Children's Hospital Of Philadelphia

This pilot trial tests whether feeding directly into the small intestine (transpyloric) is better than feeding into the stomach (gastric) for very preterm infants with bronchopulmonary dysplasia, a chronic lung condition. The study will measure how often babies have low oxygen le…

This study tests whether the antibiotic rifampin can safely lower high calcium levels in the blood and urine of people with a rare genetic mutation in the CYP24A1 gene. The condition can cause kidney stones and other problems. Sixty participants aged 6 months to 65 years will tak…

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

This study tests whether specially grown immune cells (called cytotoxic T lymphocytes) can safely lower BK virus levels in 20 transplant patients with weakened immune systems. Participants have had a stem cell or kidney transplant and show signs of BK virus infection. The goal is…

This study tests a program called PrTNER that helps young people aged 15-29 who are at risk for HIV or already living with it, and who also have substance use issues. The program provides prevention, treatment, and support to help them start HIV prevention medication (PrEP) or lo…

This early-phase trial tests whether using personalized dosing models for the antibiotic vancomycin can improve treatment in children with sepsis. The standard dosing approach may lead to blood levels that are too high (risking kidney injury) or too low (risking ineffective treat…

This study is testing whether giving blood transfusions that match the patient's own RH genes can prevent the body from making harmful antibodies. About 35 people with sickle cell disease who need regular transfusions will receive specially matched donor blood. The goal is to see…

This study tests whether giving the targeted drug selpercatinib for 6 months before standard radioactive iodine therapy can improve outcomes for children, adolescents, and young adults (ages 2-25) with a specific type of thyroid cancer linked to RET gene fusions. The goal is to s…

This study tests whether adding memory immune cells (CD45RO) to a stem cell transplant can help children fight off dangerous viral and fungal infections after the procedure. The transplant uses donor stem cells that have been stripped of certain cells to prevent graft-versus-host…

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 whether a home-based digital exercise program can increase physical activity and fitness in children aged 10-17 with Fontan circulation, a heart condition. Participants will wear an activity tracker for 12 months and complete fitness tests. The goal is to see if …

This study tests a drug called lurbinectedin in people aged 10 and older whose solid tumors (like Ewing sarcoma) have returned after standard treatment. The goal is to see if the drug is safe and can shrink tumors or delay their growth. Participants must have a specific genetic c…

This study looks at two different ways to use ventilators for children with acute respiratory distress syndrome (ARDS), a serious lung condition. The goal is to see which method helps children recover faster and breathe better. About 160 children will take part, and the results w…

This study tests a personalized safety bundle (PINS) designed to reduce complications when placing a breathing tube in newborns in the NICU. The bundle includes a tailored plan covering risk assessment, when to intubate, medications, equipment, and provider expertise. Researchers…

This study tests whether giving children with leukemia a special type of donor immune cell (CD45RA-depleted) after a stem cell transplant can help their immune system recover faster and lower the risk of serious viral or fungal infections. The trial enrolls 50 children under 25 w…

This expanded access program offers a stem cell transplant using donor cells that have been specially processed to remove certain immune cells, reducing the risk of graft-versus-host disease. It is for patients with serious blood disorders, immune deficiencies, or metabolic disea…

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 guidance to extend their…

This study tests whether a tailored exercise program combined with a dietary supplement called nicotinamide riboside (NR) can improve muscle mass, strength, and fitness in adolescents and young adults who had a bone marrow transplant for leukemia or other blood disorders. About 8…

This study tests whether giving targeted drugs before radioactive iodine (RAI) can make thyroid cancer tumors absorb more RAI, potentially improving treatment. It includes 32 children and teens with metastatic differentiated thyroid cancer that has spread to the lungs. Researcher…

This phase 2 trial tests whether giving larotrectinib for 6 months can make thyroid cancer cells more responsive to radioactive iodine therapy. It enrolls 13 children and young adults with a specific genetic change (NTRK fusion) whose cancer has spread to the lungs. The goal is t…

This early-phase trial tests the safety and best dose of cabozantinib combined with high-dose ifosfamide in 30 children and adults with sarcomas (like Ewing sarcoma or osteosarcoma) that have returned or not responded to standard chemotherapy. The goal is to find a dose that cont…

This study tests a special breathing method (ventilation bundle) for children whose hearts stop beating in the hospital. The goal is to see if it helps more children survive and have better brain function. About 1,530 children who need CPR and a breathing tube will take part. The…

This study tests whether a combination of clinical guidelines, feedback, and education can reduce the overuse of vancomycin in children with suspected sepsis in intensive care units. Over 52,000 patients and many clinicians across five hospitals will take part. The goal is to low…

This study tests whether a special ultrasound with a contrast dye can safely and effectively measure brain health in infants with hydrocephalus, a condition where fluid builds up in the brain. About 20 infants under 1.5 years old will receive an injection of the contrast agent th…

This study offers expanded access to a PET scan using a radioactive tracer called 18F-DOPA to locate small, insulin-producing lesions in the pancreas. It is for patients with congenital hyperinsulinism, Beckwith-Wiedemann syndrome, or insulinoma who have low blood sugar. The goal…

This early-phase trial at Children's Hospital of Philadelphia is testing whether a radioactive tracer called 18F-Fluciclovine (Axumin) can improve PET-MRI scans for children and young adults with low-grade glioma. About 30 participants will receive the tracer before starting trea…

This study looks at whether a home sleep apnea test is as accurate as the standard overnight sleep lab test for children ages 5 to 12. Researchers will compare results from both tests and see how they affect treatment choices. The goal is to find a more comfortable and convenient…

This study tests whether a special ultrasound with contrast dye can show blood flow to the hip joint in real time during surgery for hip dysplasia in infants. About 125 infants aged 4 to 24 months will receive the ultrasound during their hip reduction surgery. The goal is to see …

This study is testing a small device called a nanosensor that measures oxygen levels in muscle tissue. The goal is to see if it can accurately assess mitochondrial function in people with mitochondrial myopathy compared to healthy volunteers. If it works, this sensor could become…

This early-stage study tests a small nanosensor placed under the skin in the forearm to measure oxygen levels in muscle, which reflects how well mitochondria are working. Researchers will compare results from 24 people—some with mitochondrial myopathy and some healthy—to see if t…

This study looks at whether a special ultrasound with contrast dye can find internal belly injuries in children after a hard hit or fall, just as well as a CT scan. CT scans use radiation and require moving the child, while this ultrasound can be done at the bedside with no radia…

This study tests whether a special PET scan called 18F-Fluciclovine can help doctors tell the difference between actual tumor growth and harmless changes caused by treatment in children with high-grade glioma. About 30 children aged 1 to 21 will receive the scan after radiation t…

This pilot study tests a depression prevention program called Brief Interpersonal Psychotherapy-Adolescent Skills Training (B-IPT-AST) for teens aged 13-17 with mild symptoms. The program includes 6 sessions (4 group, 2 individual) and is compared to usual services. Researchers w…

This study is testing a mobile health platform designed to help children ages 8-12 who don't get enough sleep (less than 8.5 hours per night) sleep longer. The goal is to see if improving sleep can help prevent obesity. The study will enroll 5,000 children and track their sleep d…

This study tests whether daily symptom tracking via smartphone can help teens with concussion get quicker access to specialty care. About 210 teens aged 13-18 who visit the emergency department with a concussion and are at high risk for long-term symptoms will report their sympto…

This study tests a special medical food powder (Encala®) in 18 adults with pancreatic cancer who have trouble absorbing fat, causing stomach issues and weight loss. The goal is to see if the powder is easy to take and helps improve fat absorption and quality of life. This is an e…

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 a group-based parent training program called SPACE-Pain, originally developed for anxiety, to see if it is feasible and acceptable for parents of children aged 10-17 with chronic pain. The program teaches parents to reduce accommodation while staying warm and sup…

This study tests whether regular massage therapy can improve sleep for children aged 12-21 who are hospitalized for cancer treatment or stem cell transplant. Seventy participants will either receive standard care or a 20-30 minute massage five days a week for three weeks. Sleep w…

This study is testing a precise form of radiation called proton beam 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 will rece…

This study tests whether a program called PRAISE, led by school staff, can reduce bullying and aggression in 3rd-5th graders. Over 1,000 students will participate. The program teaches social and emotional skills to help kids handle peer conflicts without aggression. Researchers w…

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. Participants choose a goal and bring a family member or friend to support them. The goal is to see if f…

This study tests whether a 12-week physical activity program can help teens aged 13–18 with chronic kidney disease (stages II–V) become more active. Participants will wear a Fitbit to track their moderate-to-vigorous exercise. The goal is to see if the program increases daily act…

This study explores the link between sleep duration and blood sugar control in adolescents aged 12-20 with type 2 diabetes. Researchers will first measure sleep and blood sugar in 67 teens, then test a program using Fitbits and text messages to encourage longer sleep in 15 teens …

This study observes critically ill children in the ICU who are already undergoing continuous EEG monitoring to detect brain seizures. Researchers aim to identify which children are most at risk for seizures and evaluate the safety of current seizure management strategies. The goa…

This study creates a registry to collect health information from people with Autosomal Dominant Polycystic Kidney Disease (ADPKD), the most common genetic cause of kidney failure. While ADPKD was once thought to only affect adults, researchers now know it begins in childhood, but…

This study is creating a central database and tissue bank for rare diseases affecting the kidneys and liver, such as ARPKD and Joubert syndrome. Researchers will collect medical information, genetic samples, and tissues from 200 participants to help doctors and scientists better …

This study is building a large collection of medical data and biological samples from 1,000 people with synovial sarcoma, a rare cancer. Researchers will use this information for future genetic studies to better understand the disease and improve treatments. Participants provide …

This study monitors brain oxygen levels and electrical activity in 900 newborns undergoing surgery for birth defects. Using special sensors, researchers track changes before, during, and after surgery to understand when the brain may not get enough oxygen. The goal is to identify…

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 activities like walking down stairs, and track daily …

This study looks at whether online driver training can help reduce crash risk for new teen drivers. Researchers will enroll 1,300 adolescents aged 16-17 in Pennsylvania who have a learner's permit. Participants will complete online safety education or skills training, and their d…

This study compares two training and support strategies to help hospital staff use a breathing device called a supraglottic airway (SA) during newborn resuscitation. Over 36,000 newborns and their care teams across many hospitals will take part. The goal is to see which approach …

This study tests whether simple digital tools—like letters and text messages—can encourage adolescent and young adult survivors of childhood cancer to schedule and attend long-term follow-up appointments. About 530 participants who are overdue for care will receive different comb…

This observational study aims to understand stomach electrical activity in children aged 8-25 with digestive issues like slow stomach emptying or chronic pain. Researchers will use a non-invasive device (BSGM) to record stomach signals for up to 4 hours and compare them to health…

This study uses MRI brain scans to measure chemicals like glutathione in people with primary mitochondrial disease, a genetic disorder that affects energy production and can damage the brain. Researchers will compare 30 participants with the disease to healthy volunteers to see i…

This study follows 200 people with Alexander Disease, a rare brain disorder, to learn how the disease 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 will …

This study tracks a quality improvement project across many children's hospitals to see if new safety steps reduce complications when placing breathing tubes in children. Researchers will compare the number of tube-related problems before and after the changes. Up to 150,000 chil…

This study looks at how to help hospital units reduce the use of vancomycin, an antibiotic, in preterm infants. Researchers will compare two approaches to implementing new guidelines across 13 NICUs. The goal is to see which method works better at safely lowering antibiotic use.

This study at Children's Hospital of Philadelphia is observing 90 children aged 8-18 with and without irritable bowel syndrome (IBS). Participants wear a wireless patch on their belly to measure gut muscle activity, record their diet, and provide stool and urine samples. The goal…

This study looks at whether a special bed that gently moves your legs (passive exercise) can improve how the body uses oxygen in people with mitochondrial disease. Researchers will compare patients to healthy volunteers and also test the bed in children in the ICU. The goal is to…

This study is observing 600 people of any age with STXBP1 or SYNGAP1 gene mutations to better understand how these disorders affect development, seizures, and quality of life. No treatment is given; instead, researchers will collect data from routine clinical assessments over tim…

This study collects medical information and biological samples (like blood or tissue) from up to 12,000 people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this data to find new genetic causes, develop biomarkers for future trial…

This study tests a digital 'Smart Checklist' designed to help doctors and nurses safely place breathing tubes in children in intensive care. The checklist pulls patient information from electronic health records and gives real-time warnings about high-risk situations. Researchers…

This study looks for brain activity patterns (biomarkers) in girls with Rett syndrome. Researchers will use EEG to measure brain waves and see if these patterns change with symptoms or stay stable over time. About 200 girls with Rett syndrome and typically developing girls will t…

This study watches how doctors and nurses perform CPR on children who have had a cardiac arrest. Researchers will check if following American Heart Association guidelines for chest compression depth, rate, and timing leads to better outcomes. Up to 7,000 children across multiple …

This study tests two strategies to help doctors prescribe SMART therapy—a single inhaler for daily control and quick relief—for children with moderate to severe asthma. Researchers will use electronic reminders and education, plus a community health worker and nurse care manager,…

This study tests whether giving doctors extra training and electronic reminders helps them prescribe a modern asthma treatment called SMART for children with moderate to severe asthma. The study involves 18 children ages 5 to 18 at primary care clinics. Researchers will compare h…

This observational study aims to better understand primary mitochondrial myopathy, a rare muscle disease. Researchers will follow 1300 patients and healthy controls, measuring muscle strength, balance, and daily function over time. The goal is to develop and validate tools to tra…