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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A 2-part Phase 2 Study of Safety, Pharmacokinetics and Biomarkers in Children with Duchenne Muscular Dystrophy including a Randomized, Double-Blind, Placebo-Controlled Part A, Followed by an Open-Label Part B
Primary Investigator: Marks MD, Warren
IRB ID: 2023-003 (OPEN)
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A Double-Blind, Placebo-Controlled, Dose Escalation Study to Assess the Efficacy, Safety and Pharmacokinetics of Voclosporin in Adolescents with Lupus Nephritis (VOCAL)
Primary Investigator: Gillespie MD, Robert
IRB ID: 2022-047 (OPEN)
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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 (BEYOND)
Primary Investigator: Marks MD, Warren
IRB ID: Sarepta_SRP-9001-401_Marks (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: Marks MD, Warren
IRB ID: 2019-007 (OPEN)
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A Long-term, Open-label Extension (OLE) 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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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 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: 2011-191/2023-095 (OPEN)
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A Multicenter Multinational Observational Study of Children with Hypochondroplasia
Primary Investigator: Steelman MD, Joel
IRB ID: IRB# 2024-023 (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: Eames MD, Gretchen
IRB ID: 2015-077 (OPEN)
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A Multimodal Biomarker-based Predictive Model for Infantile Spasm
Primary Investigator: Keator MD, Cynthia
IRB ID: 2022-006 (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: Whitworth MD, M. Suzanne
IRB ID: 2020-031 (OPEN)
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A Phase 1/2 Study of Tiragolumab (NSC# 827799, IND# 161266) and Atezolizumab (NSC# 783608, IND# 161266) in Patients with Relapsed or Refractory SMARCB1 or SMARCA4 Deficient Tumors
Primary Investigator: Granger MD, Meaghan
IRB ID: 2022-094 (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 Randomized Study of Irinotecan/Temozolomide/Dinutuximab with or without Eflornithine (DFMO) (IND# 141913) in Children with Relapsed, Refractory or Progressive Neuroblastoma
Primary Investigator: Granger MD, Meaghan
IRB ID: 2019-049 (TEMPORARILY CLOSED TO ACCRUAL)
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A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of NBI-921352 as Adjunctive Therapy in Subjects with SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE)
Primary Investigator: Perry MD, M. Scott
IRB ID: 2020-071 (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 (TEMPORARILY CLOSED TO ACCRUAL)
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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: Zhao, MD, Sibo
IRB ID: 2019-072 (TEMPORARILY CLOSED TO ACCRUAL)
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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 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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A Phase 2 Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of SPR001 (Tildacerfont) in Children Aged 2 to 17 Years with Congenital Adrenal Hyperplasia
Primary Investigator: Thornton MD, Paul Stephen
IRB ID: 2021-078 (OPEN)
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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, 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: Johnson MD, Clarissa
IRB ID: 2019-024 (OPEN)
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A Phase 2/3 Adaptive, Double-blind, Placebo Controlled Study to Evaluate the Efficacy and Safety of VX-147 in Subjects Aged 12 Years and Older With APOL1-mediated Proteinuric Kidney Disease
Primary Investigator: Gillespie MD, Robert
IRB ID: 2023-069 (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 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: Murray MD, Jeffrey
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: Heym MD, Kenneth
IRB ID: 2020-072 (TEMPORARILY CLOSED TO ACCRUAL)
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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 Randomized, Placebo-Controlled Trial Evaluating Memantine (IND# 149832) for Neurocognitive Protection in Children Undergoing Cranial Radiotherapy as Part of Treatment for Primary Central Nervous System Tumors
Primary Investigator: Zhao, MD, Sibo
IRB ID: 2022-019 (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: Vallance MD, Kelly
IRB ID: 2017-030 (OPEN)
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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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A Phase 3 Study of Selumetinib 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 - ACNS1931
Primary Investigator: Zhao, MD, Sibo
IRB ID: 2021-017 (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: Heym MD, Kenneth
IRB ID: 2019-059 (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 received a 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, Fixed-Dose, Multicenter Study to Examine the Efficacy and Safety of Zx008 in Subjects with CDKL5 Deficiency Disorder Followed by an Open-Label Extension
Primary Investigator: Perry MD, M. Scott
IRB ID: 2021-093 (OPEN)
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A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel-group Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
Primary Investigator: Roy, Sani
IRB ID: 2023-073 (OPEN)
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A phase 3, single-arm open label trial to assess the efficacy, safety, PK and PD of dasiglucagon when administered as a rescue therapy for severe hypoglycemia in children with T1D
Primary Investigator: Thornton MD, Paul Stephen
IRB ID: 2022-048 (OPEN)
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A Phase I Study of Tagraxofusp With or Without Chemotherapy in Pediatric Patients with Relapsed or Refractory CD123 Expressing Hematologic Malignancies
Primary Investigator: Heym MD, Kenneth
IRB ID: 2023-008 (OPEN)
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A Phase I Study with an Expansion Cohort/Randomized Phase II Study of the Combinations of Ipilimumab, Nivolumab and Brentuximab Vedotin in Patients with Relapsed/Refractory Hodgkin Lymphoma
Primary Investigator: Albritton MD, Karen
IRB ID: 2020-087 (TEMPORARILY CLOSED TO ACCRUAL)
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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: Beam MD, Donald
IRB ID: 2020-048 (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 Data Registry for the Congenital Cardiovascular Interventional Study Consortium: Risk Stratification for Cardiac Catheterization Procedures
Primary Investigator: Kuo MD, James
IRB ID: 2016-064 (OPEN)
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A 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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A prospective study to evaluate biological and clinical effects of significantly corrected CFTR function in infants and young children (BEGIN-OB-19).
Primary Investigator: Schultz MD, Karen D
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: Marks MD, Warren
IRB ID: 2019-052 (OPEN)
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A Prospective, Long-Term, Interventional, Active Extension Study to Evaluate the Safety and Tolerability of NBI-921352 as Adjunctive Therapy in Subjects with SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE)
Primary Investigator: Perry MD, M. Scott
IRB ID: 2022-021 (OPEN)
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A Prospective, Non-interventional (NIS), Long-term, Post-Authorisation Safety Study (PASS) of Patients Treated with Lonapegsomatropin.
Primary Investigator: Thornton MD, Paul Stephen
IRB ID: 2023-053 (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: Vallance MD, Kelly
IRB ID: 2018-089 (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-CPO) 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 (OPEN)
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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: COG_ACCL1931_Albritton (OPEN)
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A Randomized, Controlled, Open-Label, Phase 3 Study to Evaluate the Efficacy and Safety of INZ-701 in Children Ages >6 Months to <13 Years with Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency
Primary Investigator: Steelman MD, Joel
IRB ID: 2023-031 (OPEN)
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A Randomized, Double-Blind Evaluation of the Pharmacokinetics, Safety, and Antiviral Efficacy of Tenofovir Alafenamide (TAF) in Adolescents with Chronic Hepatitis B Virus Infection
Primary Investigator: Whitworth MD, M. Suzanne
IRB ID: 2016-091 (OPEN)
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A Randomized, Double-Blind, Placebo- Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants with Spinal Muscular Atrophy with Open-Label Extension (RESILIENT)
Primary Investigator: Acord, Stephanie
IRB ID: 2022-074 (OPEN)
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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: Thornton MD, Paul Stephen
IRB ID: 2020-039 (OPEN)
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A randomized, double-blind, placebo-controlled, multicenter phase III study to evaluate the efficacy and safety of ABX464 once daily for induction treatment in subjects with moderately to severely active ulcerative colitis
Primary Investigator: Huang, MD, Clifton
IRB ID: 2022-092 (OPEN)
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A randomized, double-blind, placebo-controlled, multicenter phase III study to evaluate the efficacy and safety of ABX464 once daily for maintenance treatment in patient with moderate to severely active ulcerative Colitis
Primary Investigator: Huang, MD, Clifton
IRB ID: 2022-103 (OPEN)
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A retrospective cohort study of response and survival in pediatric patients with relapsed and refractory acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LLy) treated with contemporary salvage therapy
Primary Investigator: Heym MD, Kenneth
IRB ID: 2021-016 (OPEN)
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A RETROSPECTIVE FOLLOW-UP STUDY OF THE DURABILITY OF ANTIVIRAL THERAPY ON LONG-TERM HEARING AND NEURODEVELOPMENTAL OUTCOMES AMONG PATIENTS TREATED FOR CONGENITAL CYTOMEGALOVIRUS INFECTION AS INFANTS OR TODDLERS
Primary Investigator: Eger MD, Lynne
IRB ID: 2023-093 (OPEN)
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A RETROSPECTIVE STUDY OF INOTUZUMAB OZOGAMICIN IN PATIENTS WITH DOWN SYNDROME AND ACUTE LYMPHOBLASTIC LEUKEMIA
Primary Investigator: Pacenta, Holly
IRB ID: 2023-062 (OPEN)
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Activa® Dystonia Therapy
Primary Investigator: Marks MD, Warren
IRB ID: 593 (OPEN)
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ALTE22C1: 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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An Open-label Intermediate-size Treatment Protocol for the Urgent Treatment of Seriously Ill Patients with Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) with Triheptanoin (UX007)
Primary Investigator: Lacy MD, Adrian
IRB ID: 2020-028 (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: 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, Single Arm, Multicenter, Phase III Study on the Efficacy, Safety, and Pharmacokinetics of FP-001 42 mg Controlled Release in Patients with Central (Gonadotropin-Dependent) Precocious Puberty
Primary Investigator: Radack MD, Jill Goldfarb
IRB ID: 2022-114 (OPEN)
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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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AOST2032: 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 (TEMPORARILY CLOSED TO ACCRUAL)
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Ascendis Growth Hormond Deficiency-3385/0043
Primary Investigator: Thornton MD, Paul Stephen
IRB ID: 2023-054 (OPEN)
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ASCT2031: A Multi-Center, Phase 3, Randomized Trial of Matched Unrelated Donor (MUD) versus HLA-Haploidentical Related (Haplo) Myeloablative Hematopoietic Cell Transplantation for Children, Adolescents, and Young Adults (AYA) with Acute Leukemia or Myelodysplastic Syndrome (MDS)
Primary Investigator: Howrey MD, Richard P.
IRB ID: 2022-125 (OPEN)
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ASND0034: A Multicenter, Phase 2, Randomized, Open-label, Active-controlled, Parallel Group Clinical Trial to Investigate the Safety, Tolerability, and Efficacy of Different Dose Levels of Once-weekly Lonapegsomatropin Compared to Daily Somatropin in Prepubertal Individuals with Turner Syndrome
Primary Investigator: Thornton MD, Paul Stephen
IRB ID: 2022-097 (OPEN)
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Assessment of Clonal Hematopoiesis and its Relationship to Cardiovascular Disease in Hodgkin Lymphoma Survivors. A COG Groupwide Observational Study.
Primary Investigator: Beam MD, Donald
IRB ID: COG_ALTE21C1_Beam (OPEN)
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ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
Primary Investigator: McCavit MD, Timothy
IRB ID: 2021-019 (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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CHART: Congenital Hyperinsulinism: A Retrospective Longitudinal Study
Primary Investigator:
IRB ID: 2021-079 (OPEN)
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Children’s Mercy Kansas City Cardiac High Acuity Monitoring Program (CHAMP).
Primary Investigator: Roten, Lisa
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: Muyskens MD, Steve
IRB ID: 2015-047 (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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COG Study ALTE03N1: Key Adverse Events after Childhood Cancer
Primary Investigator: Granger MD, Meaghan
IRB ID: 543 (OPEN)
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COG_AHOD2131_Albritton
Primary Investigator: Albritton MD, Karen
IRB ID: 2023-056 (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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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: CCHCS_PWS Adrenal Function_Roy (OPEN)
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Cystic Fibrosis Patient Registry
Primary Investigator: Schultz MD, Karen D
IRB ID: 394 (OPEN)
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descriptive study on infants with confirmed or suspected meningoencephalitis due to parechovirus (PeV) infection
Primary Investigator: Whitworth MD, M. Suzanne
IRB ID: 2023-007 (OPEN)
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Domperidone Use in Patients with Gastrointestinal Disorders
Primary Investigator: Osuntokun MD, Bankole Oluseyi
IRB ID: 2018-088 (OPEN)
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Early MRSA therapy in CF (streamlined eradication with repeat cycle) (Staph Aureus Resistance-Treat Early and Repeat (STAR-TER))
Primary Investigator: Schultz MD, Karen D
IRB ID: 2018-075 (OPEN)
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Electrophysiological Biomarkers of GABA Metabolism in Children with Genetic Epilepsy Syndromes
Primary Investigator: Papadelis, PhD, Christos
IRB ID: 2022-055 (OPEN)
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Electrophysiological Biomarkers of GABA metabolism in children with SCN1A+ Dravet Syndrome
Primary Investigator: Papadelis, PhD, Christos
IRB ID: 2022-051 (OPEN)
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Epilepsy Genetics Database
Primary Investigator: Keator MD, Cynthia
IRB ID: 2023-074 (OPEN)
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Evaluation of the outcome of surgical intervention on the upper extremity in young patients with hemiplegia
Primary Investigator: Marshall MD, James
IRB ID: 2020-098 (OPEN)
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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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Exploration of Perceived Stress, Sleep, Fatigue, and Biomarkers in Childhood Brain Tumor Survivors
Primary Investigator: Bashore NP, Lisa M
IRB ID: 2018-010 (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 Thromboembolic Disease
Primary Investigator: Torres MD, Marcela
IRB ID: 2021-065 (OPEN)
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Genzyme Rare Diseases Registry
Primary Investigator: Gillespie MD, Robert
IRB ID: 2009-043 (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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Hyperinsulinism Center Registry for Research and Quality Data Collection
Primary Investigator: Thornton MD, Paul Stephen
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: 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: CCHMC_Novel Gene Mutations_Thornton (OPEN)
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Improve Care Now
Primary Investigator: Ogunmola MD, Nick
IRB ID: 2021-066 (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: Heym MD, Kenneth
IRB ID: 2017-048 (OPEN)
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International Registry for Severe Chronic Neutropenia
Primary Investigator: Beam MD, Donald
IRB ID: 94 (OPEN)
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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: Granger MD, Meaghan
IRB ID: 2018-048 (TEMPORARILY CLOSED TO ACCRUAL)
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Kaposiform Hemangioendothelioma: Hematology and Oncology Survey (KHHAOS) Study
Primary Investigator: Heym MD, Kenneth
IRB ID: 2021-051 (OPEN)
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Long-term Antipsychotic Pediatric Safety Trial (LAPS)
Primary Investigator: Bailey PhD, Laurie
IRB ID: 2019-078 (OPEN)
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Long-Term Follow-Up of Patients Who Have Participated in Children's Oncology Group Studies
Primary Investigator: Beam MD, Donald
IRB ID: 2010-109 (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 Evaluation of the Threat of Established and Emerging Respiratory Viral Infections in Pediatric Transplant Recipients
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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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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NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 study of LOXO-101 (Larotrectinib) in patients with tumors harboring NTRK fusions (APEC1621A)
Primary Investigator: Vallance MD, Kelly
IRB ID: 2018-006 (OPEN)
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NCI-COG Pediatric Match (Molecular Analysis For Therapy Choice) - Phase 2 Subprotocol Of AG-120 (Ivosidenib) in Patients with Tumors Harboring IDH1 Mutations (APEC1621K)
Primary Investigator: Vallance MD, Kelly
IRB ID: 2020-055 (OPEN)
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NCI-COG Pediatric Match (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of Loxo-292 in Patients with Tumors Harboring Ret Gene Alterations (APEC1621N)
Primary Investigator: Vallance MD, Kelly
IRB ID: 2020-099 (OPEN)
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NCI-COG Pediatric Match (Molecular Analysis For Therapy Choice) - Phase 2 Subprotocol of Tipifarnib in Patients with Tumors Harboring Hrasgenomic Alterations (APEC1621M)
Primary Investigator: Vallance MD, Kelly
IRB ID: 2020-070 (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 (APEC1621F)
Primary Investigator: Vallance MD, Kelly
IRB ID: 2018-003 (OPEN)
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NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Screening Protocol (APEC1621SC)
Primary Investigator: Vallance MD, Kelly
IRB ID: 2017-065 (OPEN)
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Neuromuscular Observational Research (MOVR) Data Hub Protocol
Primary Investigator: Marks MD, Warren
IRB ID: 2019-055 (OPEN)
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Neuropsychological, Social, Emotional, and Behavioral Outcomes in Children with Cancer (ALTE07C1)
Primary Investigator: Murray MD, Jeffrey
IRB ID: 2009-013 (OPEN)
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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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Observational study of pediatric thrombotic disease: the Throm-PED registry
Primary Investigator: Torres MD, Marcela
IRB ID: IPTN_Throm-PED 2018_Torres (OPEN)
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ON-TRK: A prOspective Non-interventional study in patients with locally advanced or metastatic TRK fusion cancer treated with larotrectinib.
Primary Investigator: Vallance MD, Kelly
IRB ID: 2023-065 (OPEN)
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Parental Attitudes in Neonatal Clinical Trial Enrollment: Decision-Making Preferences and Reasoning among Participants and Non-Participants
Primary Investigator: Riley MD, David
IRB ID: 2017-016 (OPEN)
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Paroxysmal Nocturnal Hemoglobinuria / IPIG registry study/USA
Primary Investigator: Beam MD, Donald
IRB ID: 2022-093 (OPEN)
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Pediatric Hepatic Malignancy International Therapeutic Trial (PHITT)
Primary Investigator: Akers DO, Lauren
IRB ID: 2018-066 (TEMPORARILY CLOSED TO ACCRUAL)
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Pediatric International DBS (PEDiDBS) Registry
Primary Investigator: Marks MD, Warren
IRB ID: 2016-013 (OPEN)
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PEPN22P1: A Pharmacokinetic Study of VinCRIStine in Infants Dosed According to BSA-Banded Infant Dosing Tables and Older Children Dosed by Traditional BSA Methods
Primary Investigator: Pacenta, Holly
IRB ID: 2022-112 (OPEN)
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Phase 1 Study to Evaluate the Safety and Pharmacokinetics of Palbociclib (Ibrance®) in Combination with Irinotecan and Temozolomide in Pediatric Patients with Recurrent or Refractory Solid Tumors
Primary Investigator: Albritton MD, Karen
IRB ID: 2021-077 (OPEN)
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Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited CD34+ Human Hematopoietic Stem and Progenitor Cells (EDIT-301) in Subjects with Severe Sickle Cell Disease
Primary Investigator: Eames MD, Gretchen
IRB ID: 2022-023 (OPEN)
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Phase 3 trial for patients with Dyskinetic Cerebral Palsy
Primary Investigator: Marks MD, Warren
IRB ID: 2022-027 (OPEN)
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Predicting ECMO NeuroLogICal Injuries using mAchiNe Learning (PELICAN)
Primary Investigator: Nkwantibisa, Raymond
IRB ID: 2023-075 (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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Protocol for a Research Sample Repository for Hematopoietic Cell Tranplantation, Other Cellular Therapies, and Marrow Toxic Injuries
Primary Investigator: Eames MD, Gretchen
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: 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
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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Reducing Ethnic Disparities in Acute Leukemia (REDIAL) Consortium Retrospective Chart Review
Primary Investigator: Heym MD, Kenneth
IRB ID: 2019-051 (OPEN)
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Regeneron Esophagitis 0456/0513
Primary Investigator: Osuntokun MD, Bankole Oluseyi
IRB ID: 2023-011 (OPEN)
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Retrospective data analysis of neurophysiological data for intraoperative or epilepsy monitoring
Primary Investigator: Papadelis, PhD, Christos
IRB ID: 2022-059 (OPEN)
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Retrospective non-interventional study of stiripentol use in Dravet patients in the USA.
Primary Investigator: Perry MD, M. Scott
IRB ID: 2022-113 (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 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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Seizures and Children's Outcomes after Stroke
Primary Investigator: Herring, Rachelle
IRB ID: UCSF_SCOUTS_Herring (OPEN)
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SELCLAX
Primary Investigator: Heym MD, Kenneth
IRB ID: 2021-056 (OPEN)
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Sickle cell Transplant Evaluation of Long term and LAte-effects Registry (STELLAR)
Primary Investigator: Eames MD, Gretchen
IRB ID: 2020-086 (OPEN)
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Single-center, single blinded, prospective, randomized clinical trial aimed to compare the choice, safety, and efficacy of selective serotonin reuptake inhibitors (SSRIs) dosing based on results of pharmacogenomic (PGX) testing in pediatric patients with depression.
Primary Investigator:
IRB ID: 2019-083 (TEMPORARILY CLOSED TO ACCRUAL)
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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: St. Jude_SJiMB21_Zhao (OPEN)
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Standardizing Treatments for Pulmonary Exacerbations: A platform for evaluating treatment decisions to improve outcomes (STOP360-IP-22)
Primary Investigator: Schultz MD, Karen D
IRB ID: 2022-091 (OPEN)
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Stroke Study
Primary Investigator: Torres MD, Marcela
IRB ID: 2023-029 UCSF_FOCAS_Torres (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: CCMC_LCH Targeted therapy_Ray (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 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 risk factors for disease severity of RSV infection among pediatric hematopoietic cell or solid organ transplant recipients
Primary Investigator: Whitworth MD, M. Suzanne
IRB ID: 2021-014 (OPEN)
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The Use of Fluorodopa 18F Positron Emission Tomography Combined with Computed Tomography in Congenital Hyperinsulinism
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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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: Torres MD, Marcela
IRB ID: 2012-008 (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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Tumor-Agnostic Precision Immunooncology and Somatic Targeting Rational for You (Tapistry) Phase II Platform Trial
Primary Investigator: Ray MD, Anish
IRB ID: 2020-064 (OPEN)
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US Selumetinib Registry
Primary Investigator: Vallance MD, Kelly
IRB ID: 2023-026 (OPEN)
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UTSW_ASH RESEARCH_Johnson
Primary Investigator: Johnson MD, Clarissa
IRB ID: ASH _ASH Data Hub_Johnson (OPEN)
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Venetoclax EAP IND 152442
Primary Investigator: Heym MD, Kenneth
IRB ID: 2020-080 (OPEN)