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?

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

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

• 20140106 (former CFZ008), Phase 1b Study of Carfilzomib in Combination with Induction Chemotherapy in Children with Relapsed or Refractory Acute Lymphoblastic Leukemia

• A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy

• 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 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, 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 2/3 Open-label Study to Evaluate the Safety, Tolerability, Efficacy and PK of Remdesivir in Participants from Birth to < 18 years of age with COVID-19

• 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 Study of PTC923 in Subjects with Phenylketonuria

• 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, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation

• A Phase 3, Open-label, and Rollover Study to Evaluate the Long-term Safety and Tolerability of Lumacaftor/Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Homozygous for F508del and 12 to &lt;24 Months of Age at Treatment Initiation

• 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 3, Randomized, Double-Blind, Multinational, Placebo-Controlled Study to Evaluate Efficacy and Safety of Teplizumab (PRV-031), a Humanized, FcR Non-Binding, anti-CD3 Monoclonal Antibody, in Children and Adolescents with Newly Diagnosed Type1 Diabetes (T1D)

• A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory Duchenne Muscular Dystrophy (DMD)

• 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 RANDOMIZED, MULTICENTER, DOUBLE-BLIND, PARALLEL, ACTIVECONTROL STUDY OF THE EFFECTS OF SPARSENTAN, A DUAL ENDOTHELIN RECEPTOR AND ANGIOTENSIN RECEPTOR BLOCKER, ON RENAL OUTCOMES IN PATIENTS WITH PRIMARY FOCAL SEGMENTAL GLOMERULOSCLEROSIS (FSGS)

• A Randomized Controlled Trial Assessing Video Enhanced Handoff Between the Emergency Department and Inpatient Units to Reduce Length of Stay for Patients with Bronchiolitis Admitted on Heated High Flow Nasal Cannula

• 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 Trial of Targeted Temperature Management with Whole Body Hypothermia for Moderate and Severe Neonatal Encephalopathy in Premature Infants 33-35 Weeks Gestational Age-A Bayesian Study

• 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 Study to Investigate the Efficacy and Safety of Dupilumab in Pediatric Patients with Active Eosinophilic Esophagitis

• 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 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

• 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

• AAML1421, A Phase 1/2 Study of CPX-351 (NSC# 775341; IND #129443) Alone Followed by Fludarabine, Cytarabine and G-CSF (FLAG) for Children with Relapsed Acute Myeloid Leukemia (AML)

• 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

• ADVL1521, A Phase 2 Study of the MEK inhibitor Trametinib (IND #119346, NSC# 763093) in Children with Relapsed or Refractory Juvenile Myelomonocytic Leukemia

• 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

• ADVL1711, A Phase 1/2 Study of Lenvatinib in Combination With Everolimus in Recurrent and Refractory Pediatric Solid Tumors, Including CNS 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

• 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

• AHOD1721 (CA209744), Risk-based, response-adapted, Phase II open-label trial of nivolumab + brentuximab vedotin (N + Bv) for children, adolescents, and young adults with relapsed/refractory (R/R) CD30 + classic Hodgkin lymphoma (cHL) after failure of first-line therapy, followed by brentuximab + bendamustine (Bv + B) for participants with a suboptimal response.

• 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, MULTICENTER STUDY TO EVALUATE THE PHARMACOKINETICS OF SINGLE AND MULTIPLE INTRAVENOUS DOSES OF PANTOPRAZOLE IN TWO AGE COHORTS OF HOSPITALIZED PEDIATRIC SUBJECTS 1 TO 16 YEARS OF AGE WHO ARE CANDIDATES FOR ACID SUPPRESSION THERAPY

• 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

• ANHL12P1, A Randomized Phase II study of Brentuximab Vedotin (NSC# 749710) and Crizotinib (NSC# 749005) in Patients with Newly Diagnosed Anaplastic Large Cell Lymphoma (ALCL) IND #117117

• AOST1321, Phase 2 Study of Denosumab (IND#127430, NSC# 744010), a RANK Ligand Antibody, for Recurrent or Refractory Osteosarcoma

• AOST1421, A Phase II Study of Human-Mouse Chimeric Anti-Disialoganglioside Monoclonal Antibody ch14.18 (Dinutuximab, NSC# 764038, IND# 4308) in Combination with Sargramostim (GM-CSF) in Patients with Recurrent Osteosarcoma

• 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)

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

• An Open label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443

• An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy for Seizures in Patients with Rare Seizure Disorders Such as Epileptic Encephalopathies Including Dravet Syndrome and Lennox- Gastaut Syndrome

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

• 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)

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

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

• BP-U-004, Phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative selection of TCR αβ+T cells in pediatric patients affected by hematological disorders

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

• Bedside Ultrasound Evaluation and Indicators of Progression in Crotalid Snakebites

• 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

• Bispectral Index and End Tidal Anesthetic Gas Concentration in Pediatric Patients undergoing Sevoflurane Anesthesia Clinical Investigation Plan

• 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

• Phase II Open Label Trial to Determine Safety & Efficacy of Tisagenlecleucel in Pediatric Non-Hodgkin Lymphoma Patients (BIANCA)

• CDRB436G2201, Phase II open-label global study to evaluate the effect of dabrafenib in combination with trametinib in children and adolescent patients with BRAF V600 mutation positive relapsed or refractory High Grade Glioma (HGG)

• CHIlled Platelet Study (CHIPS)

• CMPEACE (Pegzilarginase Effect on Arginase 1 Deficiency Clinical Endpoints): A Randomized, Double-Blind, Placebo-Controlled Phase 3 Study of the Efficacy and Safety of Pegzilarginase in Children and Adults with Arginase 1 Deficiency

• COVID-19 Anticoagulation in Children - Thromboprophylaxis (COVAC-TP) Trial

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

• CRAD001CUS224T, Phase II Study of Everolimus (RAD001, AFINITOR®) for Children with Recurrent or Progressive Ependymoma

• Camp based multi-component intervention for families of young children with type 1 diabetes

• Circulatory Support in Pediatric Heart Failure Patients Using the Jarvik 2015 VAD: A Feasibility Study

• Compatibility of C7 with the Ketogenic Diet in Patients Diagnosed with Glucose Transporter Type 1 Deficiency G1D

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

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

• Depression Prevention in Adolescents: Intervention Optimization in Preparation for Implementation Study

• 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

• Dietary treatment of Glucose Transporter Type 1 Deficiency (G1D).

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

• 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

• Early MRSA therapy in CF (streamlined eradication with repeat cycle)(STaph Aureus Resistance-Treat Early and Repeat (STAR-TER)

• 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

• Evaluation of Breastfeeding in Infants with Cleft Palate

• Evaluation of Healthy Term Infants to Define Impairment Thresholds and Promote Unbiased Assessments of High Risk Infants: A Quality Assurance measure for Network Follow-Up Assessments (Short Name: Term Reference Study)

• 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

• Examining Lamina Propria Procurement with Various Combinations of Endoscopic Biopsy Sampling Techniques in Pediatric Eosinophilic Esophagitis Patients

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

• FUVID Study

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

• Feasibility, acceptability, and pilot trial of a tele‐coaching intervention to improve treatment adherence in cystic fibrosis

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

• GC P#05.01.020, A Multicenter, Randomized, Phase III Registration Trial of Transplantation of NiCord®, Ex Vivo Expanded, Umbilical Cord Blood-derived, Stem and Progenitor Cells, versus Unmanipulated Umbilical Cord Blood for Patients with Hematological Malignancies

• Gender Disparity and Hormones in Cystic Fibrosis

• HALO Post-Approval Study: A single arm, non-randomized, multi-center clinical study of the SJMTM Masters HPTM 15mm Rotatable Mechanical Heart Valve as aortic valve replacement therapy

• 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)

• Impact of treatment of mild sleep-disordered breathing on children’s health

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

• KIDCARE Kawasaki Disease Comparative Effectiveness Trial

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

• 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)

• Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen

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

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

• 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)

• MRX-502: Randomized Double-blind Placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) – MARCH-PFIC

• 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)

• Everolimus for Children With Recurrent or Progressive Ependymoma

• NN7999-3774 Safety, Efficacy and Pharmacokinetics of N9-GP in Previously Treated Children with Hemophilia B

• 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)

• 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

• Optimization of Oxygen Saturation Targets for Preterm Resuscitation in the Delivery Room: A Randomized Controlled Trial

• 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)

• PBMTC SUP1701, Antiviral Cellular Therapy for Enhancing T-cell Reconstitution Before or After Hematopoietic Stem Cell Transplantation (ACES)

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

• Penicillin De-Labeling in the Pediatric Primary Care Setting

• Permanent gastric electrical stimulation in children and adolescents

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

• Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy

• 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

• Physical Activity in Children at Risk of Post-thrombotic Syndrome: A Pilot Randomized Controlled Trial

• Post-Intensive Care Syndrome Pediatrics, Longitudinal Cohort Study

• 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 Addendum I5Q-MC-CGAS(1.1) A Randomized, Double-Blind, Placebo-Controlled Study of Galcanezumab in Patients 6 to 17 Years of Age with Episodic Migraine – the REBUILD Study

• 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.

• Safety and Efficacy of Cerebellar tDCS in Children with Autism Spectrum Disorder

• 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.

• Therapeutic Hypothermia After Pediatric Cardiac Arrest (THAPCA)

• TransIT, Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia PBMTC NMD1601

• 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 Depressive Symptoms in Adolescents with Type 1 Diabetes using an Internet-Based Cognitive Behavioral Therapy Intervention

• Trial of Pamrevlumab (FG-3019), a Monoclonal Antibody to Connective Tissue Growth Factor, in Non-Ambulatory Subjects with Duchenne Muscular Dystrophy

• 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

• X4P-001-103, A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients with WHIM Syndrome with Open-Label Extension

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

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