The cure rate for childhood cancer has improved greatly in the last two decades largely because of treatment in national clinical trials. In clinical trials, patients receive treatment and doctors carry out research on how the treatment affects patients. A person’s progress is closely monitored during the trial. Once the treatment portion of the trial has been completed, patients may continue to be followed in order to gather information regarding specific endpoints. These endpoints are defined prior to the study being started and may include time for disease progression and/or overall survival.

The Children's Oncology Group has nearly 100 active clinical trials open at any given time and more than 90% of children with cancer are cared for at a COG site. These trials include front-line treatment for many types of childhood cancers, studies aimed at determining the underlying biology of these diseases, and trials involving new and emerging treatments, supportive care, and survivorship.

In addition to running clinical trials with the Children’s Oncology Group, our team also has trials open with Pharmaceutical sponsors such as Gradalis, Incyte and Iderra. Our team also works on trials initiated by our own physicians.

Below is a list of open clinical trials available at Cleveland Clinic Children’s Department of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation. This list is constantly changing, so please contact the other individual study PIs listed below.

Pediatric Hematology Oncology Clinical Trials

Cancer Control Trials

  • ALTE07C1: Neuropsychological, Social, Emotional and Behavior Outcomes in Children with Cancer
  • ALTE16C1: Effects of Modern Chemotherapy Regimens on Spermatogenesis and Steroidogenesis in Adolescent and Young Adult (AYA) Survivors of Osteosarcoma
  • ALTE1631: A randomized study investigating the effects of a web-based intervention on moderate and vigorous physical activity among children and adolescents with Acute Lymphoblastic Leukemia ALL
  • ACCL1633: The Effectiveness of Lactobacillus plantarum (LBP, IND# 17339) in Preventing Acute Graft-versus-Host Disease (GvHD) in Children undergoing Alternative Hematopoietic Progenitor Cell Transplantation (HCT)
  • ACCL1333: A Phase III Randomized, Open Label, Multi-center Study of the Safety and Efficacy of Apixaban for Thromboembolism Prevention versus No Systemic Anticoagulant Prophylaxis during Induction Chemotherapy in Children with Newly Diagnosed  (ALL) or Lymphoma (T or B cell) Treated with Pegylated Asparaginase

Central Nervous System Tumor Trials

  • ACNS1422: A Phase 2 Study of Reduced Therapy for Newly Diagnosed Average-Risk WNT-Driven Medulloblastoma Patients
  • HeadStart 4: (Tanya Tekautz, MD) Headstart 4  Newly Diagnosed children (Less than10 y.o.) with medulloblastoma and other CNS Embryonal tumors
  • ACNS1721: A Phase 2 Study of Veliparib (ABT-888, IND # 139199) and Local Irradiation, Followed by Maintenance Veliparib and Temozolomide, in Patients with Newly Diagnosed High-Grade Glioma (HGG) without H3 K27M or BRAFV600E Mutations

Leukemia Trials

  • AALL1131: A Phase III Randomized Trial for Newly Diagnosed High Risk B-Lymphoblastic Leukemia (B-ALL) Including a Stratum Evaluating Dasatinib (IND#73789, NSC#732517) in Patients with Ph-like Tyrosine Kinase Inhibitor (TKI) Sensitive Mutations
  • AALL1521: A Phase 2 Study of the JAK1/JAK2 Inhibitor Ruxolitinib With Chemotherapy in Children With De Novo High-Risk CRLF2-Rearranged and/or JAK Pathway–Mutant Acute Lymphoblastic Leukemia
  • AAML1531: Risk-stratified Therapy for Acute Myeloid Leukemia in Down Syndrome
  • AALL1631: International Phase 3 Trial in Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia Ph+ ALL Testing Imatinib in Combination with Two Different Cytotoxic Chemotherapy Backbones
  • AALL1621: 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)
  • ALTE1631: A Randomized Web-based Physical Activity Intervention among Children and Adolescents with Acute Lymphoblastic Leukemia
  • IRB 5024: REGISTRY: Collection of Blood and Bone Marrow From Normal Volunteers and Patients for Research Purposes
  • AALL1731: 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)
  • ACCL1333: A Phase III Randomized, Open Label, Multi-center Study of the Safety and Efficacy of Apixaban for Thromboembolism Prevention versus No Systemic Anticoagulant Prophylaxis during Induction Chemotherapy in Children with Newly Diagnosed Acute Lymphoblastic Leukemia (ALL) or Lymphoma (T or B cell) Treated with Pegylated Asparaginase

Lymphoma Trials

  • ANHL12P1: A Randomized Phase 2 Trial of Brentuximab Vedotin (SGN35, NSC# 749710), or Crizotinib (NSC#749005, commercially labeled) in Combination with Chemotherapy for Newly Diagnosed Patients with Anaplastic Large Cell Lymphoma (ALCL) IND # 117117
  • 18-1183: Registry: Sequential Banking of Blood and Tissue Samples from Pediatric and Young Adult Patients with Solid Tumors 
  • 18-486: (Ilia Buhtoiarov, MD) Quantification of therapeutic responses in pediatric and AYA Hodgkin Lymphoma patients via detection of circulating tumor DNA
  • AALL1731: 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)
  • S1826: A Phase III, 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

Solid Tumor Trials

  • ANBL1232: Utilizing Response-and Biology-based Risk Factors to Guide Therapy in Patients with Non-High-Risk Neuroblastoma
  • ANBL00B1: Neuroblastoma Classification Biology Studies
  • ADVL1622: Phase 2 Trial of XL184 (Cabozantinib) an Oral Small-Molecule Inhibitor of Multiple Kinases, in Children and Young Adults with Refractory Sarcomas, Wilms Tumor, and Other Rare Tumors
  • AGCT1531: 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
  • AHEP1531: Pediatric Hepatic Malignancy International Therapeutic Trial (PHITT)
  • ANBL1531: A Phase 3 Study of 131I-Metaiodobenzylguanidine (131I-MIBG) or Crizotinib Added to Intensive Therapy for Children with Newly Diagnosed High-Risk Neuroblastoma (NBL) (IND# 134379)
  • ARST1431: A Randomized Phase 3 Study of Vincristine, Dactinomycin, Cyclophosphamide (VAC) Alternating with Vincristine and Irinotecan (VI) Versus VAC/VI Plus Temsirolimus (TORI, Torisel, NSC# 683864) in Patients with Intermediate Risk (IR) Rhabdomyosarcoma (RMS)
  • AGCT1532: A Randomized Phase 3 Trial of Accelerated versus Standard BEP Chemotherapy for Patients with Intermediate and Poor-risk Metastatic Germ Cell Tumors
  • ONC201: (Peter Anderson, MD) Phase 2 Study of ONC201 in Neuroendocrine Tumors
  • CL-PTL-130: (Peter Anderson, MD) A Multi-center Phase III, Randomized, Open-Label Trial of Vigil (bi-shRNAfurin and GMCSF Augmented Autologous Tumor Cell Immunotherapy) in combination with Irinotecan and Temozolimide as a Second-Line Regimen for Ewing’s Sarcoma
  • AREN1721: A Randomized Phase 2 Trial of Axitinib/Nivolumab combination therapy vs single agent Axitinib or Nivolumab for the treatment of TFE/Translocation Renal Cell Carcinoma across all age groups
  • NMTRC014: (Stacey Zahler, DO) NMTT Phase II NMRTC-014 Neuroblastoma Maintenance therapy trial using Difluoromethylornithine (DFMO)
  • APEC1621: NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)
  • 18-1183: Registry: Sequential Banking of Blood and Tissue Samples from Pediatric and Young Adult Patients with Solid Tumors 
  • IRB 15-1580: CASE 11Z15: Tissue and Body Fluid Analysis from Patients with Cancer and Other Risk- Associated Lesions
  • ANBL1821: A Phase 2 Randomized Study of Irinotecan/Temozolomide/Dinutuximab with or without Eflornithine (DFMO) (IND# 141913) in Children with Relapsed, Refractory or Progressive Neuroblastoma
  • TPX-0005-01: (Peter Anderson, MD) A Phase 1/2, Open-Label, Multi-Center, First-in-Human Study of the Safety, Tolerability, Pharmacokinetics, and Anti-Tumor Activity of TPX-0005 in Patients with Advanced Solid Tumors Harboring ALK, ROS1, or NTRK1-3 Rearrangements (TRIDENT-1)
  • TK216: (Peter Anderson, MD) A Phase 1, Dose Escalation Study of Intravenous TK216 in Patients With Relapsed or Refractory Ewing Sarcoma
  • ADVL1921: (Stacey Zahler, DO) Phase 1 Study to Evaluate the Safety and Pharmacokinetics of Palbociclib (Ibrance®) in Combination with Irinotecan and Temozolomide in Pediatric Patients with Recurrent or Refractory Solid Tumors

Survivor Trials 

  • Microbiome: (Seth Rotz, MD) Evaluating the Microbiome of Long-Term Childhood Cancer Survivors and the Relationship to Metabolic Syndrome

Non-Cancer Trials 

  • PTEN: (Rabi Hanna, MD) A Randomized Double-Blind Controlled Trial of Everolimus in Children and Adolescents with PTEN Mutations (RAD001XUS257T)
  • B0661037: (Ravi Talati, DO) A Randomized, Open-label, active controlled, safety and extrapolated efficacy study in pediatric subjects requiring anticoagulation for the treatment of a venous thromboembolic event
  • NOVA1Z17: (Rabi Hanna, MD) A phase II, open-label, non-controlled, intra-patient doseescalation 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