ONITT, A Randomized Phase I/II Study of Onivyde in Combination with Talazoparib or Temozolomide in Children and Young Adults with Recurrent Solid Malignancies and Ewing Sarcoma
NCT04901702 | A phase I/II study which will evaluate two treatment regimens; nanoliposomal irinotecan (nal-IRN, onivyde) plus talazoparib (TAL) and onivyde (ONI) plus temozolomide (TMZ) A dose finding phase I study will be open to patients with recurrent or refractory solid tumors. In the phase II study, patients with RR Ewing sarcoma will be randomized to receive either ONI plus TAL or ONI plus TMZ. | - Phase 1: refractory or recurrent non-central nervous system tumors
- Phase 2: Refractory or recurrent Ewing sarcoma
- Age: > 12 months and < 30 years
| St. Jude Children's Research Hospital | AeRang Kim, M.D. | Solid tumors |
PEPN22P1: A Pharmacokinetic Study of Vincristine in Infants Dosed According to BSA-Banded Infant Dosing Tables and Older Children Dosed by Traditional BSA Methods
NCT05359237 | A correlative study of vincristine pharmacokinetics to evaluate a new infant dosing method and determine whether it results in uniform drug exposure (AUC) across the age groups. | - Age: ≤ 12 years
- Newly diagnosed and relapsed cancer diagnosis that is being treated with vincristine at the 1.5 mg/m2 dose level
| Children's Oncology Group | AeRang Kim, M.D. | Any |
PEPN2121: A Phase 1/2 Study of Tiragolumab and Atezolizumab in Patients with Relapsed or Refractory SMARCB1 or SMARCA4 Deficient Tumors
NCT05286801 | A Phase 1/2 study to evaluate the safety of tiragolumab as monotherapy in pediatric patients (<18 years) with SMARCB1 or SMARCA4 deficient tumors (Part A), to evaluate antitumor activity of the combination of tiragolumab and atezolizumab in patients with SMARCB1 or SMARCA4 deficient tumors (Part B), and to evaluate the safety and adverse event profile of this combination therapy in subjects with SMARCB1 or SMARCA4 deficient tumors, with a particular focus in pediatric patients < 12 years of age. | - Patients must have SMARCB1 (INI1) or SMARCA4 deficient tumors
- Part A age: ≥ 12 years and < 18 years
- Part B age: ≥ 18 years
| Children's Oncology Group | AeRang Kim, M.D. | Renal medullary carcinoma, malignant rhabdoid tumor (extra-CNS), atypical teratoid rhabdoid tumor (CNS), poorly differentiated chordoma, epithelioid sarcoma, other SMARCB1 or SMARCA4 deficient tumors |
A Pilot 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
NCT04791228 | This is a pilot trial of LTLD combined with MR-HIFU hyperthermia followed by ablation therapy in subjects with relapsed/refractory solid tumors. | - Relapsed/refractory solid tumors
- Target lesion(s) must be located at sites accessible to MR-HIFU.
- Age: ≥ 12 years
| Children's National Hospital | AeRang Kim, M.D. | Solid tumors |
PEPN2111: A Phase 1/2 Trial of CBL0137 (NSC# 825802, IND# 155843) in Patients with Relapsed or Refractory Solid Tumors including CNS Tumors and Lymphoma
NCT04870944 | This phase I/II trial evaluates the best dose, side effects and possible benefit of CBL0137 in treating patients with relapsed, or refractory solid tumors, including central nervous system (CNS) tumors or lymphoma. | - Parts A and B1: ≥ 12 months and ≤ 21 years old
- Part B2 (relapsed/refractory osteosarcoma): ≥ 12 months and ≤ 30 years old
| Children's Oncology Group | AeRang Kim, M.D. | Solid tumors, lymphoma |
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
NCT05302921 | 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 and at least 2 sites of measurable disease
| Children's National Hospital | Marie Nelson, M.D. | Ewing sarcoma, osteosarcoma, rhabdomyosarcoma, and 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 |
Pivotal/Phase II Clinical Trial of Magnetic Resonance-Guided Focused Ultrasound (MR-HIFU) Treatment of Painful Osteoid Osteoma in Children and Young Adults
NCT04658771 | 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 |
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 |
VAL-10-001: Pilot Pharmacokinetic Study of VAL-413 (Orotecan®) in Patients with Recurrent Pediatric Solid Tumors
NCT04337177 | An open-label Phase I clinical trial designed to establish the recommended Phase II dose and to characterize the pharmacokinetic profile of VAL-413 when combined with temozolomide | - Recurrent Pediatric Solid Tumors
- ≥ 1 year of age or ≤ 30 years of age
| Valent Technologies LLC | AeRang Kim, M.D. | Solid tumors, including CNS tumors |
JZP712-101: A Phase 1/2 Study of Lurbinectedin Monotherapy in Pediatric and Young Adult Participants With Relapsed/Refractory Ewing Sarcoma
NCT05734066 | A Phase 1/2, open-label study to evaluate the safety, tolerability, pharmacokinetics (PK), recommended phase 2 dose (RP2D), and efficacy of lurbinectedin monotherapy in pediatric participants with previously treated solid tumors followed by expansion to assess efficacy and safety in pediatric and young adult participants with relapsed/refractory Ewing sarcoma
| - Phase 1 Part 1: Recurrent/refractory solid tumors
- Phase 1 Part 2 and Phase 2: recurrent/refractory Ewing sarcoma
- Phase 1 Part 1: participants must be ≥ 2 to < 18 years of age.
- Phase 1 Part 2: participants must be ≥ 2 to ≤ 30 years of age.
Phase 2: participants must be ≥ 2 to ≤ 30 years of age.
| Jazz Pharmaceuticals | AeRang Kim, M.D. | Solid tumors |
J20112: Neoadjuvant Nivolumab Plus Ipilimumab for Newly Diagnosed Malignant Peripheral Nerve Sheath Tumor
NCT04465643 | An open label study to evaluate the safety and feasibility of neoadjuvant Nivolumab plus Ipilimumab prior to conventional therapy (surgery, chemotherapy, radiation therapy or a combination of these) in patients with Neurofibromatosis Type 1 (NF1) and newly diagnosed pre-malignant and malignant peripheral nerve sheath tumors (MPNST) for whom surgery for resection of tumor is indicated | - Patients must have histologically confirmed diagnosis of atypical neurofibromatous neoplasms of uncertain biologic potential (ANNUBP), low grade MPNST or high grade MPNST
- ≥ 12 years of age
| Johns Hopkins University School of Medicine | AeRang Kim, M.D. | ANNUBP, low grade MPNST or high grade MPNST |