A Phase II, Open-label, Multi-center Study of Erivedge (Vismodegib) in the Treatment of Pediatric Patients With Refractory Pontine Glioma.

Inclusion Criteria:

- Subjects must have radiographically proven diffuse intrinsic pontine glioma and
confirmation of residual disease after initial therapy or at the time of
recurrence/progression as confirmed by MRI of the brain

- Subjects must be age ≥3 years and ≤ 18 years

- Diffuse intrinsic pontine glioma with measurable disease after receiving radiotherapy
either concurrent with or followed by ≤ 2 prior courses of chemotherapy

- Measurable disease as defined by:

Measurable tumor >10mm by MRI

- Karnofsky performance status (PS) 60-100% (for patients > 16 years of age) OR Lansky
PS 60-100% (for patients ≤ 16 years of age)

- Body surface area > 0.67 m2 and ≤ 2.21 m2

- Life expectancy of at least 2 months

- A negative urine pregnancy test is required for female participants of child bearing
potential (≥13 years of age or after onset of menses)

- Acceptable liver function as defined by:

1. Bilirubin ≤ 1.5 times upper limit of normal

2. AST (SGOT), ALT (SGPT) and Alkaline phosphatase ≤ 2.5 times upper limit of
normal

- Creatinine clearance or radioisotope GFR ≥ 70 mL/min OR serum creatinine based on age
as follows:

- 0.8 mg/dL (for patients ≤ 5 years of age)

- 1.0 mg/dL (for patients 6 to 10 years of age)

- 1.2 mg/dL (for patients 11 to 15 years of age)

- 1.5 mg/dL (for patients > 15 years of age)

- Acceptable hematologic status as defined by:

1. Granulocyte ≥ 1500 cells/mm3

2. Platelet count ≥ 100,000 (plt/mm3)

3. Serum albumin ≥ 2.5 g/dL

- Urinalysis:

a. No clinically significant abnormalities

- Acceptable coagulation status as defined by:

1. PT/INR less than 1.5

2. PTT within normal limits

- Subjects must be able to swallow and retain oral medication

- Female post-pubertal study subjects need to agree to use one of the more effective
birth control methods during treatment and for 7 (seven) months after treatment is
stopped. These methods include total abstinence (no sex), oral contraceptives ("the
pill"), an intrauterine device (IUD), levonorgestrol implants (Norplant), or
medroxyprogesterone acetate injections (Depo-provera shots).

- Male post-pubertal study subjects need to agree to use condoms with spermicide, even
after a vasectomy, during sexual intercourse with female partners while being treated
with Erivedge capsule and for 2 months after the last dose to avoid exposing an
embryo or fetus to Vismodegib.

- Voluntarily signed and dated a written IRB-approved informed consent by parent or
legal guardian of subject

Exclusion Criteria:

- Concurrent anti-cancer therapy (chemotherapy, radiation therapy, surgery,
immunotherapy, hormonal therapy, biologic therapy) other than the ones specified in
the protocol. Patients must have discontinued the above cancer therapies for
generally about 3 weeks (8 weeks for radiotherapy) prior to the first dose of study
medication, as well as recovered from toxicity (to ≤ than grade 2 except for
alopecia) induced by previous treatments.

- Currently receiving another investigational medicinal product.

- Uncontrolled concurrent illness including, but not limited to:

1. Active, uncontrolled bacterial, viral, or fungal infections requiring systemic
therapy

2. Diarrhea of any cause ≥ CTCAE grade 2

3. Psychiatric illness/social situations that would compromise patient safety or
limit compliance with study requirements including maintenance of a
compliance/pill diary

4. Any kind of malabsorption syndrome significantly affecting gastrointestinal
function

- Pregnant or nursing female patients. NOTE: If a female patient becomes pregnant or
suspects that she is pregnant while participating in this study, she should stop
taking study drug and immediately inform her treating physician immediately.

- Prior therapy with a Hedgehog inhibitor

- Unwillingness or inability to comply with procedures required in this protocol

- Serious nonmalignant disease (e.g., hydronephrosis, liver failure, or other
conditions) that could compromise protocol objectives in the opinion of the
investigator and/or the sponsor.

- History of Congestive Heart Failure (CHF) or ventricular arrhythmia requiring
medication.