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

Benign Hematology

  • B0661037: (Pfizer) A Randomized, Open-Label, Active Controlled, Safety and Extrapolated Efficacy Study in Pediatric Subjects Requiring Anticoagulation for the Treatment of a Venous Thromboembolic Event

Bone Marrow Transplant

  • CBMT1Z15: Natural History and Biology of Long-Term Effect Following Hematopoietic Cell Transplant for Childhood Hematologic Malignancies
  • FHCC1Z14: Multi-center, Open-label Randomized Study of Single or Double Myeloablative Cord Blood Transplantation with or without Infusion of Off-the-shelf Ex Vivo Expanded Cryopreserved Cord Blood Progenitor Cells in Patients with Hematologic Malignancies
  • JAZZ1Z16: A Phase 3, Randomized, Adaptive Study Comparing the Efficacy and Safety of Defibrotide vs Best Supportive Care in the Preventative of Hepatic Veno-Occlusive Disease in Adult and Pediatric Patients Undergoing Hematopoietic Stem Cell Transplant
  • MESO1Z16: A Single-arm, Prospective Study of Remestemcel-L, Ex-vivo Cultured Adult Human Mesenchymal Stromal Cells, for the Treatment of Pediatric Patients Who Have Failed to Respond to Steroid Treatment for Acute GVHD
  • THKO1Z15: Single-Arm Study to Assess the Efficacy of UVADEX (methoxsalen) Sterile Solution in Conjunction with the THERAKOS CELLEX Photopheresis System in Pediatric Patients with Steroid-Refractory aGVHD

For questions about bone marrow trials, please contact Dr. Rabi Hanna.

Leukemia/Lymphoma Trials

  • COG AALL0932: Treatment of Patients with Newly Diagnosed Standard Risk B-precursor Acute Lymphoblastic Leukemia
  • COG AALL1131: a Phase III Randomized Trial for Newly Diagnosed High Risk B0precursor Acute Lymphoblastic Leukemia (ALL) Testing Clofarabine (IND# 73789, NSC# 606869) in the Very High Risk Stratum
  • COG AALL1231: A Phase III Randomized Trial Investigating Bortezomib (NSC# 681239; IND# 58443) on a Modified Augmented BFM (ABFM) Backbone in Newly Diagnosed T-Lymphoblastic Leukemia (T-ALL) AND T-Lymphoblastic Lymphoma (T-LLy)
  • COG AALL1331: Risk-Stratified Randomized Phase III Testing of Blinatumomab (IND# 117467, NSC# 765986) in First Relapse of Childhood B-Lymphoblastic Leukemia
  • COG/Incyte 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
  • COG AALL15P1: A Groupwide Pilot Study to Test the Tolerability and Biologic Activity of the Addition of Azacitidine to Chemotherapy in Infants with Acute Lymphoblastic Leukemia (ALL) and KMT2A (MLL) Gene Rearrangement
  • COG AALL1631: International Phase 3 Trial in Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia Ph+ ALL Testing Imatinib in Combination with Two Different Cytotoxic Chemotherapy Backbones
  • COG AAML1331: A Phase III Study for Patients with Newly Diagnosed Acute Promyelocytic Leukemia (APL) Using Arsenic Trioxide and All-Trans Retinoic Acid
  • COG AAML1421: A Phase ½ Study of CPX-351 (NSC# 775431, IND# 129443) Alone Followed by Fludarabine, Cytarabine, and G-CSF (FLAG) for Children with Relapsed Acute Myeloid Leukemia (AML)
  • COG AAML 1531: Risk-stratified Therapy for Acute Myeloid Leukemia in Down Syndrome
  • COG AHOD1331: A Randomized Phase 3 Study of Bretuximab Vedotin (SGN-35, IND# 117117) for Newly Diagnosed High-Risk Classical Hodgkin Lymphoma (cHL) in Children and Young Adults
  • COG ALTE11C1: Longitudinal Assessment of Ovarian Reserve in Adolescents with Lymphoma
  • COG ALTE1631: Web-based Exercise Study for Children and Adolescents with Acute Lymphoblastic Leukemia

Neuro-Oncology Trials

  • COG ACNS0332: Efficacy of Carboplatin Administered Concomitantly with Radiation and Isotretinoin as a Pro-Apoptic Agent in Other Than Average Risk Medulloblastoma/PNET Patients
  • COG ACNS0831: Phase III Randomized Trial of Post-Radiation Chemotherapy in Patients with Newly Diagnosed Ependymoma Ages 1 to 21 years
  • COG ACNS1422: A Phase 2 Study of Reduced Therapy for Newly Diagnosed Average-Risk WNT-Driven Medulloblastoma Patients
  • Head Start 4: (Nationwide Children’s, Tanya Tekautz, MD) Newly Diagnosed Children (less than 10 years old) with Medulloblastoma and Other Central Nervous System Embryonal Tumors

Solid Tumor Trials

  • 2125-RST-101: (Idera Pharmaceuticals, Peter Anderson, MD) A Phase 1b Study of Intratumoral IMO-2125 in Patients with Refractory Solid Tumors
  • CL-PTL-121: (Gradalis, Peter Anderson, MD) Randomized Phase IIb Trial of Vigil™ (bishRNAfurin and GMCSF Augmented Autologous Tumor Cell Immunotherapy) versus gemcitabine + docetaxel for Ewing’s Sarcoma
  • COG ADVL1622: Phase 2 Trial of XL184 (Cabonzantinib) an Oral Small-Molecule Inhibitor of Multiple Kinases, in Children and Young Adults with Refractory Sarcomas, Wilms Tumor, and Other Rare Tumors
  • COG AEWS1221: Randomized Phase II Trial Evaluating the Addition of the IGF-1R Monoclonal Antibody Ganitumab (AMG 479, NSC# 750008, IND# 120449) to Multiagent Chemotherapy for Patients with Newly Diagnosed Metastatic Ewing Sarcoma
  • COG 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
  • COG AHEP0731: Treatment of Children with All Stages of Hepatoblastoma with Temsirolimus (IND# 122782, NSC# 683864) Added to High Risk Stratum Treatment
  • COG ANBL1231: Utilizing Response- and Biology-based Risk Factors to Guide Therapy in Patients with Non-High-Risk Neuroblastoma
  • COG AOST1321: Phase 2 Study of Denosumab (IND# 127430, NSC# 744010), a RANK Ligand Antibody, for Recurrent or Refractory Osteosarcoma
  • COG ARST1321: Pazopanib Neoadjuvant Trial in Non-Rhabdomyosarcoma Soft Tissue Sarcomas (PAZNTIS): A Phase II/III Randomized Trial of Preoperative Chemoradiation or Preoperative Radiation Plus or Minus Pazopanib (NSC# 737754, IND# 118613)
  • COG Pediatric MATCH Protocols: NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice Master Version)
  • ONC201: (PI Initiated Trial, Peter Anderson, MD) Phase 2 Study of ONC201 in Neuroendocrine Tumors

Other Trials

  • RDCRN Protocol #7904: (Rabi Hanna, MD)A Randomized Double-Blind Controlled Trial of Everolimus in Children and Adolescents with PTEN Mutations
  • SHP-609-302: (Shire, Rabi Hanna, MD) An Open Label Extension of Study HGT-HIT-094 Evaluating Long-term Safety and Clinical Outcomes of Intrathecal Idursulfase Administered in Conjunction with Elaprase® in patients with hunter Syndrome and Cognitive Impairment