Boston Children's Hospital

This study is testing whether low-dose injections of a medication called IL-2 can help control moderate-to-severe Crohn's disease. Researchers will give daily injections for 8 weeks to 30 participants who haven't responded well to standard treatments. The main goals are to find t…

This study is testing a small cartridge called RELiZORB that attaches to a feeding tube. It's designed to help children with short bowel syndrome digest and absorb fats from their tube feeding. The goal is to see if better nutrient absorption can reduce or eliminate the child's n…

This study follows people with cystic fibrosis who had to stop or change their previous treatment due to mental health issues or liver problems. Researchers want to see if a newer triple therapy medication is safer for these patients. They'll monitor 200 participants for one year…

This study is testing a digital sensor that attaches to a standard asthma inhaler. The sensor and app provide real-time feedback on a person's inhaler technique and send medication reminders. Researchers want to see if this 'digital coach' helps people use their inhalers correctl…

This study is testing a new nutrition education program for children recently diagnosed with celiac disease. It aims to teach families how to choose healthier, less-processed gluten-free foods instead of relying on packaged rice-based products. Researchers want to see if this app…

This study is testing if metformin, a common diabetes medication, can help reduce pelvic pain in young women with endometriosis when used alongside their regular hormone therapy. Researchers will compare metformin to a placebo (sugar pill) over six months in 14 participants to se…

This study compares how two types of care affect young children who must leave their families. Researchers will follow 220 children under age 2 in Brazil, randomly placing them into either high-quality foster care or an improved institutional care setting. Both caregivers receive…

This study is testing whether certain blended foods given through a feeding tube can bind to and reduce bile acids in the stomach. High bile levels may contribute to lung damage if stomach contents are accidentally inhaled (aspiration). Researchers will compare different diets in…

This study is testing whether drinking whole milk or nonfat milk for one year affects children's weight, body fat, and risk factors for heart disease and diabetes. Researchers will enroll 200 children aged 9-12 who are above average weight and randomly assign them to drink three …

This study is looking at whether thickening infant formula with rice cereal helps babies who have experienced frightening breathing episodes called BRUEs. Researchers will follow 250 infants under 1 year old for a year, comparing those who receive thickened feeds to those who don…

This study is investigating whether thickening liquids like formula helps children who have trouble swallowing safely. Researchers will measure swallowing function in up to 200 children aged 0-21 years who have experienced choking spells or swallowing difficulties. The goal is to…

This study is testing whether doctors can safely give shorter courses of antibiotics to children with complex medical conditions who get pneumonia. Instead of a standard fixed length, treatment will be tailored to stop when each child is clinically stable. The goal is to reduce o…

This study is testing if injecting a child's own mitochondria—the energy-producing parts of cells—into their heart muscle can help reduce damage caused by poor blood flow. The goal is to help children who are on a heart-lung bypass machine (ECMO) recover better and come off the m…

This study is testing whether giving a drug called abatacept for a longer period of time is better at preventing severe complications after a stem cell transplant. It will involve about 160 patients, aged 2 and up, who have a blood cancer and are receiving a transplant from a par…

This study aims to find a better way to predict if a child under anesthesia needs IV fluids to treat low blood pressure. Researchers will test a simple breathing maneuver on 48 children having surgery at Boston Children's Hospital. The goal is to help doctors give the right treat…

This study aims to develop better MRI methods to assess the health of the placenta in pregnancies where the baby is not growing properly (Intrauterine Growth Restriction or IUGR). Researchers will scan 80 pregnant women to see if MRI measurements of blood flow and oxygen in the p…

This study is testing a new, non-invasive device that uses light to monitor oxygen levels and energy use in a newborn's brain. Researchers want to see if this device can help doctors better monitor babies at risk of brain injury from low oxygen during birth. The goal is to see if…

This study is testing a short, online group program for parents of young children (ages 2.5 to 4.5). The goal is to help parents reduce how often they use screens like tablets or phones to calm their kids or keep them occupied. Researchers believe less 'regulatory screen use' wil…

This study is testing whether a newer, longer-lasting numbing medicine (liposomal bupivacaine) works better than a standard numbing medicine to control pain after spinal fusion surgery for scoliosis. Researchers want to see if the newer medicine helps teens use fewer opioid paink…

This study is testing if adding an oral form of the drug ketamine to standard pain medications can better control the severe pain of sickle cell crises in hospitalized adolescents and young adults. It aims to see if this approach is practical and safe for patients aged 12-24 who …

This study is comparing two different pain management plans for children aged 3-17 having tonsil surgery. One plan uses strong opioid painkillers, while the other tries to avoid them by using different medications. The goal is to see if the opioid-free plan controls pain just as …

This study is testing whether using positive, encouraging language is better than standard language at reducing anxiety and negative behaviors in children when they are being put to sleep with anesthesia gas through a mask. Researchers will enroll 128 healthy children, aged 5 to …

This study is testing whether a resilience-building program can help teenagers and young adults with sickle cell disease better manage their condition. The program focuses on providing mental health support to help participants cope with depression, anxiety, and pain. Researchers…

This study aims to see which of two medications works better to reduce coughing, choking, and lung infection risk in children with neurological conditions who are tube-fed. Researchers will compare prucalopride (a gut motility drug) to famotidine (an acid reducer) over 10 weeks i…

This study aims to understand how a rare, severe genetic epilepsy called EPM1 progresses over time. Researchers will remotely track symptoms and collect blood and urine samples from up to 200 participants across the US who have a confirmed genetic diagnosis. The goal is to gather…

This study is testing new MRI software designed to create brain scans much faster while maintaining image quality. Researchers aim to improve how doctors measure brain tumors in children with low-grade gliomas. The study involves 300 participants, including both patients and heal…

This study tests a new system to help families report safety concerns while their child is hospitalized. Researchers created a mobile tool that lets families easily share concerns via text, email, or QR code. The goal is to catch more safety issues that staff might miss, especial…

This study aims to learn more about Chopra-Amiel-Gordon Syndrome (CAGS), a rare genetic condition that causes intellectual disability and other health issues. Researchers will follow 125 people with confirmed or suspected CAGS over time, collecting detailed information about thei…

This study aims to improve how doctors counsel families facing the possibility of an extremely premature birth. Researchers will first interview families to learn their preferred language and then use that information to create new training programs for doctors. The goal is to he…

This study aims to understand why newborns with severe congenital heart disease are at high risk for dangerous blood clots after surgery. Researchers will collect blood samples from 10 newborns to analyze tiny molecules that may control clotting. The goal is to uncover the biolog…

This study is testing a new, non-invasive bedside monitor that uses light to measure blood flow and oxygen use in the brains of premature infants. It aims to see if this technology can better track brain health in babies who have had a brain bleed or developed fluid buildup, comp…

This study aims to understand why some patients develop abnormal blood clots during and after heart surgery. Researchers will examine immune cells and inflammatory signals in 100 patients of all ages undergoing complex cardiac procedures. The goal is to identify potential causes …

This study aims to understand how a specific type of white blood cell, called a neutrophil, behaves in children with sepsis, a life-threatening infection. Researchers will compare blood samples from children with sepsis to those without to see how these cells change and potential…

This study aims to create a mathematical model that helps doctors better predict how much blood children will need during heart surgery and identify risks of blood cell damage. Researchers will observe 50 children under 18 having heart surgery at Boston Children's Hospital to col…

This study aims to understand how a rare genetic brain disorder called SSADH deficiency changes over time in patients of all ages. Researchers will follow 55 patients for five years, collecting health information, brain scans, and body fluid samples to map the disease's natural c…

This study aims to test new MRI technology designed specifically for pregnant women to get better quality images of babies in the womb. Researchers will compare these new MRI images against standard ultrasound and current MRI pictures. The study involves 300 pregnant women, inclu…

This study aims to understand the quality of life of children and young adults living with intestinal failure, a rare condition where the gut cannot absorb enough nutrients. Researchers will follow 750 participants from multiple hospitals in the US and Canada for up to five years…

This study aims to better understand a specific type of white blood cell (neutrophil) in children with sepsis, a life-threatening body-wide infection. Researchers will compare these cells from the blood and lungs of children with sepsis to those from children without it. The goal…

This study is testing a new program called 'Bedtime Stories' that sends sleep education messages to caregivers' phones. Researchers want to see if this approach is practical and helpful for families, especially those from underserved communities. They will enroll 75 caregivers in…

This study aims to better understand rare genetic disorders that cause both epilepsy and movement problems in children. Researchers are collecting medical history and genetic information from 500 children worldwide who have these conditions. By analyzing this data, they hope to f…

This study is testing a new mobile app called OnTrackCF to see if it's easy to use and helpful for adults with cystic fibrosis (CF). Researchers want to know if people will use the app's features to help manage their daily CF medication routine. The goal is to gather feedback to …

This research program aims to better understand the genetic causes of congenital myopathies—rare muscle disorders present from birth. Researchers are studying DNA from 4,000 participants worldwide to identify genes involved in these conditions. The goal is to improve future diagn…

This study aims to understand how four common sedatives and painkillers behave in the bodies of children on ECMO, a form of heart-lung life support. Researchers will measure drug levels in the blood of up to 20 pediatric patients receiving these medications. The goal is to gather…

This study is creating a registry to collect information on how well robotic-assisted surgery and custom-made rods work for children and young adults with spine deformities. It will track 700 patients up to age 21 to see how accurate and safe the surgeries are, and how they affec…

This study aims to understand why some patients develop long-term pain after a common eye surgery for keratoconus. Researchers will use brain scans and special eye imaging to track how pain signals change and how nerves in the eye heal after the procedure. The goal is to learn ho…

This study aims to gather detailed health information and biological samples from children and young adults (under 30) with early-onset hereditary spastic paraplegia (HSP), a group of rare genetic disorders that cause progressive stiffness and weakness in the legs. By tracking pa…

This study is testing a virtual support center called VIGOR that helps community hospitals provide advanced genetic care to sick newborns. The goal is to give hospitals without specialized genetics teams access to expert advice, education, and treatment options. Researchers want …

This study aims to understand the genetic causes of hereditary spastic paraplegia (HSP), a group of rare inherited disorders that cause progressive muscle stiffness and weakness. Researchers will analyze DNA from 200 participants who have been diagnosed with progressive spasticit…

This study aims to understand the genetic causes of strabismus (misaligned eyes) and nystagmus (involuntary eye movements) by studying families where these conditions occur. Researchers will enroll families with multiple affected members to collect DNA samples. The goal is to ide…

This study aims to find the genes responsible for a group of disorders that affect eye movement and cranial nerves from birth. Researchers will collect genetic information from up to 20,000 people with these conditions and their family members. The goal is to better understand th…

This study aims to discover if interstitial cystitis/bladder pain syndrome (IC/BPS) has a genetic cause. Researchers are enrolling 1000 people, including those diagnosed with IC/BPS and their family members (both with and without symptoms). Participants provide DNA samples (blood…

This study aims to better understand how two specific genetic forms of hereditary spastic paraplegia (HSP) progress over time. Researchers will enroll 100 people with confirmed SPG4 or SPG5A HSP to collect detailed health information and blood samples. The goal is to build a rese…

This study aims to discover genetic factors that cause epilepsy, particularly severe forms that begin in childhood. Researchers will analyze DNA from 5,000 patients with epilepsy to identify genetic patterns. The findings could lead to better diagnosis and future treatments, but …

This small pilot study aims to test new brain imaging methods to monitor sickle cell disease. Researchers want to see if advanced scans can measure how well oxygen reaches the brain and how treatments affect this process. The study involves children with sickle cell disease, incl…

This study aims to see if at-home saliva tests can reliably measure cannabis use in remote clinical trials. Researchers will recruit 200 young adults who use cannabis weekly to compare saliva test results with urine tests and self-reported use. The goal is to find easier, remote-…