Boston Children's Hospital

This study tests whether feeding preterm infants with chronic lung disease through a tube placed past the stomach (into the small intestine) helps their breathing more than feeding through a tube in the stomach. About 50 high-risk preterm babies born before 32 weeks will receive …

This study tests whether giving more doses of the drug abatacept over a longer period can better prevent graft-versus-host disease (GVHD), a serious complication where donor immune cells attack the patient's body. About 160 people receiving stem cell transplants from partially ma…

This study compares two surgical methods to fix a testicle that hasn't dropped into the scrotum and is located in the abdomen. Boys aged 4 months to 3 years with one undescended testicle will be randomly assigned to either a single surgery or a two-stage procedure. Researchers wi…

This study is testing whether whole milk or nonfat milk is better for body composition and health in 200 children aged 9-12 who have a higher BMI. Families receive weekly milk deliveries and monthly check-ins. The goal is to see which milk type helps reduce fat mass and improve m…

This study tests a new approach for children on ECMO life support who have heart damage. Doctors will take a small sample of the child's own mitochondria (the energy parts of cells) and inject them directly into the heart muscle. The goal is to see if this is safe and helps the h…

This study tests 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 helps them absorb more nutrients and reduce their need for IV feeding. About 32 children aged 2 to 18 will use…

This study looks at whether children with medical complexity who have pneumonia can safely stop antibiotics sooner if they are clinically stable. Researchers will track how many families agree to the approach and monitor for worsening symptoms or return to the hospital. The goal …

This study tests whether a fluorescent dye called indocyanine green (ICG) can help diagnose biliary atresia, a rare liver disease in infants where bile ducts are blocked. After injection, the dye is normally removed through bile and appears in stool. In infants with biliary atres…

This study tests a new software called Wave-CAIPI that aims to speed up MRI scans for children with low-grade gliomas. Researchers will compare the fast scans to standard ones to see if they provide the same image quality. The goal is to make imaging quicker and less stressful fo…

This study compares different doses of two sedative drugs, propofol and dexmedetomidine, in children aged 1-12 undergoing MRI scans. The goal is to find the combination that provides adequate sedation with fewer side effects and faster recovery. Researchers will measure drug cons…

This study tests a device that treats stretch marks by gently abrading the skin from underneath. Ten people with stretch marks will receive one treatment and be checked at 1 week, 3 months, and 1 year. The goal is to see if the device safely improves the appearance of stretch mar…

This study tests a nutrition education program for children ages 2 to 18 recently diagnosed with celiac disease. The program focuses on eating naturally gluten-free foods and avoiding ultra-processed gluten-free options. Researchers will compare this approach to standard gluten-f…

This study tests a clinical decision support system (CDSS) that helps doctors decide when to remove breathing tubes from children after heart surgery. About 330 children under 12 who have been on a ventilator for at least 48 hours after surgery will take part. The system gives tw…

This study tests a program called PRISM that aims to help teens and young adults with sickle cell disease build resilience and cope better. Researchers will check if the program is easy to use and liked by participants. They will also see if it helps reduce depression, anxiety, a…

This study looks at children born with esophageal atresia who develop narrowing (strictures) after surgery. Doctors use a balloon to stretch the narrowed area, but it's unclear how long the balloon should stay inflated. The trial will compare 30 seconds versus 180 seconds of infl…

This study is testing a behavioral therapy called 'Being Brave' to help reduce anxiety in autistic children aged 3 to 6. The therapy involves 16 weekly sessions with parents and children, using visual aids and practice exercises. Researchers will also look at biological markers t…

This study looks at 40 children who get most of their nutrition through a feeding tube. Researchers want to see if a thicker blenderized tube feed helps reduce stomach symptoms like nausea and bloating compared to a thinner one. Each child will try both types over 16 weeks, and p…

This study looks at two treatments for a specific type of front knee pain in teenage female athletes. One group gets a cortisone shot plus physical therapy, the other gets a saline (salt water) shot plus physical therapy. The goal is to see which approach better reduces pain and …

This pilot study tests a brief, virtual therapy for caregivers of children aged 30-54 months. The goal is to reduce using screens to calm or distract children, which may hinder their ability to manage emotions. Over three 60-minute group sessions, caregivers learn strategies like…

This pilot study tests whether metformin, a drug typically used for diabetes, can reduce pelvic pain in young women with endometriosis. Fourteen participants will take either metformin or a placebo daily for six months and track their symptoms. The goal is to see if this non-horm…

This study tests whether a low dose of Interleukin-2 (IL-2) can safely reduce symptoms in people with moderate-to-severe Crohn's disease. About 30 participants aged 12 to 80 will receive the drug to find the best dose that works with the fewest side effects. The goal is to improv…

This study tests whether using positive language (like 'blow out the birthday candles') instead of standard language during anesthesia induction reduces anxiety and negative behaviors in children aged 5-10. 128 healthy children having non-emergency surgery will be randomly assign…

This study looks at whether problems with how the stomach and esophagus move increase the risk of aspiration (food or liquid going into the lungs) in children with brain-related disabilities. It compares two drugs, prucalopride and famotidine, to see which better reduces symptoms…

This study is testing a smartphone app called OnTrackCF, designed to help adults with cystic fibrosis remember to take their CFTR modulator medication and track their symptoms. The app sends up to 3 daily support messages and shows users their adherence levels. Researchers want t…

This study tests if oral ketamine can help control severe pain from sickle-cell crises in teens and young adults (ages 12–24). Participants receive the drug every 8 hours for 48 hours alongside standard pain care. The goal is to find better ways to manage pain and reduce the need…

This study compares two numbing medicines injected during scoliosis surgery to see which one better controls pain and reduces the need for strong opioid painkillers. About 128 teens aged 10 to 17 will be randomly assigned to receive either a long-acting numbing drug (liposomal bu…

This study is testing whether simple saliva tests done at home can accurately show when someone has not used cannabis. Researchers will enroll 200 people aged 18-30 who use cannabis at least once a week. Participants will provide saliva and urine samples over 3-4 weeks, and resea…

This study is a registry that collects information from children and teens up to age 21 who are having spine surgery with robotic and navigation tools. The goal is to track how accurately screws are placed and how much radiation patients and surgeons receive. No new treatment is …

This study aims to find out if chronic pelvic pain conditions like prostatitis and interstitial cystitis can be inherited. Researchers will collect DNA and urine from 500 participants, including family members, to look for genetic links. The goal is to better understand the cause…

This study tests a new discharge protocol for preterm infants with bronchopulmonary dysplasia (BPD), a chronic lung disease. The protocol was developed by national experts to improve the transition from NICU to home. Researchers will track follow-up rates, hospital readmissions, …

This study tests a handheld camera that looks at tiny blood vessels in children undergoing heart surgery for certain birth defects. Standard monitors only check large vessels, but this camera may reveal hidden problems. About 40 children will participate to see if the camera help…

This study looks at whether a specific genetic change (IL-4Ra R576) is linked to more severe eczema. Researchers will measure skin symptoms, immune markers, and skin bacteria in 111 people aged 6 to 65 with atopic dermatitis. No new treatment is being tested; the goal is to bette…

This study aims to figure out where bloodstream infections in children with central lines actually come from. By comparing the DNA of bacteria from the blood with bacteria from the gut, mouth, and skin, researchers hope to tell whether the infection started from the line itself o…

This study aims to find new genetic causes of epilepsy in infants whose seizures start before 12 months of age. Researchers will analyze the DNA of 600 babies to see if a genetic diagnosis can be made and how that affects medical care and family well-being. The goal is to shorten…

This study looks at how different amounts of clear liquids (like water or juice) affect stomach contents in teens aged 13-17 before an upper GI endoscopy. Participants are randomly assigned to drink 3, 5, 7, or 10 ml of clear fluid per kilogram of body weight two hours before the…

This pilot study is testing a program called CRUSH, which is a sexual health curriculum designed specifically for autistic adults aged 18 to 30. The program includes group sessions and one-on-one coaching to teach about relationships and sexual health. The goal is to see if the p…

This study is creating a registry of up to 1,000 people with rare lymphatic system disorders. Researchers will collect information from medical records and yearly surveys to understand how these diseases progress, what treatments are used, and how they affect quality of life. The…

This study follows 10,000 children with congenital heart disease who need surgery for non-heart issues. Researchers want to learn what complications occur and how hospitals use resources. The goal is to create better risk predictions and improve care for these patients.

This study is creating a registry for people with Shwachman-Diamond Syndrome (SDS) or similar conditions. Researchers will collect medical records and biological samples from up to 5,000 participants to understand how the disease progresses and what treatments work best. The goal…

This study looks at whether ultrasound can reliably measure how much is in a child's stomach before surgery, which could help prevent rare but serious complications like breathing in stomach contents. About 60 children aged 2 to 18 will have stomach ultrasounds, and some will als…

This study looks at how blenderized tube feeds (real food pureed and given through a feeding tube) affect acid reflux in children aged 1-21 who currently use formula. Researchers want to understand if these diets reduce reflux episodes and improve symptoms. The goal is to gather …

This study looks at how the intestines develop in premature and full-term babies up to 2 years old. Researchers will collect tissue, blood, and stool samples from infants already having intestinal surgery or scopes. The goal is to better understand normal development and diseases…

This study at Boston Children's Hospital is enrolling up to 10,000 people with rare or undiagnosed conditions, along with their family members. Researchers will analyze DNA samples to find the genetic causes of these illnesses. The goal is to improve diagnosis and understanding o…

This research study at Boston Children's Hospital is looking at the genes and proteins involved in congenital myopathies—rare muscle diseases that are present from birth. Researchers will analyze DNA from up to 4,000 participants, including patients and their family members, to f…

This study tests a new way for families of hospitalized children to report safety concerns using a mobile tool (email, text, or QR code). The goal is to catch errors and problems that hospital staff often miss, especially for families who speak languages other than English. About…

This study tracks the quality of life of 750 children with intestinal failure across North America. Researchers will use surveys to measure physical, emotional, and social well-being, and look at how medical and family factors affect these scores. The goal is to identify what hel…

This study aims to uncover the genetic causes of hereditary spastic paraplegia (HSP), a group of rare neurological diseases that cause progressive muscle stiffness and weakness. Researchers at Boston Children's Hospital will analyze DNA from 200 people with HSP to find genetic va…

This study looks at whether thickening the milk or formula of infants who have had a brief unexplained event (a scary but temporary episode of stopped breathing or color change) can reduce repeat episodes and hospital visits. Researchers will follow 250 babies under 1 year old fo…

This study is collecting health data and blood samples from 100 people with specific types of hereditary spastic paraplegia (SPG4 and SPG5A). The goal is to create a shared database and biobank to better understand how the disease progresses. This information will help prepare fo…

This study is following 200 people with a confirmed genetic diagnosis of EPM1, a rare and progressive epilepsy disorder. Researchers will collect clinical data and biological samples (blood and urine) over time to understand how the disease progresses. The goal is to identify bio…

This study tests a non-invasive light-based device to measure blood flow and oxygen use in the brains of premature infants who have bleeding or fluid buildup. Researchers will compare these measurements in affected babies, those with other types of fluid buildup, and healthy newb…

This study is creating a shared database of children who have received deep brain stimulation (DBS) for movement disorders like dystonia, epilepsy, or Tourette syndrome. By pooling data from multiple hospitals, researchers hope to answer key questions about who benefits most and …

This study looks at neutrophils, a type of white blood cell, in children with sepsis (a severe infection). Researchers want to understand how these cells change and may cause lung injury. By comparing sepsis patients to healthy controls, they hope to find clues for future treatme…

This study looks at how different thicknesses of liquids (thin, slightly thick, mildly thick, moderately thick) change the way children with swallowing problems swallow. Researchers will use special tools to measure swallowing safety in 200 children, including infants who had a b…

This study collects health information and biological samples from up to 700 people under 30 with early-onset hereditary spastic paraplegia (HSP). Researchers aim to better understand how the disease progresses over time and create a registry for future studies. Participants prov…

This study collects information from 500 children worldwide who have both epilepsy and movement disorders caused by specific gene changes. Researchers will analyze medical records to find patterns linking symptoms to genetics. The goal is to create a shared database that helps do…

This study looks at how placing young children (under 24 months) into foster care quickly, along with training for their foster parents, affects their thinking, emotions, and brain development. Researchers will follow 220 children in Brazil, comparing those in foster care to thos…

This study tests a new program called Bedtime Stories that sends tailored sleep health messages to caregivers of school-aged children (ages 5-11) from low-income and historically marginalized communities. The goal is to see if the program is easy to use and helps improve children…

This study looks at the genes of 300 infants who had brain injury around birth (neonatal encephalopathy) and their parents. Researchers want to find out if genetic differences explain why some babies have mild symptoms while others have severe long-term problems. The study does n…

This study will collect leftover blood samples from 10 newborns with severe heart defects who have surgery at Boston Children's Hospital. Researchers want to understand why these babies are more likely to develop dangerous blood clots after surgery. They will look at tiny molecul…

This study aims to better understand Chopra-Amiel-Gordon Syndrome (CAGS), a rare genetic disorder. Researchers will track symptoms, brain images, and learning challenges in 125 people with confirmed or suspected CAGS over time. No treatments are being tested—the goal is to collec…

This study aims to find genes that cause eye misalignment (strabismus) and related conditions involving the cranial nerves and brainstem. Researchers will analyze DNA from up to 20,000 participants with these disorders. The goal is to better understand the genetic causes, which c…

This study is testing a virtual genome center (VIGOR) to help sick newborns in community hospitals get genetic testing and expert advice without traveling to a big specialty center. The goal is to see if this approach works well and can be used more widely, especially for low-inc…

This study tracks up to 55 people with SSADH deficiency, a rare genetic disorder that affects brain function. Researchers will measure symptoms, brain activity, and chemical levels in the body over time to see how the disease changes with age. The goal is to find markers that pre…

Researchers at Boston Children's Hospital are recruiting 5,000 people with epilepsy to study their genes. By comparing DNA from patients and their parents, they hope to find genetic changes linked to epilepsy. This study does not test a new drug or treatment, but aims to better u…

This study looks at how common sedatives and painkillers behave in children on ECMO, a life-support machine. Researchers will measure drug levels in the blood of 20 pediatric patients to understand dosing needs. The goal is to improve safety and effectiveness of these drugs durin…

This study looks at neutrophils—a type of white blood cell—in children with sepsis, a severe infection that can harm organs. Researchers want to learn how these cells change and cause damage. The goal is to find clues that could lead to better treatments. The study will compare 7…

This study looks at how to manage blood transfusions better for children undergoing heart surgery. Researchers will create a math model to predict the right amount and type of blood to give, and to understand the risk of red blood cell damage. The goal is to make transfusions saf…

This study looks at leftover blood samples from 100 patients having heart surgery to see how certain immune cells (monocytes) change during the procedure. The goal is to understand links between inflammation and blood clotting risks. No new treatments are being tested; it is pure…

This study looks at whether foods that bind to bile acid can lower its levels in the stomach and saliva of children who rely on feeding tubes. The goal is to reduce the risk of breathing problems caused by stomach contents entering the lungs. About 60 children will receive differ…

This study looks at how the brain and eye nerves change after corneal crosslinking (CXL), a procedure for keratoconus. Researchers will use brain scans (fMRI) and eye imaging to track pain and nerve healing. The goal is to better understand recovery, not to test a new treatment.

This study aims to find genetic factors that may cause interstitial cystitis (also called bladder pain syndrome). Researchers are collecting blood or saliva samples and medical information from 1,000 people with the condition and their family members across North America. No trea…

This study aims to improve the way doctors counsel families when a baby may be born extremely early (between 22 and 25 weeks of pregnancy). Researchers will train doctors in better communication skills and then ask parents about their knowledge, satisfaction, and anxiety after co…

This study aims to find genetic variants linked to strabismus (crossed eyes) and nystagmus (involuntary eye movements). Researchers will analyze DNA from families with at least three members who have strabismus, or families with infantile esotropia or nystagmus. By comparing affe…

This study uses MRI scans to measure how well oxygen moves through the placenta in pregnancies complicated by intrauterine growth restriction (IUGR), where the baby is not growing as expected. Researchers will compare MRI results with placenta tissue after birth and track the bab…

This study tests new brain imaging tools to measure oxygen flow in children with sickle cell disease. Researchers will compare healthy kids, untreated patients, and those who had gene therapy. The goal is to find better ways to decide when treatment is needed and how well it work…

This study tests a new, non-invasive device that measures oxygen levels in the brains of newborns at risk for brain injury. Researchers will compare readings from babies receiving cooling therapy, those with brain injury not getting cooling, and healthy newborns. The goal is to s…

This study is testing a new MRI receiver coil designed specifically for pregnant women to see if it can produce clearer and faster images of the fetus. Researchers will compare the quality of these research MRI scans to standard clinical fetal MRI and ultrasound. The study will e…

This study looks at whether a non-invasive lung maneuver can help doctors predict if a child under general anesthesia will benefit from receiving fluids. The goal is to avoid giving unnecessary fluids when they won't help. The study will include 48 children having surgery at Bost…

This study follows 200 people with cystic fibrosis (ages 6 and up) who had mental health or liver issues while taking the drug elexacaftor/tezacaftor/ivacaftor (ETI) and had to stop or change it. They will now start a newer triple therapy called vanzacaftor/tezacaftor/deutivacaft…