ADVL1711: A Phase 1/2 Study of Lenvatinib in Combination With Everolimus in Recurrent and Refractory Pediatric Solid Tumors, Including CNS Tumors
NCT03245151 | This is a Phase 1/2 study of Lenvatinib in combination with Everolimus in pediatric patients with recurrent or refractory solid tumors. The main purpose of this study is to look at the effects of this combination of study drugs on the body, to determine the highest safe dose of study drugs that can be given in combination and to assess any shrinkage in tumors. | Phase I- Recurrent/refractory solid tumors or brain tumors
- Ages ≥ 2 years old to < 18 years old
Phase 2- Recurrent or refractory Ewing sarcoma/pPNET, rhabdomyosarcoma or high grade glioma
- Ages ≥ 2 years old to ≤ 21 years old
| Eisai, Inc. | AeRang Kim, M.D. | Ewing sarcoma, pPNET, rhabdomyosarcoma, high grade glioma, solid tumors, brain tumors, diffuse intrinsic pontine glioma |
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 ≥ 18 years
| Ascentage Pharma Group Inc. | AeRang Kim, M.D. | Malignant peripheral nerve sheath tumor |
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 expansion cohort a recommended phase II dose of TK216 in combination with vincristine. | - Relapsed/refractory Ewing sarcoma
- Ages ≥ 10 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 |
| ADVL1722: A Phase 2, multicenter, open-label study to assess safety andpreliminary activity of eribulin mesylate in pediatric subjects withrelapsed/refractory rhabdomyosarcoma (RMS), nonrhabdomyosarcomasoft tissue sarcoma (NRSTS) and Ewing sarcoma(EWS) NCT03441360 | This is a Phase 2 study of eribulin mesylate in pediatric patients with relapsed/refractory rhabdomyosarcoma (RMS), non-rhabdomyosarcoma soft tissue sarcoma (NRSTS) and Ewing sarcoma (EWS). | - Recurrent/refractory Ewing sarcoma, nonrhabdomyosarcoma soft tissue sarcoma, or rhabdomyosarcoma
- Ages ≥ 12 months old to < 18 years old
| Eisai, Inc. | AeRang Kim, M.D. | Ewing sarcoma, nonrhabdomyosarcoma soft tissue sarcoma, rhabdomyosarcoma |
| 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 Multicenter, Open-label, Randomized Phase 2 Study to Compare the Efficacy and Safety of Lenvatinib in Combination with Ifosfamide and Etoposide versus Ifosfamide and Etoposide in Children, Adolescents and Young Adults with Relapsed or Refractory Osteosarcoma (OLIE)
NCT04154189 | A Phase 2 study to evaluate the efficacy and safety of Lenvatinib in combination with ifosfamide and etoposide in children, adolescents, and young adults with relapsed or refractory osteosarcoma. | - Relapsed/refractory osteosarcoma
- Ages 2 years to ≤ 25 years
| Eisai, Inc. | AeRang Kim, M.D. | Osteosarcoma |
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 |
Safety and Feasibility Study of Using MR-guided High Intensity Focused Ultrasound (HIFU) for the Ablation of Relapsed or Refractory Pediatric Solid Tumors
NCT02076906 | To determine the safety and tolerability of MR-HIFU in children with relapsed/refractory primary or metastatic solid tumors in bone or soft tissue in close proximity to bone. | - Relapsed/refractory solid tumors including desmoid tumors
- Target lesion(s) must be located in bone or soft tissue in close proximity to bone
- Ages ≤ 30 years
| Children's National Hospital | AeRang Kim, M.D. | Solid tumors, desmoid tumors |
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 |
ADVL1414: A Phase 1 Study of Selinexor (KPT-330, IND #125052), a Selective XPO1 Inhibitor, in Recurrent and Refractory Pediatric Solid Tumors, including CNS Tumors
NCT02323880 | This phase 1 trial of selinexor in relapsed or refractory pediatric solid tumors or high-grade gliomas. | - Part A: Recurrent/refractory solid tumors, including lymphoma and CNS tumors
- Part B: Recurrent/refractory high grade glioma (WHO Grade III/IV) including disseminated tumors (excluding diffuse intrinsic pontine glioma), not requiring surgical resection
- Part C: Patients with recurrent/refractory high grade glioma (WHO Grade III/IV) and requiring surgicalresection (excluding diffuse intrinsic pontine glioma and disseminated tumors)
- Ages ≥ 12 months old to ≤ 21 years old
| Children's Oncology Group | Jeffrey Dome, M.D., Ph.D. | High grade glioma, all solid tumors, all CNS, lymphoma |
ADVL1514: A Phase 1 Study of ABI-009 (nab-rapamycin) in Pediatric Patients with Recurrent or Refractory Solid Tumors, including CNS Tumors as a Single Agent and in Combination with Temozolomide and Irinotecan
NCT02975882 | This phase 1 of the nanoparticle albumin-bound rapamycin when given together with temozolomide and irinotecan in treating pediatric patients with refractory or relapsed solid tumors | - Recurrent/refractory solid tumors, including CNS tumors
- Ages ≥ 12 months old to ≤ 21 years old
| Children's Oncology Group | AeRang Kim, M.D. | All CNS, all solid tumors |
ADVL1615: A Phase 1 Study of Pevonedistat, a NEDD8 Activating Enzyme Inhibitor, in Combination with Temozolomide and Irinotecan in Pediatric Patients with Recurrent or Refractory Solid Tumors
NCT03323034 | This is a phase 1 dose escalation of pevonedistat as a single agent and in combination with irinotecan and temozolomide for pediatric patients with refractory or recurrent solid tumors, including CNS tumors and lymphoma. | - Open cohort A2
- Recurrent/refractory solid tumors, including CNS tumors and lymphoma, for which no standard therapy is available are eligible
- Ages ≥ 6 months and <12 months
| Children's Oncology Group | AeRang Kim, M.D. | All CNS, all solid tumor, lymphoma |
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 |