Active Research
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(RTTI_Tiratricol-EAP-MCT8- 2022-4) Expanded Access Program for Tiratricol in Patients with Monocarboxylate Transporter 8 Deficiency also known as Allan-Herndon-Dudley syndrome (AHDS)
Primary Investigator: Thornton MD, Paul Stephen
IRB ID: 2023-070 (OPEN)
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10-CBA, 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: Eames MD, Gretchen
IRB ID: 2023-095 (OPEN)
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111-902, A Multicenter Multinational Observational Study of Children with Hypochondroplasia
Primary Investigator: Steelman MD, Joel
IRB ID: 2024-023 (OPEN)
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A COLLABORATIVE PHASE 2 STUDY OF VENETOCLAX IN COMBINATION WITH CONVENTIONAL CHEMOTHERAPY IN PEDIATRIC PATIENTS WITH ACUTE MYELOID LEUKEMIA
Primary Investigator: Heym MD, Kenneth
IRB ID: 2024-032 (OPEN)
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A Feasibility and Randomized Phase 2/3 Study of the VEFGR2/MET Inhibitor Cabozantinib in Combination with Cytotoxic Chemotherapy for Newly Diagnosed Osteosarcoma
Primary Investigator: Albritton MD, Karen
IRB ID: 2023-034 (OPEN)
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A master protocol for a Phase 3, multicenter, open-label, long-term extension study to evaluate the long-term efficacy and safety of mirikizumab in children and adolescents with moderate-to-severe ulcerative colitis or Crohn's disease
Primary Investigator: Huang, MD, Clifton
IRB ID: 2021-008 (OPEN)
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A Multimodal Biomarker-based Predictive Model for Infantile Spasms
Primary Investigator: Keator MD, Cynthia
IRB ID: 2022-006 (OPEN)
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A Phase 1/2 Trial of CBL0137 (NSC# 825802, IND# 155843) in Patients with Relapsed or Refractory Solid Tumors including CNS Tumors and Lymphoma
Primary Investigator: Zhao, MD, Sibo
IRB ID: 2022-024 (OPEN)
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A Phase 1/2 Trial of Selinexor (KPT-330) and Radiation Therapy in Newly-Diagnosed Pediatric Diffuse Intrinsic Pontine Glioma (DIPG) and High-Grade Glioma (HGG) ACNS1821
Primary Investigator: Zhao, MD, Sibo
IRB ID: 2022-054 (OPEN)
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A Phase 2 Study of Blinatumomab (NSC# 765986, IND# 125462) in Combination with Nivolumab (NSC # 748726, IND# 125462), a Checkpoint Inhibitor of PD-1, in B-ALL Patients Aged ≥ 1 to < 31 Years Old with First Relapse
Primary Investigator: Heym MD, Kenneth
IRB ID: 2021-006 (OPEN)
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A PHASE 2 STUDY OF DS-8201A (NSC# 807708, IND# 153036) IN ADOLESCENTS, OR YOUNG ADULTS WITH RECURRENT HER2+ OSTEOSARCOMA
Primary Investigator: Granger MD, Meaghan
IRB ID: 2021-086 (TEMPORARILY CLOSED TO ACCRUAL)
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A Phase 2 Trial of Chemotherapy followed by Response-Based Whole Ventricular & Spinal Canal Irradiation (WVSCI) for Patients with Localized Non-Germinomatous Central Nervous System Germ Cell Tumor
Primary Investigator: Zhao, MD, Sibo
IRB ID: 2021-071 (OPEN)
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A PHASE 2, TWO-STAGE, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE-ASCENDING DOSE STUDY TO EVALUATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PRELIMINARY EFFICACY OF RLS-0071 IN NEWBORNS WITH MODERATE OR SEVERE HYPOXIC-ISCHEMIC ENCEPHALOPATHY UNDERGOING THERAPEUTIC HYPOTHERMIA WITH LONG-TERM FOLLOW-UP (STAR)
Primary Investigator: Riley MD, David
IRB ID: Premier_RLS-0071-202 (ReAlta)_Riley (OPEN)
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A Phase 3 Randomized Controlled Trial Comparing Open vs Thoracoscopic Management of Pulmonary Metastases in Patients with Osteosarcoma
Primary Investigator: Albritton MD, Karen
IRB ID: 2022-122 (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: Heym MD, Kenneth
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: Heym MD, Kenneth
IRB ID: 2020-022 (OPEN)
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A Phase 3 Study of 131I-Metaiodobenzylguanidine (131I-MIBG) or ALK Inhibitor Therapy Added to Intensive Therapy for Children with Newly Diagnosed High-Risk Neuroblastoma (NBL) (IND# 134379)
Primary Investigator: Granger MD, Meaghan
IRB ID: 2018-048 (OPEN)
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A Phase 3 Study of Selumetinib (NSC# 748727, IND# 77782) or Selumetinib in Combination with Vinblastine for non-NF1, non-TSC Patients with Recurrent or Progressive Low-Grade Gliomas (LGGs) Lacking BRAFV600E or IDH1 Mutations
Primary Investigator: Zhao, MD, Sibo
IRB ID: 2021-017 (TEMPORARILY CLOSED TO ACCRUAL)
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A Phase 3, Open-label Study Evaluating the Long term Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis
Primary Investigator: Pancham MD, Krishna
IRB ID: 2022-056 (OPEN)
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A Phase 3, open-label, single-arm, repeated-dose study to evaluate the safety and tolerability, pharmacokinetics, and antiviral activity of maribavir for the treatment of cytomegalovirus (CMV) infection in children and adolescents who have receiveda hematopoietic stem cell transplant (HSCT) or a solid organ transplant (SOT)
Primary Investigator: Howrey MD, Richard P.
IRB ID: 2022-044 (OPEN)
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A Phase 3, Randomized, Double-Blind, Placebo Controlled Study to Assess the Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Dyskinesia Due to Cerebral Palsy
Primary Investigator: Marks MD, Warren
IRB ID: 2022-027 (OPEN)
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A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Investigate the Efficacy, Safety, and Tolerability of LP352 in the Treatment of Seizures in Children and Adults with Developmental and Epileptic Encephalopathies
Primary Investigator: Keator MD, Cynthia
IRB ID: 2025-027 (OPEN)
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A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Investigate the Efficacy, Safety, and Tolerability of LP352 in the Treatment of Seizures in Children and Adults with Dravet Syndrome
Primary Investigator: Perry MD, M. Scott
IRB ID: 2025-024 (OPEN)
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A phenomenological study of Cultural Humility in Patient and Physician Encounters
Primary Investigator: Campbell, Throy
IRB ID: 2023-081 (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: Wilson MD, Don
IRB ID: 2013-045 (OPEN)
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A PROSPECTIVE, LONG-TERM REGISTRY OF PATIENTS WITH A DIAGNOSIS OF SPINAL MUSCULAR ATROPHY (SMA) - (RESTORE)
Primary Investigator: Acord, Stephanie
IRB ID: 2019-052 (OPEN)
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A Randomized Phase 3 Trial of Vinorelbine, Dactinomycin, and Cyclophosphamide (VINO-AC) Plus Maintenance Chemotherapy with Vinorelbine and Oral Cyclophosphamide (VINO-C) vs Vincristine, Dactinomycin and Cyclophosphamide (VAC) plus VINO-CPO Maintenance in Patients with High Risk Rhabdomyosarcoma (HR RMS)
Primary Investigator: Vallance MD, Kelly
IRB ID: 2021-083 (TEMPORARILY CLOSED TO ACCRUAL)
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A RETROSPECTIVE STUDY OF INOTUZUMAB OZOGAMICIN IN PATIENTS WITH DOWN SYNDROME AND ACUTELYMPHOBLASTIC LEUKEMIA
Primary Investigator: Pacenta, Holly
IRB ID: 2023-062 (OPEN)
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A Safety and Feasibility Study to Evaluate Blood-Brain Barrier Disruption Using Exablate MR-Guided Focused Ultrasound in Combination with Doxorubicin in Treating Pediatric Patients with Diffuse Intrinsic Pontine Gliomas
Primary Investigator: Zhao, MD, Sibo
IRB ID: 2024-119 (OPEN)
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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)
Primary Investigator: Heym MD, Kenneth
IRB ID: 2017-049 (OPEN)
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AALL2121: A Phase 2 study of revumenib (SNDX-5613) in combination with chemotherapy for patients with relapsed or refractory KMT2A-rearranged infant leukemia
Primary Investigator: Pacenta, Holly
IRB ID: 2024-114 (TEMPORARILY CLOSED TO ACCRUAL)
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ABX464-107: A randomized, double-blind, multicenter phase III study to evaluate the long-term efficacy and safety of ABX464 25 mg or 50 mg once daily as a maintenance therapy in subjects with moderately to severely active ulcerative colitis.
Primary Investigator: Huang, MD, Clifton
IRB ID: 2022-103 (OPEN)
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ACCL1931: A Randomized Trial of Levocarnitine Prophylaxis to Prevent Asparaginase-Associated Hepatotoxicity in Adolescents and Young Adults Receiving Acute Lymphoblastic Leukemia Therapy
Primary Investigator: Albritton MD, Karen
IRB ID: 2023-096 (OPEN)
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ACP-101-303 (OLE): A Long-term, Open-label Extension Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
Primary Investigator: Roy, Sani
IRB ID: 2023-068 (OPEN)
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Activa Dystonia Therapy HDE
Primary Investigator: Marks MD, Warren
IRB ID: 593 (OPEN)
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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
Primary Investigator: Vallance MD, Kelly
IRB ID: 2017-030 (OPEN)
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AHOD2131: A Randomized Phase 3 Interim Response Adapted Trial Comparing Standard Therapy with Immuno-oncology Therapy for Children and Adults with Newly Diagnosed Stage I and II Classic Hodgkin Lymphoma
Primary Investigator: Albritton MD, Karen
IRB ID: 2023-056 (OPEN)
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ALTE05N1, Umbrella Long-Term Follow-up Protocol
Primary Investigator: Beam MD, Donald
IRB ID: 2010-109 (OPEN)
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ALTE2131 Triptorelin and Protection of Ovarian Reserve in Adolescents and Young Adults with Cancer An NCORP Phase 3 Study
Primary Investigator: Albritton MD, Karen
IRB ID: 2025-025 (OPEN)
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ALTE21C1: Assessment of Clonal Hematopoiesis and its Relationship to Cardiovascular Disease in Hodgkin Lymphoma Survivors
Primary Investigator: Beam MD, Donald
IRB ID: 2023-067 (OPEN)
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An International Pilot Study of Chemotherapy and Tyrosine Kinase Inhibitors with Blinatumomab in Patients with Newly-Diagnosed Philadelphia Chromosome-Positive or ABL-class Philadelphia Chromosome-Like B-cell Acute Lymphoblastic Leukemia
Primary Investigator: Heym MD, Kenneth
IRB ID: COG_AALL2131_Heym 2025-080 (OPEN)
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An Open Label, Expanded Access Protocol Using 131I-Metaiodobenzylguanidine (131I-MIBG) Therapy in Patients with Refractory Neuroblastoma, Pheochromocytoma, or Paraganglioma (not eligible for approved treatment)
Primary Investigator: Granger MD, Meaghan
IRB ID: 2012-026 (OPEN)
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An Open-label, Multi-center, Phase IV, Rollover Study for Patients with Sickle Cell Disease who have Completed a prior Novartis-Sponsored Crizanlizumab Study
Primary Investigator: Johnson MD, Clarissa
IRB ID: 2021-060 (OPEN)
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An Open-label, Randomized, Active-Controlled, Phase 3 Study of Setrusumab Compared With Bisphosphonates in Pediatric Subjects 2 to < 5 Years of Age With Osteogenesis Imperfecta Types I, III or IV.
Primary Investigator: Steelman MD, Joel
IRB ID: 2023-024 (OPEN)
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ANBL2131: A Phase 3 Study of Dinutuximab Added to Intensive Multimodal Therapy for Children with Newly Diagnosed High-Risk Neuroblastoma
Primary Investigator: Greer MD, Chelsee
IRB ID: 2024-050 (OPEN)
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APAL2020SC: Pediatric Acute Leukemia (PedAL) Screening Trial – Developing New Therapies for Relapsed Leukemias
Primary Investigator: Vallance MD, Kelly
IRB ID: 2024-008 (OPEN)
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APEC1621SC: NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Screening Protocol
Primary Investigator: Vallance MD, Kelly
IRB ID: 2017-065 (OPEN)
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ARAR2221: A Phase 2 Study Using Chemoimmunotherapy with Gemcitabine, Cisplatin and Nivolumab in Newly Diagnosed Nasopharyngeal Carcinoma (NPC)
Primary Investigator: Greer MD, Chelsee
IRB ID: 2024-071 (OPEN)
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ASH Data Hub: A Multi-Center Data Hub of Individuals Living with Hematologic Disease
Primary Investigator: Johnson MD, Clarissa
IRB ID: 2022-116 (OPEN)
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BioMarin 111-210: A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
Primary Investigator: Steelman MD, Joel
IRB ID: 2024-116 (OPEN)
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BioMarin_111-903:A Multicenter, Observational Study to Characterize Growth in Children with Idiopathic Short Stature.
Primary Investigator: Steelman MD, Joel
IRB ID: 2024-115 (TEMPORARILY CLOSED TO ACCRUAL)
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Blood Collection for National Marrow Donor Program
Primary Investigator: Eames MD, Gretchen
IRB ID: 2019-062 (OPEN)
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BN44620: A PHASE IV OPEN-LABEL STUDY EVALUATING THE EFFECTIVENESS AND SAFETY OF RISDIPLAM ADMINISTERED AS AN EARLY INTERVENTION IN PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY AFTER GENE THERAPY
Primary Investigator: Acord, Stephanie
IRB ID: 2023-078 (OPEN)
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Brain functional reorganization as a result of deep-brain stimulation therapy in children with dystonia
Primary Investigator: Papadelis, PhD, Christos
IRB ID: 2021-009 (OPEN)
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CAMPFIRE: Children’s and Young Adult Master Protocol for Innovative Pediatric Research
Primary Investigator: Vallance MD, Kelly
IRB ID: 2020-066 (OPEN)
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CHAT: A Multi-Institutional Children’s Healthcare Advancements in Thrombosis Registry
Primary Investigator: Torres MD, Marcela
IRB ID: 2022-077 (OPEN)
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Clinical Assessment of Flow in Tetralogy of Fallot by Time-Resolved Volumetric Phase Contrast MRI.
Primary Investigator: Muyskens MD, Steve
IRB ID: 2015-047 (OPEN)
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Clinical Characteristics and Outcomes of Community-Acquired Pneumonia (CAP) Requiring Hospitalization among Non-Lung Pediatric Solid Organ Transplant Recipients (pSOT).
Primary Investigator: Rister, Nicholas
IRB ID: 2025-138 (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: Howrey MD, Richard P.
IRB ID: 2018-041 (OPEN)
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CNTO1959PBCRD3007, A Phase 3, Multicenter, Randomized, Platform Study of p19 Inhibition of the IL-23 Pathway to Establish Efficacy in Pediatric Crohn’s Disease (MACARONI-23) (Intervention Specific Appendix to Master Protocol PLATFORMPBCRD3001/I6T-MC-PIBD)
Primary Investigator: Huang, MD, Clifton
IRB ID: 2024-064 (OPEN)
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COG_ALTE22C1 (DS-PALS Survivors)_Chronic Health Conditions in Down Syndrome-Associated Acute Leukemia: The Down Syndrome Phenotyping Acute Leukemia Study in Survivors (DS-PALS Survivors)
Primary Investigator: Beam MD, Donald
IRB ID: 2024-009 (OPEN)
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Cognitive and Neuroimaging Biosignatures of Well-being in Youth with and without Seizures
Primary Investigator: Cooper, Crystal
IRB ID: 2020-104 (OPEN)
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Combined Abnormal General Movement Assessment and High-Resolution Brain Magnetic Resonance Imaging as a Predictor of a Diagnosis of Cerebral Palsy among Patients withHypoxic-Ischemic Encephalopathy
Primary Investigator: Johnson MD, Yvette
IRB ID: CCMC_MRI and GMA_Johnson (OPEN)
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Congenital Adrenal Hyperplasia Registry for Research and Quality Data Collection
Primary Investigator: Thornton MD, Paul Stephen
IRB ID: CCHCS_CAH Registry_Thornton (OPEN)
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COntemporary Infant and Neonatal Dialysis Study (COINED)
Primary Investigator: Razzouk MD, Randa
IRB ID: 2023-047 (OPEN)
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Cook Children’s Health Care System Stroke and Thrombosis Registry
Primary Investigator: Torres MD, Marcela
IRB ID: 2020-037 (OPEN)
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Correlation of non-invasive diastolic parameters with invasive filling pressures in single ventricle patients
Primary Investigator: Loar, Robert
IRB ID: 2020-103 (OPEN)
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Cortisol Production in Children and Adolescents with Prader-Willi Syndrome
Primary Investigator: Roy, Sani
IRB ID: 2024-002 (OPEN)
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CYP-001: Copper Histidinate Treatment for Menkes Disease
Primary Investigator: Stigall, William
IRB ID: 2024-088 (OPEN)
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Does treatment with Diazoxide elevate the plasma glucose levels in persons with GLUT1 Deficiency Syndrome? An open label, multiple ascending dose study
Primary Investigator: Thornton MD, Paul Stephen
IRB ID: CCMC_Glut1DS Diazoxide_Thornton (OPEN)
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DSM-PROT-005200: A Multi-Site Clinical Evaluation of the Simplexa COVID-19/Flu A/B & RSV Direct Assay in Symptomatic Patients
Primary Investigator: Pence, Morgan
IRB ID: 2024-102 (OPEN)
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Electrophysiological Biomarkers of GABA Metabolism in Children with Genetic Epilepsy Syndromes
Primary Investigator: Papadelis, PhD, Christos
IRB ID: 2022-051 (OPEN)
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Electrophysiological Biomarkers of GABA metabolism in children with SCN1A+ Dravet Syndrome
Primary Investigator: Papadelis, PhD, Christos
IRB ID: 2022-055 (OPEN)
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EMAtS registry
Primary Investigator: Tran, Lihn
IRB ID: 2024-111 (OPEN)
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ENDEAVOR: A CLINICAL STUDY TO EVALUATE THE SAFETY AND EFFICACY OF ETX101, AN AAV9 DELIVERED GENE THERAPY IN INFANTS AND CHILDREN WITH SCN1A POSITIVE DRAVET SYNDROME
Primary Investigator: Perry MD, M. Scott
IRB ID: 2022-076 (OPEN)
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Evaluation of the outcome of surgical intervention on the upper extremity in young patients with hemipelgia
Primary Investigator: Marshall MD, James
IRB ID: 2020-098 (OPEN)
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Exploration of Perceived Stress, Sleep, Fatigue, Physical Activity, and Biomarkers in Childhood Brain Tumor Survivors
Primary Investigator: Bashore NP, Lisa M
IRB ID: 2018-010 (OPEN)
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Focal Cerebral Arteriopathy Steroid (FOCAS) Trial (Comparative Effectiveness Trial)
Primary Investigator: Torres MD, Marcela
IRB ID: 2023-029 (OPEN)
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Fort Worth AYA Oncology Coalition Registry Database
Primary Investigator: Albritton MD, Karen
IRB ID: 2019-017 (OPEN)
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Functional Characterization of Children with Chronic Venous Thromboembolic Disease
Primary Investigator: Torres MD, Marcela
IRB ID: 2021-065 (OPEN)
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Gastroschisis Outcomes of Delivery Bank
Primary Investigator: Riley MD, David
IRB ID: 2024-066 (OPEN)
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Genzyme Rare Disease Registries
Primary Investigator: Gillespie MD, Robert
IRB ID: 2009-043 (OPEN)
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GOOD Study: Gastroschisis Outcomes of Delivery Study
Primary Investigator: Riley MD, David
IRB ID: 2023-004 (OPEN)
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H-40889 Clinical Predictors of Treatment-Related Toxicity Among Children Receiving Cancer Therapy
Primary Investigator: Heym MD, Kenneth
IRB ID: 2019-051 (OPEN)
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Health Effects After Anthracycline and Radiation Therapy (HEART): Dexrazoxane and Prevention of Anthracycline-related Cardiomyopathy
Primary Investigator: Beam MD, Donald
IRB ID: 2014-023 (OPEN)
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HPC-Cord Blood 6637-01, 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: Eames MD, Gretchen
IRB ID: 2015-077 (OPEN)
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Hyperinsulinism Center Registry for Research and Quality Data Collection
Primary Investigator: Thornton MD, Paul Stephen
IRB ID: 2011-183 (OPEN)
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I6T-MC-AMAY, A Phase 3, Multicenter, Randomized, Platform Study of p19 Inhibition of the IL-23 Pathway to Establish Efficacy in Pediatric Crohn’s Disease (ISA Title: A Phase 3, Multicenter, Randomized Clinical Study to Evaluate Mirikizumab inPediatric Crohn’s Disease)
Primary Investigator: Huang, MD, Clifton
IRB ID: 2024-065 (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: Papadelis, PhD, Christos
IRB ID: 2019-070 (OPEN)
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Identification of and Genomic Editing of Novel Gene Mutations in Congenital Hyperinsulinism
Primary Investigator: Thornton MD, Paul Stephen
IRB ID: 2023-059 (OPEN)
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Improving Diagnosis and Treatment in CPVT
Primary Investigator: Barker, Gregory
IRB ID: 2022-002 (OPEN)
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International Paediatric Stroke Study-Towards the Establishment of Standards of Practice and the Initiation of Multi-Center, Multi-National Clinical Trials for Neonates and Children with Stroke (IPSS)
Primary Investigator: Torres MD, Marcela
IRB ID: 2012-008 (OPEN)
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IPIG 5674-0001 Observational Registry Protocol: The International PNH Interest Group PNH Registry
Primary Investigator: Beam MD, Donald
IRB ID: 2022-093 (OPEN)
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Kaposiform Hemangioendothelioma: Hematology and Oncology Survey Study (KHHAOS Study)
Primary Investigator: Heym MD, Kenneth
IRB ID: 2021-051 (OPEN)
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KiteLock™ RCT: SAFETY AND EFFECTIVENESS OF 4% TETRASODIUM ETHYLENEDIAMINETETRAACETIC ACID CATHETER LOCK SOLUTION IN PREVENTING CENTRAL VENOUS CATHETER OCCLUSIONS IN CHILDREN WITH INTESTINAL FAILURE: A RANDOMIZED CONTROLLED TRIAL
Primary Investigator: Osuntokun MD, Bankole Oluseyi
IRB ID: 2024-035 (OPEN)
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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
Primary Investigator: Pacenta, Holly
IRB ID: 2020-101 (OPEN)
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MULTI-CENTER MOLECULAR DIAGNOSIS AND HOST RESPONSE OF RESPIRATORY VIRAL INFECTIONS IN PEDIATRIC TRANSPLANT RECIPIENTS (VIPER)
Primary Investigator: Whitworth MD, M. Suzanne
IRB ID: 2022-080 (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: Papadelis, PhD, Christos
IRB ID: 2019-068 (OPEN)
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n/a
Primary Investigator: Vallance MD, Kelly
IRB ID: 2025-046 (OPEN)
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NANT 2021-01: PHASE II STUDY OF EX-VIVO EXPANDED ALLOGENEIC UNIVERSAL DONOR TGFβi NK CELL INFUSIONS IN COMBINATION WITH TEMOZOLOMIDE, IRINOTECAN, DINUTUXIMAB, AND SARGRAMOSTIM IN PATIENTS WITH RELAPSED OR REFRACTORY NEUROBLASTOMA The STING (Sequential Temozolomide, Irinotecan, NK cells and GD2 mAb) Trial
Primary Investigator: Greer MD, Chelsee
IRB ID: 2024-080 (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: Roten, Lisa
IRB ID: 2017-032 (OPEN)
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National Registry of Pediatric Cancer Patients Diagnosed with COVID-19
Primary Investigator:
IRB ID: 2020-034 (OPEN)
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Nationwide_Epilepsy Multi-Site: Comprehensive Profiling of Neurological Disorders of Somatic Etiology Accelerator Protocol
Primary Investigator: Perry MD, M. Scott
IRB ID: 2024-046 (OPEN)
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n-Lorem_An open-label, single center, single participant study of an experimental antisense oligonucleotide treatment for TUBB4A-related leukodystrophy
Primary Investigator: Acosta MD, Fernando
IRB ID: 2024-122 (OPEN)
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NN7415-4616: Open-label study investigating efficacy, safety and pharmacokinetics of concizumab prophylaxis in children below 12 years with haemophilia A or B with or without inhibitors
Primary Investigator: McCavit MD, Timothy
IRB ID: 2024-012 (OPEN)
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Observational study of pediatric thrombotic disease: the Throm-PED registry
Primary Investigator: Torres MD, Marcela
IRB ID: 2021-085 (OPEN)
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Participation in the Cystic Fibrosis Patient Registry
Primary Investigator: Schultz MD, Karen D
IRB ID: 394 (OPEN)
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Pediatric International DBS Registry (PEDiDBS)
Primary Investigator: Marks MD, Warren
IRB ID: 2016-013 (OPEN)
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Pediatric Selective Cytopheretic Device (SCD-PED, QUELIMMUNE) for Critically Ill Children with Acute Kidney Injury: A Humanitarian Device Exemption (HDE) Surveillance Registry
Primary Investigator: Gillespie MD, Robert
IRB ID: 2025-038, 2025-039 (OPEN)
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PERC_Epilepsy Genetics Database
Primary Investigator: Keator MD, Cynthia
IRB ID: 2023-074 (TEMPORARILY CLOSED TO ACCRUAL)
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Phase 3 Accelerated BEP Trial: A randomised phase 3 trial of accelerated versus standard BEP chemotherapy for patients with intermediate and poor-risk metastatic germ cell tumours
Primary Investigator: Vallance MD, Kelly
IRB ID: 2018-089 (OPEN)
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PLATFORMPBCRD3001/I6T-MC-PIBD, A Phase 3, Multicenter, Randomized, Platform Study of p19 Inhibition of the IL-23 Pathway to Establish Efficacy in Pediatric Crohn’s Disease
Primary Investigator: Huang, MD, Clifton
IRB ID: 2024-063 (OPEN)
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Prader-Willi Syndrome Registry
Primary Investigator: Roy, Sani
IRB ID: CCHCS_PWS-Registry_Roy (OPEN)
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Prader-Willi Syndrome-Clinical Investigation Collaborative (PWS-CLIC) STUDYTRAX Clinical Database
Primary Investigator: Roy, Sani
IRB ID: FPWR_PWS StudyTrax_Roy (OPEN)
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Presurgical Mapping of the Eloquent Cortex in Children with Drug Resistant Epilepsy Using Videogames
Primary Investigator: Papadelis, PhD, Christos
IRB ID: 2024-028 (OPEN)
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Project DANCES (Diversity in Aya caNCEr Survivors)
Primary Investigator:
IRB ID: 2024-048 (OPEN)
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Prospective Phase 3 Study of Patients with Newly Diagnosed Very Low-risk and Low-risk Fusion Negative Rhabdomyosarcoma (ARST2032)
Primary Investigator: Vallance MD, Kelly
IRB ID: 2022-072 (OPEN)
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PROTOCOL FOR A RESEARCH DATABASE FOR HEMATOPOIETIC CELL TRANSPLANTATION, OTHER CELLULAR THERAPIES AND MARROW TOXIC INJURIES
Primary Investigator: Eames MD, Gretchen
IRB ID: 2019-061 (OPEN)
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PRWCC_Foresight, Outcomes in Pediatric and Young Adult B-Cell Malignancies After Commercially Available Immunotherapy
Primary Investigator: Pacenta, Holly
IRB ID: 2024-059 (OPEN)
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Quantitative Assessment of Sucking for Early Diagnosis of Brain Injury in Infants at High Risk
Primary Investigator: Papadelis, PhD, Christos
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 (TXCH LCH0115)
Primary Investigator: Ray MD, Anish
IRB ID: 2019-067 (OPEN)
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RBDD-PLG Project; International Retrospective and Prospective Study of Individuals affected with Hypoplasminogenemia
Primary Investigator: Torres MD, Marcela
IRB ID: 2019-028 (OPEN)
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Retrospective data analysis of neurophysiological data for intraoperative or epilepsy monitoring. An international, retrospective, multi-site study.
Primary Investigator: Papadelis, PhD, Christos
IRB ID: 2022-059 (OPEN)
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Robot-assisted hand training to induce manual functional change and cerebral neural plasticity in children with cerebral palsy
Primary Investigator: Papadelis, PhD, Christos
IRB ID: 2021-012 (OPEN)
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Safety, Accuracy, and Feasibility of Continuous Glucose Monitoring In the Neonatal Intensive Care Unit
Primary Investigator: Thornton MD, Paul Stephen
IRB ID: CCHCS_CGM_Thornton (OPEN)
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Seizures and Children's Outcomes After Stroke (SCOUTS)
Primary Investigator: Herring, Rachelle
IRB ID: 2022-067 (OPEN)
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Sensory and Affective Processing in Children with Autism Spectrum Disorder
Primary Investigator: Papadelis, PhD, Christos
IRB ID: 2024-047 (OPEN)
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Signatera Efficacy Study
Primary Investigator:
IRB ID: Dell Childrens_Signatera Efficacy_Ray (OPEN)
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SJiMB21, Phase 2 Study of Molecular and Clinical Risk-Directed Therapy for Infants and Young Children with Newly Diagnosed Medulloblastoma
Primary Investigator: Zhao, MD, Sibo
IRB ID: 2023-057 (OPEN)
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SRP-9001-401: A Long-term Multicenter Prospective Observational Study Evaluating the Comparative Effectiveness and Safety of Sarepta Gene Transfer Therapy vs. Standard of Care in Participants with Duchenne Muscular Dystrophy under Conditions of Routine Clinical Practice
Primary Investigator: Marks MD, Warren
IRB ID: 2023-084 (OPEN)
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STK-001-DS-301_Stoke_Dravet Syndrome
Primary Investigator: Perry MD, M. Scott
IRB ID: Stoke_STK-001-DS-301_Perry (OPEN)
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STOP360-IP-22: Standardizing Treatments for Pulmonary Exacerbations: A platform for evaluating treatment decisions to improve outcomes (STOP360)
Primary Investigator: Schultz MD, Karen D
IRB ID: 2022-091 (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: Wilson MD, Don
IRB ID: 2013-028 (OPEN)
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Targeted approach to Langerhans Cell Histiocytosis (LCH) using MEK inhibitor, Trametinib.
Primary Investigator: Ray MD, Anish
IRB ID: 2023-058 (OPEN)
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TDN_REACH-OB-23: A Research Study to Advance the CF Therapeutics Pipeline for People without Modulators
Primary Investigator: Schultz MD, Karen D
IRB ID: 2024-070 (OPEN)
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Test protocol
Primary Investigator:
IRB ID: CCHCS_Test_PI (OPEN)
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Testicular tissue cryopreservation for fertility preservation in patients facing infertility-causing diseases or treatment regimens
Primary Investigator: Palmer, MD, Blake
IRB ID: 2020-053 (OPEN)
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The American Thrombosis and Hemostasis Network (ATHN) National Web Tracker
Primary Investigator: Sanders MD, Joann
IRB ID: 2011-160 (OPEN)
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The Cardiac High Acuity Monitoring Program (CHAMP) Data Repository (CHAMP App Repository)
Primary Investigator: Roten, Lisa
IRB ID: 2017-033 (OPEN)
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The Impact of Massage Therapy on Post-Surgical Pain, Anxiety, Quality of Life, and Opioid Analgesia Exposure on Children after Thoracic or Lumbar Surgery
Primary Investigator:
IRB ID: 2020-088 (OPEN)
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The Use of Fluorine F 18 6-fluorodopamine Positron Emission Tomography Combined with Computed Tomography in Congenital Hyperinsulinism and Insulinoma
Primary Investigator: Thornton MD, Paul Stephen
IRB ID: 2012-060 (OPEN)
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Tissue Banking- Collecting and Banking Human Tissue Samples for Future Use
Primary Investigator: Zhao, MD, Sibo
IRB ID: 2020-093 (OPEN)
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to assess the efficacy and safety of DefenCath in pediatric population who are on hemodialysis for kidney failure.
Primary Investigator: Gillespie MD, Robert
IRB ID: 2025-036 (OPEN)
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Treatment of Newly Diagnosed Diffuse Anaplastic Wilms Tumors (DAWT) and Relapsed Favorable Histology Wilms Tumors (FHWT)
Primary Investigator: Vallance MD, Kelly
IRB ID: 2020-097 (OPEN)
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U.S. Cohort of Parechovirus Meningoencephalitis Among Neonates and Young Infants (STU-2022-0580)
Primary Investigator: Whitworth MD, M. Suzanne
IRB ID: 2023-007 (OPEN)
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Unknown
Primary Investigator: Roy, Sani
IRB ID: 2025-040 _Aardvark_AVK-101-301_Roy (OPEN)
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unknown
Primary Investigator: Pancham MD, Krishna
IRB ID: 2025-011 (OPEN)
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Unknown
Primary Investigator: Whitworth MD, M. Suzanne
IRB ID: Childrens National_RWE Maribavir_Whitworth (OPEN)
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Urine Neutrophil Gelatinase-Associated Lipocalin (NGAL) and serum Cystatin C levels in children after Cardiopulmonary Bypass (CPB)
Primary Investigator: Marshall MD, James
IRB ID: 2020-041 (OPEN)
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Using data from a network of patients, families, clinicians, researchers and health care organizations to improve care and outcomes for children, adolescents and young adults with Inflammatory Bowel Disease
Primary Investigator: Ogunmola MD, Nick
IRB ID: 2021-066 (OPEN)
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Utility of RESPONDR® TNF, a laboratory developed diagnostic test, in a real-world inflammatory bowel disease population
Primary Investigator: Huang, MD, Clifton
IRB ID: Prometheus Laboratories_RESPONDR 24IBD02_Huang (OPEN)
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UTSW_PELICAN: Predicting ECMO NeuroLogICal Injuries using mAchiNe Learning (PELICAN)
Primary Investigator: Nkwantibisa, Raymond
IRB ID: 2023-075 (OPEN)
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UX143-CL301: An Operationally Seamless, Randomized Phase 2/3 Study Consisting of a Phase 2 Single-Blind, Dose-Evaluation Phase and a Phase 3 Double-Blind, Placebo-controlled Phase to Assess the Efficacy and Safety of Setrusumab in Subjects with Osteogenesis Imperfecta
Primary Investigator: Steelman MD, Joel
IRB ID: 2023-061 (OPEN)
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VOG, A Retrospective Evaluation of High-Flow Intracranial Arteriovenous Malformations in Neonates: Clinical Management and Outcomes in North America
Primary Investigator: Riley MD, David
IRB ID: 2024-052 (OPEN)
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VX21-147-301: Phase 2/3 Adaptive, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of VX-147 in Adult and Pediatric Subjects With APOL1-mediated Proteinuric Kidney Disease
Primary Investigator: Gillespie MD, Robert
IRB ID: 2023-069 (OPEN)
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X4P-001-110: A Phase 3, randomized, double-blind, placebo-controlled, multicenter study of mavorixafor in participants with congenital and acquired primary autoimmune and idiopathic chronic neutropenic disorders who are experiencing recurrent and/or serious infections.
Primary Investigator: Beam MD, Donald
IRB ID: 2024-091 (OPEN)