A Randomized Phase 3 Study of Combination Antineoplaston Therapy [Antineoplastons A10 (Atengenal) and AS2-1 (Astugenal)] vs. Temozolomide in Subjects With Recurrent and / or Progressive Optic Pathway Glioma After Carboplatin or Cisplatin Therapy
1. Children age ≥ 6 months < 18 years are eligible if they have 1) received prior
treatment with carboplatin or cisplatin, which was terminated secondary to toxicity
or progression of OPG or 2) developed recurrence of OPG after completion of
carboplatin or cisplatin therapy.
2. Children with or without prior RT are eligible.
3. Histological confirmation of OPG is required unless the risks of obtaining a
diagnostic biopsy are prohibitive.
4. Evidence of OPG (≥ 5 mm), as diagnosed by MRI of the brain, with and without
gadolinium contrast, within four weeks of protocol study entry is required. The MRI
is interpreted by two independent neuroradiologists. If there is disagreement, a
third independent neuroradiologist will adjudicate. Baseline MR spectroscopy (MRS)
and positron emission tomography (PET) scan are also performed.
5. Children who are receiving corticosteroids and, for at least one week prior to entry
into the protocol study have been on the lowest dose of corticosteroids that
preserves optimal neurologic function, are eligible.
6. Children with a life expectancy of > 6 months are eligible.
7. Children ≤ 14 years of age with a Lansky performance status of > 60 are eligible.
Children > 14 years of age with a Karnofsky performance status of > 60 are eligible.
8. Children with normal organ and marrow function (as defined below) are eligible.
- hemoglobin ≥ 10 g/dL
- leukocytes > 2000/mm3
- absolute neutrophil count (ANC) >1,500/ mm3
- serum NA+, K+, BUN within institutional normal limits
- platelets >75,000/ mm3
- total bilirubin < 1.5 mg/dL
- AST(SGOT)/ALT(SGPT) <3 times institutional upper limit of normal (ULN)
- serum creatinine < 1.5 mg/dL
9. At the recommended therapeutic dose, the effects of ANP therapy on the developing
human fetus are unknown. For this reason, women of child-bearing potential who agree
to use adequate contraception (hormonal or barrier method of birth control;
abstinence) prior to protocol study entry and for the duration of protocol study are
eligible. Should a woman become pregnant or suspect she is pregnant while
participating in this protocol study, she will inform her treating physician
10. Children who are able to understand a written informed consent document, and are
willing to sign it, are eligible. A subject with a parent or guardian who is able to
understand a written informed consent document, and who is willing to sign it on the
subject's behalf, is eligible.
1. Children receiving prior ANP or TMZ therapy are not eligible.
2. Children with an uncontrolled intercurrent illness including, but not limited to,
ongoing or active infection, uncontrolled hypertension (> grade 2) or psychiatric
illness and/or social situations that would limit compliance with protocol study
requirements are not eligible.
3. Children with a history of congestive heart failure, deep venous thrombosis, or other
cardiovascular or renal conditions that would contradict administration of high dose
intravenous sodium or insertion of a subclavian venous catheter are not eligible.
4. Pregnant women are not eligible because the teratogenic and abortifacient effects of
ANP therapy in humans are unknown. Because there is an unknown but potential risk for
adverse events in nursing infants secondary to the mother receiving ANP therapy,
breastfeeding is discontinued if the mother receives ANP therapy.