Pediatrics Clinical Trials
Leukemia/Lymphoma clinical trials
2022-0312: Phase II study of pedi-cRIB: mini-hyper-CVD with condensed rituximab, inotuzumab ozogamicin and blinatumomab (cRIB) for relapsed therapy for pediatric with B-Cell lineage acute lymphocytic leukemia
2022-0804 or COGAPAL2020SC: Pediatric Acute Leukemia (PedAL) screening trial - developing new therapies for relapsed leukemias.
2023-0369: A Phase II Children's Oncology Group (COG) study of tovorafenib (DAY101) in relapsed and refractory Langerhans cell histiocytosis.
2021-0916: A Phase II study of the combination of azacitidine and pembrolizumab for patients relapsed/refractory Hodgkin's lymphoma.
2016-0211: Lead in and Phase II study of Clofarabine, Etoposide, Cyclophosphamide (CEC), Liposomal Vincristine (VCR), Dexamethasone and Bortezomib in relapsed/refractory acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LL).
2016-0772: Phase I study of palbociclib in combination with various agents in patients with relapsed/refractory leukemias.
2020-0686: A Phase II study to determine the response kinetics, safety and efficacy of brentuximab vedotin and nivolumab alone and then combined with rituximab, cyclophosphamide, doxorubicin and prednisone for patients with untreated primary mediastinal large b-cell. Also known as the PACIFIC Trial.
2015-0313: A Phase II study of personalized natural killer (NK) cell immunotherapy in Cord Blood Transplantation.
2014-0845: Phase II clinical trial with HCVAD+Blinatumomab+Inotuzumab for B-ALL
COGAALL1732: A Phase III Randomized Trial of Inotuzumab Ozogamicin (IND#:133494, 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
2020-0818 or COGAAML1831: Phase III 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
2021-1059: A Phase I/II study investigating the all oral combination of the menin inhibitor SNDX-5613 with decitabine/cedazuridine (ASTX727) and venetoclax in Acute Myeloid Leukemia (SAVE-ML).
2018-1092: Bispecific NK Engager (AFM13) combined with cord blood-derived NK cells for refractory Hodgkin and other CD30+ lymphomas
2017-0937: Cardioprotection with Dexrazoxane in Acute Myeloid Leukemia (AML), High-Risk Myelodysplastic Syndrome (MDS), Myeloid Blast Phase of Chronic Myeloid Leukemia (CML), Ph+ AML, and Myeloid Blast Phase of Myeloproliferative Neoplasms
2018-1061: Multiple part clinical trial of brentuximab vedotin in classical Hodgkin lymphoma
2021-0620: Muscle vibration for vincristine associated neuropathy
Neuro-oncology/CNS clinical trials
2024-0303: GCC1949¡ªa Phase II trial of indoximod with chemotherapy and radiation for children with progressive brain tumors or newly diagnosed DIPG. Indoximod was developed to inhibit the IDO (indoleamine 2,3-dioxygenase) enzymatic pathway, which is important in the natural regulation of immune responses.
2024-0094: NCI PBTC-061-a Phase II trial of G207 + 5 Gy radiation for children with high-grade gliomas.
2023-0688: Phase I trial of engineered HSV G207 in children with recurrent or refractory cerebellar brain tumors.
2023-0163: A Phase Ib trial of eribulin in combination with Irinotecan and temozolamide in children with relapsed or refractory solid tumors
2022-0043: 67Cu-SARTATE? Peptide Receptor Radionuclide Therapy administered to pediatric patients with high-risk neuroblastoma. It is a multi-center, dose-escalation, open-label, non-randomized, Phase I/IIa theranostic clinical trial
2020-0220 or COGACNS1833: A Phase III Randomized Non-Inferiority Study of Carboplatin and Vincristine versus Selumetinib (NSC# 748727, IND# 77782) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) not associated with BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1).
2021-0716 or COGACNS2021: A Phase II 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.
COGACNS1831: A Phase III Randomized Study of Selumetinib (IND # 77782) versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)
COGACNS1721:- A Phase II 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 BRAFV600 Mutation
COGACNS1833: A Phase III Randomized Non-Inferiority Study of Carboplatin and Vincristine versus Selumetinib (NSC# 748727, IND# 77782) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) not associated with BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1)
Non-neural solid tumor clinical trials
2021-0390 or AREN1921: Treatment of Newly Diagnosed Diffuse Anaplastic Wilms Tumors (DAWT) and Relapsed Favorable Histology Wilms Tumors (FHWT).
2022-0256 or AOST2031: A Phase III Randomized Controlled Trial Comparing Open vs Thoracoscopic Management of Pulmonary Metastases in Patients with Osteosarcoma.
2022-0573 or ARST2032: A Prospective Phase III Study of Patients with Newly Diagnosed Very Low-Risk and Low-Risk Fusion Negative Rhabdomyosarcoma.
2023-0468 or ARET2121: Intravitreal Melphalan for Intraocular Retinoblastoma. This Phase II trial tests the safety and side effects of adding melphalan to standard chemotherapy in the early treatment of patients with retinoblastoma (RB).
2020-0793: Phase II evolutionary inspired therapy for newly diagnosed, metastatic, fusion positive rhabdomyosarcoma.
2020-0439: Phase II study of avapritinib in patients with CKIT or PDGFRA mutation-positive malignant solid tumors.
2021-0873: A Phase II multi-center open-label basket trial of ABI-009 (nab sirolimus) for adult and adolescent patients with solid tumors harboring pathogenic inactivating alterations in tuberous sclerosis complex (1 or 2) genes.
2020-1340: Phase I Study of Safety, PK, PD and Preliminary Clinical Activity of RP-6306 in Patients with Advanced Solid Tumors (MYTHIC Study ¨C CCNE1 altered)
ARST1921 or 2020-1195: A safety, pharmacokinetic and efficacy study of a secretase inhibitor, nirogacestat (PF-03084014; IND#146375) in children and adolescents with progressive, surgically unresectable desmoid tumors.
COGARST1431 A Randomized Phase III Study of Vincristine, Dactinomycin, Cyclophosphamide (VAC) Alternating with Vincristine and Irinotecan (VI) Versus VAC/VI Plus Temsirolimus (TORI, Torisel, NSC# 683864, IND# 122782) in Patients with Intermediate Risk (IR) Rhabdomyosarcoma (RMS)
COGAPEC1621 Pediatric MATCH (Molecular Analysis for Therapy Choice)
COGAGCT1531 A Phase III 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
2019-1048: A Phase I/II , 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, or NTRK1-3 Alterations
2018-0643: Phase II study of Nivolumab with Ipilimumab in solid tumors of high mutational burden
2015-0158: PLX8394 in patients with advanced, unresectable solid tumors
2020-0833: A Phase I/II open-label multicenter study to assess the safety, tolerability , pharmacokinetics, pharmacodynamics and efficacy of PC14586 in patients with advanced solid tumors harboring a p53 Y220C mutation
Phase I studies/investigational cancer therapeutics
2011-0953: A Phase I/IIa Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of PLX8394 in Patients with Advanced, Unresectable Solid Tumors.
2017-0567: The Targeted Agent and Profiling Utilization Registry (TAPUR) Study is a non-randomized clinical trial that aims to describe the performance (both safety and efficacy) of commercially available, targeted anticancer drugs prescribed for treatment of patients with advanced solid tumors, multiple myeloma or B cell non-Hodgkin lymphoma with a genomic variant known to be a target of an FDA-approved anti-cancer drug or to predict sensitivity to an FDA-approved anti-cancer drug.
Sarcoma and solid tumor clinical trials
2021-0409: A Phase 2 Trial of Atezolizumab and Cabozantinib in Adolescents and Young Adults with Recurrent/metastatic Osteosarcoma (TACOS)
2021-0443: Phase I/II study of CAR.70- engineered IL15-transduced cord blood-derived NK cells in conjunction with lymphodepleting chemotherapy for the management of advanced solid tumors
2021-0619: A first-in-human phase 1 trial of T-Cell membrane-anchored tumor targeted Il12 (Attil12)- T-Cell therapy in subjects with advanced/metastatic soft tissue and bone sarcoma
2018-0224: A Phase I/II study of gemcitabine and docetaxel in combination with hydroxychloroquine (Autophagy Inhibitor) in patients with recurrent osteosarcoma.
2018-0643: A Randomized, Open-Label, Phase 2 Study of Nivolumab in Combination with Ipilimumab or Nivolumab Monotherapy in Participants with Advanced alone is effective and safe in the treatment of solid tumors with High Tumor Mutational Burden (TMB-H).
2017-0085: Ex-vivo expanded allogeneic NK cells for the treatment of solid tumors of pediatric origin in Children and Young Adults.
COGAGCT1531: A Phase III Study of active surveillance for low risk and a randomized trial of carboplatin versus cisplatin for standard risk pediatric and adult patients with germ cell tumors.
2017-0672: ?Clinical study to assess efficacy and safety of LN-145 (Autologous Centrally Manufactured Tumor Infiltrating Lymphocytes) Across Multiple Tumor Types).
2015-0720: Phase I study of Aerosol Gemcitabine in patients with Solid Tumors and Pulmonary Metastases.
Stem cell and cell therapy trials for pediatric/adult patients
2021-0712: A Phase II hematopoietic cell transplantation using treosulfan-based conditioning for the treatment of bone marrow failure diseases
2019-1122: Randomized controlled pilot study of two doses of cord blood tissue-derived mesenchymal stromal cells combined with ruxolitinib versus ruxolitinib alone for steroid-refractory aGVHD.
2015-0313: A Phase II study of personalized natural killer (NK) cell immunotherapy in Cord Blood Transplantation.
2018-0964: 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 (PBMTC ONC1701).
2019-0353: A Phase II Study of Venetoclax in Combination with Azacitidine in the Post-Transplant Setting for High-Risk Acute Leukemia Patients.
2013-0657: A Phase II Most Closely HLA Matched Allogeneic CMV Specific Cytotoxic T-Lymphocytes (CTL) to treat CMV Infection after Hematopoietic Stem Cell Transplantation (HSCT).
2017-0350: Administration of Off-the-Shelf, Expanded, Most Closely HLA Matched, Third Party Adenovirus Specific T Cells for Therapy of Adenovirus Related Disease in Immunocompromised Patients.
2018-0221: A Phase I/II Study of Anti-viral Central Memory CD8 Veto Cells in Haploidentical Hematopoietic Stem Cell Transplantation.
2020-0138: A randomized trial of low versus moderate exposure busulfan for infants with severe combined immunodeficiency (SCID) receiving TCR¦Á¦Â+/CD19+ depleted transplantation: A Phase II study by the Primary Immune Deficiency Treatment Consortium (PIDTC) and Pediatric Blood and Marrow Transplant Consortium (PBMTC) PIDTC ¡°CSIDE¡± Protocol (Conditioning SCID Infants Diagnosed Early) PBMTC NMD 1801
Endocrine clinical trials
2017-0202: A Phase I oral LOXO-292 advanced solid tumors, including RET-fusion non-small cell lung cancer and medullary thyroid cancer, and other tumors with increased RET activity.
Observational, wellness/psychosocial and biobanking (non-therapeutic) studies:
COGAPEC14B1: The Project: Everychild Protocol: A Registry, Eligibility Screening, Biology and Outcome Study
Registry trials for pediatric patients
PA13-0014: Circulating tumor cells in pediatric cancer patients as predictors of chemotherapy response and metastatic disease.
2014-0938: This protocol seeks to gather biospecimens over a period of years for all tumor types. During the clinical research study, samples of normal and tumor tissue, as well as blood, bone, marrow, and bodily fluid will be collected over time.
Branko Cuglievan, M.D., assistant professor and section chief for Pediatric Leukemia and Lymphoma, greets a clinical trial patient before starting an exam.
The Children¡¯s Cancer Hospital continues to be highly regarded on the U.S. News and World Report¡¯s annual list of the . In news released in October 2024, the hospital was ranked No. 24.