Clinical Trials

General inquiries: 314-454-6018

Research area	Principle investigator(s)	Email
Brain tumors (neuro-oncology)	Mohamed S. Abdelbaki, MD	mohameda@wustl.edu
Cancer predisposition	Todd Druley, MD, PhD	druley_t@wustl.edu
	Andrea Stacy, MS, CGC	astacy@wustl.edu
Hematological malignancy (leukemia & lymphoma)	Jeffrey Magee MD, PhD	mageej@wustl.edu
	Laura Schuettpelz MD, PhD	schuettpelz_l@wustl.edu
Hemostasis and thrombosis (clotting/bleeding disorders)	David Wilson, MD, PhD	wilson_d@wustl.edu
Immune disorders	Jeffrey Bednarski, MD, PhD	bednarski_j@wustl.edu
Sickle cell disease	Monica Hulbert, MD	monicahulbert@wustl.edu
Solid tumors	Frederick Huang, MD	huang_f@wustl.edu
Stem cell transplantation and cellular therapy	Shalini Shenoy, MD	shalinishenoy@wustl.edu
	Sima Bhatt, MD	bhatts@wustl.edu
Survivorship	Robert Hayashi, MD	hayashi_r@wustl.edu

Studies

All cancers

Principal investigator	Title
Robert Hayashi, MD	Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening Trial)

Brain tumors

Principal investigator	Title
Neuro/Onc, IDS pharmacy	Single-arm, multicenter Phase I/Ib studyof avelumab + lenvatinib in children with primary CNS tumors
Margaret Shatara, MD	Phase 1/2 Study of Bempegaldesleukin in Combination with Nivolumab in Children, Adolescents, and Young Adults with Recurrent or Refractory Malignancies (PIVOT IO 020)
Mohamed S. Abdelbaki, MD	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
Robert Hayashi, MD	Selinexor in Treating Younger Patients with Recurrent or Refractory Solid Tumors or High Grade Glioma
Karen Gauvain, MD	Study of RXDX-101 in Children with Recurrent of Refractory Solid Tumors and Primary CNS Tumors, with or without TRK, ROS1, or ALK Fusions
Karen Gauvain, MD	Using MRI-Guided Laser Heat Ablation to Induce Disruption of the Peritumoral Blood Brain Barrier to Enhance Delivery and Efficacy of Treatment of Pediatric Brain Tumors
Joshua Rubin, MD, PhD	A Study Comparing Two Carboplatin Containing Regimens for Children and Young Adults with Previously Untreated Low Grade Glioma
Karen Gauvain, MD	Phase II Pediatric Study With Dabrafenib in Combination With Trametinib in Patients With HGG and LGG
Joshua Rubin, MD, PhD	Maintenance Chemotherapy or Observation Following Induction Chemotherapy and Radiation Therapy in Treating Younger Patients With Newly Diagnosed Ependymoma
Karen Gauvain, MD	Reduced Craniospinal Radiation Therapy and Chemotherapy in Treating Younger Patients With Newly Diagnosed WNT-Driven Medulloblastoma
Karen Gauvain, MD	CA209-908 – Phase Ib/II Clinical Trial of Nivolumab Monotherapy and Nivolumab in Combination with Ipilimumab in Pediatric Subjects with High Grade Primary CNS Malignancies
Karen Gauvain, MD	PNOC 001 – Phase II Study of Everolimus for Recurrent or Progressive Low-Grade Gliomas in Children
Karen Gauvain, MD	H3.3K27M Peptide Vaccine for Children With Newly Diagnosed DIPG and Other Gliomas
Karen Gauvain, MD	PNOC013 – A Safety and Pharmacokinetic Study of Single Agent REGN2810 in Pediatric Patients with Relapsed or Refractory Solid or Central Nervous System (CNS) Tumors and a Safety and Efficacy Trial of REGN2810 in Combination with Radiotherapy in Pediatric Patients with Newly Diagnosed Diffuse Intrinsic Pontine Glioma, Newly Diagnosed High-Grade Glioma, or Recurrent High-Grade Glioma
Karen Gauvain, MD	Phase I-II Study of MEK162 for Children with Ras/Raf Pathway Activated Tumors
Karen Gauvain, MD	Modified Measles Virus (MV-NIS) for Children and Young Adults With Recurrent Medulloblastoma or Recurrent ATRT
Karen Gauvain, MD	Vemurafenib in Children With Recurrent/Refractory BRAFV600E-mutant Gliomas
Joshua Rubin, MD, PhD	Study Assessing the Feasibility of a Surgery and Chemotherapy-Only in Children With Wnt Positive Medulloblastoma
Joshua Rubin, MD, PhD	Cognitive Biomarkers in Pediatric Brain Tumor Patients
Shalini Shenoy, MD	ANBL1531: Iobenguane I-131 or Crizotinib and Standard Therapy in Treating Younger Patients with Newly-Diagnosed High-Risk Neuroblastoma or Ganglioneuroblastoma
Karen Gauvain, MD	Expanded Access to ONC201 for Patients with H3 K27M-mutant and/or Midline High Grade Gliomas
Karen Gauvain, MD	REGN2810 in Pediatric Patients with Relapsed, Refractory Solid, or Central Nervous System (CNS) Tumors and Safety and Efficacy of REGN2810 in Combination with Radiotherapy in Pediatric Patients with Newly Diagnosed or Recurrent Glioma
Karen Gauvain, MD	An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type I
Karen Gauvain, MD	Veliparib, Radiation Therapy, and Temozolimide in Treating Participants with Newly Diagnosed Malignant Glioma Without H3 K27M or BRAFV600E Mutations
Karen Gauvain, MD	Cabozantinib for Plexiform Neurofibromas (PN) in Subjects With NF1 in Children and Adults

Cancer predisposition

Principal Investigator	Title
Robert Hayashi, MD	Phenotypic Characterizations of Cancer Predisposition Syndromes

Hematologic malignancies

Principal investigator	Title
Neha Mehta-Shah, MD / Robert Hayashi, MD	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
Robert Hayashi, MD	AAML18P1: Stopping Tyrosine Kinase Inhibitors (TKI) to Assess Treatment-Free Remission (TFR) in Pediatric Chronic Myeloid Leukemia – Chronic Phase (CML-CP)
Robert Hayashi, MD	Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients with Down Syndrome
Robert Hayashi, MD	Trentinoin and Arsenic Trioxide in Treating Patients with Untreated Acute Promyelocytic Leukemia
Robert Hayashi, MD	A Phase II Study of Ruxolitinib with Chemotherapy in Children with Acute Lymphoblastic Leukemia
Robert Hayashi, MD	Blinatumomab in Treating Younger Patients with Relapsed B-Cell Acute Lymphoblastic Leukemia
Robert Hayashi, MD	Combination Chemotherapy in Treating Young Patients with Newly Diagnosed High-Risk B Acute Lymphoblastic Leukemia and Ph-Like TKI Sensitive Mutations
Robert Hayashi, MD	A Study of Brentuximab Vedotin + Adriamycin, Vinblastine, and Dacarbazine in Pediatric Participants With Advanced Stage Newly Diagnosed Hodgkin Lymphoma
Robert Hayashi, MD	Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening Trial)
Robert Hayashi, MD	A Study of Nivolumab Plus Brentuximab Vedotin in Patients Between 5 and 30 Years Old, With Hodgkin’s Lymphoma (cHL), Relapsed or Refractory From First Line Treatment
Robert Hayashi, MD	Brentuximab Vedotin and Combination Chemotherapy in Treating Children and Young Adults With Stage IIB or Stage IIIB-IVB Hodgkin Lymphoma
Robert Hayashi, MD	A Study of Pembrolizumab (MK-3475) in Pediatric Participants with an Advanced Solid Tumor or Lymphoma
Robert Hayashi, MD	Post Transplant Lymphoproliferative Disease: Analysis of Outcome to Chemotherapy Treatment
Robert Hayashi, MD	Rituximab and LMP-Specific T-Cells in Treating Pediatric Solid Organ Recipients with EBV-Positive Cluster of Differentiation (CD) 20-Positive Post-Transplant Lymphoproliferative Disorder
Shalini Shenoy, MD	A Study of Venetoclax in Combination With Navitoclax and Chemotherapy in Subjects With Relapsed Acute Lymphoblastic Leukemia or Relapsed/Refractory Lymphoblastic Lymphoma
Jeffrey Magee, MD	Matched Targeted Therapy for High-Risk Leukemias and MDS
Robert Hayashi, MD	A Study of Nivolumab Plus Brentuximab Vedotin in Patients Between 5 and 30 Years old, with Hodgkin’s Lymphoma (cHL), Relapsed or Refractory From First Line Treatment

Sickle cell disease

Principal investigator	Title
Shalini Shenoy, MD	A Multi-center Retrospective Registry of Children with Sickle Cell Disease following Hematopoietic Cell Transplantation: a Sickle Transplant Alliance for Research (STAR) Project
Allison King, MD, PhD	Specifying Interventions From the Sickle Cell Disease Implementation Consortium
Shalini Shenoy, MD	A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in Severe Sickle Cell Disease

Solid tumors

Principal investigator	Title
Amy Armstrong, MD	Blood Based Biomarkers for Minimal Residual Disease Detection in Pediatric Sarcomas
Amy Armstrong, MD	Phase 1 trial of the LSD1 Inhibitor Seclidenstat (SP-2577) with and without Topotecan and Cyclophosphamide in patients with relasped or refractory Ewing Sarcoma and select Sarcomas
Mohamed S. Abdelbaki, MD	A Phase 1/2, Single-arm Study to Evaluate the Safety, Pharmacokinetics, and Antitumor Activity of Avapritinib in Pediatric Patients With Solid Tumors Dependent on KIT or PDGFRA Signaling
Amy Armstrong, MD	An Open-Label, Multicenter Phase 1/2 Study of Surufatinib in Combination with Gemcitabine in Pediatric, Adolescent, and Young Adult Patients with Recurrent or Refractory Solid Tumors
Robert Hayashi, MD	A Phase 1/2 Trial of CBL0137 (NSC# 825802, IND# 155843) in Patients with Relapsed or Refractory Solid Tumors including CNS Tumors and Lymphoma
Robert Hayashi, MD	A Phase 1/ 2 Study of BAY 1895344 (elimusertib, IND#152153, NSC#810486) in Pediatric Patients with Relapsed or Refractory Solid Tumors
Robert Hayashi, MD	A Phase 1/2 Study of Tegavivint (IND#156033, NSC#826393) in Children, Adolescents, and Young Adults with Recurrent or Refractory Solid Tumors, Including Lymphomas and Desmoid Tumors
Amy Armstrong, MD	A Maintenance Protocol of Sirolimus in Combination with Metronomic Chemotherapy in Children with High-Risk Solid Tumors
Frederick Huang, MD	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)
Frederick Huang, MD	Response and Biology-Based Risk Factor-Guided Therapy in Treating Younger Patients With Non-high Risk Neuroblastoma
Robert Hayashi, MD	Selinexor in Treating Younger Patients with Recurrent or Refractory Solid Tumors or High Grade Gliomas
Karen Gauvain, MD	Study of RXDX-101 in Children with Recurrent of Refractory Solid Tumors and Primary CNS Tumors, with or without TRK, ROS1, or ALK Fusions
Robert Hayashi, MD	PI3K/mTOR Inhibitor LY3023414 in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With TSC or PI3K/MTOR Mutations (A Pediatric MATCH Treatment Trial)
Robert Hayashi, MD	Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening Trial)
Robert Hayashi, MD	Active Surveillance, Bleomycin, Carboplatin, Etoposide, or Cisplatin in Treating Pediatric and Adult Patients With Germ Cell Tumors
Frederick Huang, MD	Combination Chemotherapy With or Without Ganitumab in Treating Patients With Newly Diagnosed Metastatic Ewing Sarcoma
Todd Druley, MD, PhD	International Pleuropulmonary Blastoma (PPB) Treatment and Biology Registry
Alok Kothari, MD	Treating Relapsed/Recurrent/Refractory Pediatric Solid Tumors With Sorafenib in Combination With Irinotecan
Alok Kothari, MD	Erlotinib in Combination With Temozolomide in Treating Relapsed/Recurrent/Refractory Pediatric Solid Tumors
Robert Hayashi, MD	Nivolumab with or without Ipilimumab in Treating Younger Patients with Recurrent or Refractory Solid Tumors or Sarcomas
Robert Hayashi, MD	WEE1 Inhibitor MK-1775 and Irinotecan Hydrochloride in Treating Younger Patients with Relapsed or Refractory Solid Tumors
Robert Hayashi, MD	A Study of Pembrolizumab (MK-3475) in Pediatric Participants with an Advanced Solid Tumor or Lymphoma
Robert Hayashi, MD	PI3K/mTOR Inhibitor LY3023414 in Treating Patients with Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders with TSC or PI3k/MTOR Mutations (A Pediatric MATCH Treatment Trial)
Robert Hayashi, MD	ADVL1614 – A Phase 1/2 Study of VX15/2503 (IND# 136181) in Children, Adolescents or Young Adults with Recurrent or Relapsed Solid Tumors
Robert Hayashi, MD	Prexasertib in Treating Pediatric Patients with Recurrent or Refractory Solid Tumors
Robert Hayashi, MD	Nanoparticle Albumin-Bound Rapamycin, Temozolomide, and Irinotecan Hydrochloride in Treating Pediatric Patients with Recurrent or Refractory Solid Tumors
Robert Hayashi, MD	A Study of Ramucirumab (LY3009806) in Children with Refractory Solid Tumors
Robert Hayashi, MD	Pevonedistat, Irinotecan Hydrochloride, and Temozolomide in Treating Patients With Recurrent or Refractory Solid Tumors or Lymphoma
Robert Hayashi, MD	Entinostat in Treating Pediatric Patients with Recurrent or Refractory Solid Tumors
Robert Hayashi, MD	Accelerated v’s Standard BEP Chemotherapy for Patients with Intermediate and Poor-risk Metastatic Germ Cell Tumours
Robert Hayashi, MD	Active Surveillance, Bleomycin, Carboplatin, Etoposide, or Cisplatin in Treating Pediatric and Adult Patients with Germ Cell Tumors
Robert Hayashi, MD	Pazopanib Paediatric Phase II Trial Children’s Oncology Group (COG) in Solid Tumors
Robert Hayashi, MD	Biomarkers in Tumor Tissue Samples From Patients With Newly Diagnosed Neuroblastoma or Ganglioneuroblastoma
Robert Hayashi, MD	Study of Kidney Tumors in Younger Patients
Robert Hayashi, MD	Accelerated v’s Standard BEP Chemotherapy for Patients With Intermediate and Poor-risk Metastatic Germ Cell Tumours (P3BEP)
Robert Hayashi, MD	Larotrectinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With NTRK Fusions (A Pediatric MATCH Treatment Trial)
Robert Hayashi, MD	Erdafitinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With FGFR Mutations (A Pediatric MATCH Treatment Trial)
Robert Hayashi, MD	Selumetinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Activating MAPK Pathway Mutations (A Pediatric MATCH Treatment Trial)
Robert Hayashi, MD	Ensartinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With ALK or ROS1 Genomic Alterations (A Pediatric MATCH Treatment Trial)
Robert Hayashi, MD	Vemurafenib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With BRAF V600 Mutations (A Pediatric MATCH Treatment Trial)
Robert Hayashi, MD	Olaparib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Defects in DNA Damage Repair Genes (A Pediatric MATCH Treatment Trial)
Robert Hayashi, MD	Palbociclib in Treating Patients With Relapsed or Refractory Rb Positive Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Activating Alterations in Cell Cycle Genes (A Pediatric MATCH Treatment Trial)

Stem cell transplant & cellular therapy

Principal investigator	Title
Sima Bhatt, MD	BMT CTN 1904- Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure Diseases
Sima Bhatt, MD	Real-World Treatment Patterns and Outcomes in Patients with Hemophagocytic Lymphohistiocytosis (HLH) and other Clinical Conditions Treated with Emapalumab across Treatment Centers in the US (REAL-HLH Study)
Shalini Shenoy, MD	A Pilot Study in the Treatment of Refractory BK Infections with Related Donor BK Cytotoxic T-Lymphocytes in Children, Adolescent, and Young Adult Recipients
Shalini Shenoy, MD	A Pilot Study in the Treatment of Refractory EBV Infection with Related Donor EBV Cytotoxic T-Lymphocytes in Children, Adolescent, and Young Adult Recipients
Peter Westervelt, MD, PhD; Shalini Shenoy, MD	Clinical Transplant-related Long-term Outcomes of Alternative Donor Allogeneic Transplant (CTRL-ALT-D)
Shalini Shenoy, MD	NYMC 579: A Pilot Study in the Treatment of Refractory Adenovirus (ADV) Infection with Related Donor ADV Cytotoxic T-Lymphocytes (ADV-CTLs) in Children, Adolescents and Young Adult Recipients
Shalini Shenoy, MD	A Pilot Study in the Treatment of Refractory Cytomegalovirus (CMV) Infections with Related Donor CMV Specific Cytotoxic T-cells (CTLs) in Children, Adolescents, and Young Adult Recipients – NYMC 580
Shalini Shenoy, MD	Bone Marrow Transplantation vs Standard of Care in Patients With Severe Sickle Cell Disease (STRIDE2) (BMT CTN 1503)
Shalini Shenoy, MD	Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
Jeffrey Bednarski, MD, PhD	Cytokine Induced Memory-like NK Cell Adoptive Therapy for Relapsed AML After Allogeneic Hematopoietic Cell Transplant
Shalini Shenoy, MD	Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases
Shalini Shenoy, MD	UVADEX^® and ECP for the Treatment of Pediatric Patients With Steroid Refractory Acute Graft Versus Host Disease
Shalini Shenoy, MD	An Expanded Access Program of Ruxolitinib for the Treatment of Graft-Versus-Host Disease Following Allogeneic Hematopoietic Stem Cell Transplant
Robert Hayashi, MD	Prevalence of Ototoxicity following Bone Marrow Transplantation and Stem Cell Transplantation in Pediatrics
Andrew Cluster, MD	Anti-Angiogenic Therapy Post Transplant (ASCR) for Pediatric Solid Tumors (LASCR)
Shalini Shenoy, MD	Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMT CTN 1507)
Shalini Shenoy, MD	Optimizing Cord Blood and Haploidentical Aplastic Anemia Transplantation (BMT CTN 1502) (CHAMP)
Sima Bhatt, MD	CAR-T Cell Therapy for Acute Pre-B Leukemia and Lymphoma
Jeffrey Bednarski, MD, PhD	Reduced Intensity Transplant for Immune Deficiency Disorders (Immune Disorder HSCT Protocol)
Shalini Shenoy, MD	Phase ½ Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)
Shalini Shenoy, MD	A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in Severe Sickle Cell Disease

Survivorship

Principal investigator	Title
Robert Hayashi, MD	Long-Term Follow-Up of Patients Who Have Participated in Children’s Oncology Group Studies
Joshua Rubin, MD, PhD	Neuropsychological and Behavioral Testing in Younger Patients With Cancer
Robert Hayashi, MD	Genetic Analysis in Identifying Late-Occurring Complications in Childhood Cancer Survivors
Robert Hayashi, MD	Prevalence of Ototoxicity following Bone Marrow Transplantation and Stem Cell Transplantation in Pediatrics
Robert Hayashi, MD	Childhood Cancer Survivor Study (CCSS) – Expansion
Robert Hayashi, MD	Knowledge and perceptions of infertility in adolescent female cancer survivors and parents
Robert Hayashi, MD	Late Onset and Asymmetric Sensorineural Hearing Loss in Pediatric Cancer Survivors Following Treatment with Cisplatin Containing Chemotherapy Regimens
Caroline Mohrmann, PNP	Patient, Family, and Provider Decision-Making in the Setting of Chemotherapy-Induced Hearing Loss
Robert Hayashi, MD	Effects of Modern Chemotherapy Regimens on Spermatogenesis and Steroidogenesis in Adolescent and Young Adult (AYA) Survivors of Osteosarcoma
Miranda Camet	Retrospective Review of Audiologic Test Results for Pediatric Patients with Beckwith-Wiedemann Syndrome and hemihypertrophy
Shalini Shenoy, MD	A Multi-center Retrospective Registry of Children with Sickle Cell Disease following Hematopoietic Cell Transplantation: a Sickle Transplant Alliance for Research (STAR) Project

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