Last updated on August 2018

A Study of Pomalidomide (CC-4047) Monotherapy for Children and Young Adults With Recurrent or Progressive Primary Brain Tumors


Brief description of study

A Phase 2 study that 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.

The study will consist of 4 parallel groups, one for each of the following primary brain tumor types: high-grade glioma, medulloblastoma, ependymoma and DIPG. A Simon's Optimal two-stage study design will be applied to each group.

Detailed Study Description

This is a Phase 2 multi-center, open-label, parallel-group study that 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 after at least one prior standard therapy.

The study will consist of 4 parallel groups, one for each of the following primary brain tumor types: high-grade glioma, medulloblastoma, ependymoma and DIPG. A Simon's Optimal two-stage study design will be applied to each group and enrollment will occur as follows:

  • Stage 1: Nine subjects will be enrolled.
  • Stage 2: If during Stage 1, 2 subjects achieves either an objective response (either CR or PR) within the first 6 cycles of treatment (within first 3 cycles for DIPG), or a long-term SD, an additional 11 subjects shall be enrolled; otherwise no additional subjects will be enrolled into that group.
  • If a total of 5 or more subjects across all 20 subjects in a given group (Stage 1 and 2) evaluable for the primary endpoint are observed as having either an objective response (either CR or PR) within the first 6 cycles of treatment (within first 3 cycles for DIPG) or a long-term SD, pomalidomide will be considered effective in that disease indication. Subjects who do not meet the criteria for an objective response or disease progression by the end of Cycle 6 (end of Cycle 3 for DIPG subjects) will be considered as having long-term SD.

Response evaluations will be based on MRI results obtained at each site and will be assessed both locally and by an independent central reviewer. Corticosteroid use and clinical assessments (ie, neurologic status) will also be considered when determining overall response.

Tumor assessments will be conducted by standard MRI with and without contrast using three MRI sequences (T1-weighted pre- and post-contrast, T2-weighted, fluid-attenuated inversion recovery [FLAIR]). Overall radiographic objective response will be assessed utilizing the sequence(s) best representative of tumor in the opinion of the neuroradiologist.

Once treatment has been discontinued, subjects will be followed up for up to 5 years from enrollment of the last subject.

Subjects who withdraw from either stage for reasons other than disease progression prior to completing Cycle 1 of study treatment will be replaced.

An independent Data Monitoring Committee will monitor the study conduct.

Clinical Study Identifier: NCT03257631

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