Table. Clinical Trials of Checkpoint Inhibitors in Patients <21 Years
TitleTreatmentDiseasePhaseEnrollmentStatusNational Clinical Trials Database Number
Phase I Study of Ipilimumab (Anti-CTLA-4) in Children and Adolescents With Treatment-Resistant CancerIpilimumabSarcoma, Wilm tumor, lymphoma, neuroblastoma133Completed (November 2013)NCT01445379
Phase 2 Study of Ipilimumab in Children and Adolescents (12 to <18 Years) With Previously Treated or Untreated, Unresectable Stage III or Stage IV Malignant MelanomaIpilimumabMelanoma214Completed (July 2016)NCT01696045
Pilot Study of Nivolumab in Pediatric Patients With Hypermutant CancersNivolumabRefractory or recurrent hypermutated malignancies, biallelic mismatch repair deficiency positive patients1/220Accruing (Start date May 2017)NCT02992964
A Phase I/II Clinical Trial of Nivolumab in Progressive/Relapsed Pediatric Solid TumorsNivolumabOsteosarcoma, Ewing sarcoma, neuroblastoma, rhabdomyosarcoma1/230Not yet accruing (Posted September 2016)NCT02901145
Phase Ib/II Clinical Trial of Nivolumab Monotherapy and Nivolumab in Combination With Ipilimumab in Pediatric Subjects With High Grade Primary CNS MalignanciesNivolumab ± ipilimumabHigh grade primary CNS malignancies2170Accruing (Start date June 2017)NCT03130959
European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory TumorsNivolumab + lirilumabRefractory or recurrent malignancies1/2397Accruing (Start date August 2016)NCT02813135
A Phase I/II Trial Evaluating Anti-PD1 (Nivolumab) in Combination with Metronomic Chemotherapy in Children and Teenagers with Refractory/Relapsing Solid Tumors or LymphomaNivolumab ± cyclophosphamide ± vinblastine ± capecitabineSolid tumors, lymphoma1/2102Accruing (Start date September 2018)NCT03585465
A Phase I Study of 131-1 Meta-iodobenzylguanidine (mIBG) Followed by Nivolumab and Chimeric 14.18 Anti-GD2 Monoclonal Antibody Produced in Chinese Hamster Ovary Cells (ch14.18/CHO) in Children With Relapsed/Refractory NeuroblastomaNivolumabNeuroblastoma136Accruing (Start date May 2018)NCT02914405
A Phase I/II Study of Pembrolizumab (MK-3475) in Children With Advanced Melanoma or a PD-L1 Positive Advanced, Relapsed or Refractory Solid Tumor or Lymphoma (KEYNOTE-051)PembrolizumabMelanoma, lymphoma, solid tumors, classical Hodgkin lymphoma, microsatellite-instability-high solid tumor1/2310Accruing (Start date March 2015)NCT02332668
A Phase I, Open-Label, Single Institution Study to Assess the Safety, Tolerability, and Pharmacokinetics of Durvalumab in Pediatric Patients With Relapsed or Refractory Solid Tumors, Lymphoma, and Central Nervous System TumorsDurvalumabSolid tumors, lymphoma, CNS tumors136Accruing (Start date July 2016)NCT02793466
A Phase I/II Clinical Trial of MDV9300 (Pidilizumab) in Diffuse Intrinsic Pontine GliomaPidilizumabDiffuse pontine glioma1/250Active, not accruing (Estimated study completion date April 2019)NCT01952769
  • CNS, central nervous system.

  • Note: Information on these clinical trials is available at the U.S. National Library of Medicine