A dose escalation scale consisting of 5 dosage levels is being used to determine the maximum
tolerated dose (MTD) of SU101. A minimum of 3 and a maximum of 6 patients will be enrolled
at each dose level. MTD is defined as the dose level immediately below that at which 2 or
more patients exhibit dose limiting toxicity.
Each treatment cycle is 21 days. Patients receive a 96 hour continuous IV infusion of SU101
on days 1-4.
SU101 is a member of a novel class of antineoplastic agents, platelet-derived growth factor
(PDGF) receptor inhibitors. Preclinical data suggests that SU101 might be an effective
agent against neuroglial tumors as well as a variety of sarcomas. A pediatric phase I trial
of SU101 in children with these malignancies will be conducted to find the maximum tolerated
dose of SU101 and define the toxicity profile of this agent. In addition, we will define
the pharmacokinetics of SU101 and its active metabolite SU0020 in pediatric patients and
gather preliminary information regarding response.
Histologically proven primary CNS malignancy, neuroblastoma or sarcoma that is refractory
to standard therapy or for which no standard therapy exists and disease can not be cured
Recovered from toxic affects of all prior therapy.
No investigational agent within past 2 weeks.
BIOLOGY THERAPY: Not specified.
No myelosuppressive therapy within past 3 weeks.
No nitrosourea within past 6 weeks.
ENDOCRINE THERAPY: If receiving dexamethasone dose must be stable for at least 2 weeks.
RADIOTHERAPY: Not specified.
SURGERY: Not specified.
Age: 3 to 21.
Performance status: ECOG 0-2.
Life expectancy: At least 8 weeks.
AGC greater than 1500/mm(3).
Hemoglobin greater than or equal to 8.0 g/dL percent.
Platelet count greater than 100,000/mm(3).
For patients with bone marrow involvement or history of bone marrow transplantation or
craniospinal radiotherapy: AGC greater than 750/mm(3), Hemoglobin greater than 6.0 g/dL,
Platelet count greater than 50,000/mm(3).
SGOT, SGPT or alkaline phosphatase less than 3 times upper limit of normal.
Bilirubin no less than or equal to 1.5 times upper limit of normal.
Ages 3-5 Creatinine no greater than 0.8 mg/dL.
Ages 5-10 Creatinine no greater than 1.0 mg/dL.
Ages 10-15 Creatinine no greater than 1.2 mg/dL.
Ages 16-21 Creatinine no greater than 1.5 mg/dL.
All patients or their legal guardians (if the patient is under 18 years old) must sign a
document of informed consent indicating their understanding of the investigational nature
and the risks of this study.
For patients with brain tumors who are over 18 years of age, a DPA should be signed.
Not pregnant or nursing.
Not allergic to etoposide.
No acute or chronic medical or psychiatric condition or laboratory abnormality that may
increase the risks associated with study participation/study drug administration or may
interfere with the interpretation of study results.