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	David Wilson, MD, PhD	wilson_d@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 & 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 & hematology	Monica Hulbert, MD	monicahulbert@wustl.edu
Solid tumors	Amy Armstrong, MD	armstrongae@wustl.edu
Stem cell transplantation & cellular therapy	Shalini Shenoy, MD	shalinishenoy@wustl.edu
Survivorship	Robert Hayashi, MD	hayashi_r@wustl.edu

Studies

All cancers

Principal investigator	Title
Allison King, MD, PhD	SPARK: Symptom Screening Linked to Care Pathways for Children With Cancer: a Cluster Randomized 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)

Brain tumors

Rare Brain Tumor Consortium Registry and Biobank

To learn more about this opportunity, please contact Andrew Cluster, MD

Principal investigator	Title
Andrew Cluster, MD	A Phase 3 Study of Sodium Thiosulfate for Reduction of Cisplatin-Induced Ototoxicity in Children With Average-Risk Medulloblastoma and Reduced Therapy in Children With Medulloblastoma With Low-Risk Features
Andrew Cluster, MD	A Phase 3 Randomized Study of Selumetinib Versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)
Andrew Cluster, MD	A Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine Versus Selumetinib (NSC# 748727) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) Not Associated With BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1)
Andrew Cluster, MD	A Phase 3 Study of Selumetinib (NSC# 748727) 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
Robert J. Hayashi, MD	A Phase I/II Study of Pembrolizumab (MK-3475) in Children With Advanced Melanoma or a PD-L1 Positive Advanced, Relapsed or Refractory Solid Tumor or Lymphoma (KEYNOTE-051)
Robert J. Hayashi, MD	The Project: EveryChild Protocol: A Registry, Eligibility Screening, Biology and Outcome Study
Robert J. Hayashi, MD	NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Screening Protocol APEC1621SC, APEC1621A, APEC1621D, APEC1621F, APEC1621K, APEC1621M, APEC1621N
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
Andrew Cluster, MD	HeadStart4: Newly Diagnosed Children (<10 y/o) With Medulloblastoma and Other CNS Embryonal Tumors Clinical and Molecular Risk-Tailored Intensive and Compressed Induction Chemotherapy Followed by Consolidation With Randomization to Either Single Cycle or to Three Tandem Cycles of Marrow-Ablative Chemotherapy With Autologous Hematopoietic Progenitor Cell Rescue
Robert J. Hayashi, MD	Classification of Acute Lymphoblastic Leukemia Consent for Collection of Additional Bone Marrow and Consent for Study Entry and Shipment of Samples
Robert J. Hayashi, MD	A Pharmacokinetic Study of VinCRIStine in Infants Dosed According to BSA-Banded Infant Dosing Tables and Older Children Dosed by Traditional BSA Methods
Mohamed S. Abdelbaki, MD	FIREFLY-1: A Phase 2, Open-Label, Multicenter Study to Evaluate the Safety and Efficacy of the Oral Pan-RAF Inhibitor DAY101 in Pediatric Patients With BRAF-Altered, Recurrent or Progressive Low-Grade Glioma and Advanced Solid Tumors
Margaret S. Shatara, MD	A Pilot Trial of Real Time Drug Screening and Genomic Testing to Determine an Individualized Treatment Plan in Children and Young Adults With Relapsed Medulloblastoma
Allison King, MD, MPH, PhD	Symptom Screening Linked to Care Pathways for Children With Cancer: a Cluster Randomized Trial
Andrew Cluster, MD	A Prospective Multi-Institutional Study to Assess the Risk of Radiation Induced Vasculopathy and Stroke as well as Stroke Recurrence in Children with Cancer who Received Radiation Therapy to the Neck and/or Brain
Andrew Cluster, MD	A Phase 1/2, Open-Label, Safety, Tolerability, Pharmacokinetics, and Anti-Tumor Activity Study of Repotrectinib in Pediatric and Young Adult Subjects With Advanced or Metastatic Malignancies Harboring ALK, ROS1, NTRK1-3 Alterations
Amy Armstrong, MD	A Phase II Trial of Poly-ICLC for Progressive, Previously Treated Low-Grade Gliomas in Children and Young Adults With Neurofibromatosis Type 1
Andrew Cluster, MD	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)
Andrew Cluster, MD	A Combination Therapy Trial Using an Adaptive Platform Design for Children and Young Adults With Diffuse Midline Gliomas (DMGs) Including Diffuse Intrinsic Pontine Gliomas (DIPGs) at Initial Diagnosis, Post-Radiation Therapy and at Time of Progression
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
Margaret S. Shatara, 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
Andrew Cluster, MD	Phase II Pediatric Study With Dabrafenib in Combination With Trametinib in Patients With HGG and LGG
Andrew Cluster, MD	Maintenance Chemotherapy or Observation Following Induction Chemotherapy and Radiation Therapy in Treating Younger Patients With Newly Diagnosed Ependymoma
Andrew Cluster, MD	Reduced Craniospinal Radiation Therapy and Chemotherapy in Treating Younger Patients With Newly Diagnosed WNT-Driven Medulloblastoma
Margaret S. Shatara, 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
Mohamed S. Abdelbaki, MD	PNOC 001 – Phase II Study of Everolimus for Recurrent or Progressive Low-Grade Gliomas in Children
Margaret S. Shatara, MD	A Pilot Study to Assess the Safety, Feasibility, and Preliminary Efficacy of a Neoepitope-based Personalized DNA Vaccine Approach in Pediatric Patients With Recurrent Brain Tumors
Mohamed S. Abdelbaki, 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
Andrew Cluster, MD	Phase I-II Study of MEK162 for Children with Ras/Raf Pathway Activated Tumors
Andrew Cluster, MD	Modified Measles Virus (MV-NIS) for Children and Young Adults With Recurrent Medulloblastoma or Recurrent ATRT
Stephanie M. Perkins, MD	Cognitive Biomarkers in Pediatric Brain Tumor Patients
Frederick S. Huang, MD	ANBL1531: Iobenguane I-131 or Crizotinib and Standard Therapy in Treating Younger Patients with Newly-Diagnosed High-Risk Neuroblastoma or Ganglioneuroblastoma
Andrew Cluster, MD	Expanded Access to ONC201 for Patients with H3 K27M-mutant and/or Midline High Grade Gliomas
Mohamed S. Abdelbaki, 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

Cancer predisposition

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

Hematologic malignancies

Principal investigator	Title
Robert J. Hayashi, MD	AALL1731: A Phase 3 Trial Investigating Blinatumomab (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)
Robert J. Hayashi, MD	AALL1732: A Phase 3 Randomized Trial of Inotuzumab Ozogamicin (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
Shalini Shenoy, MD	AAML1831: 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
Robert J. Hayashi, MD	AALL1821: A Phase 2 Study of Blinatumomab (NSC# 765986) in Combination With Nivolumab (NSC # 748726), a Checkpoint Inhibitor of PD-1, in B-ALL Patients Aged >/= 1 to < 31 Years Old With First Relapse
Robert J. Hayashi, MD	ANHL1931: A Randomized Phase 3 Trial of Nivolumab (NSC# 748726) in Combination With Chemo-Immunotherapy for the Treatment of Newly Diagnosed Primary Mediastinal B-Cell Lymphoma
Robert J. Hayashi, MD	AALL1631: International Phase 3 Trial in Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Ph+ALL) Testing Imatinib in Combination With Two Different Cytotoxic Chemotherapy Backbones
John DiPersio, MD, PhD	SNDX5613-0700: A Phase 1/2, Open-label, Dose-Escalation and Dose-Expansion Cohort Study of SNDX-5613 in Patients With Relapsed/Refractory Leukemias, Including Those Harboring an MLL/KMT2A Gene Rearrangement or Nucleophosmin 1 (NPM1) Mutation
Robert J. Hayashi, MD	AALL1621: A Phase 2 Study of Inotuzumab Ozogamicin (NSC# 772518) in Children and Young Adults With Relapsed or Refractory CD22+ B-Acute Lymphoblastic Leukemia (B-ALL)
Amanda Cashen, MD	WUSM A L L Bank: Tissue Acquisition for Analysis of Prognostic Factors, Immunology, and Genetic Progression of HIV-1 Associated Malignancies
Peter Westervelt, MD	WUSM AML Bank: Tissue Acquisition for Analysis of Genetic Progression Factors in Hematologic Diseases
Robert J. Hayashi, MD	PEPN22P1: A Pharmacokinetic Study of VinCRIStine in Infants Dosed According to BSA-Banded Infant Dosing Tables and Older Children Dosed by Traditional BSA Methods
Robert J. Hayashi, MD	ADVL1822: A Phase 1/2, Multicenter, Dose-Escalating Study To Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy Of Quizartinib Administered in Combination With Re-Induction Chemotherapy, and as a Single-Agent Continuation Therapy, in Pediatric Relapsed/Refractory AML Subjects Aged 1 Month to <18 Years (and Young Adults Aged up to 21 Years) With FLT3-ITD Mutations
Robert J. Hayashi, MD	APAL2020SC: The Pediatric Acute Leukemia (PedAL) Screening Trial – A Study to Test Bone Marrow and Blood in Children With Leukemia That Has Come Back After Treatment or Is Difficult to Treat – A Leukemia & Lymphoma Society and Children’s Oncology Group Study
Neha Mehta-Shah, MD / Robert Hayashi, MD	E4412: 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	APEC1621: 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	ADVL1621: A Study of Pembrolizumab (MK-3475) in Pediatric Participants with an Advanced Solid Tumor or Lymphoma
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

Late effects

Principal investigator	Title
Robert Hayashi, MD	StepByStep: A Randomized Trial of a Mobile Health and Social Media Physical Activity Intervention Among Adolescent and Young Adult Childhood Cancer Survivors
Robert Hayashi, MD	SurvivorLink: Scalability of an Electronic Personal Health Record for Cancer Survivors and Caregivers at Pediatric Cancer Centers (SurvivorLink Scalability)

Sickle cell disease & hematology

Principal investigator	Title
Monica Hulbert, MD	Hibiscus: An Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral Etavopivat, a Pyruvate Kinase Activator in Patients With Sickle Cell Disease (HIBISCUS)
David Wilson, MD, PhD	X4P: A Phase 1b/2, Open-Label, Multicenter Study of Mavorixafor in Patients With Congenital Neutropenia and Chronic Neutropenia Disorders
Sana Saif Ur Rehman, MD	GRNDaD: Globin research Network for DATA and Discovery
Surbhi Saini, MD	Throm-PED: Observational study of pediatric thrombotic disease
Monica Hulbert, MD	MRI study: Cerebral Hemodynamics in Children with Hemoglobinopathies Undergoing Hematopoietic Stem Cell Transplant
Shalini Shenoy, MD	STAR: 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

Solid tumors

Principal investigator	Title
Robert Hayashi, MD	Intravitreal Melphalan for Intraocular Retinoblastoma
Frederick Huang, MD	A Safety, Pharmacokinetic and Efficacy Study of a y-Secretase Inhibitor, Nirogacestat (PF-03084014) in Children and Adolescents With Progressive, Surgically Unresectable Desmoid Tumors
Robert Hayashi, MD	A Phase 2 Study of DS-8201a (NSC#807708, IND#153036) in Adolescents, or Young Adults with Recurrent HER2+ Osteosarcoma
Amy Armstrong, MD	Treatment of Newly Diagnosed Diffuse Anaplastic Wilms Tumors (DAWT) and Relapsed Favorable Histology Wilms Tumors (FHWT)
Frederick Huang, MD	A Pilot Study of Dinutuximab, Sargramostim (GM-CSF), and Isotretinoin in Combination With Irinotecan and Temozolomide in the Post-Consolidation Setting for High-Risk Neuroblastoma
Bryan Sisk, MD	EPIK-P2: A Phase II Double-blind Study With an Upfront, 16-week Randomized, Placebo-controlled Period, to Assess the Efficacy, Safety and Pharmacokinetics of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (PROS)
Robert Hayashi, MD	A Pharmacokinetic Study of VinCRIStine in Infants Dosed According to BSA-Banded Infant Dosing Tables and Older Children Dosed by Traditional BSA Methods
Robert Hayashi, MD	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
Amy Armstrong, MD	A Prospective Phase 3 Study of Patients with Newly Diagnosed Very Low-risk and Low-risk Fusion Negative Rhabdomyosarcoma
Amy Armstrong, MD	Phase 1/2 Study to Evaluate Palbociclib (Ibrance®) in Combination with Irinotecan and Temozolomide and/or in Combination with Topotecan and Cyclophosphamide in Pediatric Patients with Recurrent or Refractory Solid Tumors
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
Amy Armstrong, 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
David Wilson, MD, PhD	International Pleuropulmonary Blastoma (PPB) Treatment and Biology Registry
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	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	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	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
Shalini Shenoy, MD	17-SIBS – Identifying Predictors of Poor Health-Related Quality-of-Life Among Pediatric Hematopoietic Stem Cell Donors
Shalini Shenoy, MD	A Phase II Pilot Trial to Estimate Survival After a Non-total Body Irradiation (TBI) Based Conditioning Regimen in Patients Diagnosed With B-acute Lymphoblastic Leukemia (ALL) Who Are Pre-allogeneic Hematopoietic Cell Transplantation (HCT) Next-generation-sequence (NGS) Minimal Residual Disease (MRD) Negative
Shalini Shenoy, MD	A Phase 2, Randomized, Multicenter Study to Evaluate the Efficacy and Safety of KD025 in Subjects With Chronic Graft Versus Host Disease (cGVHD) After At Least 2 Prior Lines of Systemic Therapy (The ROCKstar Study)
Shalini Shenoy, MD	Early HLA Matched Sibling Hematopoietic Stem Cell Transplantation for Children With Sickle Cell Disease: A Sickle Transplant Advocacy and Research Alliance (STAR) Trial
Robert Hayashi, MD	Managed Access Program (MAP) to provide access to tisagenlecleucel in line with locally approved indications with out of specification leukapheresis product and/or manufactured tisagenlecleucel out of specification for commercial release (CAR-T MAP Cohort)
Robert Hayashi, MD	AALL1721: A Phase II Trial of Tisagenlecleucel in First-Line High-Risk (HR) Pediatric and Young Adult Patients With B-cell Acute Lymphoblastic Leukemia (B-ALL) Who are Minimal Residual Disease (MRD) Positive at the End of Consolidation (EOC) Therapy
Jeffrey Bednarski, MD, PhD	A Randomized Trial of Low Versus Moderate Exposure Busulfan for Infants With Severe Combined Immunodeficiency (SCID) Receiving TCRαβ+/CD19+ Depleted Transplantation: A Phase II Study by the Primary Immune Deficiency Treatment Consortium (PIDTC) and Pediatric Blood and Marrow Transplant Consortium (PBMTC)
Jeffrey Bednarski, MD, PhD	Severe Combined Immune Deficiency: Prospective and Longitudinal Study of Genotypes, Management and Outcomes (PIDTC 6907)
Jeffrey Bednarski, MD, PhD	A Prospective Natural History Study of Diagnosis, Treatment and Outcomes of Children with SCID Disorders (PIDCT 6901)
Jeffrey Bednarski, MD, PhD	Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995 (PIDTC 6903)
Jeffrey Bednarski, MD, PhD	A Pilot Study of Haploidentical Hematopoietic Stem Cell Transplantation With Ex Vivo TCR Alpha/Beta and CD19 Depletion in Pediatric Hematologic Malignancies
Jeffrey Bednarski, MD, PhD	EXCEL trial: A Phase II Pilot Study of Donor-Derived Ex-Vivo Expanded Natural Killer Cell Infusions in Children and Young Adults with High Risk Acute Myeloid Leukemia Receiving Myeloablative HLA-Haploidentical Hematopoietic Cell Transplant: A Multicenter Pediatric Transplantation and Cellular Therapy Consortium (PTCTC) Study- PTCTC CT2001
Shalini Shenoy, MD	BMT CTN 1904- Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure Diseases
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
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	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
Jeffrey Bednarski, MD, PhD	Reduced Intensity Transplant for Immune Deficiency Disorders (Immune Disorder HSCT Protocol)

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	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