Recurrent Childhood Medulloblastoma Clinical Trial
Official title:
A Phase II Clinical Trial Evaluating the Efficacy and Safety of GDC-0449 in Children With Recurrent or Refractory Medulloblastoma
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.
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.
;
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Withdrawn |
NCT02194452 -
Efficacy of 68Ga-DOTATOC Positron Emission Tomography (PET) CT in Children and Young Adults With Brain Tumors
|
N/A | |
| Completed |
NCT00053963 -
FR901228 in Treating Children With Refractory or Recurrent Solid Tumors or Leukemia
|
Phase 1 | |
| Completed |
NCT00095940 -
Lapatinib in Treating Young Patients With Recurrent or Refractory Central Nervous System Tumors
|
Phase 1/Phase 2 | |
| Completed |
NCT00052780 -
Temozolomide and O6-Benzylguanine in Treating Children With Recurrent Brain Tumors
|
Phase 1 | |
| Completed |
NCT00004078 -
Irinotecan in Treating Children With Refractory Solid Tumors
|
Phase 2 | |
| Completed |
NCT00100880 -
Lenalidomide in Treating Young Patients With Recurrent, Progressive, or Refractory CNS Tumors
|
Phase 1 | |
| Completed |
NCT00070525 -
Tipifarnib in Treating Young Patients With Recurrent or Progressive High-Grade Glioma, Medulloblastoma, Primitive Neuroectodermal Tumor, or Brain Stem Glioma
|
Phase 2 | |
| Terminated |
NCT01088763 -
Gamma-Secretase Inhibitor RO4929097 in Treating Young Patients With Relapsed or Refractory Solid Tumors, CNS Tumors, Lymphoma, or T-Cell Leukemia
|
Phase 1 | |
| Completed |
NCT00326664 -
AZD2171 in Treating Young Patients With Recurrent, Progressive, or Refractory Primary CNS Tumors
|
Phase 1 | |
| Completed |
NCT00101270 -
Oxaliplatin and Irinotecan in Treating Young Patients With Refractory Solid Tumors or Lymphomas
|
Phase 1 | |
| Completed |
NCT02271711 -
Expanded Natural Killer Cell Infusion in Treating Younger Patients With Recurrent/Refractory Brain Tumors
|
Phase 1 | |
| Active, not recruiting |
NCT03210714 -
Erdafitinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With FGFR Mutations (A Pediatric MATCH Treatment Trial)
|
Phase 2 | |
| Completed |
NCT02444546 -
Wild-Type Reovirus in Combination With Sargramostim in Treating Younger Patients With High-Grade Relapsed or Refractory Brain Tumors
|
Phase 1 | |
| Completed |
NCT00363272 -
Ispinesib in Treating Young Patients With Relapsed or Refractory Solid Tumors or Lymphoma
|
Phase 1 | |
| Completed |
NCT00946335 -
ABT-888 and Temozolomide in Treating Young Patients With Recurrent or Refractory CNS Tumors
|
Phase 1 | |
| Completed |
NCT00063973 -
Cilengitide in Treating Children With Refractory Primary Brain Tumors
|
Phase 1 | |
| Completed |
NCT00822458 -
GDC-0449 in Treating Young Patients With Medulloblastoma That is Recurrent or Did Not Respond to Previous Treatment
|
Phase 1 | |
| Active, not recruiting |
NCT00638898 -
Busulfan, Melphalan, Topotecan Hydrochloride, and a Stem Cell Transplant in Treating Patients With Newly Diagnosed or Relapsed Solid Tumor
|
Phase 1 | |
| Terminated |
NCT02684071 -
Phase II Study of Intraventricular Methotrexate in Children With Recurrent or Progressive Malignant Brain Tumors
|
Phase 2 | |
| Completed |
NCT01076530 -
Vorinostat and Temozolomide in Treating Young Patients With Relapsed or Refractory Primary Brain Tumors or Spinal Cord Tumors
|
Phase 1 |