A Study of Pomalidomide Monotherapy for Children and Young Adults With Recurrent or Progressive Primary Brain Tumors

Summary

This study will assess the efficacy, safety and tolerability of pomalidomide in children and young adults aged 1 to \< 21 years with recurrent or progressive primary brain tumors in one of four primary brain tumor types: high-grade glioma (HGG), medulloblastoma, ependymoma and diffuse intrinsic pontine glioma (DIPG).

Timeline

Start

2017-09-18

Primary completion

2023-09-14

Completion

2023-09-14

Outcome

Missed primary endpoint

paper Treatment with POM monotherapy did not meet the primary measure of success in any cohort. PMID 34168987 ↗

Publications

• Fangusaro J, Cefalo MG, Garre ML, Marshall LV, Massimino M, Benettaib B, Biserna N, Poon J, Quan J, Conlin E, Lewandowski J, Simcock M, Jeste N, Hargrave DR, Doz F, Warren KE. Phase 2 Study of Pomalidomide (CC-4047) Monotherapy for Children and Young Adults With Recurrent or Progressive Primary Brain Tumors. Front Oncol. 2021 Jun 8;11:660892. doi: 10.3389/fonc.2021.660892. eCollection 2021.
• Fangusaro J, Mitchell DA, Kocak M, Robinson GW, Baxter PA, Hwang EI, Huang J, Onar-Thomas A, Dunkel IJ, Fouladi M, Warren KE. Phase 1 study of pomalidomide in children with recurrent, refractory, and progressive central nervous system tumors: A Pediatric Brain Tumor Consortium trial. Pediatr Blood Cancer. 2021 Feb;68(2):e28756. doi: 10.1002/pbc.28756. Epub 2020 Oct 7.

Drugs

Indications