The purpose of this study is to learn if a vaccine made from the patient's own tumor cells, then genetically modified to secrete granulocyte-macrophage colony-stimulating factor (GM-CSF), will delay...
In the Congenital Myopathy Research Program at Boston Children's Hospital and Harvard Medical School, the researchers are studying the congenital myopathies (neuromuscular diseases present from birth)...
Angelman Syndrome (AS) is a developmental disorder that is caused by a deficiency of a maternally transmitted gene. It is inherited at birth, and affects movement, speech, and social demeanor. This...
This research study is a long-term follow-up to an earlier study of developmental care. For that study, high risk preterm newborns were randomly assigned to a standard care group, which received the...
Interstitial cystitis (IC), also called Bladder Pain syndrome (BPS), or chronic pelvic pain syndrome(CPPS) is a common condition with no known cause or cure. Twin studies and family accounts have...
The purpose of this study is to identify genes and proteins responsible for neuromuscular disorders by studying genetic material from individuals with neuromuscular disease, as well as their family...
Chronic Prostatitis/Chronic Pelvic Pain Syndrome (CP/CPPS) is a condition with several causes of which some remain unknown. It is believed that some types of CP may be genetic or passed down ...
Children and adolescents with inflammatory bowel disease (IBD) have high rates of depressive symptoms and more trouble with daily functioning than those without physical illness. The proposed study...
The Study is designed to collect information about adverse events that occur in children undergoing anesthesia in participating hospitals. Demographic information will be collected on all anesthetics....
The purpose of this study is to determine the radiographic and clinical outcomes of Scoliosis surgical and non-operative treatment in patients with Cerebral Palsy.
The Kids-DOTT trial is a randomized controlled clinical trial whose primary objective is to evaluate non-inferiority of shortened-duration (6 weeks) versus conventional-duration (3 months)...
The primary objective is to confirm the long-term functionality of implantation of the Medtronic Melody TPV at 5 years is no worse than the historical control established through literature review....
This study aims to determine whether respiratory system transfer impedance (Ztr) may fill an important clinical function by providing a reproducible, valid, and sensitive measure of airway obstruction...
The purpose of the study is to collect phenotypic (observable characteristics) and genetic information about individuals with Autism Spectrum Disorders (ASDs) and their families.
Hutchinson-Gilford Progeria Syndrome (Progeria) is a rare autosomal disease that results in premature death at a median age of 13 years due to cardiovascular and cerebralvascular compromise. The...
The goal of the Bronchopulmonary Dysplasia (BPD) Patient Registry is to collect data on individuals with neonatal lung disease to better understand the illness and ultimately improve their care and...
Scientist have begun to realize that many types of bacteria often live together as a complex community, and the investigators wish to apply that idea to the bacteria in the respiratory system of...
The purpose of this study is to determine if the use of sirolimus in the treatment of children and young adults with complicated vascular anomalies will prove to be safe and provide objective response...
Researchers are working on ways to treat SCID patients who don't have a matched brother or sister. One of the goals is to avoid the problems that happen with stem cell transplant from parents and...
This study is working towards gaining a better understanding of the genetic and environmental factors involved in autism spectrum disorders (ASD), which includes autism, pervasive developmental...
This study is a prospective evaluation of children with Severe Combined Immune Deficiency (SCID) who are treated under a variety of protocols used by participating institutions. In order to determine...
Congenital heart defects (CHD) are the most common major human birth malformation, affecting ~8 per 1,000 live births. CHD are associated with significant morbidity and mortality, and are second only...
Brain tumors are the leading cause of death from solid tumors in children. Tumor imaging is important in the management of these tumors, but current imaging methods have limitations in providing the...
The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC...
Individuals with a past diagnosis of severe combined immune deficiency (including many cases of "leaky SCID", Omenn syndrome, and reticular dysgenesis) who have undergone blood and marrow transplant,...
Patients with relapsed medulloblastoma, ependymoma and ATRT have a very poor prognosis whether treated with conventional chemotherapy, high-dose chemotherapy with stem cell rescue, irradiation or...
The Wiskott-Aldrich Syndrome (WAS) is an inherited disorder that results in defects of the blood and bone marrow. It affects boys because the genetic mistake is carried on the X chromosome. Normal...
The Prophylactic Antibiotic Regimens in Tumor Surgery (PARITY) trial is the first ever international multi-center randomized controlled trial in bone cancer surgery. In order to avoid amputation for...
Neurally adjusted ventilatory assist (NAVA) is an FDA approved mode of mechanical ventilation. This mode of ventilation is currently in routine use in adult, pediatric and neonatal intensive care...
The goal of this research study is to collect blood and urine samples from people who have either the R117H type of CF or the non-G551D gating type of CF to be kept for future research.We will also...
The purpose of this study is to determine whether adding an interactive biofeedback video game to anger control cognitive behavioral therapy is an effective and feasible treatment.
This study is being conducted to learn about the effects of SC-PEG, which is a new form of a chemotherapy drug called asparaginase. Asparaginase is used to treat ALL and lymphoblastic lymphoma. The...
The purpose of this study is to learn about the relationship between family factors and developmental and psychosocial outcomes in children with congenital heart disease at 6 years of age. A secondary...
Amblyopia is the leading cause of monocular visual impairment in children and adults. Despite conventional treatment with patching or eye drops, many older children and adults do not achieve normal...
Medical scientists want to find better ways to treat neuroblastoma and to find ways to prevent the tumor from growing back. To do this, they need more information about the characteristics of...
The overall objective of this drug trial is to determine whether the treatment of acute hyperammonemia with N-carbamyl-L-glutamate (NCG, Carglumic acid) in propionic acidemia (PA), methylmalonic...
The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to determine which of...
Dyskeratosis congenita is a disease that affects numerous parts of the body, most typically causing failure of the blood system. Lung disease, liver disease and cancer are other frequent causes of...
The purpose of this study is to analyze the long-term outcomes of surgical treatment of idiopathic scoliosis of all curve patterns treated by either anterior or posterior procedures. In addition, to...
The purpose of this study is to evaluate the safety of single ascending IV doses of a Factor IX (FIX) Gene Therapy in up to 16 Adults with Hemophilia B.
In order to learn more about women's health issues that occur over the lifespan, the Women's Health Study: from Adolescence to Adulthood is building a biorepository and database. The biorepository...
The purpose of this study is to compare the functional, clinical and radiographic outcomes associated with trans-articular drilling versus retro-articular drilling, two commonly employed techniques of...
The purpose of this early feasibility study is to determine how a new transcatheter pulmonary valve will move and perform once implanted in the right ventricular outflow tract.
To determine whether EEGs during infancy is a reliable biomarker to identify TSC patients that will develop infantile spasms/epilepsy in the near future and thus are appropriate candidates for an...
The investigators are enrolling 3-12 month old infants with a diagnosis of tuberous sclerosis complex (TSC) for a new study on early markers of autism. The study is looking for early signs for autism...
The investigators will perform a double blind, placebo controlled clinical trial with Xolair (omalizumab) at four centers to safely and rapidly desensitize patients with severe peanut allergy. The...
This research study is a Three arm Phase I clinical trial, which tests the safety of an investigational drug or combination of drugs and also tries to define the appropriate dose of the combination of...
Investigators at Boston Children's Hospital are conducting research in order to better understand the genetic factors which may contribute to disorders related to epilepsy. These findings may help...
Cardiomyopathy is a disease of the heart muscle. It is rare, but it can be serious. Cardiomyopathy in children can result in death, disability, heart transplantation or serious heart rhythm disorders....
The goal of this project is to evaluate the feasibility, acceptability, and effect size of a new computerized Motivational Enhancement Therapy (cMET) intervention for alcohol-involved adolescents in...
The BMN 165 clinical development program has been designed to demonstrate the safety and efficacy of BMN 165 in reducing blood Phe concentrations in adults with PKU.
The main objectives of this large phase IIb/III paediatric study are to assess the efficacy and safety of dabigatran etexilate relative to standard of care and to document the appropriateness of the...
This trial assessed the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector (also called elivaldogene...
The purpose of this trial is to determine whether treatment with valsartan will have beneficial effect in early hypertrophic cardiomyopathy (HCM) by assessing many domains that reflect myocardial...
This proposal is to evaluate the reliability and validity of the Vanderbilt ADHD screening tool for use with adolescents and young adults aged 13-21 years. The Vanderbilt is a previously developed,...
We plan to investigate whether the Isolated Orthosis for Thumb Actuation (IOTA) can effectively facilitate the ability of participants to perform a specific set of clinically relevant tasks conducted...
The investigators hypothesize that Recorded Home Oximetry (RHO) utilization will not increase rates of respiratory-related re-hospitalizations and ED visits, and will not impair growth compared to...
This study will compare three treatment regimens containing metaiodobenzylguanidine (MIBG) and compare their effects on tumor response and associated side effects, to determine if one therapy is...
Legg-Calvé-Perthes disease is a childhood hip disorder which is common enough to be a significant public health problem (affects 1 in 740 boys between ages 0-14), but uncommon enough to have a...
Oxycodone is an oral opioid analgesic that is most commonly prescribed for the management of pain in post-operative patients at Boston Children's Hospital. Oxycodone has been widely used in adults and...
The purpose of this study is to describe the range and incidence of symptoms, treatments, and complications related to pyruvate kinase deficiency (PKD). Eligible patients are those of all ages with...
Wiskott - Aldrich syndrome (WAS) is a rare serious medical condition that causes problems both with the immune system and with easy bruising and bleeding. The immune abnormalities cause patients with...
The purpose of the study is to demonstrate the efficacy and safety, and to assess the pharmacokinetics of adalimumab administered subcutaneously (SC) in pediatric subjects with moderate to severe...
Chronic granulomatous disease (CGD) is an inherited immune system abnormality in which bone marrow transplantation (BMT) has been shown to be curative. However the risks of transplantation are high...
Heart transplantation (HT) is a lifesaving procedure for patients with end-stage heart failure and provides a better survival and quality of life if compared to medical treatment. HT is subject to...
This research study involves participants who have acute lymphoblastic or acute myelogenous leukemia that has relapsed or has become resistant (or refractory) to standard therapies. This research...
Abnormalities in the gene encoding Factor VIII (FVIII) results in hemophilia A, an X-linked recessive bleeding disorder with a prevalence of 1 in 5000 males. Hemophilia A patients are classified into...
TriVox Health is an online system designed to make it easy for healthcare providers to monitor patients' disease symptoms and functioning over time and in between in-person visits (http://www.youtube...
Context: Chest wall deformities in children are relatively common. One such deformity, known as Pectus Excavatum (PE), involves a concavity of the chest and is the most frequent of these abnormalities...
This study, "A Phase II Study of Cabozantinib (XL l84) for Plexiform Neurofibromas in Subjects with Neurofibromatosis Type I in Children and Adults diagnosed with Neurofibromatosis Type 1 (NF1) and...
In this research study we want to learn more about which treatment works better for patients diagnosed with a vascular tumor called Kaposiform Hemangioendothelioma (KHE) or other high risk vascular...
This is a prospective cohort study designed to define the impact of HIV infection and antiretroviral therapy (ART) on young adults with perinatal HIV infection as they transition into adulthood. A...
This is a registry study to evaluate the long-term safety and effectiveness of adalimumab in pediatric patients with moderately to severely active CD who are treated as recommended in the local...
The primary objective of this study is to evaluate if Broncho-Vaxom® given to high risk infants for 10 days, monthly, for two consecutive years can increase time to occurrence of the first episode of...
The purpose of this study is to determine the safety and maximum tolerated dose (MTD) of Interleukin-2 in subjects with moderate-to-severe ulcerative colitis.
Study ALD-103 will be a multi-site, global, prospective and retrospective data collection study that is designed to evaluate outcomes of allo-HSCT in male subjects with CALD ≤17 years of age.
The primary objective of this study is to evaluate the long-term safety and efficacy of UX007 in participants with LC-FAOD. The secondary objectives of this study are to evaluate the effect of UX007...
Given the widespread use of anticonvulsants in the pediatric chronic pain population and the absence of scientific data supporting their use, the investigators propose a randomized, double blind, two...
The purpose of the study is to 1) define the operating characteristics of fungal biomarker assays in pediatric patients at high-risk for developing invasive candidiasis, 2) determine the change in...
This retrospective medical chart review (RECENSUS) of approximately 100 XLMTM patients (with a goal to obtain 50 deceased and 20 living records) will provide further knowledge about the clinical...
Chronic Granulomatous Disease (CGD) is an inherited immunodeficiency disorder which results from defects that prevent white blood cells from effectively killing bacteria, fungi and other...
This is a randomized trial designed to evaluate testicular outcomes after a single stage versus a two-stage surgical procedure to fix an undescended, one-sided testicle located in the abdomen. Also to...
Respiratory disorders are the leading cause of respiratory failure in children. Thousands of children are admitted to a pediatric intensive care unit each year and placed on mechanical ventilators....
Patients are being asked to be in this research study because medical researchers hope that by gathering information about a large number of children with pulmonary hypertension over time, their...
This randomized controlled trial will compare proximal femoral resection-interposition arthroplasty to proximal femoral resection with subtrochanteric valgus osteotomy for the treatment of painful...
This is a randomized clinical trial of epiphysiodesis techniques: percutaneous transphyseal screw epiphysiodesis versus percutaneous drill epiphysiodesis for the correction of leg length discrepancy....
This pilot study aims to assess the safety and treatment effect of acute dosing with IV Remodulin in neonates with persistent pulmonary hypertension of the newborn (PPHN).
In this study, doctors are trying to see if a study drug called rituximab (Rituxan®) will lower the number of B cells in the body. Doctors are also trying to see if decreasing B cells with rituximab ...
Central line-associated bloodstream infections (CLABSIs) are the most common healthcare-associated infection in children and are associated with morbidity and mortality. This study will attempt to...
The investigators goal is to determine the efficacy of school/classroom based environmental intervention in reducing asthma morbidity in urban schoolchildren.
The objectives of this program are: to characterize and describe the Mucopolysaccharidosis IV type A (MPS IVA) population as a whole, including the heterogeneity, progression, and natural history of...
The ultimate goal of this project is to develop methods that allow informed decision-making on the delivery time of fetuses that are at increased risk of stillbirth due to IUGR. In placenta related...
This study will combine three drugs: sorafenib, cyclophosphamide and topotecan.
Adding sorafenib to cyclophosphamide and topotecan may increase the effectiveness of this combination. The...
The purpose of this study is to examine the treatment, both surgical and non-surgical, of patients with any form of early onset scoliosis. Such treatment may include the use of growth friendly devices...
This is a first-in-human, multiple-dose 2-part study to assess the safety, tolerability, efficacy, and pharmacokinetics of SRP-4053 in Duchenne muscular dystrophy (DMD) patients with deletions...
Previous research and position statements have outlined the necessity of balance and gait testing in the post-concussion evaluation of athletes. However, many of the currently available balance...
Literature provides overwhelming evidence supporting the use of chlorhexidine gluconate (CHG) a rapid onset, broad spectrum, topical antiseptic for reducing healthcare-associated infections (HAIs)....
The primary aims of this phase I/II, randomized, placebo controlled study are the assessment of safety and tolerability of universal donor FMT compared to placebo in pediatric and young adult subjects...
Recently, researchers and clinicians have examined many different forms of concussion testing aimed to assess if a brain injury has occurred and to what degree it affects the individual being tested....
This study is aimed at investigating the efficacy of placebo for symptom relief in children with abdominal pain related functional gastrointestinal disorders.
The purpose of this study is to find a dose of intravitreal bevacizumab that is lower than currently used for severe retinopathy of prematurity (ROP), is effective in this study, and can be tested in...
This study will enable investigators to find out if brain structure and characteristics are affected by the shape of the infant's head, and if changes in the brain occur with helmet therapy.
Bronchoscopy-guided tissue sampling is a central technique in many diseases including diagnosing and staging lung cancers, diagnosing interstitial lung diseases, and acute and/or chronic rejections...
Lymphatic anomalies are a rare subset of vascular anomalies that are poorly understood. the understanding of the natural history, long-term outcomes, risk factors for morbidity and mortality, and the...
Neonates, children with single ventricle congenital heart disease, and those undergoing multiple complex cardiac surgeries are at high risk of increased perioperative blood loss, and blood product...
Continuation of the CARRA Registry as described in the protocol will support data collection on patients with pediatric-onset rheumatic diseases. The CARRA Registry will form the basis for future...
The Genomic Sequencing for Childhood Risk and Newborn Illness (the BabySeq Project) is a research study exploring the use of genomic sequencing in newborns. The National Institutes of Health is...
This research study is evaluating a drug called clofarabine as a possible treatment for Langerhans Cell Histiocytosis (LCH) and and other histiocytic disorders.
Background:
- Without an explanation for severe and sometimes life-threatening symptoms, patients and their families are left in a state of unknown. The NIH helped create a network of medical...
To determine the lowest nasal cannula flow rate in which upper airway deadspace is reduced. Hypothesis - The lowest flow rate of high flow nasal cannula (HFNC) will reduce upper airway (extrathoracic)...
The purpose of this study is to comprehensively characterize PMS using standardized medical, cognitive, and behavioral measures and to track the natural history of the syndrome using repeated...
The purpose of this study is to determine cross-sectional and longitudinal medical, behavioral, and cognitive differences between PTEN ASD and other groups, as well as to identify cognitive, neural...
The purpose of this study is to characterize the developmental phenotype of ASD and ID and to identify biomarkers using advanced MRI methodology and electrophysiological biomarkers of synaptic...
Study AG348-C-003 is a multicenter study designed to evaluate the safety and efficacy of different dose levels of AG-348 (mitapivat) in participants with PK deficiency.
This was a Phase 2/3, open-label, multicenter study to evaluate the pharmacokinetics (PK), efficacy, and safety of ombitasvir/paritaprevir/ritonavir (OBV/PTV/RTV) with or without dasabuvir (DSV) and...
The main objective of this study is to evaluate the efficacy of SRP-4045 and SRP-4053 compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and...
This Registry will enroll adolescent and pediatric participants who received at least one Gilead Hepatitis C Virus (HCV) direct acting antiviral (DAA) while participating in a Gilead-sponsored chronic...
Children undergoing hematopoietic cell transplantation (HCT) for cancer or blood disorders frequently develop gastrointestinal, metabolic and infectious complications related to preparative high-dose...
This research study is evaluating the use of specialized testing of solid tumors including sequencing. The process of performing these specialized tests is called tumor profiling. The tumor profiling...
Due to different study designs, the sponsor separated Part C into a separate registration (NCT04958642), leaving Parts A/B here in NCT02534844.
This study is to find out how safe and effective VTS...
This is a longitudinal, observational study of patients with Hemophilia A or B who are planning to switch to a newly approved coagulation factor replacement product, or who have recently switched...
The goal is to determine how lifestyle and exercise impact the well-being of individuals with hypertrophic cardiomyopathy (HCM) and long QT syndrome (LQTS).
Ancillary study Aim: To understand how the...
This study will determine if air-displacement plethysmography (ADP) provides accurate measurement of body composition (percent body fat and fat-free mass) in pediatric patients with intestinal failure...
The purpose of this study is to evaluate the effects of early adenotonsillectomy (eAT) on the behavior, sleep-disordered breathing symptoms and quality of life for children who snore, but do not have...
This randomized, double-blind, controlled, outpatient two-period, two-treatment crossover study is designed to evaluate the efficacy and safety of amifampridine phosphate in patients (ages 2 and above...
This study was designed to evaluate the safety, tolerability, pharmacodynamics and pharmacokinetics of riociguat at age-, sex- and body-weight-adjusted doses of 0.5 mg, 1.0 mg, 1.5 mg, 2.0 mg and 2.5...
This trial is a randomized, double-blind, placebo controlled trial designed to test whether two years treatment of preschool children aged 2-3 years of age at high risk for asthma with omalizumab ...
This NANT trial will determine the maximum tolerated dose (MTD) of autologous expanded natural killer (NK) cells when combined with standard dosing of dinutuximab and will assess the feasibility of...
This is a phase I/II dose-escalation trial of everolimus in combination with lonafarnib in Hutchinson-Gilford Progeria Syndrome (HGPS) and progeroid laminopathies (henceforth "progeria"). The study...
This research study is studying stereotactic body radiotherapy (SBRT) as a possible treatment for lung relapse of Ewing sarcoma, rhabdomyosarcoma, osteosarcoma, non-rhabdomyosarcoma soft tissue...
The purpose of the proposed study is to acquire additional data (including the assessment of anti-rHuPH20 antibodies) on the long-term safety of HYQVIA and to assess the prescribed treatment regimens...
The primary objective is to evaluate the long-term safety and tolerability of nusinersen (ISIS 396443) administered by intrathecal (IT) injection to participants with Spinal Muscular Atrophy (SMA) who...
This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-401 administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations...
This study uses frequency domain near-infrared spectroscopy coupled with diffuse correlation spectroscopy (FDNIRS-DCS) technology for monitoring cerebral blood flow (CBF) and cerebral oxygen...
Rapid onset Obesity, Hypoventilation, Hypothalamic dysfunction and Autonomic Dysregulation (ROHHAD) is a syndrome named in 2007. The hallmark of the syndrome is the rapid onset obesity and...
This is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) plus Best supportive care (BSC) versus placebo plus...
This is a Phase 2, open-label, single arm trial of pamrevlumab (FG-3019) to estimate pamrevlumab's safety and efficacy in non-ambulatory participants with DMD.
The TOPP-2 registry is an international, non-interventional, prospective registry including children and adolescents newly diagnosed with pulmonary hypertension (PH) to gain further insights in the...
The primary objective of the study is to assess the long-term safety of dupilumab in pediatric participants with AD.
The secondary objectives of the study are:
To assess the long-term efficacy of...
This multicenter, open-label study will evaluate the safety, efficacy and pharmacokinetics of prophylactic emicizumab treatment in participants previously treated with episodic or prophylactic...
Few studies are specifically designed to address health concerns relevant during pregnancy. The consequence is a lack of evidence on best clinical practice. This includes mothers and their babies when...
This is a pilot study to determine if a standard bolus dose and infusion of dexmedetomidine can significantly decrease the dose of propofol (infusion) required for accomplishing an MRI. The...
This is an observational, multicenter study to determine the prevalence of Cerebrotendinous Xanthomatosis (CTX) in patient populations diagnosed with early-onset idiopathic bilateral cataracts....
This research study is for participants who are undergoing allogeneic hematopoietic stem cell transplantation (HSCT) and are at risk for developing acute graft-versus-host disease (GVHD). GVHD is a...
The objective of this trial is to assess the safety and effectiveness of the Edwards Pericardial Aortic Bioprosthesis Model 11000A, in the pulmonary position in pediatric and adult subjects five years...
The goal of this trial is to compare the efficacy of the Bridge-Enhanced Anterior Cruciate Ligament Repair (BEAR™) technique with the current method of treatment for anterior cruciate ligament (ACL)...
This is a study to examine the effect of combining chronic oral azithromycin with inhaled tobramycin in adolescent and adult subjects with cystic fibrosis who are chronically infected with P....
This is a multicenter, double-blind, parallel-group, placebo-controlled, study to assess the efficacy, safety, and PK of ZX008 when used as adjunctive therapy for uncontrolled seizures in pediatric...
This study will determine whether vitamin D3 prevents severe asthma attacks in children who have a serum vitamin D (25(OH)D) level <30 ng/ml and who are being treated with inhaled corticosteroids for...
This is a multi-center, long-term safety and efficacy follow-up study for participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product in a parent clinical study.
...
The primary aim is to utilize near-infrared spectroscopy (NIRS) in patients under general anesthesia to measure changes in brain blood flow in the bilateral somatosensory cortices and the prefrontal...
The overarching purpose of this study is to advance understanding of the natural history of Rett syndrome (RTT), MECP2-duplication disorder (MECP2 Dup), RTT-related disorders including CDKL5, FOXG1,...
Laparoscopic cholecystectomy is associated with considerable postoperative pain and surgeon-administered local anesthetic infiltration is the standard practice for achieving post-operative analgesia....
Although the last decade has brought major advances with respect to our knowledge of certain risks associated with fetal exposure to psychiatric medications, critical information regarding the long...
There is currently no standard of care or best practice for managing post-operative pain for patients undergoing hip arthroscopy. Perioperative pain with these procedures can be substantial. It has...
The current study proposes adding BMP-2 (INFUSE), an anabolic agent, at the surgical site of TPA (tibial pseudarthrosis) repair in children with NF1, compared to a control group of patients treated...
This is a nonrandomized study of ruxolitinib in combination with a standard multi-agent chemotherapy regimen for the treatment of B-cell acute lymphoblastic leukemia. Part 1 of the study will optimize...
The purpose of this study is to advance understanding of the natural history of Rett syndrome (RTT), MECP2-duplication disorder (MECP2 Dup), CDKL5, FOXG1, and individuals with MECP2 mutations who do...
This study will evaluate the clinical efficacy and safety of udenafil, an orally administered, potent and selective inhibitor of PDE5, versus placebo for the treatment of adolescent subjects who have...
The Gene Discovery Core at The Manton Center for Orphan Disease Research based at Boston Children's Hospital studies families with rare, poorly understood or undiagnosed, but suspected genetic...
This is a multi-center, prospective, single cohort, observational study of pediatric heart transplant recipients designed to determine the impact of preformed versus de novo human leukocyte antigen ...
This is a prospective, single center, safety and feasibility trial to evaluate the use of autologous umbilical vein as shunts or conduits in neonatal cardiac surgery. Subjects will be identified here...
Pediatric ACL: Understanding Treatment Outcomes (PLUTO) is a multi-center, prospective cohort study. Specific aims of PLUTO are to evaluate the safety and comparative effectiveness of non-operative...
The purpose of this study is to determine whether adding low dose methotrexate to anti -TNF therapy is more effective than treatment with anti-TNF therapy alone in inducing and maintaining steroid...
Treatment for pediatric acute myeloid leukemia (AML) involves intensive chemotherapy regimens that result in periods of profound neutropenia leaving patients susceptible to severe infectious...
Treatment for pediatric acute myeloid leukemia (AML) involves intensive chemotherapy regimens that result in periods of profound neutropenia leaving patients susceptible to severe infectious...
Cystic fibrosis (CF), a life-shortening genetic disease, is marked by acute episodes during which symptoms of lung infection increase and lung function decreases. These pulmonary exacerbations are...
The purpose of this research will be to evaluate the reliability and validity of the HASS tool in a cohort of patients' ages 7 to18 years old against the gold standard of spirometry, and in ages 2 to...
The purpose of this study is to examine whether the duration of treatment with phenobarbital has an impact on neurodevelopmental and epilepsy outcomes, as well as parent and family well-being, after...
The purpose of this study is to improve the ability of the investigators to monitor brain health in newborn babies at risk of brain injuries. The researchers will be using an investigational system of...
Parents of infants who have been thrombocytopenic for 3-4 days will be approached for consent to enter the study. For the purposes of the study, thrombocytopenia will be defined as a platelet count ...
The primary goal of this project is to develop a tailored screening, brief intervention and referral to treatment (SBIRT) model for youth with chronic medical conditions (YCMC) for delivery at point...
The trial objective is to demonstrate the safety of idarucizumab, as assessed by the occurence of patients with drug related adverse events (including immune reactions) and all-cause mortality in...
Background:
Doctors and nurses who work in non-mental health settings need ways to know when patients are at risk. Researchers created the Ask Suicide-Screening Questions (ASQ) to be used in an...
The main purpose is to study brain plasticity (the changes that occur in the brain through experience) in individuals with autism spectrum disorder (ASD). Research suggests that during development,...
The goal of Advanced Fetal Imaging - Phase II is to advance fetal MRI imaging by designing MRI coils specifically for pregnant women and testing recently developed MRI image acquisition techniques....
The purpose of this study is to determine the feasibility of comparing outcomes of patients treated de novo with immunosuppressive therapy (IST) versus matched unrelated donor (MUD) hematopoietic stem...
Study design is a Phase IIb prospective multi-center, randomized, placebo-controlled, double-blind clinical trial. The goal will be to enroll 80 infants with Tuberous Sclerosis Complex who are less...
The investigators propose a robust therapeutic intervention to ameliorate myocardial ischemia/ reperfusion injury and significantly decrease morbidity and mortality in patients requiring...
The purpose of this initiative is to improve care and outcomes for infants with HLHS by expanding the NPC-QIC national registry to gather clinical care process, outcome, and developmental data on...
The main objective of this proposed investigation is to evaluate blood ropivacaine concentrations in infants and toddlers following the initiation of, and over the course of, continuous paravertebral...
This is a single site, non-randomized, prospective, open-label, interventional pilot/feasibility study. Patients recruited will have medically-refractory focal neocortical epilepsy, defined on the...
This proposal sets forth the platform for a Precision Medicine clinical trial through the New Approaches to Neuroblastoma Therapy (NANT) consortium. The plan is to utilize NANT's established multi...
Miscommunications are a leading cause of serious medical errors in hospitals, contributing to more than 60% of sentinel events, the most serious adverse events reported to the Joint Commission....
In this research study, the investigators want to learn more about whether the Respiratory Volume Monitor (ExSpiron) can accurately measure breathing in infants less than 12 months of age. The...
For participants enrolled prior to Version 6 of the protocol: This was a Phase II multi-center, randomized, double-blind, 24-week, 3-arm, parallel group, placebo-controlled study to investigate the...
The purpose of this study is to collect and compare information on how and when adolescent heart transplant recipients take their prescribed medication. The investigators want to find out if regular...
This study evaluates the safety of Viaskin Peanut 250 mcg in the treatment of peanut allergy in children from 4 to 11 years of age. Subjects will receive either Viaskin Peanut 250 mcg or a placebo for...
The overall goals are to change the paradigm for development of mechanism targeted pharmacotherapy in neurodevelopmental disorders and provide a definitive test of the mGluR theory in humans by...
The objectives of this study are to illustrate the clinical, neuro/electrophysiologic, biochemical, and developmental status and progression of patients with Creatine Transporter Deficiency (CTD) and...
This is a prospective, multicenter, open-label, randomized, controlled, parallel Phase 3 study with an open-label single-arm extension period to evaluate pharmacokinetics (PK), safety and efficacy of...
In proximal urea cycle disorders (UCD), particularly ornithine transcarbamylase deficiency (OTCD), hyperammonemia (HA) causes increased brain glutamine (Gln) which perturbation is thought to be at the...
This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-402, administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations...
The purpose of this study is to evaluate the safety and efficacy of teduglutide treatment of children with short bowel syndrome (SBS) who completed the TED-C13-003 study over a long-term period. It...
The primary aim of this study is to compare the propofol requirements of children who receive propofol with that of children who receive dexmedetomidine prior to propofol, for sedation for upper and...
This study will follow participants who completed the TED-C14-006 study. The purpose of this study is to evaluate the long-term safety and efficacy of teduglutide in pediatric participants with Short...
PumpKIN is a multicenter, prospective, single-arm feasibility study; Evaluating the investigational Jarvik 2015 VAD in pediatric patients with heart failure. This feasibility trial will enroll 10...
This trial of pitavastatin will determine efficacy and safety in this high risk population and provide evidence for clinicians to target this treatable risk factor to achieve an impact on early...
The use of aerobic exercise treatment in the chronic phase of concussion recovery is well-described in the literature but there are limited existing data on the effect of exercise treatment in the...
This is a Phase I trial to evaluate the safety and efficacy of oral encapsulated fecal microbiota transplantation (FMT) in the treatment of peanut allergy. In this research the investigators would...
A pediatric study in collaboration with Boston Children's Hospital to review the performance of two novel hydration status measurement devices against standard clinical assessment methods, through...
This is a prospective, multi-center, controlled, randomized, non-inferiority study to evaluate the clinical effectiveness of Conventional versus Mirasol-treated apheresis platelets in subjects with...
This is a Phase 2, randomized, double-blind, placebo-controlled, parallel-group, dose-finding study of SC411 in children with sickle cell disease (SCD). The primary objective of the study is to...
The purpose of this study is to address the needs for efficient and informative measurement for evaluating adolescent Screening, Brief Intervention, and Referral to Treatment (SBIRT) strategies. The...
To compare the efficacy of 1 hour/day of binocular game play 5 days per week plus spectacle correction with spectacle correction only, for treatment of amblyopia in children 4 to <13 years of age.
Phosphatase and TENsin homolog (PTEN) gene germline mutations are associated with a spectrum of clinical manifestations characterized by neurocognitive deficits, intellectual disability, autism...
The purpose of this study is to demonstrate the safety, tolerability, and efficacy of AR101 through oral immunotherapy (OIT) in peanut-allergic children and adults who have completed the ARC003 study.
Data strongly suggests that aggressive and early treatment of status epilepticus (SE) is crucial for seizure abortion and prevention of long-term neurologic sequelae. We propose the creation of a...
The purpose of the study is to characterize the clinical course of homocystinuria in pediatric and adult patients aged 5 to 65 years under current clinical management practices
This clinical research study is being conducted by Spark Therapeutics, Inc. to determine the safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals with hemophilia...
Post-traumatic headaches (PTH) are the most common complaint after traumatic brain injury, possibly generated by a number of stressors to the trigeminovascular and cervical plexus networks, including...
Gastrointestinal (GI) dysfunction affects up to 50% of medical and surgical critically ill children. GI dysfunction, specifically gastric dysmotility and loss of epithelial barrier integrity, is...
This is an open-label, follow-up study for subjects who completed the PEPITES study. Subjects will be offered enrollment in this follow-up study to receive Viaskin Peanut 250 μg for 2 additional years...
This study is a 12-month (52 week) safety extension study to supplement the FUEL Phase III clinical trial to provide safety information regarding the long-term use of udenafil in adolescents with...
Acute lymphoblastic leukemia (ALL) is the most common cancer diagnosed in children. The cancer comes from a cell in the blood called a lymphocyte. Normal lymphocytes are produced in the bone marrow ...
This is a multi-center prospective, single-arm, non-randomized, pivotal study that will continue to access the feasibility of the Xeltis Bioabsorbable Pulmonary Valved Conduit in subjects requiring...
This study will have 2 parts: Pharmacokinetics (PK) Lead-in Phase and the Treatment Phase.
The primary objective of the PK Lead-in Phase is to evaluate the steady state PK and confirm the dose of...
Each year, approximately 1 child in every 100 is born with Congenital Heart Disease (CHD), making it the most common birth defect. With recent medical advances, more children with CHD survive early...
This is a phase II, open label, multi-center, intra-patient dose escalation study to characterize the pharmacokinetics after oral administration of eltrombopag in combination with immunosuppressive...
Craniosynostosis (CS) is a common malformation occurring in ~4 per 10,000 live births in which the sutures between skull bones close too early, causing long-term problems with brain and skull growth....
The purpose of this study is to determine the effectiveness of behavioral feedback plus economic incentives to promote treatment adherence among a large diverse population of adolescents and young...
This pilot study is being conducted to determine whether the intraoperative presence of a Bispectral Index Monitor (BIS), a non-invasive monitor, affects the perioperative and postoperative indices...
It is difficult to assess pain and agitation in the NICU population because for a multitude of reasons including the pre-verbal nature of the patient population, the atypical pain response of...
This protocol is designed to compare the effectiveness of two dietary interventions for patients with Crohn's disease (CD): the Specific Carbohydrate Diet (SCD) and a Mediterranean style diet (MSD)...
The purpose of this study is to identify genes associated with impaired development and function of the cranial nerves and brainstem, which may result in misalignment of the eyes (strabismus) and...
This is a Phase 2/3, open-label, multicenter study to evaluate the pharmacokinetics, efficacy,and safety of glecaprevir (GLE)/pibrentasvir (PIB) for 8, 12, or 16 weeks in Hepatitis C virus (HCV)...
Approximately 400 Congenital heart disease patients will participate in the research study which will include one or more research visits for neurodevelopmental testing, brain MRI, and collection of...
In the landmark Boston Circulatory Arrest Study, neurologic and developmental status was measured following infant heart surgery and then prospectively at ages 1, 2.5, 4, 8, and 16 years, with...
The overall purpose of this project is to advance understanding of the neurophysiological features of Rett syndrome (RTT), MECP2 Duplication (MECP2 Dup) and RTT-related disorders (CDKL5, FOXG1) to...
Patients under the age of 5, with a diagnosis of hypoplastic left heart syndrome (HLHS), unbalanced atrioventricular canal (uAVC), or borderline left heart who are undergoing staged LV recruitment...
There is considerable evidence that most general anaesthetics modulate brain development in animal studies. The impact is greater with longer durations of exposure and in younger animals. There is...
The goal of this study is to identify significant clinical and laboratory risk factors in pediatric patients with significant upper gastrointestinal bleeding. This is defined as bleeding that...
This is an open label, Phase 1/2 study of oral miransertib (MK-7075) administered to participants at least 2 years of age with PIK3CA-related Overgrowth Spectrum (PROS) and Proteus Syndrome (PS) ...
This study seeks to identify genetic causes of conditions that affect the gonads and genitals, and to study the impact on families of receiving genetic results.
This study will assess the tolerability and palatability of an amino acid based oral rehydration solution (enterade®) compared to current oral rehydration solution among children with short bowel...
The radiation exposure resulting from medical imaging is a topic of some concern. Nuclear medicine provides potentially life-saving information regarding physiological processes, and is of particular...
The radiation exposure resulting from medical imaging is a topic of some concern. Nuclear medicine provides potentially life-saving information regarding physiological processes, and is of particular...
The radiation exposure resulting from medical imaging is a topic of some concern. Nuclear medicine provides potentially life-saving information regarding physiological processes, and is of particular...
The radiation exposure resulting from medical imaging is a topic of some concern. Nuclear medicine provides potentially life-saving information regarding physiological processes, and is of particular...
Lorlatinib is a novel inhibitor across ALK variants, including those resistant to crizotinib. In this first pediatric phase 1 trial of lorlatinib, the drug will be utilized as a single agent and in...
This is a Phase 3 study to assess the safety and efficacy of Tadekinig alfa in patients with monogenic, interleukin-18 (IL 18) driven autoinflammation due to Nucleotide-binding oligomerization domain,...
The goal of the Fontan Imaging Biomarkers (FIB) study is to identify the associations of blood and urine biomarkers to imaging parameters of ventricular mechanics.
Infants often present to the hospital with episodes of coughing, choking, gagging, change in muscle tone, and/or change in skin color, known as brief resolved unexplained event. Many studies have...
This study will explore whether ivabradine lowers heart rate, and thus improves exercise capacity, in survivors of lymphoma who have an elevated resting heart rate as a side effect of prior radiation...
The purpose of this study is to evaluate vedolizumab pharmacokinetics (PK), safety and tolerability in pediatric participants with moderately to severely active UC or CD.
Cerebrovascular events, such as stroke, are a devastating complication of Fabry disease that results in part from storage of complex lipids in both large and small vessels. Understanding how the...
SMART T1D is a research study that offers interactive diabetes education for teens with type 1 diabetes in peer groups led by diabetes nurse educators. Participants in the study will be randomly...
This study proposes to evaluate the accuracy of contrast-enhanced ultrasound (CEUS) in diagnosing abdominal solid organ injuries in pediatric patients. 146 subjects will be enrolled across...
The measurement of how much oxygen a baby consumes provides important information about the health of the baby, and of how much energy they are consuming. Currently, there is no device which measures...
The goal of this study will be to assess the safety and efficacy of high-dose interval Vitamin D3 therapy in children and young adults with Inflammatory Bowel Disease being treated with serial...
This is a multi-center, randomized phase III study to evaluate the clinical effectiveness of AeroVanc in persistent methicillin-resistant Staphylococcus aureus (MRSA) infection in patients with cystic...
The investigators propose to translate confocal microscopy for use during open heart surgery. This tool will help discriminate between various types of tissues in the heart during surgery. One of the...
This study explores the effects of an eight-week exercise intervention for adolescents with Down syndrome on home exercise compliance. As well, to observe changes in fitness including muscular...
The study aims to assess the safety and efficacy of Viaskin Peanut to induce desensitization to peanut in peanut-allergic children 1 to 3 years of age after a 12-month treatment by EPicutaneous...
Prospective data collection and evaluation of complete data sets will be performed in the course of routine clinical care of a cohort of consecutive patients (children up to 16 years old) presenting...
Iron deficiency-related anemia is the most common nutritional deficiency disorder in the world, mainly affecting children, women and older adults in underdeveloped countries. To combat iron deficiency...
This is a multi-site study of how nutrition is delivered to critically ill patients in pediatric intensive care units (PICUs) around the world. Each site will include mechanically ventilated children...
In this research study, investigators want to learn more about the factors that influence children's breathing during sleep and their sleep quality. Specifically, investigators are interested in...
This is a phase II open label study that will evaluate children ≥ 1 year of age and adults with neurofibromatosis type 1 (NF1) and plexiform neurofibromas treated with the MEK inhibitor, binimetinib....
The main goal of this study is to quantitatively assess the sucking and feeding activity of infants at high risk of neurological impairment (preterm infants and term infants at risk of abnormal...
Part 1 of this trial will enroll healthy volunteers into a single ascending dose (SAD), multiple ascending dose (MAD), and Food Effect (FE) treatment groups.
The SAD treatment group is comprised of...
Acute kidney injury (AKI) is common in children after cardiac surgery with a reported incidence of 20-40%. Pediatric AKI has been found to be associated with important short and long-term adverse...
This is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an observational arm to evaluate the safety and efficacy of long-term ivacaftor treatment in subjects with cystic...
The purpose of the study is to acquire additional data on efficacy, safety, tolerability, immunogenicity, pharmacokinetic (PK) and other parameters of HYQVIA in pediatric (age ≥ 2 to <16 years)...
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat...
The primary objective of the 1701-202 STRONG SCD study is to evaluate the safety and tolerability of different dose levels of IW-1701 compared with placebo when administered daily for approximately 12...
This study evaluates the ability of contrast-enhanced ultrasound to improve the diagnosis of acute appendicitis in children compared to conventional ultrasound.
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that...
Phase 3 pivotal US trial studying open-label intravenous administration of onasemnogene abeparvovec-xioi in spinal muscular atrophy (SMA) Type 1 participants.
This is a single center, prospective, observational study to demonstrate the clinical validity of the Intrinsic LifeSciences (ILS) Intrinsic Hepcidin IDx™ Test in the diagnosis and management of iron...
This is a phase I/II open label multi-center study in which patients will receive low dose targeted busulfan followed by infusion of autologous CD34+ selected bone marrow or mobilized peripheral blood...
A national data registry of patients receiving fecal microbiota transplantation (FMT) or other gut-related-microbiota products designed to prospectively assess short and long-term safety and...
131I-Metaiodobenzylguanidine (131I-MIBG) is one of the most effective therapies utilized for neuroblastoma patients with refractory or relapsed disease. In this pediatric phase 1 trial, 131I-MIBG will...
Study Objectives
The objectives for this randomized trial are:
To determine the efficacy of daily low-dose atropine (0.01%) for slowing myopia progression over a two-year treatment period in...
This study is enrolling participants by invitation only. This is an open-label, safety extension study for subjects who participated in the ARC007 study.
Bridge-Enhanced ACL Restoration (BEAR) is a new procedure being developed to treat patients with ACL injuries. In the BEAR procedure, an implant is placed between the torn ends of the ACL and the...
This is a multicenter, cross-sectional, cohort study which will collect contemporary sweat chloride (SC) values from approximately 5000 Cystic Fibrosis (CF) patients prescribed and currently receiving...
