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.

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Find an active research study

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, 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: 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, 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, 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 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 (TEMPORARILY CLOSED TO ACCRUAL)

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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, 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 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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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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AML23: A phase 2 Study of Venetoclax in Combination with Conventional Chemotherapy in Pediatric Patients with Acute Myeloid Leukemia

Primary Investigator: Heym MD, Kenneth

IRB ID: St. Jude_AML23_Heym (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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APAl2020SC

Primary Investigator: Vallance MD, Kelly

IRB ID: 2024-008 (OPEN)

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ARAR2221 A Phase 2 Study Using Chemoimmunotherapy with Gemcitanine, Cisplatin and Nivolumab in Newly Diagnosed Nasopharyngeal Carcinona (NPC) was activated today through COG

Primary Investigator: Greer MD, Chelsee

IRB ID: 2024-071 (OPEN)

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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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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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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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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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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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COPPER HISTIDINATE TREATMENT FOR MENKES DISEASE

Primary Investigator: Stigall, William

IRB ID: 2024-088 (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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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: 2023-059 (OPEN)

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Improve Care Now

Primary Investigator: Ogunmola MD, Nick

IRB ID: 2021-066 (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 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 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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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: 2021-085 (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 1/2 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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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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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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PRWCC_Foresight_Pacenta

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

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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RCT for Intestinal Failure.

Primary Investigator: Osuntokun MD, Bankole Oluseyi

IRB ID: 2024-035 (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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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: 2022-067 (OPEN)

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SELCLAX

Primary Investigator: Heym MD, Kenneth

IRB ID: 2021-056 (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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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 (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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UTSW_ASH RESEARCH_Johnson

Primary Investigator: Johnson MD, Clarissa

IRB ID: 2022-116 (OPEN)

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Venetoclax EAP IND 152442

Primary Investigator: Heym MD, Kenneth

IRB ID: 2020-080 (OPEN)

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