Early Phase and/or Targeted Therapy Studies in Pediatric Oncology in Switzerland Study Agent(s) Phase Indication Age Open Sites Contact / PI BEACON ± Dinutuximab beta (rando 2b Neuroblastoma (relapsed or refractory) 1-21y Zürich, Nicolas Gerber 2:+ with possibility of cross- Lausanne Maja Beck-Popovic over) + backbone with TMZ, or TMZ+topotecan (rando) T-VEC Talimogene laherparepvec 1 Relapsed or refractory solid (non-CNS 0-21y Zürich, Felix Niggli (oncolytic virus) tumors) with at least 1 injectable (non- Basel Thomas Kühne visceral) lesion Venetoclax Venetoclax 1 Relapsed or refractory tumors: solid tumors 0-25y Zürich Nicolas Gerber with BCL-2 expression and AML/ALL/lymphoma Dabrafenib/ Dabrafenib and Trametinib 2 High-grade glioma (BRAF V600 mutation 6-18y Zürich Nicolas Gerber Trametinib positive, relapsed or refractory), Low Grade Glioma (BRAF V600 mutation positive, unresectable tumor requiring treatment: for LGG only as first non-surgical treatment) Larotrectinib Larotrectinib 1/2 Advanced solid tumors (CNS and Non-CNS) 0-21y (incl. of Zürich Nicolas Gerber (Phase 1 expansion cohort and Phase 2: older pat. with NTRK gene fusion required [or for infantile ped. tumor fibrosarcoma, congenital mesoblastic types to be Solid Tumors Solid nephroma, or secretory breast cancer: discussed with ETV6 rearrangement]. Phase II: also benign sponsor) tumors with NTRK fusion eligible) Claudia Althaus, Clinical Trials Manager ([email protected]) University Children's Hospital, Zürich Nicolas Gerber, Ped. Oncologist ([email protected]) 19 Dec 2019 Page 1 / 2 Solid Tumors Solid Fimepinostat Fimepinostat (start 2 days 1 Newly Diagnosed Diffuse Intrinsic Pontine 3-39y Zürich Nicolas Gerber before biopsy/surgery!) Glioma (DIPG), Recurrent Medulloblastoma, or Recurrent High-Grade Glioma (HGG). Fimepinostat is started 2 days before biopsy/tumor surgery. Target valiation in tumor tissue. Cont. therapy (after RT in DIPG, after biopsy/surgery in others) Eribulin Eribulin Mesilate + 2 refractory or recurrent RMS or NRSTS 1-18y Zürich (in Nicolas Gerber Irinotecan prep.) OLIE +- Lenvatinib (rando), 2 refractory or relapsed osteosarcoma 2-25y Zürich (in Nicolas Gerber Backbone prep.) Ifosfamide/Etoposid Bchild Bosutinib 1/2 CML (resistant or intolarant to at least one 1-18y Zürich Jean-Pierre Bourquin prior Tyrosine Kinase Inhibitor) Inotuzumab Inotuzumab Ozogamicin 1/2 relapsed/refractory CD22 positive B-cell 1-18y Zürich Jean-Pierre Bourquin precursor (BCP)-ALL or other CD22 positive B-cell malignancies Venetoclax Venetoclax 1 Relapsed or refractory tumors: 0-25y Zürich Nicolas Gerber AML/ALL/lymphoma, and solid tumors with Leukemia/lymphoma BCL-2 expression NEW: primary therapy of TCF3-HLF ALL Emapalumab Emapalumab (Anti-INFy 3 Primary Hemaphagocytic 0-18y Zürich Jana Pachlopnik Antibody) Lymphohistiocytosis Other REACH 5 Ruxolitinib 2 Moderate and severe cGvHD after SCT 0-18y Zürich (in Tayfun Güngör prep.) Further studies in preparation Claudia Althaus, Clinical Trials Manager ([email protected]) University Children's Hospital, Zürich Nicolas Gerber, Ped. Oncologist ([email protected]) 19 Dec 2019 Page 2 / 2.