Iodine I 131 Metaiodobenzylguanidine in Treating Patients With Recurrent, Progressive, or Refractory Neuroblastoma or Malignant Pheochromocytoma or Paraganglioma

NCT ID: NCT00107289

Last Updated: 2025-12-23

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 200 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2006-05-31
• Study Completion Date: 2026-05-31

Brief Summary

The purpose of this research study is to find how active and safe 131 I-MIBG is in patients with resistant neuroblastoma, malignant pheochromocytoma and malignant paraganglioma.

Conditions

Neuroblastoma; Pheochromocytoma

Keywords

metastatic pheochromocytoma; recurrent pheochromocytoma; regional pheochromocytoma; recurrent neuroblastoma; 04-148

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Radiation

Patients will be treated with IV 131I-MIBG using 1-2 high dose(s) or up to 16 low doses.

Thyroid protection is commenced seven days (+/-2 days) prior to administration of 131I-MIBG and continued for up to 42 days after each 131I-MIBG treatment. Given that this is an open access protocol, re-enrollment is allowed per PI discretion. Patients who received this treatment previously are allowed to be re-enrolled if they meet the eligibility criteria. Re-enrollment can be to the same arm previously treated on (high dose versus low dose) or to the other arm. Patients will not be eligible for re-enrollment if they have already received 16 low doses or 4 high doses of 131I-MIBG.

Group Type: EXPERIMENTAL

iobenguane I 131

Intervention Type: DRUG

A single dose of iodine I 131 metaiodobenzylguanidine (\^131I-MIBG) IV over 30 minutes to 4 hours or for 15 minutes for smaller patients on day 0. Patients undergo radiation dosimetry following the first dose of \^131I-MIBG to determine if a second dose can be safely administered. Some patients may receive a second dose of iodine I 131 metaiodobenzylguanidine (\^131I-MIBG) 6-8 weeks after the first dose. In some scenarios, extended time will be allowed before the second dose of 131I-MIBG for additional recovery and possible bridging therapy. If response is achieved and patients do not experience major toxicity. After blood radioactivity has fallen below 1 μCi/mL, patients may undergo autologous stem cell transplantation. After completion of study treatment, patients are followed at 4-6 weeks after \^131I-MIBG administration and then every 3 months for up to 1 year. Once patients are off treatment on this protocol, they will begin long term follow up through 5 years from enrollment.

Interventions

iobenguane I 131

A single dose of iodine I 131 metaiodobenzylguanidine (\^131I-MIBG) IV over 30 minutes to 4 hours or for 15 minutes for smaller patients on day 0. Patients undergo radiation dosimetry following the first dose of \^131I-MIBG to determine if a second dose can be safely administered. Some patients may receive a second dose of iodine I 131 metaiodobenzylguanidine (\^131I-MIBG) 6-8 weeks after the first dose. In some scenarios, extended time will be allowed before the second dose of 131I-MIBG for additional recovery and possible bridging therapy. If response is achieved and patients do not experience major toxicity. After blood radioactivity has fallen below 1 μCi/mL, patients may undergo autologous stem cell transplantation. After completion of study treatment, patients are followed at 4-6 weeks after \^131I-MIBG administration and then every 3 months for up to 1 year. Once patients are off treatment on this protocol, they will begin long term follow up through 5 years from enrollment.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Patients must have the diagnosis of NB in accordance with the International Criteria, i.e., either histopathology (confirmed by the MSKCC Department of Pathology) or BM involvement plus elevated urinary catecholamines.
• Must have a history of tumor progression or recurrence or failure to achieve complete response with standard therapy.
• Patients must have MIBG-avid NB and evaluable disease on MIBG scan at time of enrollment on protocol
• Prior Therapy: At least 2 weeks should have elapsed since any biologic therapy. Three weeks should have elapsed since last dose of chemotherapy.
• Age >1 year
• Determination that radiation safety restrictions during therapy period can be implemented.
• Stem cells: Patients for high does must have an autologous hematopoietic stem cell product cryopreserved and available for re-infusion after MIBG treatment. Patients for low dose do not require cryopreserved autologous hematopoietic stem cell product available. The minimum dose for peripheral blood stem cells is 2 X106 CD34+ cells/kg.
• Minimum life expectancy of eight weeks
• Signed informed consent indicating awareness of the investigational nature of this program.

• Patients must have the diagnosis of malignant CCT i.e. malignant pheochromocytoma or malignant paraganglioma
• Patients must have MIBG-avid malignant CCT and evaluable disease on MIBG scan at time of enrollment on protocol
• Prior Therapy: At least 2 weeks should have elapsed since any biologic therapy. Three weeks should have elapsed since last dose of chemotherapy.
• Age between 1 and 21 years and able to cooperate with radiation safety restrictions during therapy period
• Stem cells: Patients must have an autologous hematopoietic stem cell product cryopreserved and available for re-infusion after MIBG treatment. The minimum dose for peripheral blood stem cells is 2 X106 CD34+ cells/kg.
• Minimum life expectancy of eight weeks.
• Signed informed consent indicating awareness of the investigational nature of this program.

Exclusion Criteria

• Severe major organ toxicity. Specifically, renal, cardiac, hepatic, pulmonary, gastrointestinal and neurologic toxicity should all be grade 2 or less. A grade 3 hearing deficit is acceptable.
• Active serious infections not controlled by antibiotics.
• Pregnant women are excluded for fear of danger to the fetus. Therefore negative pregnancy test is required for all women of child-bearing age, and appropriate contraception is used during the study period.
• Inability or unwillingness to comply with radiation safety procedures or protocol requirements.

• Minimum Eligible Age: 1 Year
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Cancer Institute (NCI)

NIH

Sponsor Role: collaborator

Memorial Sloan Kettering Cancer Center

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Ellen Basu, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Memorial Sloan Kettering Cancer Center

Locations

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Ellen Basu, MD, PhD

Role: CONTACT

Phone: 212-639-5204

Shakeel Modak, MD

Role: CONTACT

Phone: 212-639-7623

Facility Contacts

Ellen Basu, MD, PhD

Role: primary

Shakeel Modak, MD

Role: backup

References

Chu BP, Horan C, Basu E, Dauer L, Williamson M, Carrasquillo JA, Pandit-Taskar N, Modak S. Feasibility of Administering High-Dose (131) I-MIBG Therapy to Children with High-Risk Neuroblastoma Without Lead-Lined Rooms. Pediatr Blood Cancer. 2016 May;63(5):801-7. doi: 10.1002/pbc.25892. Epub 2016 Jan 15.

Reference Type: DERIVED

PMID: 26773712 (View on PubMed)

Modak S, Zanzonico P, Carrasquillo JA, Kushner BH, Kramer K, Cheung NK, Larson SM, Pandit-Taskar N. Arsenic Trioxide as a Radiation Sensitizer for 131I-Metaiodobenzylguanidine Therapy: Results of a Phase II Study. J Nucl Med. 2016 Feb;57(2):231-7. doi: 10.2967/jnumed.115.161752. Epub 2016 Jan 7.

Reference Type: DERIVED

PMID: 26742708 (View on PubMed)

Related Links

http://www.mskcc.org/

Memorial Sloan Kettering Cancer Center

Other Identifiers

MSKCC-04148

Identifier Type: -

Identifier Source: secondary_id

04-148

Identifier Type: -

Identifier Source: org_study_id