Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Giselle Sholler
With
Phoenix Children's Hospital
Age
3–18
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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
You may not be if
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:
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.
Clareo Health | Erivedge (Vismodegib) in the Treatment of Pediatric Patients With Refractory Pontine Glioma
