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A Phase I Study of SU5416 in Pediatric Patients With Recurrent or Progressive Poor Prognosis Brain Tumors

Phase 1
21 Years
Not Enrolling
Brain and Central Nervous System Tumors

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Trial Information

A Phase I Study of SU5416 in Pediatric Patients With Recurrent or Progressive Poor Prognosis Brain Tumors

OBJECTIVES: I. Determine the qualitative and quantitative toxicity of SU5416 in pediatric
patients with recurrent or progressive brain tumors. II. Determine the acute and chronic
dose-limiting toxicity and cumulative toxicity of this regimen in these patients. III.
Determine the maximum tolerated dose and pharmacokinetics of this regimen in this patient
population. IV. Determine the effects of hepatic enzyme-inducing drugs, such as
anticonvulsant agents, on the pharmacokinetics of this regimen in these patients. V.
Determine the efficacy, in a preliminary manner, of this regimen in these patients.

OUTLINE: This is a dose-escalation, multicenter study. Patients are stratified according to
concurrent use of enzyme-inducing anticonvulsant drugs (yes vs no drugs or modest-induction
drugs). Patients receive SU5416 IV over 1 hour twice a week for 6 weeks. Treatment repeats
every 6 weeks for 17 courses (approximately 2 years) in the absence of unacceptable toxicity
or disease progression. Cohorts of 3-6 patients in each stratum receive escalating doses of
SU5416 until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose
preceding that at which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity.
Patients are followed every 3 months for 1 year, every 6 months for 4 years, and then
annually for 5 years.

PROJECTED ACCRUAL: A total of 50 patients (25 per stratum) will be accrued for this study.

Inclusion Criteria

DISEASE CHARACTERISTICS: Histologically proven malignant recurrent or progressive brain
tumor at initial presentation or at time of recurrence or progression for which no
standard curative therapy exists Histologic verification for brainstem gliomas may be
waived Bone marrow involvement allowed

PATIENT CHARACTERISTICS: Age: 21 and under Performance status: Karnofsky 60-100% Life
expectancy: More than 8 weeks Hematopoietic: Absolute neutrophil count greater than
1,000/mm3* Platelet count greater than 75,000/mm3* Hemoglobin greater than 9 g/dL
*Transfusion independent Hepatic: Bilirubin normal for age SGOT and SGPT less than 2.5
times normal for age PT/PTT no greater than 1.2 times upper limit of normal Albumin
greater than 3 g/dL No overt hepatic disease Renal: Creatinine no greater than 1.5 times
normal for age OR Glomerular filtration rate greater than 70 mL/min No overt renal disease
Cardiovascular: No deep venous or arterial thrombosis within the past 3 months No history
of myocardial infarction, severe or unstable angina, or severe peripheral vascular disease
No overt cardiac disease No prior cerebral bleeds Pulmonary: No pulmonary embolism within
the past 3 months No overt pulmonary disease Other: Not pregnant or nursing Negative
pregnancy test Fertile patients must use effective contraception No known allergies to
paclitaxel or other agent that uses Cremophor EL No uncontrolled infection Neurological
deficits allowed if stable for at least 1 week prior to study Greater than 3rd percentile
weight for height

PRIOR CONCURRENT THERAPY: Biologic therapy: More than 6 months since prior bone marrow
transplantation More than 1 week since prior growth factor(s) Chemotherapy: At least 3
weeks since prior myelosuppressive chemotherapy (6 weeks for nitrosoureas) and recovered
Endocrine therapy: Concurrent dexamethasone allowed if dose stable for at least 1 week
prior to study Radiotherapy: More than 3 months since prior craniospinal irradiation
greater than 24 Gy More than 3 months since prior total body irradiation More than 2 weeks
since prior focal irradiation to symptomatic metastatic sites No prior stereotactic
radiosurgery Concurrent total body irradiation allowed Surgery: See Radiotherapy Other: No
other concurrent anticancer or experimental drug therapy Concurrent anticonvulsant drugs

Type of Study:


Study Design:

Endpoint Classification: Safety Study, Primary Purpose: Treatment

Outcome Measure:

Toxicities of SU5416 in children and adolescents with refractory CNS malignancies

Safety Issue:


Principal Investigator

Mark W. Kieran, MD, PhD

Investigator Role:

Study Chair

Investigator Affiliation:

Dana-Farber Cancer Institute


United States: Food and Drug Administration

Study ID:




Start Date:

August 2000

Completion Date:

March 2006

Related Keywords:

  • Brain and Central Nervous System Tumors
  • childhood craniopharyngioma
  • childhood central nervous system germ cell tumor
  • childhood oligodendroglioma
  • childhood choroid plexus tumor
  • childhood grade I meningioma
  • childhood grade II meningioma
  • childhood grade III meningioma
  • recurrent childhood cerebellar astrocytoma
  • recurrent childhood cerebral astrocytoma
  • recurrent childhood medulloblastoma
  • recurrent childhood visual pathway and hypothalamic glioma
  • recurrent childhood ependymoma
  • Brain Neoplasms
  • Nervous System Neoplasms
  • Central Nervous System Neoplasms



Baylor College of Medicine Houston, Texas  77030
Children's Hospital of Philadelphia Philadelphia, Pennsylvania  19104
Duke Comprehensive Cancer Center Durham, North Carolina  27710
UCSF Cancer Center and Cancer Research Institute San Francisco, California  94115-0128
Dana-Farber Cancer Institute Boston, Massachusetts  02115
Children's National Medical Center Washington, District of Columbia  20010-2970
Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania  15213
Children's Hospital and Regional Medical Center - Seattle Seattle, Washington  98105
Saint Jude Children's Research Hospital Memphis, Tennessee  38105-2794