Faculty and fellows of the Division of Pediatric Hematology-Oncology are engaged in a wide range of clinical, translational and basic investigation related to pediatric cancer, disorders of the blood including clotting or bleeding problems, and the quality of life of children and their families undergoing severe medical conditions.
Research Faculty
Lars Martin Wagner, MD, Chief
Areas of interest: Solid tumors, combination therapies for sarcoma
Michael D. Deel, MD
Areas of interest: Gene regulation and molecular pathways responsible for alveolar rhabdomyosarcoma
Susan Kreissman, MD
Areas of interest: Improving the treatment of neuroblastoma through the study of new treatment plans, coordinating multidisciplinary care for retinoblastoma patients, leading national inquiry in COG neuroblastoma treatment studies.
Daniel Bryce Landi, MD
Areas of interest: Development and implementation of novel immune-based therapies
Corinne Linardic, MD, PhD
Areas of interest: The study of childhood rhabdomyosarcoma, using primary cell-based and xenograft models to understand the implicated signaling pathways and to identify potential therapeutic targets.
Kristy Pahl, MD
Areas of interest: Pediatric cancer, bleeding disorders, vascular malformations
Joanna Robles, MD
Areas of interest: Cancer and blood disorders, health disparities within pediatric hematology-oncology
Jennifer Rothman, MD
Areas of interest: Benign hematology, including congenital hemolytic anemia, sickle cell disease, immune thrombocytopenia, aplastic anemia and bone marrow failure syndromes.
Kristin M. Schroeder, MD
Areas of interest: Improving outcomes for pediatric cancer patients in low resource countries, including creating diagnostic algorithms to predict patients with lymphoma in areas where biopsy may not be readily available, as well as evaluating the factors contributing to high treatment abandonment rates among pediatric cancer patients.
Nirmish Ramesh Shah, MD
Areas of interest: Novel therapeutic options for patients with sickle cell disease; transition from pediatric to adult care for sickle cell disease; and use of mobile technology to advance patient care for sickle cell disease; use of wearables to provide objective data to combine with subjective symptoms in patients with sickle cell disease, cancer and bone marrow transplant who have pain.
John J. Strouse, MD, PhD
Areas of interest: My research focuses on the epidemiology, risk factors, and prevention of the pulmonary and central nervous system complications of sickle cell disease and includes retrospective and prospective cohort studies and clinical trials.
Jessica Muller Sun, MD
Areas of interest: Developing umbilical cord blood-derived therapies for the treatment of childhood genetic and acquired neurologic disorders.
David Sinclaire Van Mater, MD
Areas of interest: Factors that impact sarcoma formation; clinical studies that impact oncologic sequelae of neurofibromatosis.
Clinical Trials
Oncology
Leukemia and Lymphoma
Title: A Phase 3 Trial of Blinatumomab in Combination with Chemotherapy in Patients with Newly Diagnosed Standard Risk or Down syndrome B-Lymphoblastic Leukemia (B-ALL) or B-Lymphoblastic Lymphoma (B-LLy)
Study: AALL1731
ClinicalTrials.gov: NCT03914625
Patient Population: Newly diagnosed patients with standard-risk B-ALL or B-lymphoblastic lymphoma
Title: A Phase 3 Randomized Trial of Inotuzumab Ozogamicin for Newly Diagnosed High-Risk B-ALL
Study: AALL1732
ClinicalTrials.gov: NCT03959085
Patient Population: Newly diagnosed patients with high-risk B-ALL, B-lymphoblastic lymphoma, or mixed phenotype leukemia
Title: International Phase 3 Trial of Imatinib with Chemotherapy for Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia Ph+ ALL
Study: AALL1631
ClinicalTrials.gov: NCT03007147
Patient Population: Newly diagnosed patients with Ph+ ALL
Title: A Phase 3 Randomized Trial of Gemtuzumab Ozogamicin GO and CPX-351 with GO for Patients with Newly Diagnosed AML
Study: AAML1831
ClinicalTrials.gov: NCT04293562
Patient Population: Newly diagnosed patients with acute myeloid leukemia (AML)
Title: Risk-stratified Therapy for Acute Myeloid Leukemia in Down Syndrome
Study: AAML1531
ClinicalTrials.gov: NCT02521493
Patient Population: Newly diagnosed patients with Down syndrome and AML
Title: A Phase 3 Randomized Study of Nivolumab (Opdivo) Plus AVD or Brentuximab Vedotin (Adcetris) Plus AVD in Patients (Age >/=12 Years) with Newly Diagnosed Advanced Stage Classical Hodgkin Lymphoma
Study: S1826
ClinicalTrials.gov: NCT03907488
Patient Population: Patients with newly diagnosed advanced Hodgkin lymphoma
Title: A Phase 2 Study of the MEK Inhibitor Trametinib in Children with Relapsed or Refractory Juvenile Myelomonocytic Leukemia
Study: ADVL1521
ClinicalTrials.gov: NCT03190915
Patient Population: Patients with recurrent juvenile myelomonocytic leukemia (JMML)
Title: A Phase 2 Study of Inotuzumab Ozogamicin in Children and Young Adults with Relapsed or Refractory CD22+ B-Acute Lymphoblastic Leukemia (B-ALL)
Study: AALL1621
ClinicalTrials.gov: NCT02981628
Patient Population: Patients with relapsed B- ALL or B-lymphoblastic lymphoma
Solid Tumors
Title: A Phase 3 Study of 131I-Metaiodobenzylguanidine (131I-MIBG) or Crizotinib Added to Intensive Therapy for Children with Newly Diagnosed High-Risk Neuroblastoma (NBL)
Study: ANBL1531
ClinicalTrials.gov: NCT03126916
Patient Population: Newly diagnosed patients with high-risk neuroblastoma
Title: 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
Study: AGCT153131
ClinicalTrials.gov: NCT03067181
Patient Population: Newly diagnosed patients with low or standard risk germ cell tumor
Title: Pediatric Hepatic Malignancy International Therapeutic Trial (PHITT)
Study: AHEP1531
ClinicalTrials.gov: NCT03533582
Patient Population: Newly diagnosed patients with hepatoblastoma or hepatocellular carcinoma
Title: A Randomized Phase 3 Study of Vincristine, Dactinomycin, Cyclophosphamide (VAC) Alternating with Vincristine and Irinotecan (VI) Versus VAC/VI Plus Temsirolimus in Patients with Intermediate Risk (IR) Rhabdomyosarcoma (RMS)
Study: ARST1431
ClinicalTrials.gov: NCT02567435
Patient Population: Patients with newly diagnosed rhabdomyosarcoma
Title: Treatment of Newly Diagnosed Diffuse Anaplastic Wilms Tumors (DAWT) and Relapsed Favorable Histology Wilms Tumors (FHWT)
Study: AREN1921
ClinicalTrials.gov: NCT04322318
Patient Population: Newly diagnosed patients with Wilms tumor having diffuse anaplasia, or those with relapsed Wilms tumor
Title: NMTT- Neuroblastoma Maintenance Therapy Trial Using Difluoromethylornithine (DFMO)
Study: Beat Childhood Cancer 014
ClinicalTrials.gov: NCT02679144
Patient Population: Patients with high-risk neuroblastoma in first remission
Title: A Phase Ib/II Study to Evaluate the Safety, Feasibility and Efficacy of Nivolumab or Nivolumab in Combination with Azacitidine in Patients with Recurrent, Resectable Osteosarcoma
Study: MCC19487
ClinicalTrials.gov: NCT03628209
Patient Population: Patients with recurrent osteosarcoma
Title: A Phase 2 Study of DS-8201a (NSC#807708, IND#153036) in Adolescents, or Young Adults with Recurrent HER2+ Osteosarcoma
Study: PEPN1924
ClinicalTrials.gov: NCT04616560
Patient Population: Patients with recurrent osteosarcoma
Title: A Phase 1 Study of the Bromodomain (BRD) and Extra-Terminal Domain (BET) Inhibitor BMS-986158 in Pediatric Cancer
Study: BMS-986158
ClinicalTrials.gov: NCT03936465
Patient Population: Patients with recurrent brain tumors or solid tumors
Title: Expanded Access Treatment with Therapeutic MIBG for Patients with Recurrent Neuroblastoma
Study: MIBG
ClinicalTrials.gov: N/A
Patient Population: Patients with recurrent neuroblastoma
Title: Phase 1/2 Study of 9-ING-41, a Glycogen Synthase Kinase-3 Beta (GSK-3β) Inhibitor, as a Single Agent or with Irinotecan, Temozolomide/Irinotecan or Cyclophosphamide/Topotecan in Pediatric Patients with Refractory Malignancies
Study: Actuate 1902
ClinicalTrials.gov: NCT04239092
Patient Population: Patients with recurrent neuroblastoma, sarcoma, or other solid tumor
Title: Phase 1/2 Study of Radiopharmaceutical CLR 131
Study: DCL-17-001
ClinicalTrials.gov: NCT03478462
Patient Population: Patients with relapsed/refractory solid tumors
Title: Phase I/2 Study of Elimusertib
Study: PEPN2112
ClinicalTrials.gov: NCT05071209
Patient Population: Relapsed rhabdomyosarcoma, Ewing sarcoma
Title: Phase I/II Study of Oratecan Given with Temozolomide
Study: VAL
ClinicalTrials.gov: NCT04337177
Patient Population: Relapsed solid tumors
Brain Tumors
Title: 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)
Study: ACNS1831
ClinicalTrials.gov: NCT04576117
Patient Population: Newly diagnosed patients with Neurofibromatosis Type 1 and low-grade glioma
Title: A Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine versus Selumetinib in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) not associated with BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1)
Study: ACNS1833
ClinicalTrials.gov: NCT04166409
Patient Population: Newly diagnosed patients with low-grade glioma
Title: A Phase 3 Study of Selumetinib or Selumetinib in Combination with Vinblastine for non-NF1 Patients with Recurrent or Progressive Low-Grade Gliomas (LGGs)
Study: ACNS1931
ClinicalTrials.gov: NCT04576117
Patient Population: Patients with recurrent low-grade glioma
Title: Phase 1 Study of the Bromodomain (BRD) and Extra-Terminal Domain (BET) Inhibitor BMS-986158 in Pediatric Cancer
Study: BMS-986158
ClinicalTrials.gov: NCT03936465
Patient Population: Patients with recurrent brain tumors or solid tumors
Title: Developing evidence-based criteria for initiating treatment for neurofibromatosis type I-associated optic pathway glioma
Study: NF1 Natural History
ClinicalTrials.gov: N/A
Patient Population: Patients with newly diagnosed NF1 with optic pathway glioma
Title: A Phase 1b Study of PTC596 in Children with Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma
Study: CONNECT1702
ClinicalTrials.gov: NCT03605550
Patient Population: Patients with newly diagnosed DIPG and HGG
Title: A Pilot and Surgical Study of Larotrectinib for Treatment of Children with Newly-Diagnosed High-Grade Glioma with NTRK Fusion
Study: CONNECT1903
ClinicalTrials.gov: NCT04655404
Patient Population: Patients with newly diagnosed high-grade glioma with NTRK Fusion
Title: PEP-CMV in Recurrent Medulloblastoma/Malignant Glioma (PRiME)
Study: PRiME
ClinicalTrials.gov: NCT03299309
Patient Population: Patients with recurrent/progressive high-grade glioma or medulloblastoma
Title: 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 (DAY101-001)
Study: FIREFLY-1
ClinicalTrials.gov: NCT04775485
Patient Population: Patients with recurrent/progressive low-grade glioma with BRAF Fusion
Title: A Phase II Study of Panobinostat in Paediatric, Adolescent and Young Adult Patients with Solid Tumours Including Osteosarcoma, Malignant Rhabdoid Tumour/Atypical Teratoid Rhabdoid Tumours and Neuroblastoma
Study: NORTH
ClinicalTrials.gov: N/A
Patient Population: Patients with recurrent/refractory atypical teratoid/rhabdoid tumors
Hematology
- Sickle Cell Disease, Thalassemia or Other Inherited Anemia
- Immune Thrombocytopenia or Platelet Disorders
- Bone Marrow Failure
- Hemostasis/Bleeding and Clotting Disorders
Sickle Cell Disease, Thalassemia or Other Inherited Anemia
Title: A Phase 3 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the LentiGlobin BB305 Lentiviral Vector in Subjects with Severe Sickle Cell Disease
Study: HGB-220
ClinicalTrials.gov: N/A
Patient Population: Gene therapy for children and young adults with severe sickle cell disease
Title: 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
Study: CSEG101B2201
ClinicalTrials.gov: N/A
Patient Population: Children ages 6 months to less than 18 years, with SCD (all genotypes), who have experienced at least 1 VOC within the preceding 12 months
Title: A Phase 2 Open-Label Study to Evaluate Safety and Clinical Activity of FT-4202 in Patients with Thalassemia or Sickle Cell Disease
Study: 4202-Hem-201
ClinicalTrials.gov: N/A
Patient Population: Children ages 12 years and over, with SCD or thalassemia (all genotypes), on chronic RBC transfusions
Title: An Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral FT-4202, a Pyruvate Kinase Activator in Patients with Sickle Cell Disease (PRAISE)
Study: 4202-Hem-301
ClinicalTrials.gov: N/A
Patient Population: Children ages 12 years and over, with SCD (all genotypes), who have experienced at least 2 VOC within the preceding 12 months
Title: Reduced Intensity Conditioning for Haploidentical Bone Marrow Transplantation in Patients with Symptomatic Sickle Cell Disease
Study: BMT CTN PROTOCOL 1507
ClinicalTrials.gov: N/A
Patient Population: Children ages 5 years and over, with severe sickle cell disease
Title: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects with Pyruvate Kinase Deficiency Who Are Not Regularly Transfused, Followed by a 5-year Open-label Extension Period
Study: AG348-C-023
ClinicalTrials.gov: N/A
Patient Population: Children ages 1 to less than 18 years with PKD
Title: Globin Regional Network for Data and Discovery Registry
Study: GRNDaD
ClinicalTrials.gov: N/A
Patient Population: Children with SCD
Immune Thrombocytopenia or Platelet Disorders
Title: A Phase 3b, Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel-group Trial with an Open-label Extension Phase to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects with Immune Thrombocytopenia for ≥6 Months
Study: AVA-PED-301
ClinicalTrials.gov: N/A
Patient Population: Children between 1-18 years of age with chronic ITP (lasting greater than 6 months)
Title: Association of Platelet Parameters Identified by the Sysmex XN-1000 and Flow Cytometry with Concurrent and Subsequent Bleeding Severity in Children with Immune Thrombocytopenia (ITP): A Multicenter Study
Study: ITP-APPS
ClinicalTrials.gov: N/A
Patient Population: Children between 6 months to 20 years of age with ITP and platelets < 50,000
Title: Association of Platelet Parameters Identified by the Sysmex XN-1000 and Flow Cytometry with Concurrent and Subsequent Bleeding Severity in Children with Immune Thrombocytopenia (ITP): A Multicenter Study
Study: ITP-APPS
ClinicalTrials.gov: N/A
Patient Population: Children between 6 months to 20 years of age with ITP and platelets < 50,000
Bone Marrow Failure
Title: A Phase II, open-label, non-controlled, intra-patient dose- escalation study to characterize the pharmacokinetics after oral administration of eltrombopag in pediatric patients with refractory, relapsed or treatment naïve severe aplastic anemia or recurrent aplastic anemia
Study: CETB115E2201
ClinicalTrials.gov: N/A
Patient Population: Children between 1 to 18 years of age diagnosed with sever aplastic anemia
Title: NAPAAC Registry Study of Bone Marrow Failure and Myelodysplastic Syndromes
Study: NAPAAC BMF MDS registry
ClinicalTrials.gov: N/A
Patient Population: Children between 1 to 18 years of age diagnosed with bone marrow failure or MDS
Hemostasis/Bleeding and Clotting Disorders
Title: A randomized, open-label, active controlled, safety and descriptive efficacy study in pediatric subjects requiring anticoagulation for the treatment of a venous thromboembolic event
Study: B0661037
ClinicalTrials.gov: N/A
Patient Population: Children between 1 month to 18 years of age with a newly diagnosed blood clot