Clinical Research Directory

A Study of Sonrotoclax (BGB-11417) in Children With Relapsed or Refractory Acute Myeloid Leukemia and B-cell Acute Lymphoblastic Leukemia

The goal of this clinical trial is to learn if sonrotoclax (BGB-11417) is safe and may help treat children and adolescents with acute myeloid leukemia (AML) or acute lymphoblastic leukemia (ALL) that has come back after treatment or has not responded to treatment. The study will also learn how the body processes sonrotoclax when it is given with other medicines. The main questions it aims to answer are: * Is sonrotoclax safe and well tolerated when given with other anti-cancer medicines? * How does the body absorb, process, and remove sonrotoclax? * Does treatment with sonrotoclax, in combination with other medicines, help reduce or eliminate leukemia? Researchers will give sonrotoclax together with other anti-cancer medicines to participants with relapsed or refractory AML or ALL. Participants will: * Take sonrotoclax in combination with other anti-cancer medicines * Have regular clinic visits for physical exams, blood tests, heart monitoring, and other safety assessments. * Provide blood samples to measure how the body processes sonrotoclax. * Have tests to evaluate how their leukemia responds to treatment. * Continue treatment as long as it is helping and side effects remain manageable, according to the study plan.

Safety Study to Evaluate the Occurrence of EFAD in Pediatric Patients With PNAC Who Require More Than Eight Weeks of Omegaven Treatment

This study will demonstrate safety in pediatric patients with Parenteral Nutrition-Associated Cholestasis treated with Omegaven®, which is indicated as a source of calories and fatty acids in this patient population

Evaluation of Pediatric eCART Implementation

This is a study comparing 3 years of retrospective data (pre-implementation) to 2 years of prospective data after the implementation of a pediatric version of Electronic Cardiac Arrest Risk Triage (pediatric eCART), a clinical decision support (CDS) tool that uses electronic health records (EHR) to identify patients with high risk for life threatening outcomes. Up to 30,000 encounters with pediatric patients will be assessed. Acceptability of the pediatric eCART intervention will also be measured from pediatric nurse clinicians.

Little NIRVANA for Pediatric Pain and Anxiety

The aim of this study is to test the feasibility of using Little NIRVANA (augmented reality (AR)) for managing anxiety and pain for children 2-8 years old during hospital emergency peripheral intravenous (PIV) insertions. The investigators hypothesize that Little NIRVANA plus a numbing cream will reduce pain and anxiety for patients undergoing PIV placement in the emergency department (ED) when compared to the cream alone. It is anticipated that using the Little NIRVANA will not increase the ED length of stay, failed PIV placement attempts, or the need for additional intervention.

PANDA Gym: Automated Assessment of Neurodevelopment in Infants at Risk for Motor Disability

Early childhood detection of motor delays or impairments provides the opportunity for early treatment which improves health outcomes. This study will use state of the art sensors combined with machine learning algorithms to develop objective, accurate, easy-to-use tools for the early scoring of deficits and lays the foundation for the early prediction of physical disability.

VR-PAT During Home Burn Dressings - Multisite

This study will evaluate the effectiveness of smartphone Virtual Reality Pain Alleviation Therapy (VR-PAT) as a pain distraction tool during repeated at-home burn dressing changes among 100 children (age 6-17 years) with a burn injury in comparison to 100 children with a burn injury who do not use the VR-PAT.

Multicenter HomeVENT: Home Values and Experiences Navigation Track

This is a multicenter study to test a decision-making support process for families and clinicians facing decisions about chronic home ventilation for a child. The investigators hypothesize that the intervention will increase family preparedness for decision-making and will improve clinician-family shared-decision making. Half of families will be assigned to "usual care" arm and half to the "intervention" arm. Intervention families will view the study website with study staff and will answer questions related to website content. All families will be interviewed and surveyed at 1, 6 and 12 months after enrollment. Each family will designate 1-2 physician involved in the decision about home ventilation; each physician will be interviewed and surveyed at 1 month.

Regional Anesthesia for Totally Awake Upper Limb Surgery in PEDiatric Population

Peripheral loco-regional anesthesia (LRA) for anesthetic purposes is a technique widely used in adults for upper limb surgery. In pediatrics, LRA is not classically considered on its own. It is usually performed for analgesic purposes during sedation or general anesthesia. It is entirely possible to carry out awakened peripheral blocks in communicating children, of school age, thus avoiding the issues of GA in pediatrics, in particular in an emergency context. The use of Emla anesthetic skin patches for punctures (perfusion and LRA) as well as the viewing of cartoons on touch tablets during treatment can improve the perception of painless care such as LRA and treatment. In pediatrics, only ropivacaine at a concentration of 0.2% has Marketing Authorization in pediatrics in children under 12 for the production of peripheral blocks. On the basis of the experience of the Claude Galien Private Hospital in this area, it seems interesting to assess the feasibility of upper limb surgery under anesthetic LRA alone with a dose of ropivacaine at the concentration 0.35% in communicating children, of school age.

The Occurrence of Emergence Agitation in Pediatric Strabismus Surgery

The present trial conducted to demonstrate the administration of sugammadex as reversal of neuromuscular blockade agent reduces the incidence of emergence agitation (EA) comparing to neostigmine

Hospital Based Continuous Patient Monitoring System

In Ghana, and many other low and middle income countries in Africa, manual vital signs monitoring is the prevalent mode of vital signs monitoring because continuous bedside monitors are non-functional. This lack of continuous vital signs monitoring may result in missed opportunities to catch physiologic deterioration. The investigators propose to develop a dashboard that is based on the Garmin Venu 3, a consumer wearable device that reliably measures heart rate, SPO2, and respiratory rate, as an alternative to bedside monitors in hospitals in Ghana.

Innovative Prosthetic Systems for Pediatric Limb Loss to Accommodate Growth

An immediate fit adjustable prosthetic system is being investigated for children. The age range being recruited is from 3 years up to 18 years old. Phase I of this study will involve developing a final prototype by conducting in-lab testing on children with limb loss. Phase II will involve a two month home trial to further evaluate the comfort and usability of the socket. Outcome measures include a PEQ-based questionnaire, socket pressure measures and gait analysis.

Impact of a Double-reading Animated Film (Child, Parents) in Preoperative on the Anxiety of Children Upon Arrival at the Operating Room

Preoperative anxiety affects 40 to 60% of children. A literature review concluded that effective preparation strategies must involve parents, must be appropriate to their age and proposed upstream of the intervention. A double-readinganimated film aimed at informative was created for the children and their parents/legal guardians in order to solve the problem of anxiety found at the arrival of children in the operating room.

Hepatic Artery Stenosis and Thrombosis After Liver Transplantation in Children

The goal of this observational study is to investigate the incidence, current management practices, and outcomes in pediatric patients with HAC after liver transplantation. Research question: * What are the overall incidence of HAC and the effectiveness of all treatment strategies for HAC after pediatric LT? * What are the current management practices in the experience of centers, anticoagulant therapy, screening protocol, and assessment criteria for HAC after pediatric LT? The burden of participation is considered to be minimal, and limited to the questionnaires.

PRotein Optimization With eXercise to Improve MUscle Mass and Functional outcomeS

The investigators have designed a 2-center, pilot feasibility, randomized controlled trial (PROXIMUS) to determine the feasibility and safety of a larger multi center, randomized open-label trial comparing high protein combined with individualized exercise vs. standard management during the acute phase of critical illness in children. The investigators aim to determine the impact of the intervention on preservation of muscle mass; and functional status at 1 month and 6 months after randomization.

Study of CD19 Allogeneic Memory T-cell Therapy for Relapsed/Refractory CD19+ Leukemia

This is a Phase I clinical study evaluating the safety and maximum tolerated dose of a novel CAR T-cell product: allogeneic memory (CD45RA- negative) T-cells expressing a CD19-specific CAR 41BBz (CD19-CAR.CD45RA- negative T-cells) for the treatment of patients ≤ 21 years old with relapsed and/ or refractory CD19-positive leukemia. Primary Objective To determine the maximum tolerated dose (MTD) and characterize the safety profile and dose-limiting toxicities (DLTs) of treatment with allogeneic CD19-CAR.CD45RA-negative T-cells in pediatric, adolescent and young adult patients ≤ 21 years of age, with relapsed and/or refractory CD19-positive leukemia. Secondary Objectives * To evaluate the anti-leukemic activity of allogeneic CD19-CAR.CD45RA-negative T-cells. * To determine rates and severity of graft-versus-host-disease (GVHD) after treatment with allogeneic CD19-CAR.CD45RA-negative T-cells. Exploratory Objectives * To study the expansion, persistence and phenotype of allogeneic CD19-CAR.CD45RA-negative T-cells. * To characterize the cytokine profile in the peripheral blood and CSF after treatment with allogeneic CD19-CAR.CD45RA-negative T-cells. * To assess whether allogeneic CD19-CAR.CD45RA-negative T-cells acquire functional versus exhaustion-associated epigenetic programs. * To determine immune reconstitution post treatment, and the clonal structure and endogenous repertoire of allogeneic CD19-CAR.CD45RA-negative T-cells and relate inferred specificity to CAR response profiles. * To characterize incidence and mechanisms of relapse post-therapy with allogeneic CD19-CAR.CD45RA-negative T-cells.

An Endotracheal Tube Inserted to the Level of the Hypopharynx

The goal of this observational study is to assess the incidence of successful ventilation using an ETT inserted orally above the vocal cords in pediatric patients undergoing surgeries. The main question it aims to answer are: Is the use of an endotracheal tube (ETT), positioned in the hypopharynx with its tip placed just above the vocal cords an easy, safe and effective way for the ventilation of pediatric patients who are undergoing surgery under general anesthesia via orotracheal intubation? The procedures to be performed in this study are standard of care. They are also used by pediatric anesthesiologists in the oral intubation of pediatric patients suffering from tumor or abscess.

Pediatric Vaccinations

As the Covid-19 pandemic hit New York City in the spring of 2020, many families were home-bound due to the city-wide lockdown. As a result, families in New York City were unable to attend their child's healthcare appointments and many young children did not receive their regularly-scheduled vaccinations. Parts of the city-wide lockdown did eventually lift in the summer of 2020 as positive Covid-19 virus rates declined in NYC. However, regularly scheduled vaccination rates (non-Covid-related) remain low. Yet, it is critical that young children receive their mandatory vaccines in a timely manner to decrease the chance of contracting preventable illnesses. Thus, the investigators seek to increase vaccination rates among children ages 0-2 years in Sunset Park Brooklyn.

RISE With Residents

Prevention of child maltreatment (CM) remains a public health priority in Canada; approximately one-third of Canadians report exposure to at least one form of CM. Physicians play an important role in recognizing and responding to CM and its associated sequelae. However, increasing evidence indicates that physicians receive insufficient training related to recognizing and responding to CM. CM education is especially pertinent during the pediatric and psychiatry residency period but it remains unclear what the optimal approach is for preparing Canadian physicians with the knowledge and skills to effectively recognize and respond to CM. Those educational interventions that have been evaluated in medical education contexts have comparatively little emphasis on the complex overlap between IPV, children's exposure to IPV, and other forms of CM. The Violence, Evidence, Guidance, Action Project (VEGA) is a novel educational intervention that has the potential to improve the preparation of physicians to be able to effectively recognize and respond to CM in their clinical encounters and takes into account this complex overlap. The purpose of this study is to assess the acceptability and feasibility of a future randomized-controlled trial comparing two approaches to administering the VEGA intervention, facilitator-led or self-directed VEGA and whether/how these approaches can support residents' education. The investigators hypothesize that there will be significant increases in preparedness, knowledge and skills, and self-efficacy to recognize and respond to CM in both the experimental and AC arms from Time 1 (baseline) to Time 2 (immediately after the intervention) and Time 1 (baseline) to Time 3 (3 month follow-up). The investigators also predict that these improvements will be slightly attenuated in the experimental arm. Qualitative data pertaining to perceived value and impact will corroborate the quantitative findings.

Multiphase Optimization Strategy (MOST) for Treating Pediatric Post-traumatic Stress Disorder (PTSD)

The goal of this clinical trial is to test the extent to which different treatment components work to improve pediatric post-traumatic stress disorder (PTSD). It will also provide evidence for how these components work. The main research questions are: What are the effects of different components used to treat PTSD? What do these components change to produce benefits in PTSD? Researchers will: Compare components to a psychological placebo to estimate their effects and measure how they work Examine how components work alone and in conjunction with other components Participants will: Receive different combinations of components and placebo Attend weekly treatment sessions Provide information to evaluate changes in PTSD

Exercise MRI to Evaluate Cardiorespiratory Fitness in Children With Heart Disease

There are many barriers to heart-healthy lifestyles in pediatric patients with acquired and congenital heart disease. Investigators want to further understand how participants heart and skeletal muscles work together during exercise and evaluate the impact on cardiac function. To do this, the investigators will use magnetic resonance imaging (MRI) to scan the heart and skeletal muscles during exercises to assess blood flow, oxygenation and function.

HIIT in Pediatric Heart Transplant Recipients (MedBIKE™)

Heart transplantation is the long-term treatment for children and adults with advanced heart failure. Post-transplant outcomes have improved over time, such that 50% of pediatric heart transplant recipients (HTR) remain alive with a need for re-transplantation 17-years following the initial transplant. With improved short- and medium-term outcomes, focus has shifted towards optimizing long-term survival and reducing transplant-associated morbidities. This includes strategies aimed at optimizing cardiorespiratory fitness and physical activity levels. Pediatric and adult HTRs have reduced exercise capacity compared with the general population. Previous groups have shown gradual improvements in heart rate response to exercise and exercise capacity in pediatric HTRs. However, after an initial improvement, exercise capacity appears to plateau, or even decline in pediatric HTRs, and remains sub-optimal compared with the general population. Most exercise interventions in HTRs to date have focused on moderate-intensity continuous exercise (MICE), with some resistance components incorporated. More recently, high-intensity interval training (HIIT), consisting of short, intense bursts of exercise with rest periods, has been explored in the adult HTR population, with findings to date suggesting that it may yield greater improvements in cardiorespiratory fitness compared with MICE. Exercise interventions, particularly HIIT interventions, have consistently shown clinically important improvements in exercise capacity in adult HTRs that are linked with improved long-term post-transplant outcomes and well-being. Unfortunately, trials of exercise interventions in pediatric HTRs remain lacking. This study team is proposing an assessment of the feasibility of a home-based HIIT exercise program using a novel telemedicine-enable video game linked customizable cycle ergometer (MedBIKE™).

Study of the Efficacy of Moderate Sedation With Intra-NAsal Dexmedetomidine Monitored by EEG MOnitoring

This is a prospective, monocentric diagnostic study aiming to evaluate whether a sedation score obtained by a per-procedural electroencephalogram (EEG), the PSI score, could identify patients for whom a 2 μg/kg dose of DEX would not be sufficient for the successful performance of cerebral NMRI, and who could therefore benefit from a higher dosage of DEX (4 μg/kg).

Pediatric Normative Markerless Movement Analysis

The objective of this project is to collect baseline data from a normative population of 20 children that gender and age matches the population of patients expected to be treated by Mary Bridge Children's Research \& Movement Lab.

HIIT in Youth With Congenital Heart Disease (MedBIKE)

Congenital heart disease (CHD), the most common birth defect, is present in nearly 1% of the population. CHD patients are associated with intense resource utilization and premature death in adulthood. The risk of premature death is linked with reduced exercise capacity, a finding consistently noted in youth with CHD. Reduced exercise capacity in this population has also been associated with reduce physical activity and health-related quality of life. Cardiac rehabilitation (CR) in adults with acquired heart disease is an established secondary prevention strategy that improves exercise capacity. The investigators propose a prospective clinical trial of a home-based high intensity interval training (HIIT) program using a novel telemedicine-equipped video game-linked cycle ergometer (MedBIKE™) for 10 to 18 year olds with repaired moderate-complex CHD. The pilot study with the MedBIKE has shown promising results. The investigators now seek to study the efficacy of this program in a broader CHD population.