Trastuzumab Deruxtecan (DS-8201a) for the Treatment of Recurrent or Refractory Osteosarcoma, Wilms Tumor, and Desmoplastic Small Round Cell Tumor

Summary

This phase I/II trial studies the effects of trastuzumab deruxtecan (DS-8201a) in treating patients with osteosarcoma, Wilms tumor (WT) or desmoplastic small round cell tumor (DSRCT) that is newly diagnosed or has come back after a period of improvement (recurrent) or that has not responded to previous treatment (refractory). Trastuzumab deruxtecan is in a class of medications called antibody-drug conjugates. It is composed of a monoclonal antibody, called trastuzumab, linked to a chemotherapy drug, called deruxtecan. Trastuzumab attaches to HER2 positive tumor cells in a targeted way and delivers deruxtecan to kill them.

Timeline

Start

2021-03-08

Primary completion

2027-12-31

Completion

2027-12-31

Publications

• Reed DR, Janeway KA, Minard CG, Hall D, Crompton BD, Lazar AJ, Wang WL, Zylber R, Contreras A, Carrero G, Voss SD, Reid JM, Safgren SL, Militano O, Gorlick R, Pickett CA, Fox E, Weigel BJ. PEPN1924, A Phase II Study of Trastuzumab Deruxtecan in Patients With Recurrent Human Epidermal Growth Factor Receptor 2+ Osteosarcoma: A Children's Oncology Group Pediatric Early-Phase Clinical Trial Network Study. JCO Oncol Adv. 2025;2:e2500094. doi: 10.1200/oa-25-00094. Epub 2025 Oct 14.

Drugs

Indications