All Clinical Trials

• Prospective evaluation of the use of imaging and computational tools to improve risk stratification in children with bone cancer

• Is your child allergic to peanuts?

• 131I-Metaiodobenzylguanidine (131I-MIBG) Therapy for Relapsed/Refractory Neuroblastoma

• 1st Brain Injury Education and outpatient Navigation

• A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy (BOLD)

• A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen

• A Multi-Center, Placebo-Controlled, Double-Blind, Randomized Study Evaluating the Role of Oral Glutathione on Growth Parameters in Children with Cystic Fibrosis.

• A Multicenter, Open-Label Study Evaluating the Long-Term Safety, Tolerability, and Efficacy of Monthly Subcutaneous Administration of Fremanezumab for the Preventive Treatment of Episodic and Chronic Migraine in Pediatric Patients 6 to 17 Years of Age A Study of the Long-Term Safety, Tolerability, and Efficacy of Fremanezumab for Preventing Migraine in Pediatric Patients A Study to Test if Fremanezumab is Effective in Preventing Migraine in Children and Adolescents Long-Term Safety Study (Phase 3)

• A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study Comparing the Efficacy, Safety, and Tolerability of Subcutaneous Administration of Fremanezumab Versus Placebo for the Preventive Treatment of Chronic Migraine in Pediatric Patients 6 to 17 Years of Age Randomized, Double-Blind, Placebo-Controlled Study of Fremanezumab in Patients (6 to 17 Years) with Chronic Migraine A Study to Test if Fremanezumab is Effective in Preventing Chronic Migraine in Patients 6 to 17 Years of Age Efficacy, Safety, and Tolerability Study (Phase 3)

• A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study Comparing the Efficacy, Safety, and Tolerability of Subcutaneous Administration of Fremanezumab Versus Placebo for the Preventive Treatment of Episodic Migraine in Pediatric Patients 6 to 17 Years of Age Randomized, Double-Blind, Placebo-Controlled Study of Fremanezumab in Patients (6 to 17 Years) with Episodic Migraine A Study to Test if Fremanezumab is Effective in Preventing Episodic Migraine in Patients 6 to 17 Years of Age Efficacy, Safety, and Tolerability Study (Phase 3)

• A Natural History and Outcome Measure Discovery Study of Developmental and Epileptic Encephalopathies

• A PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED TRIAL EVALUATING THE EFFICACY AND SAFETY OF HUMAN ALLOGENEIC CARDIOSPHERE-DERIVED CELLS FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (HOPE-3)

• A Phase 1-2 Multi-Center Study to Assess the Efficacy and Safety of Abiraterone Acetate as Adjunctive Therapy in Pre-Pubescent Children with Classic 21-Hydroxylase Deficiency

• A Phase 1b/2, randomized, double-blind, placebo-controlled, multi-center study of STMC-103H in neonates and infants at risk for developing allergic disease

• A Phase 2 Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NS-089/NCNP-02 in Boys with Duchenne Muscular Dystrophy (DMD)

• A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

• A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subject With Duchenne Muscular Dystrophy (EMBARK)

• A Phase 3 Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of Chronocort in the Treatment of Participants Aged 16 Years and Over with Congenital Adrenal Hyperplasia.

• A Phase 3, Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone (GNX) Treatment in Children and Adults with Tuberous Sclerosis Complex (TSC)-related Epilepsy (TrustTSC)

• A Phase 3, Open-Label Study Evaluating the Efficacy and Safety of Liprotamase in Subjects with Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency.

• A Phase 3, Prospective, Open-Label, Multicenter Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Paricalcitol Oral Solution for the Treatment of Secondary Hyperparathyroidism in Pediatric Subjects Ages 0 to 9 Years with Stage 5 Chronic Kidney Disease Receiving Peritoneal Dialysis or Hemodialysis

• A Phase 4 Multi-center, Ascending Dose, Open Label Study to Determine the Pharmacokinetics of Phenylephrine and the Pharmacodynamic Effects on Blood Pressure following Intravenous Administration of Phenylephrine Hydrochloride Injection, USP in Pediatric Subjects (≥12 to 16 years of age) Undergoing General and Neuroaxial Anesthesia

• A Phase I/II Open-label Intrathecal Administration of MELPIDA to Determine the Safety and Efficacy for Patients with Spastic Paraplegia Type 50 (SPG50) caused by a Mutation in the AP4M1 gene.

• A Randomized Controlled Trial of Abdominal Ultrasound (FAST) in Children with Blunt Torso Trauma

• A Randomized Controlled Trial of The Effect of Hydrocortisone on Survival Without Bronchopulmonary Dysplasia and on Neurodevelopmental Outcomes at 22-26 Months of Age in Intubated Infants <30 Weeks Gestational Age

• A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Pediatric Subjects with Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment

• A Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safetyof Cenobamate Adjunctive Therapy in Subjects with Primary Generalized Tonic-Clonic Seizures

• A Two-Part Study of ZX008 in Children and Adults with Lennox-Gastaut Syndrome (LGS); Part 1: A Randomized, Double-blind, Placebo-controlled Trial of Two Fixed Doses of ZX008 (Fenfluramine Hydrochloride) Oral Solution as Adjunctive Therapy for Seizures in Children and Adults with LGS, Followed by Part 2: An Open-label Extension to Assess Long-Term Safety of ZX008 in Children and Adults with LGS

• A double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of intravenous ganaxolone in status epilepticus

• A multicenter safety study of unlicensed investigational cryopreserved cord blood units (CBUs) manufactured by the National Cord Blood Program (NCBP) and provided for unrelated hematopoietic stem cell transplantation of pediatric and adult patients

• A three-year, multi-center, double-blind, extension study to evaluate the long-term safety and efficacy of ligelizumab in patients who completed ligelizumab’s Phase III studies in food allergy

• AALL0932, Treatment of Patients with Newly Diagnosed Standard Risk B-Lymphoblastic Leukemia (B-ALL) or Localized B-lineage Lymphoblastic Lymphoma (B-LLy)

• AALL1131, A Phase 3 Randomized Trial for Newly Diagnosed High Risk B- Lymphoblastic Leukemia (B-ALL) Including a Stratum Evaluating Dasatinib (IND#73789, NSC#732517) in Patients with Ph-like Tyrosine Kinase Inhibitor (TKI) Sensitive Mutations

• AALL1231, A Phase III Randomized Trial Investigating Bortezomib (NSC# 681239; IND# 58443) on a Modified Augmented BFM (ABFM) Backbone in Newly Diagnosed T-Lymphoblastic Leukemia (T-ALL) and T-Lymphoblastic Lymphoma (T-LLy)

• AALL1331, Risk-Stratified Randomized Phase III Testing of Blinatumomab (IND#117467, NSC#765986) in First Relapse of Childhood B-Lymphoblastic Leukemia (B-ALL)

• AALL15P1, A Groupwide Pilot Study to Test the Tolerability and Biologic Activity of the Addition of Azacitidine (IND# 133688, NSC# 102816) to Chemotherapy in Infants with Acute Lymphoblastic Leukemia (ALL) and KMT2A (MLL) Gene Rearrangement

• AALL1621: A Phase 2 Study of Inotuzumab Ozogamicin (NSC# 772518, IND#133494) in Children and Young Adults with Relapsed or Refractory CD22+ B-Acute Lymphoblastic Leukemia (B-ALL)

• AALL1631: International Phase 3 trial in Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) testing imatinib in combination with two different cytotoxic chemotherapy backbones

• AALL1721 CTL019G2201J: A phase II trial of tisagenlecleucel in first-line high-risk (HR) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (B-ALL) who are minimal residual disease (MRD) positive at the end of consolidation (EOC) therapy

• A Study to Determine the Outcomes of Patients With Localized B Cell Lymphoblastic Lymphoma (B-LLy) When Treated With Standard Risk B-ALL Therapy

• AAML1531, Risk-stratified Therapy for Acute Myeloid Leukemia in Down Syndrome

• ACCL1033, A Comprehensive Approach to Improve Medication Adherence in Pediatric ALL

• ACCL10P1, Computerized Cognitive Training for Pediatric Brain Tumor Patients: A Pilot Study

• ACNS0332, Efficacy of Carboplatin administered Concomitantly with Radiation and Isotretinoin as a Prop-Apoptotic Agent in Other Than Average Risk Medulloblastoma/PNET Patients.

• ACNS1123, Phase 2 Trial of Response-Based Radiation Therapy for Patients with Localized Central Nervous System Germ Cell Tumors

• ACNS1422, A Phase 2 Study of Reduced Therapy for Newly Diagnosed Average-RiskWNT-Driven Medulloblastoma Patients

• Dabrafenib Combined With Trametinib After Radiation Therapy in Treating Patients With Newly-Diagnosed High-Grade Glioma

• ADVL1621, MK-3475-051, A Phase I/II Study of Pembrolizumab (MK-3475) in Children with advanced melanoma or a PD-L1 positive advanced, relapsed or refractory solid tumor or lymphoma (KEYNOTE-051)

• ADVL1622 Phase 2 Trial of XL184 (Cabozantinib) an Oral Small-Molecule Inhibitor of Multiple Kinases, in Children and Young Adults with Refractory Sarcomas, Wilms Tumor, and Other Rare Tumors

• Larotrectinib in Treating Patients With Previously Untreated TRK Fusion Solid Tumors and TRK Fusion Relapsed Acute Leukemia

• ADVL1921, Phase 1 Study to Evaluate the Safety and Pharmacokinetics of Palbociclib (Ibrance®) in Combination with Irinotecan and Temozolomide in Pediatric Patients with Recurrent or Refractory Solid Tumors (A5481092)

• AEWS1221, Randomized Phase 3 Trial Evaluating the Addition of the IGF-1R Monoclonal Antibody Ganitumab (AMG 479, NSC# 750008, IND# 120449) to Multiagent Chemotherapy for Patients with Newly Diagnosed Metastatic Ewing Sarcoma

• AG348-C-022, A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Regularly Transfused, Followed by a 5-Year Open-label Extension Period

• AG348-C-023: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Not Regularly Transfused, Followed by a 5-Year Open-label Extension Period

• AGCT1531 A Phase 3 Study of Active Surveillance for Low Risk and a Randomized Trial of Carboplatin vs. Cisplatin for Standard Risk Pediatric and Adult Patients with Germ Cell Tumors

• AGCT1532, A Randomized Phase 3 Trial of Accelerated versus Standard BEP Chemotherapy for Patients with Intermediate and Poor-risk Metastatic Germ Cell Tumors

• AHEP0731, Treatment of Children with All Stages of Hepatoblastoma with Temsirolimus (IND#122782, NSC#683864) Added to High Risk Stratum Treatment

• AHEP1531, Pediatric Hepatic Malignancy International Therapeutic Trial (PHITT)

• AHOD1331, A Randomized Phase III Study of Brentuximab Vedotin (SGN-35, IND #117117) for Newly Diagnosed High-Risk Classical Hodgkin Lymphoma (cHL) in Children and Adolescents

• ALTE15N2, LEAHRN (Late Effects After High-Risk Neuroblastoma) Study

• ALTE1631, A Randomized Web-based Physical Activity Intervention among Children and Adolescents with Acute Lymphoblastic Leukemia

• AN OPEN-LABEL, SINGLE-DOSE, PHARMACOKINETICS STUDY OF VALTOCO® WITH OPEN-LABEL SAFETY PERIOD IN PEDIATRIC SUBJECTS WITH EPILEPSY

• ANBL1221,A Phase II Randomized Trial of Irinotecan/Temozolomide with Temsirolimus (NSC# 683864, IND# 61010) or Chimeric 14.18 Antibody (ch14.18) (NSC# 623408, IND# 4308) in Children with Refractory, Relapsed or Progressive Neuroblastoma

• ANBL1232, Utilizing Response- and Biology-Based Risk Factors to Guide Therapy in Patients with Non-High-Risk Neuroblastoma

• ANBL1531, A Phase 3 Study of 131I-Metaiodobenzylguanidine (131I-MIBG) or Crizotinib Added to Intensive Therapy for Children with Newly Diagnosed High-Risk Neuroblastoma (NBL) (IND# 134379)

• ANBL1821, A Phase 2 Randomized Study of Irinotecan/Temozolomide/Dinutuximab with or without Eflornithine (DFMO) (IND# 141913) in Children with Relapsed, Refractory or Progressive Neuroblastoma

• APEC1621: NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)

• ARST1431, A Randomized Phase 3 Study of Vincristine, Dactinomycin, Cyclophosphamide (VAC) Alternating with Vincristine and Irinotecan (VI) Versus VAC/VI Plus Temsirolimus (TORI, Torisel, NSC# 683864, IND# 122782) in Patients with Intermediate Risk (IR) Rhabdomyosarcoma (RMS)

• ATLAS-NEO (EFC17574): A Phase 3, Single-arm, Multicenter, Multinational, Open-Label, One-Way Crossover Study to Investigate the Efficacy and Safety of Fitursiran Prophylaxis in Male Participants aged &gt;/= 12 Years with Severe Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX.

• Expanded Access Protocol Using 131I-MIBG Therapy for Refractory Neuroblastoma, Pheochromocytoma, or Paraganglioma

• An Open-Label safety study for previously treated ataluren (PTC124) patients with nonsense mutation dystrphinopathy

• An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia (BOLD-EXT)

• An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC)

• An Open-label, Single Arm, Multicenter, Phase III Study on the Efficacy, Safety,and Pharmacokinetics of FP-001 42 mg Controlled Release in Patients with Central (Gonadotropin-Dependent) Precocious Puberty

• Assessment of the impact of transcatheter stenting of aortic coarctation on the left ventricular afterload and work using left ventricular pressure volume loops

• Azithromycin Therapy in Preschoolers with a Severe Wheezing Episode Diagnosed at the Emergency Department (AZ-SWED)

• Clinical Transplant-Related Long-term Outcomes of Alternative Donor Allogeneic Transplantation (BMT CTN 1702)

• Balanced Fluid Resuscitation to Decrease Kidney Injury in Children with Septic Shock

• Biofire Filmarray Bone and Joint Panel Performance and Utility Compared to Standard of Care Testing for the Diagnosis of Pediatric Septic Arthritis

• Biologic effects and efficacy of Cerebellar tDCS in Children with Autism Spectrum Disorder

• CCTL019B2003I: Managed Access Program (MAP) to provide access to CTL019, for acute lymphoblastic leukemia (ALL) or large B-cell lymphoma patients with out of specification leukapheresis product and/or manufactured tisagenlecleucel out of specification for commercial release

• Study of Efficacy and Safety of CTL019 in Pediatric All Patients

• CHIlled Platelet Study (CHIPS)

• COOLPRIME: Comparative Effectiveness for Cooling Prospectively Infants with Mild Encephalopathy

• COmparison of Methods of Pulmonary blood flow Augmentation in neonates: Shunt versus Stent (The COMPASS Trial)

• COngenital Multicenter trial of Pulmonic vAlve dysfunction Studying the SAPIEN 3 interventIONal THV Post-Approval Study - COMPASSION S3 PAS

• Cerebellar tDCS in Children with Autism Spectrum Disorder: Raynor Cerebellum Project

• Cerebellar tDCS stimulation in children and adult women with Anorexia Nervosa disorder- A Pilot Study

• Clinical outcome and microbiome biomarkers predicting success with fiber food introduction in short bowel syndrome

• Congenital Multicenter trial of Pulmonic Valve dysfunction studying the SAPIEN 3 interventional THV.

• Contrast Ultrasound for Pediatric Trauma - Comparative Evaluation (CAPTURE) Study

• Cycled Phototherapy: A Safer Effective Method to Control the Serum Bilirubin of Extremely Premature Infants?

• DHREAMS: Diaphragmatic Hernia Research & Exploration; Advancing Molecular Science

• Darbepoetin Trial to Improve Red Cell Mass and Neuroprotection in Preterm Infants

• Developing an Intervention to Promote Lethal Means Safety in Suicidal Adolescents: Randomization

• Development and Validity of a Spanish Verbal Memory Test for Children

• Development of quantitative optical tools to continuously monitor cerebral autoregulation, blood flow, oxygenation and inflammation during pediatric extracorporeal life support

• Does caudal block increase the incidence of urethrocutaneous fistula formation following hypospadias repair in infants? A multi-center prospective randomized trial

• Double-Blind Randomized Controlled Trial of Acute-Course of Ketamine Versus Midazolam for Recurrence of Suicidality in Adolescents

• EPIK-P2: A Phase II double-blind study with an upfront, 16- week randomized, placebo-controlled period, to assess the efficacy, safety and pharmacokinetics of alpelisib (BYL719) in pediatric and adult patients with PIK3CA-related overgrowth spectrum (PROS)

• EPITOPE OPEN-LABEL EXTENSION STUDY TO EVALUATE THE LONG- TERM CLINICAL BENEFIT AND SAFETY OF DBV712 IN PEANUT-ALLERGIC CHILDREN (EPOPEX)

• ESTABLISHING EFFICACY OF AN INPATIENT INTERVENTION AND PHONE APP TO REDUCE SUICIDE RISK

• Effectiveness of Repositioning and Cranial Remolding in Infants with Cranial Deformation

• Effects of Hypoglossal Nerve Stimulation on Cognition and Language in Down Syndrome and Obstructive Sleep Apnea

• Evaluating the Safety and Tolerability of HAMS-AB in Mitigating Type 1 Diabetes in Newly Diagnosed Patients

• Evaluation of Magnetoencephalography Protocols and Software

• Evaluation of SEDline EEG monitoring in pediatric patients undergoing General Anesthesia or sedation

• Evaluation of [18F]FLT PET/CT as an early predictor of outcome in pediatric solid tumors

• Exogenous melatonin administration for primary prevention of delirium in critically ill pediatric patients: A Pilot Study

• FUVID Study

• Facial metric for conversation and articulation

• Feasibility and effectiveness of real-time, remote continuous glucose monitoring in adolescents with poorly controlled type 1 diabetes

• Ferric Citrate and Chronic Kidney Disease in Children (FIT4KiD)

• Focal Cerebral Arteriopathy Steroid (FOCAS) Trial

• HFNC Initiation Flow Rate Study - A Single Center, Randomized Controlled, Feasibility Study

• HYDROXYCHLOROQUINE FOR PREVENTION OF ABNORMAL GLUCOSE TOLERANCE AND DIABETES IN INDIVIDUALS AT-RISK FOR TYPE 1 DIABETES MELLITUS

• High-Dose Erythropoietin for Asphyxia and Encephalopathy (HEAL)

• INHALE-1: A 26-week Primary Treatment Phase, with 26-week Extension, Open-label, Randomized Clinical Trial Evaluating the Efficacy and Safety of Afrezza® Versus Rapid-acting Insulin Analog Injections, Both in Combination with a Basal Insulin, in Pediatric Subjects with Type 1 or Type 2 Diabetes Mellitus

• Investigational Plan for the Clinical Study of the ARTISAN® Aphakia Lens for the Correction of Aphakia in Children

• JAK Inhibitors to Preserve C-Peptide Production in New Onset T1D:A Phase 2 Multi-Center, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subtype-Selective JAK Inhibitors for Preservation of Pancreatic β Cell Function in Newly Diagnosed Type 1 Diabetes Mellitus Protocol TN 31

• LCH-IV: (NACHO) INTERNATIONAL COLLABORATIVE TREATMENT PROTOCOL FOR CHILDREN AND ADOLESCENTS WITH LANGERHANS CELL HISTIOCYTOSIS

• LIBELULA: Debio 4326-301: An open-label, single-arm, multi-center, Phase 3 study on the efficacy, safety, and pharmacokinetics of Debio 4326, a triptorelin 12-month formulation, in pediatric participants who are receiving gonadotropin-releasing hormone agonist therapy for central precocious puberty

• LOXO-RET-18036: A Phase 1/2 Study of the Oral RET Inhibitor LOXO-292 in Pediatric Patients with Advanced Solid or Primary Central Nervous System Tumors

• Oral TRK Inhibitor LOXO-101 (Larotrectinib) for Treatment of Advanced Pediatric Solid or Primary Central Nervous System Tumors (SCOUT)

• Limbix Spark: a CBT-based mobile intervention for adolescent depression in specialty care settings

• Long-Term Safety and Efficacy of Apitegromab in Patients with Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigational Trials of Apitegromab

• Low Carbohydrate Diet: The Effects on Non Alcoholic Fatty Liver Disease in Obese Teens

• Low Dose Antithymocyte Globulin (ATG) to Delay or Prevent Progression to Stage 3 T1D (TN28 Protocol)

• Low-dose propofol infusion as an abortive treatment for migraine headaches in pediatric patients

• MASA Valve Early Feasibility Clinical Study

• MEK162, Phase I-II Study of MEK 162 for Children with Low-Grade Gliomas and Other Ras/Raf/ERK Pathway Activated Tumors

• MIBG 2014-01, A Phase II Single-Arm Study of Therapeutic Iobenguane (131I) for Relapsed, High-Risk Neuroblastoma Subjects (OPTIMUM Trial)

• Maintenance of cerebral perfusion pressure in moderate to severe traumatic brain injury attenuates cerebral nervous system inflammation after pediatric traumatic brain injury

• Management of the Patent Ductus Arteriosus in Premature Infants (PDA Trial)

• Milrinone in Congenital Diaphragmatic Hernia

• Multicenter Study of Congenital Pulmonic Valve Dysfunction Studying the SAPIEN 3 THV With the Alterra Adaptive Prestent

• Multimodality biomarkers for noninvasive assessment of the Fontan patient

• NF PROTOCOL 105, A Phase II Study of Cabozantinib (XL184) for Plexiform Neurofibromas in Subjects with Neurofibromatosis Type 1 in Children and Adults

• NMTRC 014, NMTT- Neuroblastoma Maintenance Therapy Trial Using Difluoromethylornithine (DFMO)

• Nasotracheal Intubation with Videolaryngoscopy versus Direct Laryngoscopy in Infants (NasoVISI) Trial

• Neo RQI: Evaluating the Effect of Neonatal Resuscitation Quality Improvement Cart Booster Training on Provider Positive Pressure Ventilation Skills Retention

• Neurodevelopmental outcome after standard dose sevoflurane versus low-dose sevoflurane/dexmedetomidine/remifentanil anaesthesia in young children: The TREX Trial

• Non-Invasive Diagnosis of Pediatric Pulmonary Invasive Mold Infections (DOMINIC)

• Noninvasive characterization of intracranial pulsatile dynamics in neurosurgical conditions using near infrared spectroscopy and peripheral photoplethysmography.

• OPTION: A Phase 2, Open-Label, Multicenter, 2x2 Crossover Trial to assess the Safety and Efficacy of MS1819-SD in Patients with Exocrine Pancreatic with Insufficiency due to Cystic Fibrosis

• The OUtMATCH Study

• Optimizing Individual Nutrition in Preterm Very Low Birth Weight Infants

• Optimizing Individual Nutrition in Preterm Very Low Birth Weight Infants: Randomized Clinical Trial

• Overexpression of amyloid precursor protein in adipose tissue from patients with Trisomy 21

• PBMTC NMD 1801 (CSIDE), A randomized trial of low versus moderate exposure busulfan for infants with severe combined immunodeficiency (SCID) receiving TCRαβ+/CD19+ depleted transplantation: A Phase II study by the Primary Immune Deficiency Treatment Consortium (PIDTC) and Pediatric Blood and Marrow Transplant Consortium (PBMTC) PIDTC “CSIDE” Protocol (Conditioning SCID Infants Diagnosed Early)

• POCUS: Hemostatic Potential and Joint Health in Patients with Severe Hemophilia A on Novel Replacement Therapies

• PRIORITY (PReventing InhibitOR Recurrence IndefiniTelY), A Multi-center, Prospective Study Evaluating the Rate of Inhibitor Recurrence Following Successful ITI in Patients Receiving Ongoing Once Per Weekly Factor VIII Therapy Along with Emicizumab and in Patients Who Discontinue FVIII Therapy and are on Emicizumab for Prophylaxis

• PROSpect: PRone and OScillation PEdiatric Clinical Trial

• Pediatric Acute Liver Failure Immune Response Network (PALF IRN) TReatment for ImmUne Mediated PathopHysiology A Phase 2b, Double-Blind, Three Arm, Randomized, Placebo Controlled Trial with Restricted Response Adaptive Randomization Testing the Efficacy and Safety of High Dose Methylprednisolone or Equine Anti-Thymocyte Globulin as Treatment for Acute Liver Failure in Pediatric Patients

• Pediatric CRI Studies: Part 1 Assessing Dehydration with Compensatory Reserve Index (CRI) Monitoring in Children with Vomiting and Diarrhea

• Pediatric Dose Optimization for Seizures in Emergency Medical Services (PediDOSE)

• Pediatric Influence of Cooling duration on Efficacy in Cardiac Arrest Patients (P-ICECAP)

• Percutaneous Intervention Versus Observational Trial of ArterialDuctus in Low weight Infants (PIVOTAL)

• Permanent gastric electrical stimulation in children and adolescents

• Phase 1 Intrathecal Lumbar Administration of AAV9/CLN7 for Treatment of CLN7 Disease

• Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy

• Phase 3, multicenter, randomized, double-blind, group sequential, placebo-controlled study to assess efficacy and safety of rimegepant for the treatment of migraine (with or without aura) in children and adolescents 6 to 18 years of age

• Prevention of Urinary Stones with Hydration (PUSH)

• Prospective Evaluation of a Standardized Approach to Diagnosis (PREDICT) and Treatment (PATIENCE) of Nontuberculous Mycobacteria Disease in Cystic Fibrosis.

• Prospective, non-randomized pivotal clinical study to assess the safety and efficacy of the bioabsorbable pulmonary valved conduit in subjects undergoing Right Ventricular Outflow Tract (RVOT) reconstruction

• Protocol Title: A Phase 2/3 Randomized, Placebo-Controlled, Double-blind, Clinical Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Vericiguat in Pediatric Participants with Heart Failure due to Left Ventricular Systolic Dysfunction Consistent with Dilated Cardiomyopathy (VALOR)

• Quantification of Glucose-6-Phosphate (G6P) and Gamma-Aminobutyric Acid (GABA) in the brain using 7T MRI or 3T Spectroscopy in Lafora Disease and Neurogenetic Diseases.

• Quantification of N-acetylglucosamine-asparagine (GlcNAc-Asn) in the brain using MR Spectroscopy in non Aspartylglucosaminuria patients

• Quantitative Imaging for Diagnosis of Cystic Fibrosis-Associated Hepatobiliary, Pancreatic, and Splenic Disease

• RITUXIMAB-PVVR FOLLOWED BY ABATACEPT VERSUS RITUXIMAB-PVVR ALONE IN NEW ONSET TYPE 1 DIABETES (Protocol TN-25)

• Radiation-free heart catheterization using MRI

• Randomized Controlled Trial of Budesonide + Surfactant versus Surfactant Alone in Extremely Preterm Infants (“The Budesonide in Babies (BiB) Trial”)

• Randomized Trial of the Safety and Effectiveness of 3% vs. 23% Hypertonic Saline for the Treatment of Pediatric Intracranial Hypertension: A Pilot Study

• SJMB12, A Clinical and Molecular Risk-Directed Therapy for Newly Diagnosed Medulloblastoma

• SPOC-2012-001, Phase 1 Dose-escalating Study of MM-398 (Irinotecan Sucrosofate Liposome Injection) plus Intravenous Cyclophosphamide in Recurrent or Refractory Pediatric Solid Tumors

• SPOC-2013-001, Phase I Study of Fenretinide (4-HPR, NSC 374551) Lym-X-Sorb(LXS) Oral Powder Plus Ketoconazole Plus Vincristine in Patients with Recurrent or Resistant Neuroblastoma (IND #: 68,254)

• SPOC-2014-001, Expanded Access Study of Fenretinide (4-HPR, NSC 374551) Lym-X-Sorb(LXS) Oral Powder Plus Ketoconazole in Patients with Recurrent or Resistant Neuroblastoma (IND #68,254)

• Safeguarding the Brain Of Our Smallest Children – an open-label phase-III randomized trial of cerebral oximetry combined with a treatment guideline versus treatment as usual in premature infants.

• Standardizing Treatments for Pulmonary Exacerbations: A platform for evaluating treatment decisions to improve outcomes (STOP360)

• T2012-002, A Pilot Study of Vincristine Sulfate Liposome Injection (Marqibo®) in Combination with UK ALL R3 Induction Chemotherapy for Children, Adolescents, and Young Adults with Relapse of Acute Lymphoblastic Leukemia

• Nivolumab in Combination With 5-azacytidine in Childhood Relapsed/Refractory AML

• T2017-002, A TACL Phase 1/2 Study of PO Ixazomib in Combination with Chemotherapy for Childhood Relapsed or Refractory Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma

• TOPPLE T1D A multiple ascending dose trial investigating safety, tolerability and pharmacokinetics of NNC0361-0041 administered subcutaneously to patients with type 1 diabetes mellitus

• Tacrolimus Everolimus Against Tacrolimus Mycophenolate mofetil in Pediatric Heart Transplant Recipients using the Major Adverse Transplant Events Score

• Targeting the RANK/RANKL/OPG pathway for treatment of osteoporosis in Cystic Fibrosis

• The CHEC-SC Cohort Study: CHaractErizing CFTR Modulated Changes in Sweat Chloride and their Association with Clinical Outcomes.

• The use of a program like The Listening Program with bone conduction headphones changes hypersensitivity to sound and behavioral responses associated with flight/fight responses of children with Autism Spectrum Disorder therefore increasing adaptive life skills.

• TransIT Protocol: A Phase III Randomized Trial Comparing Unrelated Donor Bone Marrow Transplantation with Immune Suppressive Therapy for Newly Diagnosed Pediatric and Young Adult Patients with Severe Aplastic Anemia

• Treatment Use of the CliniMACS® CD34 Reagent System to Prepare Cells for an Unlabeled Indication Using an HLA-Compatible Related or Unrelated Donor for Allogenic Transplant

• Treatment of Obstructive Sleep Apnea with Personalized Surgery in Children with Down Syndrome (TOPS-DS)

• Trial of Indication-based Transfusion of Red Blood Cells in ECMO

• Use of the Medtronic Contegra Pulmonary Valved Conduit Device: Humanitarian Use Device (HUD)

• Utilizing A Single Session Problem-Solving Intervention with Caregivers of Pediatric Patients Receiving Chronic Transfusion to Treat Sickle Cell Disease

• VGA039-CP001, A Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study of VGA039 Following Intravenous or Subcutaneous Administration of Single Ascending Doses in Healthy Adults and Adult Patients with von Willebrand Disease.

• X4P-001-104, A Phase 1b, Open-Label, Multicenter Study of Mavorixafor in Patients with Severe Congenital Neutropenia and Chronic Neutropenia Disorders

• iotaSOFT™ Insertion System for Use with Cochlear Implant Surgery in Pediatrics

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