Conditions: Recurrent Childhood Medulloblastoma
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 12
Healthy volunteers: no

Brief 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.

Arms:
arm 1: Patients receive vismodegib PO QD on days 1-28. Treatment repeats every 28 days for up to 26 courses in the absence of disease progression or unacceptable toxicity.

Interventions: intervention 1: Laboratory Biomarker Analysis intervention 2: Pharmacological Study intervention 3: Vismodegib
Intervention descriptions: intervention 1: Correlative studies intervention 2: Correlative studies intervention 3: Given PO

Protocol document: