chores or self-care. Each participant picks a goal and brings a trusted person (like a family member or friend) to support t…

This study looks at whether massage therapy can improve sleep for children aged 12 to 21 who are in the hospital for cancer treatment or a stem cell transplant. About 70 participants will either receive standard care or add massage therapy to their routine. Researchers will track…

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 it causes fewer short-term side effects than standard radiation. About 28 people under 30 who need radiation as part of…

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 tests a digital checklist that helps doctors place breathing tubes more safely in children in intensive care. The checklist uses information from the child's medical record to guide the care team. Researchers will track whether this tool reduces serious problems like l…

This study is testing a new, minimally invasive oxygen nanosensor to measure how well muscles use oxygen in people with mitochondrial myopathy (a muscle disease) and in healthy volunteers. The goal is to see if the sensor can reliably detect differences in muscle function, which …

This study watches how doctors place breathing tubes in children in intensive care and emergency rooms. The goal is to track and reduce dangerous events during the procedure. Up to 150,000 children will be observed across many hospitals to find ways to make this common but risky …

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, and how that affects recovery. Babies are monitored before, during, and for 24 hours after surgery using special …

This study looks at why some new mothers with postpartum depression struggle to get mental health care, especially online. Researchers will interview 10 women to understand these barriers, then test a virtual patient navigation program with 30 women over two months. The goal is t…

This study looks at how exercise—both active (like cycling) and passive (using a special bed that gently moves the legs)—affects oxygen use in people with mitochondrial disease compared to healthy volunteers. Researchers will measure oxygen consumption, heart rate, and muscle ene…

This study is creating a large collection of medical data and biological samples from people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this information to discover new genetic causes, develop better diagnostic tools, and track…

This study follows 200 people with Alexander Disease, a rare brain disorder, to learn how the condition changes over time. Researchers measure movement, speech, swallowing, and quality of life, and collect blood and spinal fluid samples. The goal is to gather information that wil…

This study aims to learn more about STXBP1 and SYNGAP1 related disorders, which cause developmental delays, seizures, and intellectual disability. Researchers will follow 600 participants of any age who have a confirmed mutation in one of these genes, tracking their development, …

This study looks at whether different types of driver training can help reduce the risk of crashes for new teen drivers. Researchers will follow 1,300 teens ages 16-17 in Pennsylvania who have a learner's permit. They will track driving habits like hard braking and phone use usin…

This study watches how chest compressions are given to children (up to age 18) during cardiac arrest. Researchers want to see if following American Heart Association guidelines for compression depth, rate, and timing leads to better outcomes. No new treatments are tested—just car…

This study looks at whether a special ultrasound can give surgeons real-time information about blood flow to the hip joint during surgery in infants with hip dysplasia. About 125 infants aged 4 to 24 months will be enrolled. The goal is to see if this technique can help predict a…

This study looks at brain activity in girls with Rett syndrome to find measurable signs (biomarkers) of the disease. Researchers will use EEG tests to see if these brain signals change with the condition and if they are stable over time. About 200 girls and young women, ages 1 to…

This study tests a personalized planning tool to make placing breathing tubes safer for babies in the NICU. Researchers will compare complication rates before and after using the tool in about 3000 infants. The goal is to reduce oxygen drops and other adverse events during the pr…

This study looks at whether giving doctors extra support through their electronic health records can increase the use of a preferred asthma treatment called SMART for children with moderate to severe asthma. The researchers will track how often doctors start and continue SMART th…

This study looks at ways to help doctors prescribe a treatment called SMART for children with moderate to severe asthma. SMART uses one inhaler for both daily control and quick relief. The study will test if extra support like community health workers helps more kids get this tre…

This study looks at two different strategies to help hospital staff use a special breathing device (supraglottic airway) more quickly when newborns need help breathing after birth. Over 36,000 babies and many hospital staff will take part across multiple hospitals. The goal is to…

This study follows 1300 people with primary mitochondrial myopathy, a rare muscle disease, to learn how the condition changes over time. Researchers will measure muscle strength, balance, and daily function, and compare results with healthy volunteers. The goal is to create bette…

This study watches children with irritable bowel syndrome (IBS) to learn how their diet and gut muscle activity relate to their symptoms. Kids wear a small patch, answer questions, and provide stool and urine samples. The goal is to find patterns that could lead to personalized d…

This study looks at whether a program called PRAISE, led by school staff with coaching from researchers, can reduce bullying and aggression among 3rd to 5th graders. About 1,000 students from participating schools will take part. The program teaches kids how to handle anger and s…

This study is testing whether a protein called titin, found in urine, can serve as a reliable marker of muscle injury in people with Duchenne or Becker muscular dystrophy. Researchers will measure titin levels before and after physical activity, both in the clinic and at home, us…

This study looks at whether personalized dosing of the antibiotic vancomycin can reduce kidney injury in children with sepsis. Researchers will use new models that account for each child's kidney function to find the right dose. The trial involves 20 children in intensive care. T…

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 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 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 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 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 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…