Table 2. Pediatric immunotherapy clinical trials involving the established immunotherapeutic targets that are currently active/recruiting in the US since 2016.
B-ALL: B-cell Acute lymphoblastic leukemia; HL: Hodgkin lymphoma; NHL: Non- Hodgkin lymphoma; CLL: Chronic lymphocytic leukemia; DIPG: Diffuse Intrinsic Pontine Glioma
| Identifier | Study name | Target | Phase | Disease (s) | Intervention (s) |
|---|---|---|---|---|---|
| NCT04544592 | UCD19 CarT in Treatment of Pediatric B-ALL and B-NHL | CD19 | ½ | B-cell Acute Lymphoblastic Leukemia B-cell Non Hodgkin Lymphoma |
CD19CAR-CD3Zeta-4-1BB-Expressing Allogenic TLymphocyte Cells |
| NCT03186118 | Pilot Study of T-APCs Following CAR T Cell Immunotherapy for CD19+ Leukemia | CD19 | 1 | CD19+ Acute Leukemia | T-cell Antigen Presenting Cells expressing truncated CD19 (T-APC) |
| NCT04276870 | Phase 2 Trial of CD19 Redirected Autologous CAR T Cells (CART19) for Orphan Indications of Pediatric B Cell Acute Lymphoblastic Leukemia (B ALL) | CD19 | 2 | Hypodiploid or t(17;19) B-ALL Infants With Very High Risk KMT2A B-ALL Patients With Central Nervous System Relapse |
Murine CART19 |
| NCT03792633 | Study of huCART19 for Very High-Risk (VHR) Subsets of Pediatric B-ALL | CD19 | 2 | B-ALL | huCART19 |
| NCT03684889 | CD19-specific CAR T Cells With a Fully Human Binding Domain for CD19+ Leukemia or Lymphoma | CD19 | 1/2 | B-cell Leukemia B-cell Lymphoma |
SCRI-huCAR19v1 SCRI-huCAR19v2 |
| NCT03573700 | Evaluation of CD19-Specific CAR Engineered Autologous T-Cells for Treatment of Relapsed/Refractory CD19+ Acute Lymphoblastic Leukemia | CD19 | 1/2 | B-relapsed/refractory ALL | Cyclophosphamide Fludarabine; Mesna CD19- specific CAR T-cells (SJCAR19 product) |
| NCT03467256 | CD19 T-CAR for Treatment of Children and Young Adults With r/r B-ALL | CD19 | 1/2 | B-ALL | CD19 CAR T-Cell Fludarabine Cyclophosphamide Tocilizumab |
| NCT03774654 | CD19.CAR Allogeneic NKT for Patients With Relapsed or Refractory B-Cell Malignancies (ANCHOR) | CD19 | 1 | B-cell NHL CLL |
CD19.CAR-aNKT cells Cyclophosphamide Fludarabine |
| Identifier | Study name | Target | Phase | Disease (s) | Intervention (s) |
| NCT03016377 | Administration of Autologous CAR-T CD19 Antigen With Inducible Safety Switch in Patients With Relapsed/Refractory ALL | CD19 | 1/2 | B-ALL | iC9-CAR19 cells Rimiducid Cyclophosphamide Fludarabine |
| NCT03768310 | CD19.CAR-multiVSTs for Patients With CD19+ B-ALL or NHL Undergoing Related Allogeneic HSCT | CD19 | 1 | B-ALL B-cell NHL |
CD19.CAR-multiVST |
| NCT03056339 | Umbilical & Cord Blood (CB) Derived CAR-Engineered NK Cells for B Lymphoid Malignancies | CD19 | 1/2 | B-ALL B-cell NHL |
Fludarabine; Cyclophosphamide Mesna iC9/CAR.19/IL15-Transduced CB-NK Cells AP1903 |
| NCT03743246 | A Study to Evaluate the Safety and Efficacy of JCAR017 in Pediatric Subjects With Relapsed/Refractory (r/r) B-cell Acute Lymphoblastic Leukemia (B-ALL) and B-cell Non-Hodgkin Lymphoma (B-NHL) | CD19 | 1/2 | B-ALL B-cell NHL |
JCAR017 Fludarabine Cyclophosphamide |
| NCT04196413 | GD2 CAR T Cells in Diffuse Intrinsic Pontine Gliomas(DIPG) & Spinal Diffuse Midline Glioma(DMG) | GD2 | 1 | Glioma of Spinal Cord Glioma of Brainstem | GD2 CAR T cells Fludarabine Cyclophosphamide |
| NCT03721068 | Study of CAR T-Cells Targeting the GD2 With IL-15+iCaspase9 for Relapsed/Refractory Neuroblastoma | GD2 | 1 | Neuroblastoma | iC9.GD2.CAR.IL-15 T-cells Cyclophosphamide Fludarabine |
| NCT04099797 | C7R-GD2.CAR T Cells for Patients With GD2-expressing Brain Tumors (GAIL-B) | GD2 | 1 | DIPG High Grade Glioma |
(C7R)-GD2.CART cells Cyclophosphamide Fludarabine |
| NCT03635632 | C7R-GD2.CART Cells for Patients With Relapsed or Refractory Neuroblastoma and Other GD2 Positive Cancers (GAIL-N) | GD2 | 1 | Neuroblastoma Osteosarcoma Ewing Sarcoma Rhabdomyosarcoma |
(C7R)-GD2.CART cells Cyclophosphamide Fludarabine |
| NCT03294954 | GD2 Specific CAR and Interleukin-15 Expressing Autologous NKT Cells to Treat Children With Neuroblastoma (GINAKIT2) | GD2 | 1 | Neuroblastoma | GD2-CAR NKT cells Cyclophosphamide Fludarabine |
| NCT03860207 | Study of the Safety and Efficacy of Humanized 3F8 Bispecific Antibody (Hu3F8-BsAb) in Patients With Relapsed/Refractory Neuroblastoma, Osteosarcoma and Other Solid Tumors | GD2 | 1/2 | Neuroblastoma Osteosarcoma Other Solid Tumor Cancers |
Humanized 3F8 Bispecific Antibody |
| Identifier | Study name | Target | Phase | Disease (s) | Intervention (s) |
| NCT03363373 | Naxitamab for High-Risk Neuroblastoma Patients With Primary Refractory Disease or Incomplete Response to Salvage Treatment in Bone and/or Bone Marrow | GD2 | 2 | Neuroblastoma | GM-CSF + Naxitamab |
| NCT03690869 | REGN2810 in Pediatric Patients With Relapsed, Refractory Solid, or Central Nervous System (CNS) Tumors and Safety and Efficacy of REGN2810 in Combination With Radiotherapy in Pediatric Patients With Newly Diagnosed or Recurrent Glioma | PD-1 | 1/2 | Relapsed/refractory Solid Tumor Central Nervous System Tumor DIPG High Grade Glioma |
REGN2810 Radiation: Conventional or hypofractionated Re-irradiation |
| NCT03843294 | Tumor Associated Antigen Specific T Cells (TAA-T) With PD1 Inhibitor for Lymphoma | PD-1 | 1 | Hodgkin Lymphoma Diffuse Large B Cell Lymphoma |
TAA-T cells Nivolumab |
| NCT03407144 | Safety and Efficacy of Pembrolizumab (MK-3475) in Children and Young Adults With Classical Hodgkin Lymphoma | PD-1 | 2 | HL | Pembrolizumab Chemotherapy drugs Radiotherapy |
| NCT02992964 | Pilot Study of Nivolumab in Pediatric Patients With Hypermutant Cancers | PD-1 | 1/2 | Refractory or Recurrent Hypermutated Malignancies Biallelic Mismatch Repair Deficiency (bMMRD) Patients |
Nivolumab |
| NCT02914405 | Phase I Study of 131-I mIBG Followed by Nivolumab & Dinutuximab Beta Antibodies in Children With Relapsed/Refractory Neuroblastoma (MiniVan) | PD-1/GD2 | 1 | Neuroblastoma | Nivolumab Ch14.18/CHO |
| NCT03605589 | Pembro + Blina Combination in Pediatric and Young Adult Patients With Relapsed/Refractory Acute Leukemia or Lymphoma | CD19/PD-1 | 1 | B-cell Leukemia B-cell Lymphoma |
Blinatumomab Pembrolizumab |
| NCT03769467 | Tabelecleucel in Combination With Pembrolizumab in Subjects With Epstein-Barr Virus-associated Nasopharyngeal Carcinoma (EBV+ NPC) | CD19/PD-1 | 1/2 | Epstein-Barr Virus Infections Epstein-Barr Viraemia Epstein-Barr Virus-associated Nasopharyngeal Carcinoma |
tabelecleucel pembrolizumab |
| NCT02879695 | Blinatumomab and Nivolumab With or Without Ipilimumab in Treating Patients With Relapsed or Refractory CD19+ Precursor B-Lymphoblastic Leukemia | CD19/PD-1/CTLA-4 | 1 | B-ALL | Blinatumomab Ipilimumab Nivolumab |
| Identifier | Study name | Target | Phase | Disease (s) | Intervention (s) |
| NCT04465643 | Neoadjuvant Nivolumab Plus Ipilimumab for Newly Diagnosed Malignant Peripheral Nerve Sheath Tumor | PD-1/CTLA-4 | 1 | Nerve Sheath Tumors | Nivolumab Ipilimumab |
| NCT03837899 | Durvalumab and Tremelimumab for Pediatric Malignancies | PD-1/CTLA-4 | 1 | Pediatric Cancer Solid Tumor Pediatric Hematological Malignancies |
Durvalumab / Tremelimumab Combination Therapy |
| NCT04323046 | Immunotherapy (Nivolumab and Ipilimumab) Before and After Surgery for the Treatment of Recurrent or Progressive High Grade Glioma in Children | PD-1/CTLA-4 | 1 | Glioblastoma Malignant Glioma Grade III Glioma |
Ipilimumab Nivolumab |