| CNH-0012021: Phase II Study Investigating the Efficacy of Neoadjuvant Dual Checkpoint Inhibition and Cryoablation Therapy in Children, Adolescents, and Young Adults with Relapsed/Refractory Solid Tumors | The is a phase II, single arm, open-label, multi-site trial studying the combination of cryoablation therapy and dual checkpoint inhibition with nivolumab (anti-PD-1) and ipilimumab (anti-CTLA-4) given at the recommended phase 2 dose (RP2D) in pediatric and young adult patients with relapsed or refractory solid tumors. | - Ages ≥ 1 year and <40 years old
- Relapsed/refractory solid tumors (osteosarcoma, Ewing sarcoma, rhabdomyosarcoma) and at least 2 sites of measurable disease
| Children's National Hospital | Marie Nelson, M.D. | Ewing sarcoma, osteosarcoma, rhabdomyosarcoma |
ADVL2121 - 2020-012-GLOB2: 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
NCT05093322 | A Phase 1/2 study to establish a recommended Phase 2 dose (RP2D) of nivolumab in combination with 5-azacytidine in children with relapsed or refractory AML and to assess the clinical activity of Nivolumab in combination with 5-azacytidine in AML patients with M2/M3 disease at study entry. | - Part 1 and 2: ages ≥ 2 and ≤ 21 years old (< 30 in the osetosarcoma cohort)
- Part 1: recurrent or refractory solid tumors or lymphoma, that have a known or expected dysfunction of VEGFR-1, -2, and -3; FGFR-1; or CSF-1R pathways
- Part 2: recurrent or refractory osetosarcoma, Ewing sarcoma, rhabdomyosarcoma, or non-rhabdomyosarcoma (NRSTS)
| Hutchison MediPharma Limited | AeRang Kim, M.D. | Lymphoma, osetosarcoma, Ewing sarcoma, rhabdomyosarcoma, non-rhabdomyosarcoma (NRSTS), other solid tumors |
BA3011-001: A Phase 1/2 Dose Escalation and Dose Expansion Study of BA3011 Alone and in Combination with Nivolumab in Adult and Adolescent Patients 12 Years and Older with Advanced Solid Tumors
NCT03425279 | A Phase 1/2 study to define the safety profile, including dose-limiting toxicity (DLT), and determine the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D) and other safety parameters for BA3011 in patients with advanced solid tumors. Additionally the study aims to assess antitumor activity, safety, and further characterize the clinical activity of BA3011 alone and in combination with nivolumab. | - Phase 1: Age ≥ 18 years
Locally advanced unresectable or metastatic solid tumor - Phase 2: Age ≥ 12 years
Locally advanced unresectable or metastatic sarcoma
| BioAlta, Inc. | AeRang Kim, M.D. | Sarcomas, other solid tumors |
ADVL2021- J1S-MC- JV01: A Randomized, Open-Label Phase 1/2 Study Evaluating Ramucirumab in Pediatric Patients and Young Adults with Relapsed, Recurrent, or Refractory Desmoplastic Small Round Cell Tumor
NCT04145349 | A Phase 1/2 study to investigate the efficacy of ramucirumab in combination with low-dose cyclophosphamide and vinorelbine for the treatment of pediatric and young adult patients with desmoplastic small round cell tumor (DSRCT). | - Ages 12 months to ≤ 29 years old
- Relapsed, recurrent, or refractory DSRCT
| Eli Lilly and Company | AeRang Kim, M.D. | Desmoplastic small round cell tumor |
PEPN1924: A Phase 2 Study of DS-8201a (Nsc# 807708, Ind# 153036) in Adolescents, or Young Adults with Recurrent Her2+ Osteosarcoma
NCT04616560 | A Phase 2 study to estimate the proportion of patients with recurrent measurable osteosarcoma treated with DS-8201a who are event free (%EF) at 24 weeks, and assess the safety of DS-8201a in patients with recurrent osteosarcoma. | - Ages > 12 years and ≤ 39 years old
- Osteosarcoma and confirmed HER2 expression of >10% of osteosarcoma cells
| Children's Oncology Group | AeRang Kim, M.D. | Osteosarcoma |
PEPN2011: 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
NCT04851119 | A Phase 1/2 study to Investigate the safety, dosing and efficacy of Tegavivint in children and young adults with relapsed or refractory solid tumors including desmoid tumors and lymphoma. | - Part A: ≥ 12 months and ≤ 21 years old
- Relapsed or refractory solid tumors, including patients with non-Hodgkin lymphoma and desmoid tumors
- Part B: ≥ 12 months and ≤ 30 years
- Recurrent or refractory Ewing sarcoma, desmoid tumors, osteosarcoma, liver tumors (HCC and hepatoblastoma), Wilms tumor, and tumors with Wnt pathway aberrations.
| Children's Oncology Group | AeRang Kim, M.D. | Solid tumors, including non-Hodgkin lymphoma and desmoid tumors |
PEPN2112: A Phase 1/ 2 Study of BAY 1895344 (elimusertib, IND#152153, NSC#810486) in Pediatric Patients with Relapsed
or Refractory Solid Tumors
NCT05071209 | A Phase 1/2 study to investigate the safety, dosing and efficacy of BAY 1895344 (elimusertib) in children and young adults with relapsed or refractory solid tumors. | - Part A1: Patients with specified recurrent or refractory solid tumors or lymphomas without bone marrow involvement ≥ 12 months and < 18 years of age
- Part B1a: Patients with any Ewing Sarcoma or any EWS-fusion positive solid tumor ≥ 18 years of age
- Part B2a: Patients with alveolar rhabdomyosarcoma (ARMS) with the PAX3-FOXO1 fusion ≥ 18 years of age
- Part B3a: Patients with any (non-CNS primary) solid tumor including lymphoma with inactivation of any of the DNA Damage Repair (DDR) genes ≥ 18 years of age
| Children's Oncology Group | AeRang Kim, M.D. | Ewing sarcoma, alveolar rhabdomyosarcoma with PAX-FOXO1 fusion, non-CNS primary solid tumor including lymphoma with inactivation of DDR genes |
A Phase Ib/II Study of APG-115 in Combination with Pembrolizumab in Patients with Unresectable or Metastatic Melanomas or Advanced Solid Tumors
NCT03611868 | The study is currently a Phase 2 study assessing the overall response rate of APG-115 in combination with pembrolizumab in the treatment of patients advanced solid tumors. Children's is enrolling for Cohort F for patients with metastatic, refractory or unresectable malignant peripheral nerve sheath tumors. | - Malignant peripheral nerve sheath tumor
- Ages ≥ 12 years
| Ascentage Pharma Group Inc. | AeRang Kim, M.D. | Malignant peripheral nerve sheath tumor |
TK216-01: A Phase I, Dose Escalation Study of Intravenous TK216 in Patients with Relapsed or Refractory Ewing Sarcoma
NCT02657005 | A multicenter, open-label, Phase 1 study in which the safety and efficacy of TK216 will be evaluated in patients with relapsed or refractory Ewing sarcoma. The study is currently in the dose and schedule evaluation. | - Relapsed/refractory Ewing sarcoma
- Ages ≥ 8 years
| Oncternal Therapeutics, Inc. | AeRang Kim, M.D. | Ewing sarcoma |
Pivotal/Phase II Clinical Trial of Magnetic Resonance-Guided Focused Ultrasound (MR-HIFU) Treatment of Painful Osteoid Osteoma in Children and Young Adults
NCT: pending | A Phase 2 study to determine treatment safety and efficacy of MR-HIFU ablation of painful osteoid osteoma in children and young adults. | - Painful osteoid osteoma
- Ages ≤ 30 years
| Children's National Hospital | Karun Sharma, M.D. | Osteoid osteoma |
ADVL1721: A non-randomized, open-label, multi-center, Phase I/II study of PI3K inhibitor copanlisib in pediatric patients with relapsed/refractory solid tumors or lymphoma
NCT03458728 | Phase 1/2 study of copanlisib for pediatric patients with a relapsed/refractory solid tumor or lymphoma. | Phase 1- Recurrent/refractory solid tumors or lymphoma
- Ages 6 months to ≤ 21 years old
Phase 2- Recurrent/refractory Ewing sarcoma, rhabdomyosarcoma, osteosarcoma or neuroblastoma
- Ages 6 months old to ≤21 years old
| Bayer Pharmaceuticals | AeRang Kim, M.D. | Ewing sarcoma, rhabdomyosarcoma, osteosarcoma, neuroblastoma, lymphoma, solid tumors |
Phase I/II, Open-Label, Multicenter Study to Evaluate the Safety, Tolerability,and Preliminary Efficacy of Durvalumab Monotherapy or Durvalumab inCombination with Tremelimumab in Pediatric Patients with Advanced SolidTumors and Hematological Malignancies
NCT03837899 | This is a first time in pediatrics study primarily designed to evaluate the safety and tolerability of durvalumab and durvalumab in combination with tremelimumab at increasing doses in pediatric patients with advanced solid malignancies including lymphomas. | - Relapsed/refractory osteosarcoma, Ewing sarcoma, rhabdomyosarcoma, nonrhabdomyosarcoma soft tissue sarcoma, other sarcomas, neuroblastoma, other solid tumors, Hodgkin's lymphoma, non-Hodgkin's lymphoma, acute lymphoblastic leukemia, acute myeloid leukemia, myelodysplastic syndrome, juvenile myelomonocytic leukemia
- Ages birth to <18 years old
| AstraZeneca | Marie Nelson, M.D. | Osteosarcoma, ewing sarcoma, rhabdomyosarcoma, nonrhabdomyosarcoma soft tissue sarcoma, other sarcomas, neuroblastoma, other solid tumors, Hodgkin's lymphoma, non-Hodgkin's lymphoma, acute lymphoblastic leukemia, acute myeloid leukemia, myelodysplastic syndrome, juvenile myelomonocytic leukemia |
A Phase 2 Trial of the MEK inhibitor selumetinib (AZD6244 hydrogen sulfate) in combination with the mTOR inhibitor sirolimus for patients with unresectable or metastatic malignant peripheral nerve sheath tumors
NCT03433183 | A phase 2 trial of MEK inhibitor selumetinib in combination with the mTOR inhibitor sirolimus to determine the safety and clinical benefit in patients with unresectable or metastatic malignant peripheral nerve sheath tumors (MPNSTs). | - Unresectable or metastatic histologically confirmed sporadic or NF1 associated malignant peripheral nerve sheath tumors
- Ages ≥ 12 years
| Sarcoma Alliance for Research though Collaboration | AeRang Kim, M.D. | Malignant peripheral nerve sheath tumor |
A Phase I Study of Lyso-thermosensitive Liposomal Doxorubicin (LTLD, ThermoDox®) and Magnetic Resonance-Guided High Intensity Focused Ultrasound (MR-HIFU) for Treatment of Relapsed or Refractory Solid Tumors in Children, Adolescents, and Young Adults.
NCT02536183 | A Phase 1 trial of LTLD with MR-HIFU in children and young adults with relapsed/refractory solid tumors. | - Relapsed/refractory solid tumors
- Target lesion(s) must be located in areas accessible to HIFU
- Ages ≤ 21 years of age
| Children's National Hospital | AeRang Kim, M.D. | Solid tumors |
ADVL1823: Larotrectinib (LOXO-101, NSC# 788607, IND# 141824) for Previously Untreated TRK Fusion Pediatric Solid Tumors and TRK Fusion Relapsed Pediatric Acute Leukemias
NCT03834961 | A Phase 2 study of Larotrectinib, a highly selective oral small molecule inhibitor of the TRK family of tyrosine kinases which are encoded by the NTRK genes in select pediatric cancers. | - Cohort A: histologic diagnosis of infantile fibrosarcoma
- Cohort B: histologic diagnosis of any solid tumor including CNS tumors but excluding high grade gliomas, with an NTRK1, NTRK2, or NTRK3 fusion
- Cohort C: histologic diagnosis of relapsed/refractory acute leukemia with an NTRK1, NTRK2, or NTRK3 fusion
- Ages ≤ 30 years of age
| Children's Oncology Group | AeRang Kim, M.D. | Infantile fibrosarcoma, TRK fusion solid tumors, including CNS and TRK Fusion acute leukemias |
ADVL1921: Phase 1 study to evaluate the safety and pharmacokinetics of Palbociclib (Ibrance®) in combination with Irinotecan and Temozolomide and in combination with Topotecan and Cyclophosphamide in pediatric patients with recurrent or refractory solid tumors
NCT03709680 | This is a Phase 1 study of palbociclib in combination with chemotherapy (irinotecan and temozolomide or topotecan and cyclophosphamide) in pediatric patients with recurrent/refractory solid tumors | - Recurrent/refractory solid tumors, including CNS tumors
- Age ≥ 2 to < 21 years
| Pfizer | AeRang Kim, M.D. | All solid tumors, CNS |
PNOC-013: 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
NCT03690869 | A study evaluating REGN2810, a human monoclonal antibody targeting the PD-1 receptor in pediatric patients with relapsed or refractory solid or central nervous system tumors. | - Newly diagnosed diffuse intrinsic pontine glioma, high grade glioma, recurrent high grade glioma and solid tumors
- Primary diagnosis of high grade glioma of the spinal cord or disseminated disease not eligible
- Ages 3 to 25 years
| Pacific Neuro-Oncology Consortium | Lindsay Kilburn, M.D. | Diffuse intrinsic pontine glioma, high grade glioma, recurrent high grade glioma, solid tumors |
EH102: A Phase 1 Study of the EZH2 Inhibitor Tazemetostat in Pediatric Subjects with Relapsed or Refractory INI1-Negative Tumors or Synovial Sarcoma
NCT02601937 | This is a phase 1 study of Tazemetostat for patients with INI1 negative tumors of CNS or rhabdoid solid tumors. | - Relapsed/refractory disease of malignant rhabdoid tumors, including CNS
- Ages > 6 months to < 18 years
| Epizyme | Lindsay Kilburn, M.D. | Rhabdoid tumors, INI1 negative tumors includes CNS tumors |
OZM-077: Phase I/Ib Trial of Combined 5'Azacitidine and Carboplatin for Recurrent/Refractory Pediatric Brain and Solid Tumors
NCT03206021 | Phase 1/1b study of the combination of azacitidine and carboplatin for CNS ependymomas and childhood solid tumors. | - Recurrent/refractory brain or solid tumor including recurrent/refractory ependymomas
- Tissue from diagnosis or resection is required
- Ages 1 to < 18 years
| The Hospital for Sick Children | Eugene Hwang, M.D. | CNS ependymoma, solid tumors |