Table 1.
Current open clinical trials for refractory and relapsed neuroblastoma.
| NCT Number | Title | Drug | Molecular Target | Therapeutic Class | Study Enrollment Target | Eligibility Age Range | Primary Site/Group or Sponsor | Status |
|---|---|---|---|---|---|---|---|---|
| NCT00089245 | Radiolabeled Monoclonal Antibody Therapy in Treating Patients with Refractory, Recurrent, or Advanced CNS or Leptomeningeal Cancer | Intrathecal 131I-8H9 | B7-H3 | Systemic Radiotherapy | 120 | any | New York, NY (Memorial Sloan Kettering Cancer Center) | Active, recruiting |
| NCT00107289 | Iodine 131I Metaiodobenzylguanidine in Treating Patients with Recurrent, Progressive, or Refractory Neuroblastoma or Malignant Pheochromocytoma or Paraganglioma | 131I-MIBG | NET | Systemic Radiotherapy | 80 | over 1 year | New York, NY (Memorial Sloan Kettering Cancer Center) | Active, recruiting |
| NCT00601003 | Study of Nifurtimox to Treat Refractory or Relapsed Neuroblastoma or Medulloblastoma | Nifurtimox, cyclophosphamide, topotecan | Reactive Oxygen Species | Systemic Chemotherapy | 100 | up to 21 years | Beat Childhood Cancer (BCC) | Active, recruiting |
| NCT00638898 | Busulfan, Melphalan, Topotecan Hydrochloride, and a Stem Cell Transplant in Treating Patients with Newly Diagnosed or Relapsed Solid Tumor | Busulfan, melphalan, topotecan | N/A | Systemic Chemotherapy | 25 | 6 months to 40 years | Duarte, CA (City of Hope Cancer Center) | Active, not recruiting |
| NCT00788125 | Dasatinib, Ifosfamide, Carboplatin, and Etoposide in Treating Young Patients with Metastatic or Recurrent Malignant Solid Tumors | Carboplatin, dasatinib, etoposide, ifosfamide | Multiple Kinases (Abl, Src, c-Kit, PDGFRβ) | Molecularly Targeted Therapy | 143 | 1–25 years | Duarte, CA (City of Hope Cancer Center) | Active, not recruiting |
| NCT00877110 | Anti-GD2 3F8 Antibody and Allogeneic Natural Killer Cells for High-Risk Neuroblastoma | Cyclophosphamide, vincristine, topotecan, NK cells, 3F8 | GD2 | Systemic Chemotherapy plus Immunotherapy | 71 | any | New York, NY (Memorial Sloan Kettering Cancer Center) | Active, not recruiting |
| NCT00911560 | Bivalent Vaccine with Escalating Doses of the Immunological Adjuvant OPT-821, in Combination with Oral β-glucan for High-Risk Neuroblastoma | Adjuvant OPT-821 in a vaccine containing two antigens (GD2L and GD3L) covalently linked to KLH | GD2L, GD3L | Immunotherapy | 215 | up to 21 years | New York, NY (Memorial Sloan Kettering Cancer Center) | Active, recruiting |
| NCT01183884 | 3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic Acid for Consolidation of Second or Greater Remission of High-Risk Neuroblastoma | 3F8, GM-CSF, isotretinoin | GD2 | Immunotherapy | 63 | over 18 months | New York, NY (Memorial Sloan Kettering Cancer Center) | Active, not recruiting |
| NCT01183897 | 3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic Acid for Primary Refractory Neuroblastoma in Bone Marrow | 3F8, GM-CSF, isotretinoin | GD2 | Immunotherapy | 31 | over 18 months | New York, NY (Memorial Sloan Kettering Cancer Center) | Active, not recruiting |
| NCT01331135 | Aflac ST0901 CHOANOME—Sirolimus in Solid Tumors (Aflac ST0901) | Sirolimus | mTOR | Molecularly Targeted Therapy | 24 | up to 30 years | Atlanta, GA (Children’s Healthcare of Atlanta) | Active, not recruiting |
| NCT01419834 | Humanized 3F8 Monoclonal Antibody (Hu3F8) in Patients with High-Risk Neuroblastoma and GD2-Positive Tumors | Hu3F8 | GD2 | Immunotherapy | 74 | over 2 years | New York, NY (Memorial Sloan Kettering Cancer Center) | Active, recruiting |
| NCT01467986 | Multimodal Molecular Targeted Therapy to Treat Relapsed or Refractory High-Risk Neuroblastoma | Dasatinib, sirolimus, irinotecan, temozolomide | Multiple Kinases (Abl, Src, c-Kit, PDGFRβ), mTOR | Molecularly Targeted Therapy | 114 | up to 25 years | Regensburg, Germany (University Hospital Regensburg) | Active, recruiting |
| NCT01492673 | Cyclophosphamide, Topotecan, and Bevacizumab (CTB) in Patients with Relapsed/Refractory Ewing’s Sarcoma and Neuroblastoma | Cyclophosphamide, topotecan, bevacizumab | Angiogenesis | Molecularly Targeted Therapy | 9 | up to 21 years | New York, NY (Memorial Sloan Kettering Cancer Center) | Active, not recruiting |
| NCT01576692 | Combination Chemotherapy, Monoclonal Antibody, and Natural Killer Cells in Treating Young Patients with Recurrent or Refractory Neuroblastoma | Cyclophosphamide, topotecan, Hu14.18K322A, cisplatin, etoposide, doxorubicin, Vincristine, busulfan, melphalan, natural killer cells, GM-CSF, interleukin-2 | GD2 | Systemic Chemotherapy plus Immunotherapy | 34 | up to 21 years | Memphis, TN (St. Jude Children’s Research Hospital) | Active, not recruiting |
| NCT01601535 | Study of MLN8237 in Combination with Irinotecan and Temozolomide | Alisertib (MLN8237), irinotecan, temozolomide | Aurora A Kinase | Molecularly Targeted Therapy | 4 | 1–30 years | New Approaches to Neuroblastoma Therapy (NANT) | Active, not recruiting |
| NCT01606878 | Crizotinib and Combination Chemotherapy in Treating Younger Patients with Relapsed or Refractory Solid Tumors or Anaplastic Large Cell Lymphoma | Crizotinib, cyclophosphamide, topotecan, vincristine, doxorubicin | ALK, c-Met | Systemic Chemotherapy plus Molecularly Guided Therapy | 65 | 1–21 years | Children’s Oncology Group | Active, not recruiting |
| NCT01625351 | A Study of CD45RA+ Depleted Haploidentical Stem Cell Transplantation in Children with Relapsed or Refractory Solid Tumors and Lymphomas | Alemtuzumab, fludarabine, sirolimus, busulfan, melphalan | mTOR | Systemic Chemotherapy plus Immunotherapy | 23 | 2–21 years | Memphis, TN (St. Jude Children’s Research Hospital) | Active, not recruiting |
| NCT01662804 | Humanized 3F8 Monoclonal Antibody (Hu3F8) When Combined with Interleukin-2 in Patients with High-Risk Neuroblastoma and GD2-positive Solid Tumors | Hu3F8, IL-2 | GD2 | Immunotherapy | 14 | over 13 months | New York, NY (Memorial Sloan Kettering Cancer Center) | Active, not recruiting |
| NCT01711554 | Lenalidomide and Dinutuximab With or Without Isotretinoin in Treating Younger Patients with Refractory or Recurrent Neuroblastoma | Lenalidomide, dinutuximab, isotretinoin | GD2 | Immunotherapy | 62 | up to 21 years | New Approaches to Neuroblastoma Therapy (NANT) | Active, recruiting |
| NCT01742286 | Phase I Study of LDK378 in Pediatric, Malignancies with a Genetic Alteration in Anaplastic Lymphoma Kinase (ALK) | Ceritinib (LDK378) | ALK | Molecularly Guided Therapy | 83 | 1–17 years | Novartis Pharmaceuticals | Active, not recruiting |
| NCT01757626 | Combination Therapy of Antibody Hu3F8 With Granulocyte- Macrophage Colony Stimulating Factor (GM-CSF) in Patients with Relapsed/Refractory High-Risk Neuroblastoma | Hu3F8 with GM-CSF | GD2 | Immunotherapy | 224 | any | New York, NY (Memorial Sloan Kettering Cancer Center) | Active, recruiting |
| NCT01804634 | A Phase II Trial of Reduced Intensity Conditioning and Haploidentical BMT for High-risk Solid Tumors | Cyclophosphamide, fludarabine, XRT | N/A | Systemic Chemotherapy plus Immunotherapy | 20 | up to 40 years | Baltimore, MD (Sidney Kimmel Cancer Center at Johns Hopkins) | Active, recruiting |
| NCT01956669 | A Phase II Study of Pazopanib GW786034, NSC# 737754 in Children, Adolescents, and Young Adults with Refractory Solid Tumors | Pazopanib | Multiple Kinases (VEGFR-1, VEGFR-2, VEGFR-3, PDGFRβ, c-kit) | Molecularly Targeted Therapy | 154 | 1–18 years | Children’s Oncology Group | Active, recruiting |
| NCT02013336 | Phase 1 Study of MM-398 Plus Cyclophosphamide in Pediatric Solid Tumors | MM-398 (Irinotecan sucrosofate liposomes), cyclophosphamide | N/A | Systemic chemotherapy | 30 | 1–20 years | South Plains Oncology Consortium | Active, recruiting |
| NCT02030964 | N2012-01: Phase 1 Study of Difluoromethylornithine (DFMO) and Celecoxib with Cyclophosphamide/Topotecan (DFMO) | DFMO, celecoxib, cyclophosphamide, topotecan | ODC | Molecularly Targeted Therapy | 30 | 2–30 years | New Approaches to Neuroblastoma Therapy (NANT) | Active, not recruiting |
| NCT02034981 | Phase 2 Study Assessing Efficacy and Safety of Crizotinib in Patients Harboring an Alteration on ALK, MET or ROS1 | Crizotinib | ALK, c-Met | Molecularly Guided Therapy | 246 | over 1 year | Villejuif, France (Institut Gustave Roussy) | Active, not recruiting |
| NCT02035137 | 131I-MIBG Alone VS. 131I-MIBG With Vincristine and Irinotecan VS. 131I-MIBG With Vorinostat (N2011-01) | 131I-MIBG, vorinostat, vincristine/irinotecan | NET | Systemic radiotherapy plus Chemotherapy | 105 | 2–30 years | New Approaches to Neuroblastoma Therapy (NANT) | Active, recruiting |
| NCT02076906 | MR-guided High Intensity Focused Ultrasound (HIFU) on Pediatric Solid Tumors | MR-HIFU | N/A | N/A | 14 | up to 30 years | Washington, DC (Children’s National Medical Center) | Active, recruiting |
| NCT02095132 | WEE1 Inhibitor MK-1775 and Irinotecan Hydrochloride in Treating Younger Patients with Relapsed or Refractory Solid Tumors | Irinotecan, AZD1775 (adavosertib) | Wee1 | Molecularly Targeted Therapy | 154 | 2–21 years | Children’s Oncology Group | Active, recruiting |
| NCT02100891 | Phase 2 STIR Trial: Haploidentical Transplant and Donor Natural Killer Cells for Solid Tumors (STIR) | NK cells | N/A | Immunotherapy | 20 | any | Milwaukee, WI (Medical College of Wisconsin) | Active, recruiting |
| NCT02124772 | Study to Investigate Safety, Pharmacokinetic (PK), Pharmacodynamic (PD) and Clinical Activity of Trametinib in Subjects with Cancer or Plexiform Neurofibromas and Trametinib in Combination with Dabrafenib in Subjects with Cancers Harboring V600 Mutations | Trametinib, dabrafenib | MEK | Molecularly Guided Therapy | 142 | 1–17 years | Novartis Pharmaceuticals | Active, recruiting |
| NCT02139397 | Study of DFMO in Combination with Bortezomib for Relapsed or Refractory Neuroblastoma | DFMO, bortezomib | ODC, Proteasome | Molecularly Targeted Therapy | 38 | up to 21 years | Beat Childhood Cancer (BCC) | Active, recruiting |
| NCT02162732 | Molecular-Guided Therapy for Childhood Cancer | Precision Medicine | Molecularly Guided Therapy | 200 | 13 months to 21 years | Beat Childhood Cancer (BCC) | Active, recruiting | |
| NCT02163356 | Fenretinide Lym-X-Sorb + Ketoconazole + Vincristine for Recurrent or Resistant Neuroblastoma (SPOC2013-001) | Fenretinide, ketoconazole, vincristine | N/A | Systemic Chemotherapy | 42 | up to 30 years | South Plains Oncology Consortium | Active, recruiting |
| NCT02169609 | Safety Study of Dinutuximab Combined with Immunotherapy to Treat Neuroblastoma | Dinutuximab, GM-CSF, isotretinoin, IL-2 | GD2 | Immunotherapy | 25 | any | Barcelona, Spain (Hospital Sant Joan de Deu) | Active, not recruiting |
| NCT02173093 | Activated T Cells Armed with GD2 Bispecific Antibody in Children and Young Adults with Neuroblastoma and Osteosarcoma | IL-2, GD2Bi-aATC, GM-CSF | GD2 | Immunotherapy | 40 | 13 months to 29 years | Detroit, MI (Children’s Hospital of Michigan) | Active, recruiting |
| NCT02298348 | Sorafenib and Cyclophosphamide/Topotecan in Patients with Relapsed and Refractory Neuroblastoma (N2013-02) | Sorafenib, cyclophosphamide, topotecan | Multiple Kinases (Raf, VEGFR-2, VEGFR-3, c-Kit) | Molecularly Targeted Therapy | 18 | up to 30 years | New Approaches to Neuroblastoma Therapy (NANT) | Active, not recruiting |
| NCT02304458 | Nivolumab With or Without Ipilimumab in Treating Younger Patients with Recurrent or Refractory Solid Tumors or Sarcomas | Ipilimumab, nivolumab | PD-1, CTLA-4 | Immunotherapy | 484 | 1–30 years | Children’s Oncology Group | Active, recruiting |
| NCT02308527 | Activity Study of Bevacizumab with Temozolomide ± Irinotecan for Neuroblastoma in Children | Bevacizumab, temozolomide, irinotecan, topotecan | Angiogenesis | Molecularly Targeted Therapy | 160 | 1–21 years | Cancer Research UK | Active, recruiting |
| NCT02311621 | Engineered Neuroblastoma Cellular Immunotherapy (ENCIT)-01 | Anti-CD171 CAR-T cells | CD171 | Immunotherapy | 40 | 18 months to 26 years | Seattle, WA (Seattle Children’s Hospital) | Active, recruiting |
| NCT02332668 | A Study of Pembrolizumab (MK-3475) in Pediatric Participants with an Advanced Solid Tumor or Lymphoma (MK-3475-051/KEYNOTE-051) | Pembrolizumab | PD-1 | Immunotherapy | 310 | 6 months to 17 years | Merck Sharp & Dohme Corp. | Active, recruiting |
| NCT02343718 | Vinblastine and Temsirolimus in Pediatric Patients with Recurrent or Refractory Lymphoma or Solid Tumours Including CNS Tumours | Temsirolimus, vinblastine | mTOR | Molecularly Targeted Therapy | 7 | 1–18 years | Toronto, ON (Hospital for Sick Children) | Active, not recruiting |
| NCT02378428 | MIBG Therapy for Patients with MIBG Avid Tumors (MIBG) | 131I-MIBG | NET | Systemic Radiotherapy | 65 | 1–40 years | Columbus, OH (Nationwide Children’s Hospital) | Active, recruiting |
| NCT02390843 | Simvastatin with Topotecan and Cyclophosphamide in Relapsed and/or Refractory Pediatric Solid and CNS Tumors (AflacST1402) | Simvastatin, cyclophosphamide, topotecan | HMG-coA Reductase | Systemic Chemotherapy | 36 | 1–29 years | Atlanta, GA (Children’s Healthcare of Atlanta) | Active, recruiting |
| NCT02441088 | Theranostics: 68GaDOTATOC and 90YDOTATOC (PRRT) | 90Y-DOTA-tyr3-Octreotide (90Y-DOTATOC) | SSTR | Systemic Radiotherapy | 25 | over 6 months | Iowa City, IA (University of Iowa) | Active, not recruiting |
| NCT02452554 | Lorvotuzumab Mertansine in Treating Younger Patients with Relapsed or Refractory Wilms Tumor, Rhabdomyosarcoma, Neuroblastoma, Pleuropulmonary Blastoma, Malignant Peripheral Nerve Sheath Tumor, or Synovial Sarcoma | Lorvotuzumab mertansine (IMGN-901) | CD56 | Immunotherapy | 114 | 1–30 years | Children’s Oncology Group | Active, not recruiting |
| NCT02508038 | TCRαβ+/CD19+ Depleted Haploidentical HSCT + Zoledronate | Zoledronate, TCRαβ+/CD19+ depleted Haploidentical HSCT | N/A | Immunotherapy | 21 | 7 months to 21 years | Madison, WI (University of Wisconsin) | Active, recruiting |
| NCT02520713 | The iCat2, GAIN (Genomic Assessment Informs Novel Therapy) Consortium Study | Precision Medicine | Molecularly Guided Therapy | 825 | up to 30 years | Boston, MA (Dana-Farber Cancer Institute) | Active, recruiting | |
| NCT02536183 | A Phase I Study of Lyso-thermosensitive Liposomal Doxorubicin and MR-HIFU for Pediatric Refractory Solid Tumors | MR-HIFU hyperthermia + lyso-thermosensitive liposomal doxorubicin | N/A | Systemic Chemotherapy | 34 | up to 30 years | Washington, DC (Children’s National Medical Center) | Active, recruiting |
| NCT02541604 | A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Immunogenicity, and Preliminary Efficacy of Atezolizumab (Anti-Programmed Death-Ligand 1 [PD-L1] Antibody) in Pediatric and Young Adult Participants with Solid Tumors | Atezolizumab | PD-L1 | Immunotherapy | 90 | up to 30 years | Hoffmann-La Roche | Active, not recruiting |
| NCT02557854 | HIFU Hyperthermia with Liposomal Doxorubicin (DOXIL) for Relapsed or Refractory Pediatric and Young Adult Solid Tumors | Doxil + MR-HIFU Hyperthermia | N/A | Systemic Chemotherapy | 14 | 1–40 years | Dallas, TX (University of Texas Southwestern Medical Center) | Active, recruiting |
| NCT02573896 | Immunotherapy of Relapsed Refractory Neuroblastoma with Expanded NK Cells | NK cells, dinutuximab, lenalidomide | GD2 | Immunotherapy | 24 | 1 month to 30 years | New Approaches to Neuroblastoma Therapy (NANT) | Active, not yet recruiting |
| NCT02574728 | Sirolimus in Combination with Metronomic Chemotherapy in Children with Recurrent and/or Refractory Solid and CNS Tumors | Sirolimus, celecoxib, etoposide, cyclophosphamide | mTOR | Molecularly Targeted Therapy | 60 | 1–30 years | Atlanta, GA (Children’s Healthcare of Atlanta) | Active, recruiting |
| NCT02624388 | Study of Genistein in Pediatric Oncology Patients (UVA-Gen001) (UVA-Gen001) | Genistein | N/A | Systemic Chemotherapy | 50 | 1–21 years | Charlottesville, VA (University of Virginia) | Active, recruiting |
| NCT02630043 | Trial of Tolcapone With Oxaliplatin for Neuroblastoma | Tolcapone, oxaliplatin | N/A | Systemic Chemotherapy | 21 | up to 21 years | Beat Childhood Cancer (BCC) | Active, recruiting |
| NCT02638428 | Genomics-Based Target Therapy for Children with Relapsed or Refractory Malignancy | Ifosfamide, carboplatin, etoposide, axitinib, crizotinib, dasatinib, erlotinib, everolimus, imatinib, pazopanib, ruxolitinib, sorafenib, vandetanib, vemurafenib, trastuzumab, pazopanib, sorafenib | Precision Medicine | Molecularly Guided Therapy | 90 | up to 18 years | Seoul, Korea (Samsung Medical Center) | Active, recruiting |
| NCT02639546 | Safety and Pharmacokinetics of Cobimetinib in Pediatric and Young Adult Participants with Previously Treated Solid Tumors (iMATRIXcobi) | Cobimetinib | MEK | Molecularly Targeted Therapy | 50 | 6 months to 30 years | Hoffmann-La Roche | Active, recruiting |
| NCT02641314 | Metronomic Treatment in Children and Adolescents with Recurrent or Progressive High Risk Neuroblastoma | Propranolol, celecoxib, cyclophosphamide, vinblastine, etoposide | N/A | Systemic Chemotherapy | 26 | 2–20 years | Cologne, Germany (University of Cologne) | Active, recruiting |
| NCT02644460 | Abemaciclib in Children with DIPG or Recurrent/Refractory Solid Tumors (AflacST1501) | Abemaciclib (LY2835219) | CDK4/6 | Molecularly Targeted Therapy | 50 | 2–25 years | Atlanta, GA (Children’s Healthcare of Atlanta) | Active, recruiting |
| NCT02650401 | Study of RXDX-101 in Children with Recurrent or Refractory Solid Tumors and Primary CNS Tumors, With or Without TRK, ROS1, or ALK Fusions | RXDX-101 (Entrectinib) | TRK, ROS1, ALK | Molecularly Guided Therapy | 190 | 2–22 years | Hoffmann-La Roche | Active, recruiting |
| NCT02650648 | Humanized Anti-GD2 Antibody Hu3F8 and Allogeneic Natural Killer Cells for High-Risk Neuroblastoma | Hu3F8, cyclophosphamide, NK cells, IL-2 | GD2 | Immunotherapy | 36 | any | New York, NY (Memorial Sloan Kettering Cancer Center) | Active, recruiting |
| NCT02743429 | Phase II Study of Monoclonal Antibody ch14.18/CHO Continuous Infusion in Patients with Primary Refractory or Relapsed Neuroblastoma | ch14.18/CHO | GD2 | Immunotherapy | 40 | 1–21 years | Greifswald, Germany (University Medicine Greifswald) | Active, recruiting |
| NCT02748135 | A Two-Part Study of TB-403 in Pediatric Subjects with Relapsed or Refractory Medulloblastoma | TB-403 | N/A | Systemic Chemotherapy | 36 | 6 months to 18 years | Beat Childhood Cancer (BCC) | Active, recruiting |
| NCT02761915 | A Cancer Research UK Trial of Anti-GD2 T-cells (1RG-CART) | Anti-GD2 CAR-T cells, cyclophosphamide, fludarabine | GD2 | Immunotherapy | 27 | over 1 year | London, UK (University College London) | Active, recruiting |
| NCT02765243 | Anti-GD2 4th Generation CART Cells Targeting Refractory and/or Recurrent Neuroblastoma | Anti-GD2 CAR-T cells | GD2 | Immunotherapy | 30 | 1–14 years | China (Zhujiang Hospital) | Active, recruiting |
| NCT02780128 | Next Generation Personalized Neuroblastoma Therapy (NEPENTHE) | Ceritinib, trametinib, HDM201, ribociclib | ALK, RAS-MAPK, p53 | Molecularly Guided Therapy | 105 | 1–21 years | Philadelphia, PA (Children’s Hospital of Philadelphia) | Active, recruiting |
| NCT02813135 | European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory Tumors (ESMART) | Ribociclib, topotecan, temozolomide, everolimus, AZD1775, carboplatin, olaparib, irinotecan, AZD2014, nivolumab, Vistusertib, selumetinib, enasidenib, lirilumab | Precision Medicine | Molecularly Guided Therapy | 397 | up to 18 years | Villejuif, France (Institut Gustave Roussy) | Active, recruiting |
| NCT02909777 | Trial of CUDC-907 in Children and Young Adults with Relapsed or Refractory Solid Tumors, CNS Tumors, or Lymphoma | CUDC-907 (fimepinostat) | PI3K, HDAC | Molecularly Targeted Therapy | 44 | 1–21 years | Boston, MA (Dana-Farber Cancer Institute) | Active, recruiting |
| NCT02919046 | Study Evaluating the Efficacy and Safety With CAR-T for Relapsed or Refractory Neuroblastoma in Children | Anti-GD2 CAR-T cells | GD2 | Immunotherapy | 22 | 1–14 years | China (Nanjing Children’s Hospital) | Active, recruiting |
| NCT02982941 | Enoblituzumab (MGA271) in Children with B7-H3-expressing Solid Tumors | Enoblituzumab (MGA271) | B7-H3 | Immunotherapy | 112 | 1–35 years | MacroGenics | Active, recruiting |
| NCT02998983 | Racotumomab in Patients with High risk Neuroblastoma | Racotumomab | N-glycolil (NGc) GM3 (NGcGM3) | Immunotherapy | 39 | 1–12 years | Buenos Aires, Argentina (Hospital Universitario Austral) | Active, recruiting |
| NCT03107988 | Study of Lorlatinib (PF-06463922) | Lorlatinib, cyclophosphamide, topotecan | ALK, ROS1 | Molecularly Targeted Therapy | 40 | 1–90 years | New Approaches to Neuroblastoma Therapy (NANT) | Active, recruiting |
| NCT03155620 | Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients with Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (Pediatric MATCH) | Palbociclib, selumetinib, ensartinib, vemurafenib, olaparib, larotrectinib, LY3023414, erdafitinib | Precision Medicine | Molecularly Guided Therapy | 49 | 1–21 years | Children’s Oncology Group | Active, recruiting |
| NCT03189706 | Pilot Study of Chemoimmunotherapy for High-Risk Neuroblastoma | Hu3F8, irinotecan/temozolomide, GM-CSF | GD2 | Immunotherapy | 20 | any | New York, NY (Memorial Sloan Kettering Cancer Center) | Active, recruiting |
| NCT03209869 | Treatment of Relapsed or Refractory Neuroblastoma with Expanded Haploidentical NK Cells and Hu14.18-IL2 | Hu14.18-IL2, NK cells | GD2 | Immunotherapy | 6 | 7 months to 21 years | Madison, WI (University of Wisconsin) | Active, recruiting |
| NCT03236857 | A Study of the Safety and Pharmacokinetics of Venetoclax in Pediatric and Young Adult Patients with Relapsed or Refractory Malignancies | Venetoclax | BCL-2 | Molecularly Targeted Therapy | 135 | up to 25 years | AbbVie | Active, recruiting |
| NCT03242603 | Immunotherapy of Neuroblastoma Patients Using a Combination of Anti-GD2 and NK Cells (NKEXPGD2) | Haploidentical NK cells, ch14.18/CHO | GD2 | Immunotherapy | 5 | 6 months to 25 years | Singapore (National University Hospital) | Active, recruiting |
| NCT03273712 | Dosimetry-Guided, Peptide Receptor Radiotherapy (PRRT) With 90Y-DOTA-tyr3-Octreotide (90Y-DOTATOC) | 90Y-DOTA-tyr3-Octreotide (90YDOTATOC) | SSTR | Systemic Radiotherapy | 20 | 6 months to 90 years | Iowa City, IA (University of Iowa) | Active, recruiting |
| NCT03275402 | 131I-burtomab Radioimmunotherapy for Neuroblastoma Central Nervous System/Leptomeningeal Metastases | 131I-burtomab (8H9) | B7-H3 | Systemic Radiotherapy | 32 | up to 18 years | Y-mAbs Therapeutics | Active, not yet recruiting |
| NCT03294954 | GD2 Specific CAR and Interleukin-15 Expressing Autologous NKT Cells to Treat Children with Neuroblastoma (GINAKIT2) | NKT cells, cyclophosphamide, fludarabine | GD2 | Immunotherapy | 24 | 1–21 years | Houston, TX (Baylor College of Medicine) | Active, recruiting |
| NCT03332667 | MIBG With Dinutuximab | 131I-MIBG dinutuximab | NET, GD2 | Systemic Radiotherapy plus Immunotherapy | 24 | 1–30 years | New Approaches to Neuroblastoma Therapy (NANT) | Active, not yet recruiting |
| NCT03373097 | Anti-GD2 CAR T Cells in Pediatric Patients Affected by High Risk and/or Relapsed/Refractory Neuroblastoma | Anti-GD2 CAR-T cells | GD2 | Immunotherapy | 42 | 1–18 years | Italy (Bambino Gesù Hospital and Research Institute) | Active, recruiting |
| NCT03434262 | SJDAWN: St. Jude Children’s Research Hospital Phase 1 Study Evaluating Molecularly-Driven Doublet Therapies for Children and Young Adults with Recurrent Brain Tumors | Ribociclib, gemcitabine, trametinib, sonidegib | Precision Medicine | Molecularly Guided Therapy | 108 | 1–39 years | Memphis, TN (St. Jude Children’s Research Hospital) | Active, recruiting |
| NCT03458728 | Safety, Tolerability, Efficacy and Pharmacokinetics of Copanlisib in Pediatric Patients | Copanlisib (BAY806946) | PI3K | Molecularly Targeted Therapy | 130 | 6 months to 21 years | Bayer Pharma | Active, recruiting |
| NCT03478462 | Dose Escalation Study of CLR 131 in Children and Adolescents with Relapsed or Refractory Malignant Brain Cancer, Neuroblastoma, Rhabdomyosarcoma, Ewings Sarcoma, and Osteosarcoma | CLR 131 | N/A | Systemic Radiotherapy | 30 | 2–21 years | Madison, WI (University of Wisconsin) | Active, not yet recruiting |
| NCT03507491 | Nab-paclitaxel in Combination with Gemcitabine for Pediatric Relapsed and Refractory Solid Tumors | Nab-paclitaxel, gemcitabine | N/A | Systemic Chemotherapy | 24 | 6 months to 30 years | Atlanta, GA (Children’s Healthcare of Atlanta) | Active, not yet recruiting |
| NCT03561259 | A Study of Therapeutic Iobenguane (131I) for High-Risk Neuroblastoma at the Time of First Relapse (OPTIMUM) | 131I-MIBG | NET | Systemic Radiotherapy | 65 | over 1 year | Jubilant DraxImage Inc. | Active, recruiting |
Molecularly Targeted Therapy—Targeted agent used in non-targeted fashion; Molecularly Guided Therapy—Targeted agent used in targeted patient population based on molecular aberration or phenotype; NET—norepinephrine transporter; SSTR—somatostatin receptor; XRT—radiation therapy; ODC—ornithine decarboxylase, HIFU—high-intensity focused ultrasound, HDAC—histone deacetylase; MIBG—meta-iodobenzylguanidine; CAR-T cells—chimeric antigen receptor T cells; ALK—anaplastic lymphoma kinase; NK cells—natural killer cells.