Clinical trial NCT00293319: 131 I-MIBG in Treating Patients With Refractory or Relapsed Neuroblastoma
Official title: I-Metaiodobenzylguanidine (I-MIBG) Therapy for Refractory Neuroblastoma, A Phase II Study
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Neuroblastoma
Keywords: recurrent neuroblastoma
Lead sponsor: University of California, San Francisco
Sponsor class: OTHER
Collaborators: name: National Cancer Institute (NCI)
First posted: 2006-02-17
Enrollment: 164
Sex: ALL
Minimum age (years): 1
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: RATIONALE: Radioactive drugs, such as 131 I-MIBG, may carry radiation directly to tumor cells and not harm normal cells.

PURPOSE: This phase II trial is studying how well 131 I-MIBG works in treating patients with refractory or relapsed neuroblastoma.

Detailed description: OBJECTIVES:

Primary

* Determine if iodine I 131 metaiodobenzylguanidine can provide palliative therapy for patients with refractory or relapsed neuroblastoma.
* Determine the acute and late toxicity of this regimen in these patients.

Secondary

* Determine disease and symptom responses of patients treated with this regimen.

OUTLINE: This is a compassionate use study.

Patients receive iodine I 131 metaiodobenzylguanidine IV over 2 hours. Beginning 10 days later, patients with low neutrophil counts receive filgrastim (G-CSF) subcutaneously until blood count recovers. Patients with stable or responding disease may receive a second dose of iodine I 131 metaiodobenzylguanidine at least 6 weeks after the first dose.

After completion of study treatment, patients are followed periodically.

PROJECTED ACCRUAL: A total of 50 patients will be accrued for this study.

Eligibility criteria: DISEASE CHARACTERISTICS:

* Original diagnosis of neuroblastoma based on 1 of the following criteria:

  * Histopathology
  * Elevated urine catecholamines with typical tumor cells in the bone marrow
* Refractory or relapsed disease, meeting 1 of the following criteria:

  * Failure to respond to standard therapy (e.g., combination chemotherapy with or without radiotherapy and surgery)
  * Evidence of disease progression (i.e., any new lesion or an increase in size of \> 25% of a pre-existing lesion) at any time
* Evaluable disease by MIBG scan within 6 weeks of study entry

PATIENT CHARACTERISTICS:

* Not pregnant or nursing
* Fertile patients must use effective contraception
* Negative pregnancy test
* Bilirubin \< 2 times normal
* AST/ALT ≤ 10 times normal
* Creatinine ≤ 2 mg/dL
* Absolute neutrophil count\* ≥ 750/mm\^3 (transfusion independent)
* Platelet count\* ≥ 50,000/mm\^3 (20,000/mm\^3 if stem cells are available and platelet transfusion independent)
* Hemoglobin\* ≥ 10 g/dL (transfusion allowed)
* No dyspnea at rest
* No exercise intolerance
* No oxygen requirement
* No clinically significant cardiac dysfunction
* No disease of any major organ system that would preclude study compliance
* No active infection that meets grade 3 or 4 toxicity criteria NOTE: \*Patients with granulocytopenia and/or thrombocytopenia due to tumor metastases to the bone marrow may be eligible at the discretion of the principal investigator

PRIOR CONCURRENT THERAPY:

* See Disease Characteristics
* Recovered from prior therapy
* At least 2 weeks since prior antitumor therapy
* At least 3 months since prior radiotherapy to any of the following fields:

  * Craniospinal
  * Total abdominal
  * Whole lung
  * Total body
* At least 1 day since prior cytokine therapy (e.g., filgrastim \[G-CSF\], sargramostim \[GM-CSF\], interleukin-6, or epoetin alfa)
* Prior iodine I 131 metaiodobenzylguanidine allowed provided it was given more than 6 months ago AND patient has adequate hematopoietic stem cells available
* No concurrent hemodialysis

Primary outcomes:
measure: Ability of iodine I 131 metaiodobenzylguanidine to provide palliative therapy
measure: Acute and late toxicities
Secondary outcomes:
measure: Disease and symptom responses
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: UCSF Comprehensive Cancer Center
state: California
city: Ann Arbor
country: United States
facility: University of Michigan Comprehensive Cancer Center
state: Michigan
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
Study officials:
affiliation: University of California, San Francisco
name: Katherine K. Matthay, MD
role: STUDY_CHAIR
affiliation: Children's Hospital of Philadelphia
name: John M. Maris, MD
affiliation: University of California, San Francisco
name: Robert Goldsby, MD
References:
citation: Matthay KK, Yanik G, Messina J, Quach A, Huberty J, Cheng SC, Veatch J, Goldsby R, Brophy P, Kersun LS, Hawkins RA, Maris JM. Phase II study on the effect of disease sites, age, and prior therapy on response to iodine-131-metaiodobenzylguanidine therapy in refractory neuroblastoma. J Clin Oncol. 2007 Mar 20;25(9):1054-60. doi: 10.1200/JCO.2006.09.3484.