T2016-002: A Phase I/II Study of Nivolumab in Combination with 5-azacytidine in pediatric patients with relapsed/refractory acute myeloid leukemia
NCT03825367 | 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. | - Ages ≥ 1 and ≤ 30 years old
- Relapsed or refractory AML, CNS negative
| Therapeutic Advances in Childhood Leukemia and Lymphoma | Reuven Schore, M.D. | Acute myeloid leukemia |
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 osteosarcoma cohort)
- Part 1: recurrent or refractory solid tumors or lymphoma, that have a known or expected
| Hutchison MediPharma Limited | AeRang Kim, M.D. | Lymphoma, osteosarcoma, Ewing sarcoma, rhabdomyosarcoma, non-rhabdomyosarcoma (NRSTS), other solid tumors |
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., Ph.D. | Ewing sarcoma, alveolar rhabdomyosarcoma with PAX-FOXO1 fusion, non-CNS primary solid tumor including lymphoma with inactivation of DDR genes |
AALL1721: A Phase 2 trial of Tisagenlecleucel in First-Line High-Risk (HR) Pediatric and Young Adult Patients with B-cell Acute Lymphoblastic Leukemia (B-ALL) who are Minimal Residual Disease (MRD) Positive at the End of Consolidation (EOC) therapy.
NCT03876769 | A phase 2 trial to evaluate the efficacy of tisagenlecleucel in first-line high-risk (HR) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (B-ALL) who are minimal residual disease (MRD) positive at the end of consolidation (EOC) therapy. | - Relapsed/refractory CD19 expressing B-Cell acute lymphoblastic leukemia
- MRD positive at EOC therapy
- Ages 1 years to 25 years
| Novartis Pharmaceuticals | Reuven Schore, M.D. | B-Cell Acute Lymphoblastic Leukemia |
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 I- Recurrent/refractory solid tumors or lymphoma
- Ages 6 months to ≤ 21 years old
Phase II- Recurrent/refractory Ewing sarcoma, rhabdomyosarcoma, osteosarcoma, or neuroblastoma
- Ages 6 months old to ≤21 years old
| Bayer Pharmaceuticals | AeRang Kim, M.D., Ph.D. | Ewing sarcoma, rhabdomyosarcoma, osteosarcoma, neuroblastoma, lymphoma, solid tumors |
ADVL1822: A Phase 1/2, Multi-Center, Dose-Escalating Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Quizartinib Administered in Combination with Re-Induction Chemotherapy, and as a Single- Agent Continuation Therapy, in Pediatric Relapsed/ Refractory AML Subjects Aged 1 Month to <18 Years (And Young Adults Aged Up to 21 Years) With FLT3-ITD Mutations
NCT03793478 | This is a Phase 1/2 study of Quizartinib, administered in combination with fludarabine and cytarabine (FLA) (Re-Induction Cycle 1) and FLA (Re-Induction Cycle 2) chemotherapy for re-induction, and as a single agent continuation therapy. It is being investigated as a treatment for relapsed or refractory AML in pediatric subjects aged ≥1 month to <18 years (and young adults aged up to 21 years) old with FLT3-internal tandem duplication (ITD) mutations following failure of front-line intensive chemotherapy. | - Diagnosis of acute myeloid leukemia with FLT3-ITD mutation
- 1st recurrence/relapse of acute myeloid leukemia
- Ages 1 months to ≤ 21 years
| Daiichi Sankyo, Inc. | AeRang Kim, M.D., Ph.D. | Acute Myeloid Leukemia |
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 |
BIANCA: A Phase II, single arm, multicenter open label trial to determine the safety and efficacy of tisagenlecleucel in pediatric subjects with relapsed or refractory mature B-cell non-Hodgkin lymphoma (NHL)
NCT03610724 | A Phase II trial of tisagenlecleucel in pediatric subjects with relapsed or refractory mature B-cell non-Hodgkin lymphoma (BIANCA study). | - Relapsed/refractory B-cell non-Hodgkin lymphoma
- Ages ≤ 25 years old
| Novartis Pharmaceuticals | Hema Dave, M.D., Ph.D. | B-cell non-Hodgkin lymphoma |
T2012-002, A Pilot Study of Vincristine Sulfate Liposome Injection (Marqibo®) in Combination with Chemotherapy for Children, Adolescents, and Young Adults with Relapse of Acute Lymphoblastic Leukemia
NCT02879643 | This is a pilot study utilizing Marqibo® (vincristine sulfate liposome injection) combined with dexamethasone, mitoxantrone and asparaginase for relapsed acute lymphoblastic leukemia. | - Relapsed/refractory B-Cell acute lymphoblastic leukemia , T-Cell B-Cell acute lymphoblastic leukemia, mixed phenotypic acute leukemia, lymphoblastic lymphoma
- Ages ≥1 year old to ≤21 years old
| Therapeutic Advances in Childhood Leukemia Consortium | Reuven Schore, M.D. | B-Cell acute lymphoblastic leukemia T-Cell B-Cell acute lymphoblastic leukemia, mixed phenotypic acute leukemia, lymphoblastic lymphoma |
| T2017-002, A TACL Phase 1/2 Study of PO Ixazomib in Combination with Chemotherapy for Childhood Relapsed or Refractory Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma NCT03817320 | Phase 1/2 study of Ixazomib administered in conjunction with re-induction chemotherapy. | - Relapsed/ refractory acute lymphoblastic leukemia or lymphoblastic lymphoma
- Ages ≤21 years old but will be restricted to <18 years old for the first 9 patients enrolled on each phase
| Therapeutic Advances in Childhood Leukemia Consortium | Reuven Schore, M.D. | Acute lymphoblastic leukemia, lymphoblastic lymphoma |
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 with an NTRK1, NTRK2, or NTRK3 fusion
- Cohort B: histologic diagnosis of any solid tumor other than infantile fibrosarcoma, 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., Ph.D. | 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 |
ADVL1521: A Phase 2 Study of the MEK inhibitor Trametinib (IND #119346, NSC# 763093) in Children with Relapsed or Refractory Juvenile Myelomonocytic Leukemia (JMML)
NCT03190915 | This is a Phase 2 study of the MEK inhibitor Trametinib in children with relapsed or refractory juvenile myelomonocytic leukemia. | - Diagnosis of juvenile myelomonocytic leukemia
- Ages ≥ 1 month to < 22 years old
| Children's Oncology Group | AeRang Kim, M.D., Ph.D. | Juvenile myelomonocytic leukemia |
PEPN1812: A Phase 1 Trial of the CD123 X CD3 DART® Molecule Flotetuzumab (NSC#808294, IND#145986) in Children, Adolescents, and Young Adults with Relapsed or Refractory Acute Myeloid Leukemia
NCT04158739 | Phase 1 study of flotetuzumab, a CD123 x CD3 bispecific DART molecule, in pediatric patients with relapsed/refractory AML. | - Recurrent/refractory acute myeloid leukemia
- Patients must weigh ≥ 17 kg
- Ages < 21 years of age
| Children's Oncology Group | AeRang Kim, M.D., Ph.D. | Acute myeloid leukemia |
ADVL18P1: An Open-Label Feasibility Study to Assess the Safety and Pharmacokinetics of Enasidenib in Pediatric Patients with Relapsed/Refractory Acute Myeloid Leukemia R/R-AML with an Isocitrate Dehydrogenase-2 IDH2 Mutation
NCT04203316 | This trial studies enasidenib for patients with relapsed or refractory AML with a mutation in a protein called IDH2 | - Acute myeloid leukemia with an IDH2 mutation identified from a peripheral blood or bone marrow sample at the time of diagnosis and/or relapsed/refractory disease.
- Ages ≥ 24 months to ≤ 18 years old
| Children's Oncology Group | AeRang Kim, M.D., Ph.D. | Acute myeloid leukemia |
AALL1931: An Open Label, Multicenter Study of RC-P in Patients with Acute Lymphoblastic Leukemia ALL/Lymphoblastic Lymphoma LBL Following Hypersensitivity to E. coli-derived Asparaginases
NCT04145531 | This is a pivitol phase 2/3 study to determine the efficacy, safety and tolerability of RC-P, a novel asparaginase agent, in patients who are hypersensitive to E. coli derived asparaginases. | - Pediatric or adult patients diagnosed with B-cell acute lymphoblastic leukemia or acute lymphoblastic leukemia/lymphoma
- Patients that have had an allergic reaction to long acting E. coli-derived asparaginase or have a silent inactivation
| Jazz Pharmaceuticals | Reuven Schore, M.D. | Acute lymphoblastic leukemia, acute lymphoblastic lymphoma |
ANHL12P1: Brentuximab Vedotin or Crizotinib and Combination Chemotherapy in Treating Patients With Newly Diagnosed Stage II-IV Anaplastic Large Cell Lymphoma
NCT01979536 | This partially randomized phase II trial studies how well Brentuximab Vedotin or Crizotinib, and combination chemotherapy works in treating patients with newly diagnosed stage II-IV anaplastic large cell lymphoma. | - Newly diagnosed histologically proven anaplastic large cell lymphoma
- Stage II, III, or IV disease
- Ages < 22 years
| Children's Oncology Group | Hema Dave, M.D., Ph.D. | Anaplastic large cell lymphoma (ALK-positive), Ann Arbor stage II - IV noncutaneous childhood anaplastic large cell lymphoma, CD30-positive neoplastic cells present |
ANHL1522: Rituximab and LMP-Specific T-Cells in Treating Pediatric Solid Organ Recipients With EBV-Positive Cluster of Differentiation(CD) 20-Positive Post-Transplant Lymphoproliferative Disorder
NCT02900976 | This pilot clinical trial studies how rituximab and latent membrane protein (LMP)-specific T-cells work in treating pediatric solid organ recipients with Epstein-Barr virus-positive, cluster of differentiation (CD)20-positive post-transplant lymphoproliferative disorder. | - History of solid organ transplantation
- Newly diagnosed, relapsed/refractory polymorphic or monomorphic post-transplant lymphoproliferative disorder
- Ages < 30 years
| Children's Oncology Group | Hema Dave, M.D., Ph.D. | Epstein-Barr virus-related post-transplant lymphoproliferative disorder, monomorphic post-transplant lymphoproliferative disorder, polymorphic post-transplant lymphoproliferative disorder |