A Study of Pomalidomide Monotherapy for Children and Young Adults With Recurrent or Progressive Primary Brain Tumors
Condition: Central Nervous System Neoplasms; Medulloblastoma
MeSH terms: Brain Neoplasms; Medulloblastoma; Nervous System Neoplasms; Central Nervous System Neoplasms
Study type: Interventional
Phase: Phase 2

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).
    

Detailed description: 
      The study will consist of 4 parallel groups of participants, 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 participants will be enrolled in each brain tumor type group

        -  Stage 2: If during Stage 1, ≥ 2 participants achieves either an objective response
           (either complete response or partial response) within the first 6 cycles of treatment
           (or within the first 3 cycles for DIPG participants), or a long-term stable disease, an
           additional 11 participants shall be enrolled; otherwise no additional participants will
           be enrolled into that group.

        -  If a total of 5 or more participants across all 20 participants in a given group (Stage
           1 and 2) evaluable for the primary endpoint are observed as having either an objective
           response (either complete response or partial response) within the first 6 cycles of
           treatment (or within the first 3 cycles for DIPG participants) or a long-term stable
           disease, pomalidomide will be considered effective in that disease indication.

      Once treatment has been discontinued, participants will be followed up for up to 5 years from
      enrollment of the last participant.

      Participants who withdraw from either stage for reasons other than disease progression prior
      to completing Cycle 1 of study treatment will be replaced.
    

Intervention name(s): Pomalidomide; Pomalidomide
Intervention type(s): Drug; Drug
Intervention description(s): : Subjects will be administered pomalidomide on Days 1 to 21, followed by a 7-day rest period, of each 28-day treatment cycle and will continue treatment for up to 24 cycles or until disease progression, withdrawal of consent/assent or unresolved toxicities as described in the protocol.; Pomalidomide will be provided as gelatin capsules and as an oral suspension. The starting dose will be 2.6 mg/m²/day, administered on Days 1 to 21, followed by a 7-day rest period, of each 28-day treatment cycle.
Other intervention name(s): CC-4047; CC-4047; Pomalyst®
Intervention MeSH terms: Thalidomide; Pomalidomide
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Pediatric; Brain Tumor; Central Nervous System Neoplasms; Glioma; High-grade glioma; HGG; Ependymoma; Medulloblastoma; DIPG; Diffuse intrinsic pontine gliomaProgressive; RecurrentPomalidomide; PomalystImnovid; CC-4047; Children