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Protocols
266 protocol(s) meet the specified criteria
Age Group: C
Protocol No.TitleStatus
00055029Epilepsy Genetics Initiative (EGI) RepositoryOpen
029-PKA Phase 1/2 study to assess the pharmacokinetics and safety of multiple doses of pharmaceutical Cannabidiol Oral Solution in pediatric subjects with treatment-resistant seizure disordersOpen
09994131I-Metaiodobenzylguanidine(131I-MIBG) Therapy for Patients with Malignant Pheochromocytoma & Related Tumors: A Best Available Therapy/Compassionate Use ProtocolOpen
10-00207Discovery of Gene Mutations and Polymorphisms that Contribute to the Risk of Cardiovascular and Metabolic Disease and Macular Degeneration. Open
10-00515Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC), part of the Childhood Liver Disease Research and Education Network (ChiLDREN)Open
10-00610Clinical and neurocognitive assessment in adolescents and young adults at risk for serious psychiatric disordersOpen
10-00895A Prospective Database of Infants with CholestasisOpen
10-01241Genetics of Familial CholestasisOpen
10-01355Clinical and Molecular Investigation of Primary ImmunodeficienciesOpen
10-02012Predictors of Outcome in Patients with Strokes and Brain Injury - a Multicenter Arteriovenous Malformation Research Study (MARS)Open
10-02190Alopecia Areata Registry and Immunogenetic MechanismOpen
10-02467Genetics of Tissue-specific AutoimmunityOpen
10-02515Evolution and Progression of Myelin Injury and Cognitive Manifestations in Fabry DiseaseOpen
10-02635Risk Stratification and Identification of Immunogenetic and Microbial Markers of Rapid Disease Progression in Children with Crohn s DiseaseOpen
10-02830Genetic Risk Factors for Inflammatory Skin DiseaseOpen
10-02877Study of African Americans, Asthma, Genes, and Environments (SAGE)Open
10-04135Oral Insulin for Prevention of Diabetes in Relatives at Risk for Type 1 Diabetes Mellitus (TrialNet Protocol TN-07)Open
10-04170Induced Pluripotent Stem Cells for Cardiovascular ResearchOpen
10-04522"Preventing Inter-Generational Transmission of Stress Vulnerability and Obesity in at-risk Populations"Open
10-05034PEDIATRIC MULTIPLE SCLEROSIS COHORTOpen
10-05039 Environmental and genetic risk factors for pediatric multiple sclerosis"Open
1002703The Infant Development and Environment Study Open
10082An Expanded Access Study of the Feasibility of Using the CliniMACS® Device for CD34+ Cell Selection and T Cell Depletion for Graft-versus-Host Disease Prophylaxis in Alternative Donor Stem Cell Transplant RecipientsOpen
11-05719Molecular Genetic Analysis of Patients with Multiple Congenital Anomaly SyndromesOpen
11-06034A Randomized, Placebo-Controlled, Multi-Site Phase 2 Study Evaluating the Safety and Efficacy of Preemptive Treatment with CMX001 for the Prevention of Adenovirus Disease Following Hematopoietic Stem Cell Transplantation (The ADV HALT Trial).Open
11-06163Risk Factors for Infant Overweight in a Latino CohortOpen
11-06454Comprehensive Brain Imaging In Genetically Defined Autism PatientsOpen
11-06556Natural History of and Genetic Modifiers in Spinocerebellar AtaxiasOpen
11-06722Management of Myelomeningocele Study 2 (MOMS 2)Open
11-07311COVERED CHEATHAM PLATINUM STENT FOR PREVENTION OR TREATMENT OF AORTIC WALL INJURY ASSOCIATED WITH AORTIC COARCTATION (COAST II)Open
12-09480Long Term Investigative Follow-Up In TrialNet (LIFT) (Protocol TN-16)Open
12-10330CTLA-4 Ig (Abatacept) FOR PREVENTION OF ABNORMAL GLUCOSE TOLERANCE AND DIABETES IN RELATIVES AT-RISK FOR TYPE 1 DIABETES MELLITUS (TrialNet Protocol TN-18)Open
12-10366Study on oral microbiome and virulence genes of oral bacteria in dental caries formationOpen
12-10504The Effect of Soluble Urokinase Plasminogen Activation Receptor (suPAR) and Blocking Agents on Podocyte Activation in Normal KidneysOpen
120817Phase II Study of Everolimus for Recurrent or Progressive Low-Grade Gliomas in ChildrenOpen
12084Pediatric Myelodysplastic Syndrome and Bone Marrow Failure Disorder Registry and Tissue RepositoryOpen
13-10642Treatment of intractable epilepsy with myoclonic absences with CannabidiolOpen
13-10806Induced Pluripotent Stem Cells for Human Congenital Myelin DisordersOpen
13-11929Multicenter Phenotypic and Genetic Analysis of Vascular Overgrowth SyndromesOpen
13-11958Understanding learning development through immersion in foreign languagesOpen
13-11974CDC Public Health Surveillance for Bleeding Disorders Registry for Bleeding Disorders SurveillanceOpen
13-12160Effects of EDCs and Chronic Psychosocial Stress on Fetal GrowthOpen
13-12236Pathogen and Autoantibody Discovery in Unexplained Diseases of the Nervous SystemOpen
14-14484STAR (Service, Training, Advocacy & Research) Center for ASD and NDDsOpen
14-14533To establish a registry to obtain patient clinical data, biopsy material and stone material.Open
14-14585Predictors and Mechanisms of Serious Mental Illness: The North American Prodrome Longitudinal Study Open
14-15411Brain Vascular Malformation Consortium: Predictors of clinical course. Project 1: Modifiers of Disease Severity and Progression in Cerebral Cavernous Malformation (CCM)Open
14-15555CFCARIESFissure Caries Inhibition Study with Solea CO2-9.3 μm short-pulsed laser - A randomized, single blind, prospective, split mouth controlled, clinical trial.Open
14083An Open-Label, Dose-Escalation, Phase I/II Study to Investigate the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of the MEK Inhibitor Trametinib in Children and Adolescent Subjects with Cancer or Plexiform Neurofibromas and Trametinib in Combination with Dabrafenib in Children and Adolescents with Cancers Harboring V600 MutationsOpen
15-15769Pediatric ECMO and CefepimeOpen
15-16187Vascular effects of Infection in Pediatric Stroke (VIPS II) StudyOpen
15-16212Urinary HER2 as a biomarker for lupus nephritisOpen
15-16363Determining Risk Factors and Etiology in Pediatric Bladder and Bowel DysfunctionOpen
15-16368Multi-center Randomized Controlled Trial of Refeeding in Anorexia Nervosa.Open
15-16839Efficacy, safety, and pharmacokinetics of sulphadoxine-pyrimethamine-amodiaquine (SP-AQ), SP-AQ plus primaquine, dihydroartemisin-piperaquine (DP), DP plus methylene blue for preventing transmission of P. falciparum gametocytes in MaliOpen
15-17102Observational Study of Pediatric Rheumatic Diseases: The CARRA RegistryOpen
15-17474Brain Imaging and Cell Signaling: Insights intonthe Biology of AutismOpen
15-17491Lung Function In Latinos With Cystic FibrosisOpen
15-17756A Two-Stage Open-label Dose Escalation Safety and Efficacy Study of Adeno-Associated Virus Encoding Human aromatic L- Amino Acid Decarboxylase (AAV2-hAADC) Administered by MR-guided Infusion into the Midbrain in Pediatric patients with AADC DeficiencyOpen
15-17944Molecular Abnormalities in Neocortical EpilepsyOpen
15-18138CONGENITAL HEART DISEASE GENETIC NETWORK STUDY (CHD GENES)Open
15-18202Cryoanalgesia vs. Thoracic Epidural for Post-operative Pain Control following the Nuss ProcedureOpen
15-18415A Phase 1 study to evaluate the safety and tolerability of tandemly-purified allogeneic CD34+CD90+ HSC administered following conditioning with AMG 191 to achieve engraftment and immune reconstitution in patients with SCID Open
15-18491Surgical Repair of Proximal Hamstring Avulsion Injuries: Functional Outcomes and Ultrasound Confirmation of HealingOpen
15-18492A Pilot Study to Evaluate the Effect of Kolibree Gaming Technology on Improving Oral Hygiene and Toothbrushing Adherence in ChildrenOpen
15-18506Epigenetic Regulation of Wilson DiseaseOpen
150812PNOC 005: A Phase 1 Study of Modified Measles Virus (MV-NIS) for the Treatment of Children and Young Adults with Recurrent Medulloblastoma or Recurrent Atypical Teratoid Rhabdoid Tumors (ATRT) Open
150814Multicenter Cohort Study To Evaluate Outcomes after Receipt of Targeted Therapy Matched to an Individualized Cancer Therapy (iCat) Recommendations in Children and Young Adults with Recurrent, Refractory, or High Risk Solid Tumors: The iCat2, GAIN Consortium StudyOpen
150818Phase II Study of Clofarabine in Patients with Recurrent or Refractory Langerhans Cell Histiocytosis and LCH-related DisordersOpen
15084Discovering Therapeutic Targets Using Circulating Tumor DNA in Pediatric Patients with Solid and CNS Malignancies: A Pilot Study Open
1517377A Multicenter, Open-Label, 24-Week, Uncontrolled Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Oral Treprostinil Extended Release Tablets Following Transition from Remodulin or Inhaled Prostacyclin Therapy or as Add-on to Current PAH Therapy in De Novo Prostacyclin Pediatric Subjects Aged 7 to 17 Years with Pulmonary Arterial Hypertension Open
1517550Trial of FG-3019, a Monoclonal Antibody to Connective Tissue Growth Factor, in Non-Ambulatory Subjects with Duchenne Muscular DystrophyOpen
15981Molecular Profiling in Bone and Soft Tissue MalignanciesOpen
16-19338A PROSPECTIVE, PIVOTAL DOUBLE-BLINDED, WITHIN-SUBJECT COMPARISON OF THE SOCIAL DEVELOPMENT TESTING DEVICE AND CURRENT BEST PRACTICE CLINICAL DIAGNOSIS FOR AUTISM SPECTRUM DISORDER IN PEDIATRIC SUBJECTS 16-30 MONTHS OF AGEOpen
16-19371The Impact of Early Medical Treatment in Transgender YouthOpen
16-19542Improving Preterm Infant Outcomes with Family Integrated Care and Mobile TechnologyOpen
16-19623Melatonin for Adolescent migraine Prevention Study (MAP Study)Open
16-19770Clinical and Biological Characteristics of Hidradenitis SuppurativaOpen
16-19844Adversity and Health: Mapping a Road to Advantaged Educational and Health Outcomes in Diverse Children and Their Caregivers - A Novel Collaboration between DiverseCity and the NURTURE Center, UCSF Cardiology.Open
16-19876Clinical accuracy of a new method to wirelessly monitor skin temperature in preterm neonates using adhesive-imbedded sensorsOpen
16-19907Pediatric Optic Neuritis Prospective Outcomes Study 6 (PON1)Open
16-19931Imaging Biomarkers of Decision Making and Neuropsychiatric DiseaseOpen
16-19932FibroScan in Pediatric Cholestatic Liver Disease (FORCE) protocol developed for the ChiLDReN networkOpen
16-19936The Relation of GnRH Treatment to QTc Interval in Transgender Female Youth: A Time Series StudyOpen
16-20197Shape Up! KidsOpen
16-20340Steroids and Surfactant in Extremely Low Gestation Age Infants (SASSIE) Pilot Dose Escalation TrialOpen
16-20474Developmental Epidemiological Study of Children born through Reproductive Technology (DESCRT)Open
16-20609Strategies to Enhance New CGM use in Early childhood (SENCE): A Randomized Clinical Trial to Assess the Efficacy and Safety of Continuous Glucose Monitoring in Youth < 8 with Type 1 DiabetesOpen
16-2067618F-Fluoro-L- DOPA PET Imaging for the Detection and Localization of Focal Congenital HyperinsulinismOpen
16-20723Pilot Trial of Fetoscopic Endoluminal Tracheal Occlusion (FETO) in Severe Left Congenital Diaphragmatic Hernia (CDH)Open
16-20978Pilot to Examine Risk and Feasibility of Remote Management of BP from CKD through ESRDOpen
16-21217A Feasibility Study Evaluating a Novel Mask (Nasal Reservoir Cannula) Plus Standard Nasal Cannula vs. Standard Nasal Cannula Alone for Supplemental Oxygen Delivery in the Treatment of Hospitalized Pediatric Patients with Hypoxemia due to Severe PneumoniaOpen
16-21257TOMORROW: pediaTric use Of Macitentan tO delay disease pRogRessiOn in PAH WorldwideOpen
16081A PHASE 1/2, OPEN-LABEL, DOSE-ESCALATION AND EXPANSION STUDY OF ENTRECTINIB (RXDX-101) IN CHILDREN AND ADOLESCENTS WITH NO CURATIVE FIRST-LINE TREATMENT OPTION, RECURRENT OR REFRACTORY SOLID TUMORS AND PRIMARY CNS TUMORS, WITH OR WITHOUT TRK, ROS1, OR ALK FUSIONSOpen
160811Safety Follow-up Through 180 Days of Treatment with Remestemcel-L in Study MSB-GVHD001 in Pediatric Patients who have Failed to Respond to Steroid Treatment for Acute GVHDOpen
160814Pilot Study: Oral Microbiome and Dental Plaque Control with Livionex Dental Gel in Children Undergoing Hematopoietic Stem Cell Transplantation (HSCT)Open
160816PNOC009: A Phase I and Early Efficacy Study of Convection Enhanced Delivery of Irinotecan Liposome Injection Using Real Time Imaging with Gadolinium in Children with Diffuse Intrinsic Pontine Glioma Open
160819NF108: A Phase II Study of Binimetinib in Children and Adults with NF1 associated Plexiform Neurofibromas (PNOC010)Open
160822Comparison of High vs. Standard Dose Flu Vaccine in Pediatric Stem Cell Transplant RecipientsOpen
160825PNOC 013: A Safety and Pharmacokinetic Study of Single Agent REGN2810 in Pediatric Patients with Relapsed or Refractory Solid or Central Nervous System (CNS) Tumors and a Safety and Efficacy Trial of REGN2810 in Combination with Radiotherapy in Pediatric Patients with Newly Diagnosed Diffuse Intrinsic Pontine Glioma, Newly Diagnosed High-Grade Glioma, or Recurrent High-Grade GliomaOpen (affiliates only)
160827A Phase 3, Randomized, Adaptive Study Comparing the Efficacy and Safety of Defibrotide vs Best Supportive Care in the Prevention of Hepatic Veno-Occlusive Disease in Adult and Pediatric Patients Undergoing Hematopoietic Stem Cell TransplantOpen
160831A Phase I/II and Target Validation Study of TAK-580 (MLN2480) for Children with Low-Grade Gliomas and Other RAS/RAF/MEK/ERK Pathway Activated TumorsOpen (affiliates only)
16983Cancer Immunotherapeutics Program Tissue Acquisition ProtocolOpen
17-21497Contrast-enhanced ultrasound versus CT scan for kidney stone patient managementOpen
17-21587Milk, Growth and Microbiota: An RCT of Donor Milk vs. Formula to Supplement Breastfeeding Late Preterm NewbornsOpen
17-21772HDE Use of the Berlin Heart EXCOR® Pediatric Ventricular Assist DeviceOpen
17-22051Discovery in Unexplained and Rare DiseasesOpen
17-22141Examination of PTSD, Anxiety and Depression in Parents and Children with Pediatric Arterial Ischemic Stroke and Association with Stroke OutcomeOpen
17-22256Rate of Progression in USH2A Related Retinal Degeneration (RUSH2A)Open
17-22429Randomized, Double-Blind, Phase 3B Trial to Evaluate the Safety and Efficacy of 2 Treatment Regimens of Aztreonam 75 mg Powder and Solvent for Nebulizer Solution / Aztreonam for Inhalation Solution (AZLI) in Pediatric Subjects with Cystic Fibrosis (CF) and New Onset Respiratory Tract Pseudomonas aeruginosa (PA) Infection/ColonizationOpen
17-22447A Pilot Study to Evaluate the Effect of Livionex on Reducing Plaque Accumulation and Oral Health in ChildrenOpen
17-22535Retinal eye-tracking and targeted acuity tasks to monitor sports concussion recoveryOpen
17-22605Skeletal Effects of Pubertal Suppression In Transgender Youth (SEPSITY)Open
17-22705A Randomized, Double Blind, Phase I/II Trial of Low-Dose Interleukin-2 Immunotherapy in Established Type 1 DiabetesOpen
17-22758Pilot to Examine Risk and Feasibility of Remote Management of BP from Childhood into Early Adulthood (PERFORMANCE II)Open
17-22799A Phase I/II Feasibility Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency (ART-SCID) using a Self-Inactivating Lentiviral Vector (AProArt) to Transduce Autologous CD34 Hematopoietic CellsOpen
17-22806Gut microbiota, inflammation, and adolescent depressive symptomsOpen
17-22837Cognitive and Neuroimaging Features of Children Carrying Frontotemporal Dementia GenesOpen
17-22946User-Friendly Spirometer and Mobile App for Self-Management and Home Monitoring of Asthma Patients Phase 2Open
17-23014Multicenter, Prospectively Defined Observational Registry With Retrospective Data Collection, Evaluating Premature and Term-Near-Term Neonates With Pulmonary Hypertension Receiving Inhaled Nitric Oxide via Invasive or Noninvasive Ventilator Support at US CentersOpen
17-23312ENVISION: A Phase 3 Randomized, Double-blind, Placebo-Controlled Multicenter Study with an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyrias Open
17-23356A Pilot Trial of Using Pre-Transplant Risk Stratification and Prophylactic Defibrotide to Prevent Serious Thrombotic Microangiopathy in High-Risk Hematopoietic Stem Cell Transplant Patients Open
17-23447Is elbow immobilization necessary for pediatric traumatic elbow effusions without fracture line? Randomized controlled trial of cast vs no cast in X-ray and X-ray tomography negative injuries Open
17-23716A Phase 3, Open-label, Multicenter, 12-Month Safety and Tolerability Study of SPD489 in Preschool Children Aged 4-5 Years Diagnosed with Attention-deficit/Hyperactivity DisorderOpen
17-23723Characterizing Synergistic Effects of Physical and Cognitive Training in ChildrenOpen
17-23759Amblyopia Treatment Study (ATS20) - Binocular Dig Rush Game Treatment for Amblyopia Open
17-23787A Phase IIb Clinical Study to Assess the Pharmacokinetics, Safety, and Efficacy of the Combination Regimen of Elbasvir(EBR)/Grazoprevir (GZR) in Participants Aged 3 to less than 18 Years with Chronic Hepatitis C Infection-- MK-5172-079 Open
17-24003Longitudinal immune profiling for biomarker discovery in patients with Juvenile Dermatomyositis (JDM) Open
17-24240An Observational, Multicenter Study of the Prevalence of Cerebrotendinous Xanthomatosis (CTX) in Patient Populations Diagnosed with Early-Onset Idiopathic Bilateral CataractsOpen
170817PNOC015: An Open Label Single Arm Phase I/II study of MTX110 delivered by convection-enhanced delivery (CED) in patients with diffuse intrinsic pontine glioma (DIPG) previously treated with external beam radiation therapyOpen
170819A Phase I Study of Ad-RTS-hIL-12, an Inducible Adenoviral Vector Engineered to Express hIL-12 in the Presence of the Activator Ligand Veledimex in Pediatric Brain Tumor SubjectsOpen
170820Acupressure Intervention to reduce treatment-related symptoms in children with cancerOpen
170827A Pilot Trial Testing the Clinical Benefit of Using Molecular Profiling to Determine an Individualized Treatment Plan in Children and Young Adults with High Grade Glioma (Excluding Diffuse Intrinsic Pontine Glioma)Open
171022Fluoroethyltyrosine for the evaluation of intracranial neoplasmOpen
17254A Randomized, Double-Blind Phase 3 Study of Ibrutinib in Combination with Corticosteroids versus Placebo in Combination with Corticosteroids in Subjects with New Onset Chronic Graft Versus Host Disease (cGVHD)Open
18-24443Antiviral Cellular Therapy for Enhancing T-cell Reconstitution Before or After Hematopoietic Stem Cell TransplantationOpen
18-24453Pilot Randomized Controlled Trial of Spironolactone in Women With Nonalcoholic Steatohepatitis (NASH)Open
18-24730A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)Open
18-24731A 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 F508 del MutationOpen
18-25727Hydroxychloroquine for Prevention of Abnormal Glucose Tolerance and Diabetes in Individuals at Risk for Type 1 Diabetes MellitusOpen
180823A randomized, blinded study of fluorescence detection of pediatric primary central nervous system tumors in subjects receiving tozuleristide and imaged with the Canvas SystemOpen (affiliates only)
18103Collaboration to procure plasma and tissue from gliobastoma (GBM) patients Open
2016-6315Genomic Basis of Neurodevelopmental and Brain Outcomes in Congenital Heart DiseaseOpen
301OTC01A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OTC DeficiencyOpen
402-C-1603A Phase 2/3 Trial of the Efficacy and Safety of Bardoxolone Methyl in Patients with Alport SyndromeOpen
7323The inflammatory molecular profile for treatment response in infantile spasmsOpen
AALL0932Treatment of Patients with Newly Diagnosed Standard Risk B-Lymphoblastic Leukemia (B-ALL) or Localized B-lineage Lymphoblastic Lymphoma (B-LLy)Closed
AALL1331Risk-Stratified Randomized Phase III Testing of Blinatumomab (IND# 117467, NSC#765986) in First Relapse of Childhood B-Lymphoblastic Leukemia (B-ALL)Open
AALL1521A Phase 2 Study of the JAK1/JAK2 Inhibitor Ruxolitinib With Chemotherapy in Children With De Novo High-Risk CRLF2-Rearranged and/or JAK Pathway Mutant Acute Lymphoblastic LeukemiaOpen
AALL1621A 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)Open
AALL1631International Phase 3 trial in Philadelphia chromosome-positive Acute Lymphoblastic Leukemia (Ph+ALL) testing imatinib in combination with two different cytotoxic chemotherapy backbonesOpen
AAML1331A Phase III Study for Patients with Newly Diagnosed Acute Promyelocytic Leukemia (APL) using Arsenic Trioxide and All- Trans Retinoic AcidOpen
ABBVIE-CAPE-REGISTRYA Long-Term Non-Interventional Registry to Assess Safety and Effectiveness of Humira® (Adalimumab) in Pediatric Patients with Moderately to Severely Active Crohn's Disease (CD) – CAPEOpen
ABBVIE-M14-748An Open-Label, Multicenter Study to Evaluate the Pharmacokinetics, Safety, and Efficacy of Ombitasvir (OBV), Paritaprevir (PTV), Ritonavir (RTV) With or Without Dasabuvir (DSV) and With or Without Ribavirin (RBV) in Pediatric Subjects With Genotype 1 or 4 Chronic Hepatitis C Virus (HCV) InfectionOpen
ABC-PICUAge of Blood in Pediatric Intensive Care UnitsOpen
ACCL1633The Effectiveness of Lactobacillus plantarum (LBP, IND# 17339) in Preventing Acute Graft-versus-Host Disease (GvHD) in Children undergoing Alternative Hematopoietic Progenitor Cell Transplantation (HCT)Open
ACL-AFA Multi-Center Feasibility Trial Establishing Imaging And Biochemical Technologies As Measures Of Knee Cartilage Composition Following Acute ACL InjuryOpen
ACNS0831Phase III Randomized Trial of Post-Radiation Chemotherapy in Patients with Newly Diagnosed Ependymoma Ages 1 to 21 YearsOpen
ADVL1322A Phase II Trial of Pazopanib NSC# 737754, IND# 65747 in Children with Refractory Solid TumorsOpen
ADVL1412A Phase 1/2 Study of Nivolumab (IND#124729) in Children, Adolescents, and Young Adults with Recurrent or Refractory Solid Tumors as a Single Agent and in Combination with IpilimumabOpen
ADVL1521A Phase 2 study of the MEK inhibitor Trametinib (IND#119346, NSC#763093) in Children with Relapsed or Refractory Juvenile Myelomonocytic LeukemiaOpen
ADVL1614A Phase 1/2 Study of VX15/2503 (IND# 136181) in Children, Adolescents, or Young Adults with Recurrent or Relapsed Solid TumorsOpen
ADVL1615A Phase 1 Study of Pevonedistat (MLN4924, IND#136078), a NEDD8 Activating Enzyme (NAE) Inhibitor, in Combination With Temozolomide and Irinotecan in Pediatric Patients With Recurrent or Refractory Solid TumorsOpen
ADVL1711A Phase 1/2 Study of Lenvatinib in Combination With Everolimus in Recurrent and Refractory Pediatric Solid Tumors, Including CNS TumorsOpen
AEWS1221-CIRBRandomized 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 SarcomaOpen
AHEP1531Pediatric Hepatic Malignancy International Therapeutic Trial (PHITT)Open
ALXN-M11-001An Observational, non-interventional, multicenter, multi-national study of patients with atypical hemolytic-uremic syndrome (aHUS Registry)Open
ANBL1531ANBL1531: 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)Open
ANGELS Exploring the association between attachment, SES and healthOpen
APEC14B1The Project:Everychild Protocol: A Registry, Eligibility Screening, Biology and Outcome StudyOpen
APEC1621ANCI-COG PEDIATRIC MATCH (MOLECULAR ANALYSIS FOR THERAPY CHOICE)-PHASE 2 SUBPROTOCOL OF LOXO-101 (LAROTRECTINIB) IN PATIENTS WITH TUMORS HARBORING ACTIONABLE NTRK FUSIONSOpen
APEC1621BAPEC1621B, NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 subprotocol of JNJ-42756493 (erdafitinib) in patients with tumors harboring FGFR1/2/3/4 alterationsOpen
APEC1621CAPEC1621C: NCI-COG PEDIATRIC MATCH (MOLECULAR ANALYSIS FOR THERAPY CHOICE) - PHASE 2 SUBPROTOCOL OF TAZEMETOSTAT IN PATIENTS WITH TUMORS HARBORING ALTERATIONS IN EZH2 OR MEMBERS OF THE SWI/SNF COMPLEXOpen
APEC1621DAPEC1621D: NCI-COG PEDIATRIC MATCH (MOLECULAR ANALYSIS FOR THERAPY CHOICE)- PHASE 2 SUBPROTOCOL OF LY3023414 IN PATIENTS WITH SOLID TUMORSOpen
APEC1621GNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)- Phase 2 subprotocol of Vemurafenib in patients with tumors harboring actionable BRAF V600 mutationsOpen
APEC1621HNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 subprotocol of olaparib in patients with tumors harboring defects in DNA damage repair genesOpen
APEC1621INCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of Palbociclib in Patients With Tumors Harboring Activating Alterations in Cell Cycle GenesOpen
APEC1621JNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of BVD-523FB (Ulixertinib) in Patients With Tumors Harboring Activating MAPK Pathway MutationsOpen
APEC1621SCNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Screening ProtocolOpen
ARC003-AIMMUNEPEANUT ALLERGY ORAL IMMUNOTHERAPY STUDY OF AR101 FOR DESENSITIZATION IN CHILDREN AND ADULTS (THE PALISADE STUDY)Open
ARC004Peanut Allergy Oral Immunotherapy Study of AR101 for Desensitization in Children and Adults (PALISADE)Open
ARC007Real-World AR101 Market-Supporting Experience Study in Peanut-Allergic Children Ages 4 to 17 Years (RAMSES)Open
ARC008A MULTICENTER, OPEN-LABEL, LONG-TERM SAFETY STUDY OF AR101 CHARACTERIZED ORAL DESENSITIZATION IMMUNOTHERAPY IN SUBJECTS WHO PARTICIPATED IN A PRIOR AR101 STUDYOpen
ARC011Real-World AR101 Market-Supporting Experience Study in Peanut-Allergic Children, Active Treatment Arm Open-Label Extension Study (RAMSES OLE)Open
AUTISM-BIOMARKERAssessment of Biochemical Pathways and Biomarker Discovery in Autism Spectrum Disorder (ASD)Open
B0661037A RANDOMIZED, OPEN-LABEL, ACTIVE CONTROLLED, SAFETY AND EXTRAPOLATED EFFICACY STUDY IN PEDIATRIC SUBJECTS REQUIRING ANTICOAGULATION FOR THE TREATMENT OF A VENOUS THROMBOEMBOLIC EVENTOpen
BAMRIMagnetic resonance parameters as predictors of outcome in neonatal hypoxic-ischemic injuryOpen
BARD-APEXZZ Best African American Response to Asthma DrugsOpen
BP30153A PHASE II MULTI-CENTER, RANDOMIZED, DOUBLEBLIND, 24-WEEK, 3-ARM, PARALLEL GROUP, PLACEBO-CONTROLLED STUDY TO INVESTIGATE THE EFFICACY AND SAFETY OF RO5285119 IN CHILDREN AND ADOLESCENTS AGE 5-17 WITH AUTISM SPECTRUM DISORDER (ASD)Open
CAF-PINTCoagulation and Fibrinolysis in a Pediatric Insulin Titration TrialOpen
CCN016A multi-center, single-blind, randomized clinical trial to compare two copper IUDs:Mona Lisa NT Cu380 Mini and ParaGardOpen
CDIFFFMTNatural History of Patients undergoing Fecal Microbial Transplantation for Recurrent Clostridium Difficile InfectionOpen
CL010-168A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study to Evaluate the Safety and Efficacy of CCX168 in Patients with Anti-Neutrophil Cytoplasmic Antibody (ANCA) Associated Vasculitis Treated Concomitantly with Rituximab or Cyclophosphamide/AzathioprineOpen
CMX001-351An Intermediate-size, Expanded Access Protocol to Provide Brincidofovir for the Treatment of Serious Adenovirus Infection or Disease Open
CNTO148DML2001SIMPONI® to Arrest &#946;-cell Loss in Type 1 DiabetesOpen
CUR104An Open-Label Extension Study of CM-AT for the treatment of children with autismOpen
D1050302RLHSAFETY AND EFFECTIVENESS OF LURASIDONE IN PEDIATRIC SUBJECTS WITH SCHIZOPHRENIA AND SUBJECTS WITH IRRITABILITY ASSOCIATED WITH AUTISTIC DISORDER -D1050302Open
D1050325A 6-WEEK RANDOMIZED, PARALLEL, DOUBLE-BLIND, PLACEBO-CONTROLLED, FIXED-DOSE, MULTICENTER STUDY TO EVALUATE THE EFFICACY AND SAFETY OF LURASIDONE IN CHILDREN AND ADOLESCENT SUBJECTS WITH IRRITABILITY ASSOCIATED WITH AUTISTIC DISORDEROpen
DBSVOPThalamic DBS for Secondary DystoniaOpen
DINEDevelopmental Impact of NICU Exposures (DINE) UG3 Study Environmental Influences on Child Health Outcomes (ECHO) ProgramOpen
ES01-15002Collection of Confirmed DMD Positive and Presumptive Negative Newborn Screening DBS SpecimensOpen
ESETTEstablished Status Epilepticus Treatment TrialOpen
EXGENRectal and Oropharyngeal Swabs to Diagnose Gonococcal and Chlamydial Infection in Men and WomenOpen
EXTENDITN058AI Preserving Beta-Cell Function with Tocilizumab in New-onset Type 1 Diabetes Protocol ITN058AI Open
G1XCGDA phase I/II, non-randomized, multicenter, open-label study of G1XCGD (lentiviral vector transduced CD34+ cells) in patients with X-Linked Chronic Granulomatous DiseaseOpen
GCAGenetics of Cardiac ArrhythmiasOpen
GCA-APEXZZ Genetics of Cardiac Arrhythmias Open
GENEIMMUNEGenetic Basis of Immune System DisordersOpen
GS-US-342-1143A Phase 2, Open-Label, Multicenter, Multi-cohort Study to Investigate the Safety and Efficacy of Sofosbuvir/Velpatasvir in Adolescents and Children with Chronic HCV InfectionOpen
GS-US174-0144A Randomized, Double-Blind Evaluation of the Antiviral Efficacy, Safety, and Tolerability of Tenofovir Disoproxil Fumarate Versus Placebo in Pediatric Patients with Chronic Hepatitis B InfectionOpen
GS1113GS-US-334-1113: A Phase 4, a Long Term Follow-up Registry for Adolescent and Pediatric Subjects Who Received a Gilead Hepatitis C Virus Direct Acting Antiviral (DAA) in Gilead-Sponsored Chronic Hepatitis C Infection TrialOpen
HEALHIGH DOSE ERYTHROPOIETIN FOR ASPHYXIA AND ENCEPHALOPATHY (HEAL)Open
IMAGINE-IIA Multicenter Extension Study to Evaluate the Long-Term Safety and Durability of the Therapeutic Effect of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Pediatric Subjects with Alagille Syndrome Open
INS011-14-030A multicenter, open-label, flexible dose study to assess the long-term safety of pharmaceutical Cannabidiol Oral Solution as an adjunctive treatment for pediatric and adult subjects with a treatment-resistant seizure disorder who complete INS011-14-029, INS011-14-024, or INS011-14-025Open
INS011-15-054A Phase II Study to Assess the Efficacy and Safety of Cannabidiol Oral Solution for the Treatment of Refractory Infantile SpasmsOpen
INS011-17-103A Phase 2, Open-label, Dose-finding Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Pediatric Patients with Treatment-Resistant Childhood Absence SeizuresOpen
IXT5A Randomized Clinical Trial of Overminus 9 Spectacle Therapy for Intermittent ExotropiaOpen
JANSSENADAPTDOSEESCALA Phase 4, Multicenter, Open-label Study of Serum Infliximab Concentrations and Efficacy and Safety of Dose Escalation in Pediatric Patients with Inflammatory Bowel DiseaseOpen
JYU-ABone Marrow Cytogenetic Test Validation for UCSF Clinical LaboratoriesOpen
KNOXValidation of a hand-held spirometer to assess lung function in pediatric patients 14-14515Open
LAPSELife After Pediatric Sepsis EvaluationOpen
LIN-MD-62A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group, Safety and Efficacy Study of a Range of Linaclotide Doses Administered Orally to Children, Ages 6 to 17 Years, Who Fulfill Modified Rome III Criteria for Child/Adolescent Functional Constipation (FC)Open
LIN-MD-63A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group, Safety and Efficacy Study of a Range of Linaclotide Doses Administered Orally to Children, Ages 7 to 17 Years, With Irritable Bowel Syndrome With Constipation (IBS-C) (ie, Fulfill Rome III Criteria for Child/Adolescent IBS and Fulfill Modified Rome III Criteria for Child / Adolescent Functional Constipation)Open
LIPID-T1DCharacterization of Lipid Metabolism in Type 1 Diabetes and Relationship to Blood Sugar ControlOpen
LONG-TERM-IVFLong term health effect of assisted reproduction on childhood healthOpen
LTLT-REGISTRYOptimizing liver and cardiovascular health after pediatric liver transplant: a longitudinal cohort studyOpen
LVXSCID-NDLVXSCID-ND: A Pilot Feasibility Study of Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants Using a Self-Inactivating Lentiviral Vector to Transduce Autologous CD34+ Hematopoietic CellsOpen
M16-123An Open-Label, Multicenter Study to Evaluate the Pharmacokinetics, Safety, and Efficacy of Glecaprevir/Pibrentasvir in Pediatric Subjects with Genotypes 1 6 Chronic Hepatitis C Virus (HCV) InfectionOpen
MNK14042068A Multicenter, Double Blind, Placebo Controlled Study to Assess the Efficacy and Safety of H.P. Acthar® Gel in the Treatment of Subjects With Amyotrophic Lateral SclerosisOpen
NANT-2004-05NANT2004-05: Neuroblastoma Biology StudyOpen
NANT-2011-05NANT 2011-05: A Long Term Follow-Up Umbrella Protocol to Cover NANT Studies to Allow the Collection of Long Term Follow-Up DataOpen
NANT-2015-01Neuroblastoma Precision TrialOpen
NANT-2015-02PHASE 1 STUDY OF LORLATINIB (PF-06463922), AN ORAL SMALL MOLECULE INHIBITOR OF ALK/ROS1, FOR PATIENTS WITH ALK-DRIVEN RELAPSED OR REFRACTORY NEUROBLASTOMAOpen
NANT-2017-01NANT 2017-01: A Phase I Study of 131I-MIBG With Dinutuximab for Relapsed/Refractory NeuroblastomaOpen
NSR-XLRP-OS1Natural History of the Progression of X-Linked Retinitis PigmentosaOpen
NV25361A Phase IIIB, Randomized, Open-Label Study of Pegylated Interferon Alfa-2A in Combination with Lamivudine or Entecavir Compared with Untreated Control Patients in Children with HBEAG-Positive Chronic Hepatitis B in the Immune-Tolerant Phase.Open
P3EGSProgram in Prenatal and Pediatric Genome Sequencing (P3EGS)Open
PAKTPAKT Biospecimen RegistryOpen
PARITYProhpylactic Antibiotic Regimens in Tumor Surgery (PARITY): A Multi-Center Randomized Controlled Study Comparing Alternative Antibiotic Regimens in Patients Undergoing Tumor Resections with Endoprosthetic ReplacementsOpen
PBMTC-ONC1401The Role of KIR-favorably Mismatched Haploidentical Transplantation and KIR-polymorphisms in Determining Outcomes of Children with ALL/AML/MDS Undergoing Allogeneic Hematopoietic Cell TransplantationOpen
PBTC-029BA Phase I and Phase II and Re-Treatment Study of AZD6244 for Recurrent or Refractory Pediatric Low Grade GliomaOpen
PREMRIMagnetic Resonance Imaging in the Assessment of Brain injury in Low Birth Weight Infants Open
PRETXPTMS-PERITOProspective pilot study of metabolic syndrome in pediatric liver transplant recipientsOpen
PRODUCEPersonalized Research on Diet in Ulcerative Colitis and Crohn's DiseaseOpen
PTMS-PED-LIVER-TRANSPLANTCauses and consequences of post-transplant metabolic syndrome in pediatric liver transplant recipientsOpen
PVO-2A-201A Phase 2, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Palovarotene in Subjects with Multiple OsteochondromasOpen
RENAL-DTIDiffusion Tensor Imaging in Pediatric Renal Transplants: Testing A Potential Non-Invasive Alternative to BiopsyOpen
SHP647-302A Phase 3 Randomized, Double-blind, Placebo-controlled, Parallel-group Efficacy and Safety Study of SHP647 as Induction Therapy in Subjects with Moderate to Severe Ulcerative Colitis (FIGARO UC 302)Open
SIPSThe Impact of Seizures in Infants and Children with Ischemic Stroke (SIPS)Open
SPD489-347A Phase 3, Randomized, Double-blind, Multicenter, Parallel-group, Placebo-controlled, Fixed-dose Safety and Efficacy Study of SPD489 Compared with Placebo in Preschool Children Aged 4-5 Years with Attention-deficit/Hyperactivity DisorderOpen
TED-SBS-EXTENSIONA Prospective, Open-label, Long-term Safety and Efficacy Study of Teduglutide in Pediatric Patients with Short Bowel Syndrome Who Completed TED-C14-006Open
TIDESTYPE 1 DIABETES EXTENSION STUDYOpen
TN-19ATGGCSFAntithymocyte Globulin (ATG) and pegylated granulocyte colony stimulating factor (GCSF) in New Onset Type 1 DiabetesOpen
TN20Exploring Immune Effects of Oral Insulin in Relatives at Risk for Type 1 Diabetes Mellitus (TrialNet Protocol TN-20)Open
TRANSPLANTBIOMARKERSBiomarkers and Mechanisms of Disease and Organ Transplantation Open
UCDC-5101Longitudinal Study of Urea Cycle DisordersOpen
UX007-TRIHEPTANOINAn Open-label Long-Term Safety and Efficacy Extension Study in Subjects with Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Previously Enrolled in UX007 or Triheptanoin StudiesOpen
UX023-CL301A Randomized, Open-Label, Phase 3 Study to Assess the Efficacy and Safety of KRN23 Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients with X-linked Hypophosphatemia (XLH)Open
VEOIBD-BIOREPOSITORYVery Early Onset Inflammatory Bowel Disease (VEOIBD) Biospecimen Repository ProjectOpen
VIDPRESAEVItamin D to PREvent Severe Asthma ExacerbationsOpen
VTS301A Phase 2b/3 Prospective, Randomized, Double-Blind, Sham-Controlled Trial of VTS-270 (2-hydroxypropyl-&#946;-cyclodextrin) in Subjects with Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) DiseaseOpen
XSGO-CH01A Phase 2 Proof-of-Concept Study of CSI-Glucagon (Continuous Subcutaneous Glucagon Infusion) to Prevent Hypoglycemia With Lower Intravenous Glucose Infusion Rates in Children Up to One Year of Age With Congenital Hyperinsulinism.Open
ZX008-1501A Multicenter, Randomized, Double blind, Parallel Group, Placebo controlled Trial of Two Fixed Doses of ZX008 (fenfluramine hydrochloride oral solution) as an Adjunctive Therapy in Children and Young Adults with Dravet SyndromeOpen
ZX008-1503An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy in Children and Young Adults with Dravet SyndromeOpen
ZX008-1504A Multicenter, 2-Cohort Trial to First Assess the Pharmacokinetic and Safety Profile of a Single Dose of ZX008 (Fenfluramine Hydrochloride) Oral Solution When Added to Standard of Care (Cohort 1), Followed by a Randomized, Double-blind, Placebo-controlled Parallel Group Evaluation of the Efficacy, Safety, and Tolerability of ZX008 as Adjunctive Antiepileptic Therapy to Stiripentol Treatment in Children and Young Adults with Dravet Syndrome (Cohort 2)Open
ZX008-1601A Two-Part Study of ZX008 in Children and Adults with Lennox-Gastaut Syndrome (LGS); Part 1: A Multicenter, Randomized, Double-blind, Parallel Group, Placebocontrolled 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 Trial to Assess Long-Term Safety of ZX008 in Children and Adults with LGSOpen