Boston Children's Hospital

This study tests a new approach to help children under 18 who are on ECMO (a heart-lung machine) and have heart muscle damage. Doctors will inject the child's own mitochondria (the energy parts of cells) directly into the heart to see if it helps the heart recover and allows the …

This study tests whether giving more doses of the drug abatacept after a stem cell transplant can better prevent severe graft-versus-host disease (GVHD), a serious complication where donor cells attack the patient's body. About 160 people aged 2 and older receiving a partially ma…

This study looks at whether stomach and esophagus movement problems increase the risk of aspiration (food or liquid going into the lungs) in children with neurologic impairment. About 120 children aged 5-21 who rely on feeding tubes and are at high risk for aspiration pneumonia w…

This study looks at boys ages 4 to 36 months with one undescended testicle found in the belly. It compares doing one surgery versus two surgeries to move the testicle into the scrotum. The main goal is to see which approach leads to better testicle growth, fewer problems, and low…

This study looks at whether children with medical complexity who have pneumonia can safely stop antibiotics earlier, based on when they become clinically stable. Researchers will enroll 50 patients aged 2-25 at Boston Children's Hospital. The goal is to reduce unnecessary antibio…

This study tests a device called RELiZORB that pre-digests fat in tube feedings for children aged 2 to 18 with short bowel syndrome who rely on IV nutrition. The goal is to see if it helps them absorb more nutrients and possibly reduce or stop their need for IV nutrition. The stu…

This study looks at whether whole or nonfat milk is better for body fat, heart health, and diet in 200 children aged 9-12 who have a higher BMI. Participants drink either whole or nonfat milk for one year while researchers measure changes in fat mass, lean mass, and other health …

This study looks at whether certain foods given through a feeding tube can lower bile acid levels in the stomach and saliva of children who have trouble swallowing and are at risk for breathing in stomach contents. About 60 children who get most of their nutrition through a gastr…

This study tests whether a safe fluorescent dye called indocyanine green (ICG) can help diagnose biliary atresia, a rare liver disease in infants. After injection, the dye normally appears in stool if bile ducts are open. In babies with biliary atresia, the dye should be absent. …

This study tests a low dose of Interleukin-2 (IL-2) in 30 people aged 12-80 with moderate-to-severe Crohn's disease. The goal is to find the safest and most effective dose to reduce symptoms. Participants will be monitored for side effects and changes in disease activity over 8 w…

This study tests whether metformin, a common diabetes drug, can reduce pelvic pain in young women with endometriosis. Fourteen participants will take either metformin or a placebo daily for 6 months and track their symptoms. The goal is to see if this non-hormonal approach offers…

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 works better for reducin…

This study looks at whether a long-lasting numbing medicine (liposomal bupivacaine) can reduce the need for strong painkillers after spinal fusion surgery in teens with scoliosis. About 128 teens aged 10–17 will be randomly assigned to get either the long-lasting numbing drug or …

This study tests a brief, virtual therapy for parents of preschool children. The goal is to help parents reduce their use of screens (like tablets or phones) to calm or entertain their child. The program includes three group sessions and checks in with families before, after, and…

This study tests whether oral ketamine can help control severe pain from sickle-cell crises in adolescents and young adults aged 12-24. Participants receive ketamine every 8 hours for 48 hours alongside standard painkillers. The goal is to improve pain relief and reduce the need …

This study looks at children born with esophageal atresia, a condition where the esophagus doesn't form properly. After surgery, many develop a narrowing (stricture) that needs balloon dilation to open it up. The study will compare different inflation times to see if a longer hol…

This study looks at 40 children who get most of their nutrition through a feeding tube. Researchers want to see if changing how thick the blended food is can reduce stomach symptoms like nausea and bloating. Each child will try both thicker and thinner blends over 16 weeks to fin…

This study tests whether using positive language (like "we're going to give you a special sleepy air") instead of standard medical talk helps children aged 5-10 feel less anxious and behave better when they breathe in anesthesia through a mask. About 128 healthy children having s…

This study tests a program designed to help teens and young adults with sickle cell disease build resilience and cope better. Researchers want to see if the program is practical and helpful for participants. The goal is to reduce depression, anxiety, and pain interference.

This study looks for genes that cause problems with eye movement and certain nerve conditions present from birth. Researchers will study up to 20,000 people with these disorders to find new genes and understand how they work. The goal is to learn more about these conditions, not …

This study looks at how children with intestinal failure feel about their quality of life. Researchers will track 750 kids from hospitals across the U.S. and Canada over five years. They want to find out which medical and social factors make the biggest difference in these childr…

This study aims to find the genes that cause strabismus (crossed or misaligned eyes) and nystagmus (involuntary eye shaking). Researchers will collect DNA from families with at least three members who have strabismus, or from individuals with infantile esotropia or nystagmus and …

This study looks at how blood is managed in children under 18 during heart surgery. Researchers want to create a math model that could help doctors give the right amount and type of blood products. The goal is to better predict and treat patients who need transfusions, reducing t…

This study explores whether a non-invasive lung recruitment maneuver can predict if a child under general anesthesia will respond to intravenous fluids. Researchers aim to help anesthesiologists give the right treatment for low blood pressure and avoid unnecessary fluid administr…

This study looks at how a type of white blood cell called neutrophils changes in children with sepsis, a severe infection. Researchers will compare blood and breathing tube samples from 75 patients in the ICU with sepsis to those from children having planned surgeries. The goal i…

This study aims to find genes that may cause interstitial cystitis (also called bladder pain syndrome), a painful bladder condition with no known cause or cure. Researchers will collect blood or saliva from 1,000 people with the condition and their family members across North Ame…

This study is creating a registry of up to 1,000 people with rare lymphatic anomalies—conditions that affect the body's lymph system. Researchers will collect information from medical records and yearly surveys to understand how these diseases progress, what treatments work, and …

This study looks at how certain immune cells (monocytes) change during heart surgery that uses a heart-lung machine. Researchers will measure these cells and related chemicals in the blood before, during, and after surgery. The goal is to understand links between inflammation and…

This study aims to find the genes that cause congenital myopathies, a group of muscle diseases present from birth. Researchers will analyze DNA from up to 4,000 people with these conditions and their family members. The goal is to improve diagnosis and pave the way for future tre…

This study is testing a program called CRUSH that teaches autistic adults about dating, relationships, and sexual health. The goal is to see if the program is practical and helpful. Forty participants will attend group sessions and one-on-one coaching, and researchers will measur…

This study aims to improve how doctors talk with families when a baby may be born extremely early (22-25 weeks). Researchers will train doctors in communication skills and then measure if parents feel more informed, less anxious, and more involved in decisions. About 460 pregnant…

This study is a registry for people with Shwachman-Diamond Syndrome (SDS) or similar conditions. Researchers will collect medical information and biological samples from up to 5,000 participants to better understand the disease, its complications, and how it is treated. The goal …

This study looks at how the intestines develop normally in babies and what changes in diseases like necrotizing enterocolitis (NEC). Researchers will collect small tissue samples, blood, and stool from infants up to 2 years old who are already having intestinal surgery or a scope…

This study looks at how pain pathways in the brain and nerve regrowth in the eye change after corneal crosslinking (CXL) for keratoconus. Researchers will use brain scans (fMRI) and special eye imaging to measure these changes in 60 participants aged 8-35. The goal is to better u…

This study looks at whether a stomach ultrasound can reliably measure how much food or liquid is in a child's stomach before surgery. It involves 60 children aged 2 to 18. The goal is to see if ultrasound can help prevent rare but serious complications like breathing stomach cont…

This study looks at neutrophils, a type of white blood cell, in children with sepsis, a severe infection that can damage organs. Researchers want to understand how these cells change and cause harm, so they can develop treatments to protect the body. The study involves 72 childre…

This study is testing a smartphone app called OnTrackCF to see if it is easy and helpful for adults with cystic fibrosis. About 60 people will use the app and answer surveys. The goal is to learn if the app works well, not to treat the disease directly.

This study gathers information from 500 children worldwide who have both epilepsy and movement disorders. Researchers will review medical records and genetic data to find patterns linking these conditions. The goal is to better understand these rare diseases and guide more person…

This study looks at how common sedatives and painkillers behave in children who need ECMO, a machine that helps the heart and lungs. Researchers will measure drug levels in the blood of 20 children to understand how ECMO changes drug processing. The goal is to find the right dose…

This study collects data from many hospitals on children with congenital heart disease who need surgery or procedures not related to their heart. The goal is to better understand the risks these children face and how healthcare resources are used. By pooling information from 10,0…

This study tests new brain imaging tools to measure oxygen flow in children with sickle cell disease. Researchers will compare these tools with standard MRI scans. The goal is to find better ways to decide when treatment is needed and how well it works. The study includes healthy…

This study looks at how placing young children in foster care and giving caregivers special training affects their thinking, emotions, brain development, and mental health. Researchers will follow 220 children in Brazil who are new to the institutional care system. The goal is to…

This study follows 200 people with a confirmed genetic diagnosis of progressive myoclonus epilepsy type 1 (EPM1) to learn how the disease changes over time. Researchers will collect health data and blood or urine samples to build a biobank and identify key markers of disease prog…

This study looks at whether people with a certain gene change (IL-4Ra R576) have more severe eczema. Researchers will measure skin symptoms, immune markers, and skin bacteria in 111 participants aged 6 to 65. No new treatment is given—the goal is to better understand why some peo…

This study enrolls people with rare or undiagnosed conditions, along with their family members, to find the genetic causes of their illnesses. Researchers collect DNA samples and medical information to analyze genes and identify disease-causing changes. If a genetic cause is foun…

This study aims to collect information from 125 people with confirmed or suspected Chopra-Amiel-Gordon Syndrome (CAGS), a rare genetic condition. Researchers will track symptoms, brain images, learning challenges, and genetic data over time. The goal is to build a detailed regist…

This study tests whether a saliva (oral fluid) test can accurately show if a person has used cannabis recently, all done from home. Researchers will enroll 200 people aged 18-30 who use cannabis at least once a week. Participants will provide saliva and urine samples and report t…

This study tests a non-invasive light-based device to monitor blood flow and oxygen use in the brains of premature infants who have had a brain bleed or developed fluid buildup (hydrocephalus). Researchers will compare these babies to healthy newborns and those with other causes …

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

This study is creating a shared database of information from up to 100 children who have had or will have deep brain stimulation (DBS) for conditions like dystonia, epilepsy, or Tourette syndrome. By combining data from multiple hospitals, researchers hope to learn which children…

This study looks at why newborns with severe congenital heart disease are more likely to develop dangerous blood clots after heart surgery. Researchers will collect blood samples from 10 babies to study tiny molecules called microRNAs that control inflammation and clotting. The g…

This study follows 200 people with cystic fibrosis, ages 6 and up, who are starting a new triple therapy drug after having mental health or liver problems with a similar medicine. Researchers will track how many stop the new drug due to side effects and whether mental health or l…

This study looks at how much clear liquid (like water or juice) teenagers can drink two hours before an upper GI endoscopy without affecting safety. About 288 teens aged 13-17 will be split into four groups, each drinking a different amount. Doctors will measure the leftover stom…

This study is testing a new, non-invasive device that uses light to measure oxygen levels in the brains of newborn babies who may have suffered a brain injury. Researchers will compare these measurements with standard tests to see if the device can help doctors better monitor and…

This study is for people with certain types of hereditary spastic paraplegia (SPG4 or SPG5A). Researchers will collect medical information, blood samples, and genetic data to build a shared database. The goal is to better understand how the disease progresses and prepare for futu…

This study aims to find new genetic causes of epilepsy in babies and understand how a genetic diagnosis helps families. Researchers will enroll 600 infants who had their first seizure before 12 months old. They will use genetic testing and track development, seizure control, and …

This study tests a nutrition education program for children aged 2-18 recently diagnosed with celiac disease. The program encourages eating naturally gluten-free foods and avoiding highly processed gluten-free products. Researchers will measure changes in body composition and die…

This study tests a virtual genome center (VIGOR) to help sick newborns in community hospitals get advanced genetic testing and expert advice, even without specialized staff on site. It will enroll 750 babies with suspected genetic conditions from NICUs that serve low-income and m…

This study aims to improve how doctors decide when to deliver babies with intrauterine growth restriction (IUGR), a condition where the baby is smaller than expected and at higher risk of stillbirth. Researchers will use MRI scans to measure blood flow and oxygen levels in the pl…

This study aims to improve fetal MRI by designing special coils for pregnant women and testing new image-capture methods. Researchers will compare the quality of these research images to standard fetal MRI and ultrasound. About 300 pregnant women, both healthy and those with conc…

This study follows up to 55 people with SSADH deficiency, a rare genetic disorder that affects brain function, to see how symptoms and body chemistry change with age. Participants will have tests like blood draws and brain scans. The goal is to find patterns and markers that coul…

This study looks at four biological markers (biomarkers) in 25 children aged 3 to 6 who have both autism and an anxiety disorder. The goal is to see if these markers can help predict which children will respond well to treatment and measure how much they improve. Parents will fil…

This study tests a new MRI software that aims to take pictures of the brain much faster than current methods. Researchers want to see if the quick scans are just as clear and useful for doctors as the standard slower scans. The study includes children and adults with low-grade gl…

This study looks at children with central lines who develop bloodstream infections. Researchers will use DNA sequencing to compare bacteria from the blood with bacteria from other body sites to figure out where the infection really started. The goal is to better understand whethe…

This study aims to uncover genetic factors that contribute to epilepsy and related disorders. Researchers will analyze DNA from up to 5,000 people with epilepsy to find gene changes linked to the condition. The goal is to better understand why epilepsy varies so much from person …

This study is testing whether thickening liquids changes how safely children with swallowing problems (dysphagia) can swallow. Researchers will use special tools to measure swallowing in up to 200 children, comparing thin and thickened liquids. The goal is to find out if thicker …

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 medical errors that hospital staff often miss, especially for families who speak languages other than English. About 656 …

This study tests a computer program that helps doctors decide when to remove a breathing tube from children after heart surgery. About 330 children under 12 who have been on a ventilator for at least 48 hours will take part. The goal is to see if the tool shortens the time they n…

This study looks at whether a mobile health program called Bedtime Stories can help caregivers of children ages 5-11 improve their child's sleep. The program sends personalized sleep tips to caregivers. Researchers want to see if the program is easy to use and if it leads to bett…

This study looks at whether thickening the milk or formula of infants who have had a brief unexplained event (BRUE) can reduce symptoms and hospital visits. Researchers will follow 250 babies under 1 year old for up to 12 months, comparing those who get thickened feeds to those w…

This study aims to uncover the genetic causes of hereditary spastic paraplegia (HSP), a group of rare nerve diseases that cause progressive muscle stiffness and disability. Researchers will analyze DNA from 200 children and adults with HSP to find gene variants linked to the cond…

This study looks at the genes of 300 babies who had brain injury around birth (neonatal encephalopathy) to see if genetics play a role in how they recover. Researchers at Boston Children's Hospital will analyze DNA from babies and their parents. The goal is to find genetic causes…

This study collects health information and biological samples from up to 700 people under 30 with early-onset hereditary spastic paraplegia (HSP). Researchers hope to better understand how the disease progresses over time. The goal is to create a registry and biorepository to sup…

This study is a registry that collects information from children and teens (up to age 21) who have spine surgery using robots and special navigation tools. The goal is to see how accurate the robot-placed screws are and to track radiation exposure and patient recovery. No new tre…