NCT01239316

Vismodegib in Treating Younger Patients With Recurrent or Refractory Medulloblastoma

A Phase II Clinical Trial Evaluating the Efficacy and Safety of GDC-0449 in Children With Recurrent or Refractory Medulloblastoma

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: National Cancer Institute (NCI) · NIH
• Sex: All
• Age: 3 Years – 21 Years
• Healthy volunteers: Not accepted

Summary

This phase II trial studies how well vismodegib works in treating younger patients with recurrent or refractory medulloblastoma. Vismodegib may slow the growth of tumor cells.

Detailed description

PRIMARY OBJECTIVES: I. Estimate the efficacy of GDC-0449 (vismodegib) treatment for pediatric patients with recurrent or refractory medulloblastoma, as measured by the sustained objective response rates for patients without (stratum A) and with (stratum B) evidence of activation of Hedgehog (Hh) signaling pathway in their tumors. II. Characterize the pharmacokinetics (plasma) of GDC-0449 in children/adolescents with refractory medulloblastoma. III. To document pathologic and genomic methods to identify medulloblastomas with activation of the Hh signaling pathway. SECONDARY OBJECTIVES: I. Document and describe toxicities associated with GDC-0449 administered on a daily schedule. II. Estimate the duration of objective response and progression-free survival (PFS). III. Characterize the pharmacokinetics (cerebrospinal fluid) of GDC-0449 in children/adolescents with refractory medulloblastoma. OUTLINE: This is a multicenter study. Patients are stratified according to evidence of activation of Hedgehog signaling pathway in their tumors (without vs with vs unknown). Patients receive vismodegib orally (PO) once daily (QD) on days 1-28. Treatment repeats every 28 days for up to 26 courses in the absence of disease progression or unacceptable toxicity. Plasma and cerebrospinal fluid samples are collected periodically for pharmacokinetic and other correlative studies. After completion of study treatment, patients are followed up every other month for up to 12 months.

Conditions

• Recurrent Childhood Medulloblastoma

Interventions

Type	Name	Description
OTHER	Laboratory Biomarker Analysis	Correlative studies
OTHER	Pharmacological Study	Correlative studies
DRUG	Vismodegib	Given PO

Timeline

• Start date: 2010-11-01
• Primary completion: 2014-06-01
• Completion: 2015-08-01
• First posted: 2010-11-11
• Last updated: 2016-01-14
• Results posted: 2016-01-14

Locations

12 sites across 1 country: United States