Vismodegib in Treating Younger Patients With Recurrent or Refractory Medulloblastoma

Completed · Phase 2

Conditions studied: Recurrent Childhood Medulloblastoma

In brief

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.

Key facts

Study ID

NCT01239316

Run by

National Cancer Institute (NCI)

People needed

12

Starts

2010-11-01

Expected to finish

2015-08-01

Last updated by the study team

2016-01-14

Who can join

Age: 3 and older, up to 21. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Patients with a histologically confirmed diagnosis of medulloblastoma that is recurrent, progressive, or refractory to standard therapy and for which there is no known curative therapy
• Patient must have adequate archival formalin fixed, paraffin embedded (FFPE) primary tumor material for biology studies; specifically, adequate archival FFPE tumor material is either:
• An FFPE block, preferably in which tumor occupies an area of at least 10x10 mm if available, and is free of surgical or processing artifacts; tumor in the submitted FFPE block must not have been obtained from the CUSA trap OR
• Preferably15x5µm sections if available from an FFPE tissue block conforming to the above criteria AND
• Tissue submission must be accompanied by a copy of the pathology report
• Patients must have bi-dimensionally measureable disease in the brain or spinal cord defined as at least one lesion that can be accurately measured in at least 2 planes in order to be eligible for this study
• Patients must have a body surface area (BSA) of >= 0.67m\^2 and at most 2.5m\^2
• Patients with neurological deficits should have deficits that are stable for a minimum of 1 week prior to registration; this is to be documented in the database
• Karnofsky performance status of >= 50% in patients > 16 years, or Lansky performance status of >= 50% in patients >= 3 yrs and =< 16 years, assessed within two weeks prior to registration
• Must have recovered from prior treatment-related toxicity; no other myelosuppressive chemotherapy or immunotherapy within 4 weeks prior to study entry (6 weeks if prior nitrosurea)
• Decadron dose should also be stable or decreasing for at least 1 week (7days) prior to starting therapy
• Radiation therapy (XRT) >= 3 months prior to study entry for craniospinal irradiation; >= 8 weeks for local irradiation to primary tumor; >= 2 weeks prior to study entry for focal irradiation for symptomatic metastatic sites
• Off all colony stimulating factors > 1 week prior to study entry (granulocyte colony stimulating factor [GCSF], granulocyte macrophage colony stimulating factor [GM CSF], erythropoietin)
• Prior therapy will include primary therapy (including radiation therapy and chemotherapy) and a maximum of 2 additional salvage therapies; patients can enroll on the protocol after failure on primary therapy
• Absolute neutrophil count (ANC) >= 1000 µL
• Platelet count >= 50,000/uL (transfusion independent)
• Hemoglobin >= 8.0 gm/dL (may receive red blood cell [RBC] transfusions)
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70ml/min/1.73 m\^2 or a serum creatinine =< 1.5 mg/dL
• Serum Total Bilirubin =< 1.5 x upper limit of normal (ULN) for age
• Serum glutamic-pyruvic transaminase (SGPT) (alanine aminotransferase [ALT]) =< 2.5 times institutional ULN for age
• Serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST]) =< 2.5 times institutional ULN for age
• Alkaline Phosphatase =< 1.5 times institutional ULN
• Serum albumin >= 2.5 g/dL
• Patient must have recovered from the significant acute toxicities of all prior therapy before entering this study and meet all other eligibility criteria specified in the Inclusion and Exclusion Criteria
• Pregnancy should be avoided for 12 months after the last dose of GDC-0449 for females of child-bearing potential; female patients of childbearing potential must not be pregnant or breast-feeding; female patients of childbearing potential must have a negative serum or urine pregnancy test within 24 hours prior to beginning treatment

You may not qualify if…

• Central nervous system (CNS) embryonal tumor other than medulloblastoma; for example, patients with diagnosis of Atypical Teratoid / Rhabdoid Tumor (ATRT), primitive neuroectodermal tumor (PNET) from a non-cerebellar site within the central nervous system, ependymoblastoma, or medulloepithelioma
• Patients with any clinically significant unrelated systemic illness (serious infections or significant cardiac, pulmonary, hepatic or other organ dysfunction), that would compromise the patient's ability to tolerate protocol therapy or would likely interfere with the study procedures or results
• Patients receiving any other anticancer or investigational drug therapy, or warfarin
• Patients with inability to return for follow-up visits or obtain follow-up studies required to assess toxicity to therapy
• Other: below given criteria are confirmed by the patient history
• Inability to swallow capsules
• Malabsorption syndrome or other condition that would interfere with enteral absorption
• History of congestive heart failure
• History of ventricular arrhythmia requiring medication
• Uncontrolled hypocalcemia, hypomagnesemia, hyponatremia or hypokalemia defined as less than the lower limit of normal for the institution despite adequate electrolyte supplementation
• Clinically important history of liver disease, including viral or other hepatitis or cirrhosis

Where it is running

• Children's Hospital Los Angeles — Los Angeles, California, United States
• Lucile Packard Children's Hospital Stanford University — Palo Alto, California, United States
• Children's National Medical Center — Washington D.C., District of Columbia, United States
• Lurie Children's Hospital-Chicago — Chicago, Illinois, United States
• National Cancer Institute Pediatric Oncology Branch — Bethesda, Maryland, United States
• Memorial Sloan-Kettering Cancer Center — New York, New York, United States
• Duke University Medical Center — Durham, North Carolina, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Children's Hospital of Pittsburgh of UPMC — Pittsburgh, Pennsylvania, United States
• St. Jude Children's Research Hospital — Memphis, Tennessee, United States
• Texas Children's Hospital — Houston, Texas, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.