Boston Children's Hospital

This study compares two standard treatments—bone marrow transplant from an unrelated donor and immune suppressive therapy—as first-line therapy for children and young adults with severe aplastic anemia, a rare condition where the body stops making enough blood cells. About 53 par…

This study looks at whether people aged 12 and older with a specific gene change (IL-4RαR576) get more relief from the asthma drug dupilumab. Over 48 weeks, researchers track how many severe asthma attacks occur compared to a placebo. The goal is to see if genetic testing can hel…

This study tests adding everolimus to the standard drug lonafarnib for children with progeria, a rare condition that causes rapid aging. The goal is to find the safest dose and see if the combination improves weight gain and blood vessel health. About 80 participants will take pa…

This study looks at a device called RELiZORB that pre-digests fat in tube feedings for children with short bowel syndrome who rely on IV nutrition. The goal is to see if it is safe and helps children absorb nutrients better, possibly reducing their need for IV nutrition. About 32…

This study looks at a surgery called ETV/CPC for babies under 6 months with hydrocephalus (fluid buildup in the brain). The goal is to see if this surgery can help babies avoid needing a permanent shunt, which often fails. Researchers will track the babies' development and check …

This study tested a daily solution called T-EDTA (Kitelock™) placed into the central lines of 15 children who need IV nutrition. The goal was to see if it could prevent blood clots and serious bloodstream infections without damaging the catheter. The study was suspended, so resul…

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 if a drug called omalizumab can stop asthma from developing in young children at high risk. Researchers are giving the drug to 200 children aged 2-3 for two years, then watching them for another two years to see if they develop asthma. The goal is to find a way t…

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 program called MOMENT-V that combines two video counseling sessions with a smartphone app to help young adults reduce cannabis use and related problems. About 65 participants will either get the full program or a brief counseling session. Researchers will check…

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 looks at whether adding mental skills coaching to standard care helps adolescent athletes feel more confident about returning to sports after ACL reconstruction. About 30 teens and young adults who tear their ACL and have surgery at Boston Children's Hospital will take…

This study tested a digital platform that attaches to asthma inhalers to give real-time feedback on technique and medication reminders. About 26 people with uncontrolled asthma used the sensor and a mobile app for 3 months. The goal was to see if the platform improves inhaler ski…

This study tests whether the diabetes drug metformin is safe and practical to give to infants who suffered brain injury from lack of oxygen at birth. About 30 babies under 6 months old will receive metformin to see if it helps their brain development. The main goal is to check fo…

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 open to girls and women aged 13-25 with a confir…

This study aimed to compare two different nerve block techniques used alongside a standard pain block to manage pain after hip surgery in teenagers and young adults. The goal was to see if one method provided better pain relief or reduced the need for opioid painkillers. The stud…

This study looks at whether giving red blood cell transfusions only when a child is bleeding or not getting enough oxygen, instead of following a fixed hemoglobin level, can reduce organ failure and improve brain development. It involves 228 children under 6 years old on ECMO lif…

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 is collecting blood and tissue samples from 1,000 people with inflammatory bowel disease (IBD), graft-versus-host disease (GVHD), or functional gut disorders, as well as those receiving cell therapies like CAR-T. Researchers will analyze the immune system in detail to …

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 back at medical records of 300 children with Klippel-Feil syndrome (a rare neck condition) who had surgery at Boston Children's Hospital. The goal is to understand how doctors managed their breathing tubes during anesthesia and find the best ways to keep these ki…

This study uses ultrasound to see if pressing on the throat (cricoid pressure) during anesthesia actually blocks the esophagus and prevents stomach contents from entering the lungs in children. Researchers will check the esophagus position and size before and after pressure, and …

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 how teenagers with a knee condition called osteochondritis dissecans (OCD) feel emotionally and whether they are mentally ready to return to sports. Researchers will survey 120 patients aged 11-19 to measure stress and depression as they go through standard tr…

This study looks at how stress affects the brain and body in young people aged 11-17 with chronic widespread pain (pain lasting over 3 months). Researchers will use brain scans, saliva tests, and physical measurements to understand these connections. The goal is to find clues tha…

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…

This study looks at whether proteins in the blood can help predict which children with congenital heart disease will have serious complications after heart surgery. Researchers will measure thousands of proteins from small blood samples and combine them with patient data to build…

This study looks at how certain immune cells (neutrophils) work in patients having planned surgery. The goal is to see if differences in these cells can help predict or explain surgical site infections. About 220 adults will take part, and the research focuses on understanding, n…

This study gathers information on the traits and genetics of 1500 people with autism spectrum disorders and their families. The goal is to find links between genes and behaviors. Participants must be at least 18 months old and have an autism diagnosis or a related genetic finding…

This study looks at ways to help doctors prescribe fewer antibiotics for middle ear infections in young children. Forty pediatricians who often diagnose and treat ear infections will be split into four groups: some get personalized education and feedback, some use a digital otosc…

This study is not a treatment itself but a global online platform called DBS Matchmaker. It helps doctors from different hospitals share information about patients with rare movement disorders who are getting deep brain stimulation (DBS). The goal is to improve how doctors select…