Boston Children's Hospital

This study tests whether a bone marrow transplant from an unrelated donor works better than standard immune-suppressing drugs as the first treatment for children and young adults with severe aplastic anemia, a rare condition where the body stops making enough blood cells. About 5…

This study tests whether giving red blood cell transfusions only when a child is bleeding or not getting enough oxygen (instead of using a fixed hemoglobin level) can reduce organ damage and improve brain development. It involves 228 critically ill children under 6 years old who …

This study looks at a surgery called ETV/CPC as an alternative to standard shunts for infants with hydrocephalus in Uganda. Shunts often fail and need lifelong care, but ETV/CPC may free patients from that burden. Researchers will follow 400 infants to see how well the surgery wo…

This study looks at whether people aged 12 and older with a specific gene change (IL-4RαR576) get more help from the drug dupilumab to control their asthma. Over 48 weeks, researchers will count how many severe asthma attacks happen and check lung function. The goal is to see if …

This study tests a combination of two drugs, everolimus and lonafarnib, in children with progeria, a rare disease that causes rapid aging. The goal is to find the safest dose of everolimus when added to lonafarnib and see if the combination helps with weight gain and blood vessel…

This study tests a special cartridge that pre-digests fat in tube feeding for children with short bowel syndrome who rely on IV nutrition. The goal is to see if the device is safe and helps kids absorb more nutrients, possibly reducing their need for IV feeding and lowering the r…

This study tests a daily solution called T-EDTA (Kitelock™) placed into the central lines of children who need IV nutrition. The goal is to prevent blood clots and serious bloodstream infections without damaging the catheter. Up to 15 children aged 3 months to 18 years with a his…

This study tests a bone marrow transplant method that avoids harsh drugs and radiation, which can worsen lung and liver disease or raise cancer risk in people with dyskeratosis congenita. About 40 participants with bone marrow failure will receive this gentler approach. The goal …

This study tests whether a drug called omalizumab can prevent asthma from developing in young children at high risk. The trial involves 200 children aged 2-3 years old who have had wheezing episodes and allergies. They receive the drug or a placebo for two years, then are monitor…

This pilot study tests whether adding mental skills coaching to standard care helps adolescent athletes feel more confident returning to sports after ACL reconstruction. About 30 teens and young adults will receive the coaching and complete questionnaires measuring psychological …

This study looks at whether a 6-week, self-guided online education program can help Boston Marathon runners with patellofemoral pain (runner's knee) feel better and run better. About 104 runners will be split into two groups: one gets the program before the marathon, the other ge…

This study tests a digital platform called aflo™ that attaches to asthma inhalers to give real-time feedback on technique and medication reminders. About 26 children and adults with uncontrolled asthma will use the device for 3 months. The goal is to see if it improves how well t…

This study tests a program called MOMENT-V that combines two counseling sessions with a smartphone app to help young adults reduce cannabis use and related problems. About 65 participants from a Boston Children's Hospital clinic will either get the full program or a brief counsel…

This early-stage trial tests whether metformin, a common diabetes drug, is safe and practical for infants who suffered brain injury from oxygen loss at birth. About 30 babies aged 3-6 months will receive the drug to see if it can support brain repair and improve development. The …

This study planned to see if a TENS device (a small gadget that sends gentle electrical pulses through the skin) could reduce pain and improve quality of life in teens and young adults with endometriosis-related pelvic pain. It was designed for people aged 13-25 with a confirmed …

This small pilot study at Boston Children's Hospital tests whether a handheld device that guides deep breathing can help children with ADHD pay attention and control impulses. Ten children aged 7 to 11 will try the tool and then complete computer tasks to measure focus. The goal …

This study was designed to see which of two nerve blocks works better for pain relief after hip surgery in teens and young adults. It was withdrawn before any participants were enrolled, so no results are available. The goal was to compare a quadratus lumborum block with an erect…

This study looks at how certain immune cells (neutrophils) behave in patients having open gastrointestinal or spine surgery. Researchers want to see if differences in these cells can help explain why some people get surgical site infections. The goal is to better understand and p…

This study looks at 100 Ugandan infants under 6 months old with hydrocephalus caused by a brain infection. Researchers compare two surgeries: placing a shunt (a tube to drain fluid) versus a newer procedure called ETV/CPC. The goal is to see which surgery leads to better brain gr…

This study looks at how stress affects the brain and body in 70 teenagers (ages 11-17) with chronic widespread pain. Researchers use brain scans and saliva tests to measure stress hormones and other health markers. The goal is to understand the biological links between stress and…

This study uses ultrasound imaging to see if pressing on the cricoid cartilage (a part of the throat) actually blocks the esophagus and prevents stomach contents from entering the lungs during anesthesia in children. Researchers will also test if pressing from the side works bett…

This study looks at how young people (ages 11-19) with a knee condition called osteochondritis dissecans (OCD) feel emotionally about their diagnosis and treatment. Researchers want to see if these patients experience stress or depression and whether it affects their confidence t…

This study looks at whether a substance called UDP-glucose in urine can help doctors find kidney injury earlier in children who have had heart surgery. Current tests often miss early signs, leading to delays in care. Researchers will collect urine samples from 305 children aged 8…

This study aims to better understand immune-related gut diseases like inflammatory bowel disease (IBD), graft-versus-host disease (GVHD), and functional gut disorders. Researchers will collect blood and tissue samples from 1,000 patients to analyze immune cells and find what driv…

This study looks at whether giving doctors personalized feedback and a digital otoscope (a camera for the ear) can reduce how often they prescribe antibiotics for ear infections in young children. About 40 pediatricians who frequently diagnose and treat ear infections will be spl…

This study gathers information on the traits and genetics of 1500 people with autism spectrum disorders and their families. The goal is to better understand how genes relate to the condition. Participants must be at least 18 months old and have an autism diagnosis or a related ge…

This study looks at proteins in the blood of 60 children with congenital heart disease after heart surgery. The goal is to find which proteins can predict serious complications like fluid overload or organ injury. By combining protein data with patient information, researchers ho…

This study is not a treatment itself but a global online platform called DBS Matchmaker. It connects doctors around the world who are using deep brain stimulation (DBS) for rare movement disorders. The goal is to share knowledge and experiences to improve patient care. Up to 500 …

This study looks back at medical records of 300 children with Klippel-Feil syndrome (a rare condition causing a short, stiff neck) who had surgery at Boston Children's Hospital. The goal is to understand how often and why these children have a difficult airway during anesthesia. …

This study looks at how the painkiller oxycodone is absorbed and broken down in children aged 0-6 years after surgery. Researchers will measure drug levels in the blood and check specific genes that affect drug metabolism. The goal is to understand why some children may have more…

This study aims to understand how general anesthesia changes the way children's bodies process liquid oxycodone, a common painkiller given after surgery. Researchers will give oxycodone to 40 children aged 2-8 years at different times during anesthesia and measure drug levels in …

This study looks at whether a 4-week educational workshop can help caregivers of children who struggle to get enough healthy food. About 40 adults will attend weekly sessions and complete surveys to see if their food security and nutrition knowledge improve. The goal is to learn …

This study looks at 80 children with a known or suspected narrowing of the veins in the lungs (pulmonary vein stenosis). During a heart catheterization, doctors take extra measurements and pictures of the veins. One year later, they check how the veins are doing to see which test…