Active Research Projects
Our research team is always looking for ways to improve the health of children. There are several clinical trials that are currently under way. For questions on our research operations, please contact Dennis Carson at 682-885-7491.
Find an active research study
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16T-MC-AMBU(a) - A Multicenter, Open-Label PK Study of Mirikizumab in Pediatric Patients with Moderately to Severely Active Ulcerative Colitis. SHINE-1
Primary Investigator: Clifton Huang
IRB ID: 2019-081 (OPEN)
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A Randomized Trial in 2 parts: Double-Blind, Placebo-Controlled, Crossover Part 1 and Open-label Part 2, Evaluating the Efficacy and Safety of Dasiglucagon for the Treatment of Children with Congenital Hyperinsulinism?
Primary Investigator: Paul Stephen Thornton
IRB ID: 2019-087 (OPEN)
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A Clinical and Molecular Risk-Detected Therapy for Newly Diagnosed Medulloblastoma (SJMB12)
Primary Investigator: Jeffrey C. Murray
IRB ID: 2014-025 (OPEN)
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A Comprehensive Approach to Improve Medication Adherence in Pediatric ALL
Primary Investigator: Karen Halaburt Albritton
IRB ID: 2014-084 (OPEN)
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A double-blind, randomized, placebo-controlled trial of adjunctive ganaxolone treatment in children and young adults with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) followed by long-term open-label treatment.
Primary Investigator: M. Scott Perry
IRB ID: 2018-090 (OPEN)
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A Learning Healthcare System for Pediatric Epilepsy
Primary Investigator: M. Scott Perry
IRB ID: 2019-064 (OPEN)
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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 Trials
Primary Investigator: Mary Suzanne Whitworth
IRB ID: 2015-061 (OPEN)
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A Long-Term Non-InterventionalRegistry to Assess Safety and Effectiveness of Humira� (Adalimumab) in Pediatric Patients withModerately to Severely Active Crohn's Disease (CD).
Primary Investigator: Nicholas Ogunmola
IRB ID: 2015-052 (OPEN)
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A Long-term Observational Study Evaluating Eteplirsen in Patients with Duchenne Muscular Dystrophy under Conditions of Routine Clinical Practice
Primary Investigator: Warren A. Marks
IRB ID: 2019-007 (OPEN)
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A master protocol to test the impact of discontinuing chronic therapies in people with cystic fibrosis on highly effective CFTR modulator therapy (SIMPLIFY)
Primary Investigator: Karen Schultz
IRB ID: 2020-024 (OPEN)
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A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs) for Transplantation in Pediatric and Adult Patients with Hematologic Malignancies and Other Indications
Primary Investigator: Gretchen Eames
IRB ID: 2011-191 (OPEN)
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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
Primary Investigator: Gretchen Eames
IRB ID: 2015-077 (OPEN)
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A multi-center study to describe the epidemiology, clinical presentation, and outcomes of pediatric patients with Covid-19 across the United States.
Primary Investigator: Mary Suzanne Whitworth
IRB ID: 2020-029 (OPEN)
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A Multicenter, Open-label, Randomized Phase 2 Study to Compare the Efficacy and Safety of Lenvatinib in Combination with Ifosfamide and Etoposide versus Ifosfamide and Etoposide in Children, Adolescents and Young Adults with Relapsed or Refractory Osteosarcoma (OLIE).
Primary Investigator: Kelly L. Vallance
IRB ID: 2020-014 (OPEN)
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A natural history study of neonatal enteroviral sepsis, to identify endpoints for subsequent therapeutic studies with new drugs being developed by pharmaceutical companies.DMID 19-0026 Enteroviral Sepsis Natural History - Whitworth
Primary Investigator: Mary Suzanne Whitworth
IRB ID: 2020-031 (OPEN)
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A Phase 1 Study of Aurora Kinase A Inhibitor LY3295668 erbumine as a Single Agent and in Combination in Patients with Relapsed/Refractory Neuroblastoma
Primary Investigator: M. Meaghan Granger
IRB ID: 2020-018 (OPEN)
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A Phase 1/1b, Open-Label, Dose-Escalation and Expansion Study of Entrectinib (RXDX-101) in Children and Adolescents with Recurrent or Refractory Solid Tumors and Primary CNS Tumors
Primary Investigator: Kelly L. Vallance
IRB ID: 2017-002 (OPEN)
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A Phase 1/2 Study of Lenvatinib in Combination with Everolimus in Recurrent and Refractory Pediatric Solid Tumors, including CNS Tumors
Primary Investigator: Kelly L. Vallance
IRB ID: 2019-063 (OPEN)
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A Phase 2 Randomized Study of Irinotecan/Temozolomide/Dinutuximab with or without Eflornithine (DFMO) (IND# 141913) in Children with Relapsed, Refractory or Progressive Neuroblastoma
Primary Investigator: M. Meaghan Granger
IRB ID: 2019-049 (OPEN)
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A Phase 2 Study of Dabrafenib (NSC# 763760) with Trametinib (NSC# 763093) after Local Irradiation in Newly-Diagnosed BRAFV600-Mutant High-Grade Glioma (HGG) (IND# 145355))
Primary Investigator: Sibo Zhao
IRB ID: 2019-072 (OPEN)
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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)
Primary Investigator: Kenneth M. Heym
IRB ID: 2017-049 (TEMPORARILY CLOSED TO ACCRUAL)
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A Phase 2 Study of Veliparib (ABT-888, IND # 139199) and Local Irradiation, Followed by Maintenance Veliparib and Temozolomide, in Patients with Newly Diagnosed High-Grade Glioma (HGG) without H3 K27M or BRAFV600E Mutations
Primary Investigator: Jeffrey C. Murray
IRB ID: 2019-003 (OPEN)
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A phase 2, Multicenter, Open-Label Study to Assess Appropriate Dosing and to Evaluate Safety of Crizanlizumab, with or without Hydroxyurea/Hydroxycarbamide, in Sequential, Descending Age Groups of Pediatric Sickle Cell Disease Patients with Vaso-Occlusive Crisis
Primary Investigator: Clarissa Johnson
IRB ID: 2019-024 (OPEN)
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A Phase 2, Prospective, Interventional, Open-Label, Multi-Site, Extension Study to Assess the Long-Term Safety and Tolerability of TAK-935 as Adjunctive Therapy in Patients with RareEpilepsy
Primary Investigator: M. Scott Perry
IRB ID: 2019-029 (OPEN)
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A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Ranging Study to Evaluate the Safety and Efficacy of Eluxadoline in Pediatric Patients (Age 12 to 17 Years) with Irritable Bowel Syndrome with Diarrhea (IBS-D)
Primary Investigator: Bankole Osuntokun
IRB ID: 2018-026 (OPEN)
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A Phase 2/3 Randomized, Double-blind, Palivizumab-controlledStudy to Evaluate the Safety of MEDI8897, a MonoclonalAntibody With an Extended Half-life Against RespiratorySyncytial Virus, in High-risk Children (MEDLEY)
Primary Investigator: Karen Schultz
IRB ID: 2019-048 (OPEN)
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A Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine versus Selumetinib (NSC# 748727, IND# 77782) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) not associated with BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1)
Primary Investigator: Sibo Zhao
IRB ID: 2020-011 (OPEN)
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A Phase 3 Randomized Study of Selumetinib (IND #77782) versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG))
Primary Investigator: Jeffrey C. Murray
IRB ID: 2019-075 (OPEN)
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A Phase 3 Randomized Trial for Patients with de novo AML Comparing Standard Therapy Including Gemtuzumab Ozogamicin (GO) to CPX-351 with GO, and the Addition of the FLT3 Inhibitor Gilteritinib for Patients with FLT3 Mutations
Primary Investigator: Kenneth M. Heym
IRB ID: 2020-072 (OPEN)
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A Phase 3 Randomized Trial of Inotuzumab Ozogamicin (IND#:133494, NSC#: 772518) for Newly Diagnosed High-Risk B-ALL; Risk-Adapted Post-Induction Therapy for High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and Disseminated B-LLy
Primary Investigator: Kenneth M. Heym
IRB ID: 2020-022 (OPEN)
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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
Primary Investigator: Kelly L. Vallance
IRB ID: 2017-030 (OPEN)
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A Phase 3 Trial Investigating Blinatumomab (IND# 117467, NSC# 765986) in Combination with Chemotherapy in Patients with Newly Diagnosed Standard Risk or Down syndrome B-Lymphoblastic Leukemia (B-ALL) and the Treatment of Patients with Localized B-Lymphoblastic Lymphoma (B-LLy)
Primary Investigator: Kenneth M. Heym
IRB ID: 2019-059 (OPEN)
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A Phase 3, 2-part, Open-label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor in Subjects 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del
Primary Investigator: Karen Schultz
IRB ID: 2018-053 (TEMPORARILY CLOSED TO ACCRUAL)
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A Phase 3, Open-label Study Evaluating the Long-term Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis
Primary Investigator: Karen Schultz
IRB ID: 2019-044 (OPEN)
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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
Primary Investigator: Karen Schultz
IRB ID: 2020-009 (OPEN)
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A Phase 3, Open-label, Single Arm, Multicenter Study of Ravulizumab in the Treatment of Pediatric Patients with Persistent Thrombotic Microangiopathy (TMA) after Hematopoietic Stem Cell Transplantation (HSCT)
Primary Investigator: Gretchen Eames
IRB ID: 2020-047 (OPEN)
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A Phase I/II Study of Nivolumab in Combination with 5-azacytidine in pediatric patients with relapsed/refractory acute myeloid leukemia (BMS reference CA209-9JY)
Primary Investigator: Kenneth M. Heym
IRB ID: 2019-018 (OPEN)
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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)
Primary Investigator: M. Meaghan Granger
IRB ID: 2016-078 (OPEN)
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A Phase II Trial Of Epigenetic Priming In Patients With Newly Diagnosed Acute Myeloid Leukemia? ?
Primary Investigator: Kenneth M. Heym
IRB ID: 2017-031 (OPEN)
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A Phase III Randomized Placebo Controlled Study to Examinethe Effects of DAS181 for the Treatment of Lower RespiratoryTract Parainfluenza Infection in Immunocompromised Subjects
Primary Investigator: Richard P. Howrey
IRB ID: 2019-012 (TEMPORARILY CLOSED TO ACCRUAL)
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A Phase III Randomized, Active-comparator-controlled Clinical Trial to Study the Safety and Efficacy of MK-1986 (tedizolid phosphate) and Comparator in Subjects from Birth to
Primary Investigator: Ana Maria Rios
IRB ID: 2018-024 (OPEN)
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A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) CURRENTLY TREATED WITH COMPLEMENT INHIBITORS
Primary Investigator: Donald Beam
IRB ID: 2020-048 (OPEN)
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A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA NOT PREVIOUSLY TREATED WITH COMPLEMENT INHIBITORS
Primary Investigator: Donald Beam
IRB ID: 2020-049 (OPEN)
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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
Primary Investigator: Kenneth M. Heym
IRB ID: 2016-075 (OPEN)
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A point prevalence survey regarding antibiotic use in children for four two week blocks.
Primary Investigator: Marc A. Mazade
IRB ID: 2016-016 (OPEN)
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A Process Improvement Tool to Enhance and Monitor the Treatment of Children and Adolescents with Diabetic Ketoacidosis (DKA)
Primary Investigator: Don P. Wilson
IRB ID: 2013-045 (OPEN)
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A Prospective Data Registry for the Congenital Cardiovascular Interventional Study Consortium: Risk Stratification for Cardiac Catheterization Procedures
Primary Investigator: Duy Kuo
IRB ID: 2016-064 (OPEN)
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A prospective Study of Acute Flaccid Myelitis (AFM) to Define Natural History, Risk Factors, and Pathogenetic Mechanisms? ?
Primary Investigator: Lynne M. Eger
IRB ID: 2019-074 (OPEN)
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A prospective study to evaluate biological and clinical effects of significantly corrected CFTR function in infants and young children.?
Primary Investigator: Karen Schultz
IRB ID: 2020-052 (OPEN)
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A Prospective, Long Term Registry of Patients with a Diagnosis of Spinal Muscular Atrophy
Primary Investigator: Warren A. Marks
IRB ID: 2019-052 (OPEN)
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A Randomised Phase 3 trial of Accelerated Versus Standard BEP Chemotherapy for Patients with Intermediate and Poor-Risk Metastatic Germ Cell Tumours
Primary Investigator: Kelly L. Vallance
IRB ID: 2018-089 (TEMPORARILY CLOSED TO ACCRUAL)
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A Randomized Phase 2 Trial of Axitinib/Nivolumab Combination Therapy vs. Single Agent Axitinib or Nivolumab for the Treatment of TFE/Translocation Renal Cell Carcinoma (tRCC) Across All Age Groups
Primary Investigator: Kelly L. Vallance
IRB ID: 2019-101 (OPEN)
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A Randomized, Double-Blind Evaluation of the Pharmacokinetics,Safety, and Antiviral Efficacy of Tenofovir Alafenamide (TAF) inAdolescents with Chronic Hepatitis B Virus Infection
Primary Investigator: Mary Suzanne Whitworth
IRB ID: 2016-091 (TEMPORARILY CLOSED TO ACCRUAL)
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A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Reducing Supraphysiologic Glucocorticoid Use in Adult Subjects with Classic Congenital Adrenal Hyperplasia" ?
Primary Investigator: Paul Stephen Thornton
IRB ID: 2020-039 (OPEN)
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A Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy and Safety of Dupilumab in Pediatric Patients with Active Eosinophilic Esophagitis (EoE)
Primary Investigator: Bankole Osuntokun
IRB ID: 2020-010 (OPEN)
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A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Adult Subjects with Classic Congenital Adrenal Hyperplasia
Primary Investigator: Paul Stephen Thornton
IRB ID: 2020-038 (OPEN)
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A TACL Phase 1/2 Study of PO Ixazomib in Combination with Chemotherapy for Childhood Relapsed or Refractory Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma
Primary Investigator: Kenneth M. Heym
IRB ID: 2019-036 (OPEN)
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A Test of Children�s English/Spanish Speech Perception in Noise or Speech Maskers
Primary Investigator: Lisa Vaughan
IRB ID: 2019-040 (OPEN)
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AAML1531, Risk-stratified Therapy for Acute Myeloid Leukemia in Down Syndrome
Primary Investigator: Kenneth M. Heym
IRB ID: 2016-031 (OPEN)
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ALTE03N1 - Key Adverse Events After Childhood Cancer
Primary Investigator: Donald Beam
IRB ID: 543 (OPEN)
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ALTE07C1, Neuropsychological, Social, Emotional, and Behavioral Outcomes in Children with Cancer
Primary Investigator: Jeffrey C. Murray
IRB ID: 2009-013 (OPEN)
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ALTE1621, Pharmacologic Reversal of Ventricular Remodeling in Childhood Cancer Survivors at Risk for Heart Failure (PReVent-HF): A Phase 2b Randomized Placebo-Controlled (Carvedilol) Trial
Primary Investigator: Donald Beam
IRB ID: 2017-005 (TEMPORARILY CLOSED TO ACCRUAL)
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An Open-label Study with a Nonrandomized Single-dosePhase in Subjects with Suspected or Confirmed AerobicGram-negative Bacterial Infections followed by aRandomized, Multiple-dose, Active-controlled Phase inSubjects with Suspected or Confirmed ComplicatedUrinary Tract Infection (cUTI), to Assess the Safety,Tolerability, and Pharmacokinetics of Cefiderocol inHospitalized Pediatric Subjects 3 Months to < 18 Years ofAge?
Primary Investigator: Lynne M. Eger
IRB ID: 2019-066 (OPEN)
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An Expanded Access Protocol for Use of Triheptanoin in Patients with Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)
Primary Investigator: Adrian Lacy
IRB ID: 2019-027 (OPEN)
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An Extension Trial Evaluating the Long-term Safety and Efficacy of Dasiglucagon for the Treatment of Children with Congenital Hyperinsulinism
Primary Investigator: Paul Stephen Thornton
IRB ID: 2019-103 (OPEN)
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An Intermediate-size, Expanded Access Protocol to Provide Brincidofovir for the Treatment of Serious Adenovirus Infection or Disease
Primary Investigator: Richard P. Howrey
IRB ID: 2016-008 (OPEN)
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An Observational Study to Investigate Cognition and Quality of Life in Children and Adolescents with Dravet Syndrome
Primary Investigator: M. Scott Perry
IRB ID: 2019-032 (OPEN)
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An Open-Label Extension Study Of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy
Primary Investigator: Warren A. Marks
IRB ID: 2019-084 (OPEN)
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An Open-Label Multiple Dose Study of RZ358 in Patients With Congenital Hyperinsulinism
Primary Investigator: Paul Stephen Thornton
IRB ID: 2020-045 (OPEN)
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An Open-Label Trial of Triheptanoin in Patients with Glucose Transporter Type-1 Deficiency Syndrome (GLUT1 DS)
Primary Investigator: Adrian Lacy
IRB ID: 2013-034 (OPEN)
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An Open-label, Expanded Access Protocol for Pediatric Patients with Sickle Cell Disease Who Have No Alternative Treatment Options
Primary Investigator: Clarissa Johnson
IRB ID: 2020-085 (OPEN)
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An Open-Label, Expanded Access Protocol Using 131-I-Metaiodobenzylguanidine (131 I-MIBG) Therapy in Patients with Refractory Neuroblastoma, Pheochromocytoma, or Paraganglioma
Primary Investigator: M. Meaghan Granger
IRB ID: 2012-026 (OPEN)
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An Open-label, Non-investigational Product, Multi-center, Lead-in Study to Evaluate Prospective Bleeding and Infusion Data of Current FVIII Replacement Therapy in Adult Males with Hemophilia A
Primary Investigator: Timothy McCavit
IRB ID: 2019-105 (TEMPORARILY CLOSED TO ACCRUAL)
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An?? NIH CSER program clinical sequencing study grant collaboration
Primary Investigator: Kelly L. Vallance
IRB ID: 2018-058 (OPEN)
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ANHL12P1, A Randomized Phase II Trial of Brentuximab Vedotin or Crizotinib in combination with Chemotherapy for Newly Diagnosed Patients with ALCL (IND#117117)
Primary Investigator: Kenneth M. Heym
IRB ID: 2015-007 (TEMPORARILY CLOSED TO ACCRUAL)
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APEC1621M: PHASE 2 SUBPROTOCOL OF TIPIFARNIB IN PATIENTS WITH TUMORS HARBORING HRASGENOMIC ALTERATIONS
Primary Investigator: Kelly L. Vallance
IRB ID: 2020-070 (OPEN)
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Can we salvage young children with relapse medulloblastoma after initial radiation sparing approaches?
Primary Investigator: Jeffrey C. Murray
IRB ID: 2020-044 (OPEN)
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Children�s Mercy Kansas City Cardiac High Acuity Monitoring Program (CHAMP).
Primary Investigator: Lisa M. Roten
IRB ID: 2017-033 (OPEN)
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Clinical Assessment of Flow in Tetralogy of Fallot by Time-Resolved Volumetric Phase Contrast MRI?
Primary Investigator: Steve Muyskens
IRB ID: 2015-047 (OPEN)
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Clinical Transplant-Related Long-term Outcomes of Alternative Donor Allogeneic Transplantation (CTRL-ALT-D).
Primary Investigator: Gretchen Eames
IRB ID: 2019-054 (TEMPORARILY CLOSED TO ACCRUAL)
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Clinical, Radiologic, And Molecular Characteristics Of Anaplastic Pleomorphic Xanthoastrocytomas
Primary Investigator: Sibo Zhao
IRB ID: 2020-063 (OPEN)
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CliniMACS� CD34+ T-cell Depletion of a Haploidentical Stem Cell Product as Part of a Treatment Protocol for Patients with Bone Marrow Failure
Primary Investigator: Richard P. Howrey
IRB ID: 2018-041 (OPEN)
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CNTO148UCO3003 A Phase 3 Randomized, Open Label Study to assess the efficacy, safety, and pharmacokinetics of Golimumab Treatment, a Human anti-TNFa Monoclonal Antibody, administered subcutaneously in pediatric participants with moderately to severely active Ulcerative Colitis
Primary Investigator: Bankole Osuntokun
IRB ID: 2018-099 (OPEN)
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Cognitive and Neuroimaging Biosignatures of Well-being in Youth with and without Seizures
Primary Investigator: Crystal Cooper
IRB ID: 2020-104 (OPEN)
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Comparison Between Surgical versus Balloon Angioplasty versus Intravascular Stent Placement for Recurrent or Native Coarctation of the Aorta
Primary Investigator: Deborah A. Schutte
IRB ID: 2008-03 (OPEN)
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Comprehensive Epilepsy Center Surgical Database Project
Primary Investigator: M. Scott Perry
IRB ID: 2010-068 (OPEN)
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Cook Children�s Health Care System Stroke and Thrombosis Registry
Primary Investigator: Marcela D. Torres
IRB ID: 2020-037 (OPEN)
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Diagnosis and Treatment of Pediatric Strokes Before and After Implementation of a Pediatric Stroke Alert System at a Non-Academic Institution
Primary Investigator: Fernando Acosta Jr.
IRB ID: 2020-032 (OPEN)
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Domperidone Use in Patients with Gastrointestinal Disorders
Primary Investigator: Bankole Osuntokun
IRB ID: 2018-088 (OPEN)
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Early MRSA therapy in CF � Optimizing the Regimen (STaph Aureus Resistance-Treat Early and Repeat)
Primary Investigator: Karen Schultz
IRB ID: 2018-075 (OPEN)
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Effects of Modern Chemotherapy Regimens on Spermatogenesis and Steroidogenesis in Adolescent and Young Adult (AYA) Survivors of Osteosarcoma
Primary Investigator: Donald Beam
IRB ID: 2018-028 (OPEN)
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ELX/TEZ/IVA Expanded Access Program for Patients 6 Through 11 Years of Age With Cystic Fibrosis Who Have At Least One F508del Mutation
Primary Investigator: Karen Schultz
IRB ID: 2021-003 (OPEN)
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Evaluation Of The Efficacy Of Reirradiation�in Locally Recurrent Ependymoma
Primary Investigator: Jeffrey C. Murray
IRB ID: 2018-021 (OPEN)
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Exploration of Perceived Stress, Sleep, Fatigue, and Biomarkers in Childhood Brain Tumor Survivors
Primary Investigator: Lisa M Bashore NP
IRB ID: 2018-010 (OPEN)
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Health Effects after Anthracycline and Radiation Therapy (HEART): Dexrazoxane and Prevention of Anthracycline-related Cardiomyopathy
Primary Investigator: Donald Beam
IRB ID: 2014-023 (OPEN)
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Hyperinsulinism Center Registry for Research and Quality Data Collection
Primary Investigator: Paul Stephen Thornton
IRB ID: 2011-183 (OPEN)
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Ictal and Interictal High-Frequency Electrophysiological Activity for the Localization of the Epileptogenic Zone in Children with Refractory Epilepsy
Primary Investigator: Christos Papadelis PhD
IRB ID: 2019-070 (OPEN)
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International Phase 3 trial in Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) testing imatinib in combination with two different cytotoxic chemotherapy backbones
Primary Investigator: Kenneth M. Heym
IRB ID: 2017-048 (OPEN)
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International Registry for Severe Chronic Neutropenia
Primary Investigator: Donald Beam
IRB ID: 94 (OPEN)
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Intravenous Remodulin (Treprostinil) as Add-on Therapy for the Treatment of Persistent Pulmonary Hypertension of the Newborn: A Randomized, Placebo-Controlled, Safety and Efficacy Study
Primary Investigator: David Riley
IRB ID: 2015-001 (TEMPORARILY CLOSED TO ACCRUAL)
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Iobenguane I-131 or Crizotinib and Standard Therapy in Treating Younger Patients With Newly-Diagnosed High-Risk Neuroblastoma or Ganglioneuroblastoma
Primary Investigator: M. Meaghan Granger
IRB ID: 2018-048 (TEMPORARILY CLOSED TO ACCRUAL)
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Longitudinal Assessment of Ovarian Reserve in Adolescents with Lymphoma
Primary Investigator: Karen Halaburt Albritton
IRB ID: 2015-006 (OPEN)
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Long-Term Follow-Up of Patients Who Have Participated in Children's Oncology Group Studies
Primary Investigator: M. Meaghan Granger
IRB ID: 2010-109 (OPEN)
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MIBG 2014-01, A Phase II Single-Arm Study of Therapeutic Iobenguane (131I) for High-Risk Neuroblastoma Subjects at the Time of First Relapse
Primary Investigator: M. Meaghan Granger
IRB ID: 2018-079 (OPEN)
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Multi Center iMRI Neurosurgery Database
Primary Investigator: John Honeycutt
IRB ID: 2013-025 (OPEN)
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Multi-center Phase III, Randomized, Open-Label Trial of Vigil (bi-shRNAfurin and GMCSF Augmented Autologous Tumor Cell Immunotherapy) in combination with Irinotecan and Temozolimide as a Second-Line Regimen for Ewing's Sarcoma
Primary Investigator: Anish K. Ray
IRB ID: 2019-005 (OPEN)
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Multi-modal neuroimaging in children with cerebral palsy to assess functional and anatomical reorganization in relation to sensory and motor functions
Primary Investigator: Christos Papadelis PhD
IRB ID: 2019-068 (OPEN)
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NANT 2013-01: Immunotherapy of Relapsed Neuroblastoma with Expanded NK Cells, Lenalidomide, and ch14.18
Primary Investigator: M. Meaghan Granger
IRB ID: 2018-050 (OPEN)
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NANT 2015-02: Phase 1 Study of Lorlatinib (PF-06463922), An Oral Small Molecula Inhibitor of ALK/ROS1, for Patients with ALK-Driven Relapsed or Refractory Neuroblastoma
Primary Investigator: M. Meaghan Granger
IRB ID: 2017-022 (OPEN)
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NANT 2017-01: A Phase I Study of 131I-MIBG with Dinutuximab for Relapsed/Refractory Neuroblastoma
Primary Investigator: M. Meaghan Granger
IRB ID: 2018-032 (OPEN)
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National Pediatric Cardiology Quality Improvement Collaborative (NPC-QIC) - A Collaborative Initiative to Improve Care of Children with Complex Congenital Heart Disease
Primary Investigator: Lisa M. Roten
IRB ID: 2017-032 (OPEN)
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NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 study of JNJ-42756493 (Erdafitinib) in patients with tumors harboring FGFR alterations
Primary Investigator: Kelly L. Vallance
IRB ID: 2018-008 (OPEN)
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NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 study of LOXO-101 (Larotrectinib) in patients with tumors harboring NTRK fusions.
Primary Investigator: Kelly L. Vallance
IRB ID: 2018-006 (OPEN)
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NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 study of Olaparib in patients with tumors harboring defects in DNA damage repair genes
Primary Investigator: Kelly L. Vallance
IRB ID: 2018-007 (OPEN)
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NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 study of Tazemetostat in patients with tumors harboring alterations in EZH2 or members of the SWI/SNF complex
Primary Investigator: Kelly L. Vallance
IRB ID: 2018-005 (TEMPORARILY CLOSED TO ACCRUAL)
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NCI-COG Pediatric MATCH (Molecular Analysis For Therapy Choice) - Phase 2 Subprotocol Of Palbociclib In Patients With Tumors Harboring Activating Alterations In Cell Cycle Genes
Primary Investigator: Kelly L. Vallance
IRB ID: 2018-068 (OPEN)
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NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)- Phase 2 study of Ensartinib in patients with tumors harboring ALK or ROS1 genomic alterations
Primary Investigator: Kelly L. Vallance
IRB ID: 2018-003 (OPEN)
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NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)- Phase 2 study of LY3023414 in patients with solid tumors
Primary Investigator: Kelly L. Vallance
IRB ID: 2018-009 (OPEN)
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NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)- Phase 2 study of Selumetinib in patients with tumors harboring activating MAPK pathway mutations
Primary Investigator: Kelly L. Vallance
IRB ID: 2017-066 (TEMPORARILY CLOSED TO ACCRUAL)
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NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)- Phase 2 study of Vemurafenib in patients with tumors harboring BRAF V600 mutations
Primary Investigator: Kelly L. Vallance
IRB ID: 2018-004 (OPEN)
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NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Screening Protocol
Primary Investigator: Kelly L. Vallance
IRB ID: 2017-065 (OPEN)
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NCI-COG Pediatric MATCH (Molecular Analysis For Therapy Choice)-Phase 2 Subprotocol Of BVD-523FB (Ulixertinib) In Patients With Tumors Harboring Activating Mapk Pathway Mutations
Primary Investigator: Kelly L. Vallance
IRB ID: 2019-102 (OPEN)
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Neutrophil Gelatinase-Associated Lipocalin (NGAL) and serum Cystatin C levels in children after Cardiopulmonary Bypass (CPB)
Primary Investigator: James D. Marshall
IRB ID: 2020-041 (OPEN)
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Observational study in infants and children with SCN1A+ Dravet Syndrome
Primary Investigator: M. Scott Perry
IRB ID: 2020-056 (OPEN)
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Parental Attitudes in Neonatal Clinical Trial Enrollment:Decision-Making Preferences and Reasoning among Participants and Non-Participants
Primary Investigator: David Riley
IRB ID: 2017-016 (OPEN)
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Participation in the Cystic Fibrosis Patient Registry
Primary Investigator: Karen Schultz
IRB ID: 394 (OPEN)
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Pediatric Hepatic Malignancy International Therapeutic Trial (PHITT)
Primary Investigator: Lauren Akers
IRB ID: 2018-066 (OPEN)
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Pediatric International DBS (PEDiDBS) Registry
Primary Investigator: Warren A. Marks
IRB ID: 2016-013 (OPEN)
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PHASE 2 SUBPROTOCOL OF AG-120 (IVOSIDENIB) IN PATIENTS WITH TUMORS HARBORING IDH1 MUTATIONS
Primary Investigator: Kelly L. Vallance
IRB ID: 2020-055 (OPEN)
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Phase I Dravet Syndrome study in Children and Adolescents
Primary Investigator: M. Scott Perry
IRB ID: 2020-012 (OPEN)
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Prospective validation of a pediatric venous thrombosis risk prediction model using the CHAT: A Multi-Institutional Children�s Hospital-Acquired Thrombosis Registry
Primary Investigator: Marcela D. Torres
IRB ID: 2020-016 (OPEN)
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Protocol for a Research Database for Hematopoietic Cell Transplantation, Other Cellular Therapies and Marrow Toxic Injuries
Primary Investigator: Gretchen Eames
IRB ID: 2019-061 (OPEN)
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Protocol for a Research Sample Repository for Hematopoietic Cell Tranplantation, Other Cellular Therapies, and Marrow Toxic Injuries
Primary Investigator: Gretchen Eames
IRB ID: 2019-062 (OPEN)
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Quantitative Assessment of Sucking for Early Diagnosis of Brain Injury in Infants at High Risk
Primary Investigator: Christos Papadelis PhD
IRB ID: 2019-085 (OPEN)
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Randomization of Cytarabine Monotherapy versus Standard-of-Care Vinblastine/Prednisone for Frontline Treatment of Langerhans Cell Histiocytosis
Primary Investigator: Anish K. Ray
IRB ID: 2019-067 (TEMPORARILY CLOSED TO ACCRUAL)
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RBDD-PLG Project; International Retrospective and Prospective Study of Individuals affected with Hypoplasminogenemia
Primary Investigator: Marcela D. Torres
IRB ID: 2019-028 (OPEN)
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Survey of the National Lipid Association�s Membership - Opinions of the 2011 National Heart, Lung and Blood Institute�s Expert Panel Guidelines for Cardiovascular Health and Risk Reduction in Children and Adolescents.
Primary Investigator: Don P. Wilson
IRB ID: 2013-028 (OPEN)
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Testicular tissue cryopreservation for fertility preservation in patients facing infertility-causing diseases or treatment regimens
Primary Investigator: Blake W. Palmer
IRB ID: 2020-053 (OPEN)
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The American Thrombosis and Hemostasis Network (ATHN) National Web Tracker
Primary Investigator: Timothy McCavit
IRB ID: 2011-160 (OPEN)
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The �HEAL� Study, High-Dose Erythropoietin for Asphyxia and Encephalopathy (A randomized, placebo-controlled, double-masked 500-subject clinical trial of erythropoietin for the treatment of neonatal hypoxic-ischemic encephalopathy)
Primary Investigator: David Riley
IRB ID: 2017-010 (OPEN)
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The Use of Fluorodopa 18F Positron Emission Tomography Combined with Computed Tomography in Congenital Hyperinsulinism
Primary Investigator: Paul Stephen Thornton
IRB ID: 2012-060 (OPEN)
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Total Therapy Study XVII For Newly Diagnosed Patients With Acute Lymphoblastic Leukemia And Lymphoma
Primary Investigator: Kenneth M. Heym
IRB ID: 2018-080 (OPEN)
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Towards the Establishment of Standards of Practice and the Inititation of Multi-Center, Multi-National Clinical Trials for Neonates and Children with Stroke
Primary Investigator: Marcela D. Torres
IRB ID: 2012-008 (OPEN)
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Use of Oxandrolone to Promote Growth in Infants with Hypoplastic Left Heart Syndrome: A Phase I/II Pilot Study
Primary Investigator: Phillip Burch
IRB ID: 2019-014 (TEMPORARILY CLOSED TO ACCRUAL)
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Utilizing Response- and Biology-Based Risk Factors to Guide Therapy in Patients with Non-High-Risk Neuroblastoma
Primary Investigator: M. Meaghan Granger
IRB ID: 2015-032 (OPEN)
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Vascular effects of Infection in Pediatric Stroke Study IIVIPS II
Primary Investigator: Marcela D. Torres
IRB ID: 2018-018 (OPEN)