Children's Hospital Of Philadelphia

This early-stage study is testing a new, one-time gene therapy for people with severe beta-thalassemia, a blood disorder that requires lifelong blood transfusions. Doctors will collect a patient's own blood stem cells, add a healthy gene in the lab, and then return them. The goal…

This study is testing a new program to help doctors in pediatric intensive care units (PICUs) use a powerful antibiotic called vancomycin more carefully. The goal is to reduce unnecessary prescriptions, which can cause kidney damage, while still effectively treating children with…

This study is testing if a combination of home-based exercise training and a dietary supplement can help young people who have survived a bone marrow transplant. The goal is to rebuild lost muscle mass and improve their overall fitness and heart health. Researchers will compare t…

This study is testing whether personalized dosing models can help doctors give the right amount of vancomycin antibiotic to children with sepsis. Researchers want to see if using these models helps achieve effective drug levels while reducing the risk of kidney injury. The study …

This trial provides expanded access to stem cell transplants for children with serious blood, immune, or metabolic diseases who lack a perfectly matched sibling donor. It uses a special device to remove certain cells from the donor's blood to reduce the risk of a dangerous compli…

This study aims to find the best way to manage breathing machines for children with a severe, life-threatening lung condition called Acute Respiratory Distress Syndrome (ARDS). It will compare two different ventilator approaches in 160 children to see which one helps them recover…

This study aims to help hospitals safely use fewer antibiotics in premature babies. It tests two different support methods to help neonatal intensive care units (NICUs) follow new guidelines for reducing a specific antibiotic called vancomycin. The goal is to control antibiotic u…

This study aims to help doctors more consistently use a recommended treatment strategy for children with moderate to severe asthma. It will test different support methods, like using electronic alerts and adding community health workers, to see which helps clinics adopt the treat…

This study is testing whether a drug called larotrectinib can make radioactive iodine treatment more effective for people with advanced thyroid cancer that has spread to the lungs. Participants will take larotrectinib for six months, then receive standard radioactive iodine thera…

This study is testing if a 12-week online exercise program can help teens with chronic kidney disease become more active. Researchers want to see if getting teens to do just 15 more minutes of moderate activity each day can improve their health and quality of life. The program us…

This study is testing a new method to help patients recover their immune systems faster after a stem cell transplant for leukemia. It involves giving patients a special, filtered portion of their donor's immune cells, aiming to reduce the risk of life-threatening viral and fungal…

This study is for children and young adults with advanced thyroid cancer that has spread to the lungs. Researchers want to see if taking new 'targeted' drugs for about a month makes the cancer absorb radioactive iodine better. If it works, this could help doctors use a standard t…

This study is testing a new, standardized approach to giving breaths during CPR for children who suffer cardiac arrest in the hospital. It aims to see if this method helps more children survive and leave the hospital with good brain function. The research will involve over 1,500 …

This study is testing whether digital tools like text message reminders and online resources can help young adults who survived childhood cancer return to important long-term follow-up doctor visits. Researchers want to see which combination of reminders and support works best to…

This study is testing a personalized cell therapy called CART19 for adolescents and young adults with severe lupus that hasn't responded to standard treatments. Doctors will collect a patient's own immune cells, modify them to target lupus-causing B cells, and reinfuse them. The …

This study aims to help doctors better use a recommended asthma treatment plan for children with moderate to severe asthma. It will test whether providing doctors with computer alerts and education increases how often they prescribe this plan. The study involves children aged 5-1…

This study is testing a new safety checklist and planning tool designed to reduce complications when doctors place breathing tubes in critically ill newborns. It involves 3,000 infants across eight hospitals. The goal is to see if this personalized plan leads to fewer problems du…

This study compares two different training programs to help hospital staff quickly use a special breathing tube when a newborn is struggling to breathe. It involves over 36,000 cases across many hospitals to see which training method gets the device used more often and more relia…

This study is testing a drug called lurbinectedin to see if it is safe and can control tumors in children and adults (age 10+) whose Ewing sarcoma or related cancers have come back after initial treatment. The drug is designed to target specific genetic changes that drive these t…

This early-stage study is testing the safety and initial effectiveness of combining two drugs, cabozantinib and high-dose ifosfamide, for children and adults whose bone or soft tissue sarcomas have returned or not responded to standard chemotherapy. The main goal is to find the s…

This study is testing if a drug called selpercatinib can make a standard thyroid cancer treatment (radioactive iodine) work better for children and young adults. It focuses on patients aged 2-25 with a specific genetic change (RET fusion) in their cancer. The goal is to see if ta…

This study is testing whether a phone-based program that encourages teenagers to sleep longer can help lower their high blood pressure. Researchers are enrolling 10 teens (ages 13-18) who were recently diagnosed with high blood pressure and who get less than 7.5 hours of sleep on…

This study is testing a computer-based decision tool to help primary care doctors better manage children with concussions. The tool helps doctors identify which kids are at higher risk for longer-lasting symptoms so they can be referred to specialists sooner. Researchers will see…

This study is testing whether a special type of PET scan using a tracer called 18F-fluciclovine can help doctors more accurately tell if a child's aggressive brain tumor is actually growing again or if the changes seen on a regular MRI are just a temporary reaction to treatment. …

This study is testing a new, tiny sensor designed to measure oxygen levels inside muscle tissue. Researchers want to see if it can reliably tell the difference between healthy people and those with mitochondrial myopathy, a rare muscle disease. The goal is to create a better tool…

This study is testing a special type of ultrasound scan to see if it can safely and effectively help doctors better understand brain health in babies with hydrocephalus, a condition where fluid builds up in the brain. Researchers will use an FDA-approved contrast agent to get cle…

This study is testing a new type of ultrasound to see if it can safely replace CT scans for finding internal injuries in children who have suffered blunt force trauma to the abdomen. Researchers will compare the new bedside ultrasound, which uses a safe contrast dye and no radiat…

This study is testing whether a new type of brain scan can help doctors better see how well treatments are working for children and young adults with low-grade brain tumors. Participants will get the special scan before starting treatment and again at 3 months and 1 year. Researc…

This study is testing whether a school-based coaching program called PRAISE can reduce aggression and bullying among elementary school students. The program trains school staff to teach 3rd-5th graders skills for managing emotions, resolving conflicts peacefully, and standing up …

This study is testing a virtual support program to help mothers with postpartum depression access mental health services. First, researchers will interview 10 women to understand barriers to getting help online. Then, 30 women with depression symptoms will work with a virtual 'na…

This study is testing three different talk therapy programs to help autistic teens and young adults (ages 16-25) feel more motivated to do everyday tasks they know how to do but struggle to complete regularly. Participants will work with a therapist and a chosen support person ov…

This study is testing a new digital checklist designed to help medical teams place breathing tubes more safely in critically ill children. It will be used in six pediatric intensive care units (ICUs) with 3,000 children who need this procedure. The goal is to see if this smart ch…

This study aims to understand how the quality of chest compressions during CPR and the care provided after a child's heart stops affect their survival and brain function. Researchers will observe 7,000 children across multiple hospitals who receive CPR, measuring details like com…

This study aims to identify brain activity patterns that could serve as markers for Rett Syndrome, a rare genetic disorder affecting brain development. Researchers will measure electrical brain signals using EEG tests in girls with Rett Syndrome and compare them to typically deve…

This study aims to understand what makes IBS symptoms better or worse in children. Researchers will observe 90 kids, some with IBS and some without, to see how their diet, gut movement patterns, and gut bacteria relate to their symptoms. Participants will wear a special patch, an…

This study aims to understand how the body uses energy during exercise in people with primary mitochondrial diseases, which cause severe muscle weakness and fatigue. Researchers will compare 90 participants—some with the disease and some healthy—during different activities, inclu…

This study aims to make placing breathing tubes in critically ill children safer by observing current practices across many hospitals. It will collect data on over 150,000 procedures in pediatric and neonatal intensive care units, emergency rooms, and delivery rooms. The goal is …

This study aims to understand how mitochondrial myopathy, a rare muscle-weakening disease, progresses over time. Researchers will track 1300 patients and healthy volunteers to develop better tools for measuring symptoms like muscle strength, balance, and daily function. The goal …

This study aims to find a better way to measure muscle damage in boys with Duchenne or Becker Muscular Dystrophy (DMD/BMD). Researchers are testing if a protein called titin, found in urine, changes after physical activity and relates to disease severity. The goal is to create a …

This study aims to better understand Primary Mitochondrial Disease (PMD), a rare genetic disorder that affects the brain and nervous system. Researchers will use non-invasive MRI brain scans to measure specific chemicals in the brains of 30 people with PMD and compare them to hea…

This study aims to collect medical information and biological samples from 12,000 people worldwide with rare genetic brain disorders called leukodystrophies. Researchers will use this data to better understand these diseases, improve diagnosis, and identify targets for future tre…

This study is testing a new, minimally invasive sensor designed to measure oxygen levels in muscle tissue. Researchers want to see if this sensor can reliably detect differences between people with mitochondrial myopathy and healthy volunteers. The goal is to create a better tool…

This study aims to learn more about two rare genetic brain disorders, STXBP1 and SYNGAP1, which cause severe developmental delays and epilepsy. Researchers will follow 600 participants for up to five years, tracking their symptoms and quality of life through regular check-ups and…

This study aims to understand stomach activity in children with gastrointestinal (GI) symptoms like nausea and pain. Researchers will use a non-invasive device placed on the skin to record stomach electrical patterns for up to 4 hours in both healthy kids and those with GI disord…

This study aims to understand the natural progression of Alexander Disease, a rare disorder that affects the brain and nervous system. Researchers will follow 200 patients over time, tracking changes in their movement, speech, swallowing, and quality of life. The information gath…