MIBG for Refractory Neuroblastoma and Pheochromocytoma

NCT01850888 | Completed

• 1 other identifier: 2012LS107
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

This is a best available therapy/compassionate use single institution study designed to determine the palliative benefit and toxicity of 131I-MIBG in patients with progressive neuroblastoma and metastatic pheochromocytoma who are not eligible for therapies of higher priority. Patients may receive a range of doses depending on stem cell availability and tumor involvement of bone marrow. Response rate, toxicity, and time to progression and death will be evaluated.

Conditions

Metastatic Pheochromocytoma; Relapsed Neuroblastoma

Keywords

neuroblastoma; pheochromocytoma

Outcome Measures

Primary Outcomes (1)

• Number of patients who receive 131 I-MIBG.

  The number of patients with relapsed/refractory neuroblastoma or metastatic pheochromocytoma who receive access to 131 I-MIBG.

  2 hours

Secondary Outcomes (6)

• Disease response

  1 year

• Incidence of hematologic toxicities

  1 year

• Incidence of hepatic toxicities

  1 year

• Incidence of Thyroid Toxicity

  1 year

• Improvement of pain symptoms

  56 days

Study Arms (1)

131 I-MIBG Treatment Arm

EXPERIMENTAL

Therapeutic 131 I-Metaiodobenzylguanidine (131I-MIBG) will be infused intravenously, intravenous fluids will be administered to help maintain urine flow and isotope excretion. Potassium iodide solution will be administered to protect thyroid function. G-CSF will be used if necessary for neutrophil recovery. Hematopoietic stem cell infusion if meets the criteria.

Drug: 131 I-Metaiodobenzylguanidine (131I-MIBG); Drug: Potassium iodide solution; Drug: G-CSF; Procedure: hematopoietic stem cell infusion

Interventions

131 I-Metaiodobenzylguanidine (131I-MIBG) DRUG

Minimum dose of 10 mCi/kg and up to 18 mCi/kg maximum will be diluted in 25 ml of normal saline, and will be infused intravenously over 90-120 minutes.

Also known as: 131I-MIBG

131 I-MIBG Treatment Arm

Potassium iodide solution DRUG

For the therapeutic MIBG administration, potassium iodide solution will be administered in a loading dose of 6mg/kg orally at least 8 hours prior to the MIBG injection, and then will be given at 1mg/kg/dose every 4 hours on days 0-6, then 1 mg/kg/day through day 45 post injection. The minimum dose of potassium iodide to be given is one drop, which equals 50 mg.

Also known as: KI solution

131 I-MIBG Treatment Arm

G-CSF DRUG

It is recommended that patients with ANC less than 750 after MIBG infusion begin G-CSF 5 mcg/kg/day subcutaneously (or receive equivalent single dose of Neulasta every 14 days while neutropenic) until neutrophil recovery (generally \>5000).

Also known as: Neulasta

131 I-MIBG Treatment Arm

hematopoietic stem cell infusion PROCEDURE

The majority of patients on this protocol will have autologous PBSCs available. Allogeneic stem cells may be utilized in patients who has received a prior allogeneic transplant. The minimum quantity for peripheral blood stem cells is 1.5 x 106 CD34+ cells/kg (optimum \> 2 x 106 CD34+ cells/kg). The minimum dose for bone marrow is 1.0 x 108 mononuclear cells/kg (optimum \>2.0 x 108 mononuclear cells/kg). Infusion will be performed according to institutional guidelines.

Also known as: HSCT

131 I-MIBG Treatment Arm

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis:
• Relapsed/refractory neuroblastoma with original diagnosis based on tumor histopathology or elevated urine catecholamines with typical neuroblastoma cells in the bone marrow
• Metastatic pheochromocytoma
• Age \>1 year and able to cooperate with radiation safety restrictions during therapy period
• Karnofsky or Lansky performance status of ≥ 50%
• Life expectancy: ≥ at least 8 weeks
• Disease status: Failure to respond to standard therapy or development of progressive disease at any time.
• Disease must be evaluable by MIBG scan. A positive MIBG scan must be present within 8 weeks prior to study entry and subsequent to any intervening therapy. If the patient has only one MIBG positive lesion and that lesion was radiated, a biopsy must be done at least 4 weeks after radiation was completed and must show viable neuroblastoma.
• Stem Cells: Patients must have a hematopoietic stem cell product available for reinfusion after MIBG treatment at doses of \> 12 mCi/kg.
• Have acceptable organ function as defined below within 7 days of enrollment:
• Bone Marrow: ANC ≥750 X 109 /L and platelets ≥50,000 X 109 /L without transfusion if stem cells are not available (any ANC or platelet allowed if stem cells available)
• Renal: Creatinine ≤3x upper limit of normal
• Hepatic: Bilirubin ≤2x upper limit of normal; AST/ALT ≤10x upper limit of normal
• Cardiac: Ejection fraction ≥45% on echocardiogram
• Pulmonary: normal lung function as manifested by no dyspnea and/or oxygen saturation ≥ 88% on room air.

You may not qualify if:

• Patients with disease of any major organ system that would compromise their ability to withstand therapy.
• Because of the teratogenic potential of the study medication, no patients who are pregnant or lactating will be allowed. Patients of childbearing potential must practice an effective method of birth control while participating on this study, to avoid possible damage to the fetus.
• Known allergy to any of the agents or their ingredients used in this study.
• Patients who are on hemodialysis
• Patients with untreated positive blood cultures or progressive infections as assessed by radiographic studies

Sponsors & Collaborators

• Masonic Cancer Center, University of Minnesota: lead

Study Sites (1)

University of Minnesota Masonic Cancer Center

Minneapolis, Minnesota, 55455, United States

Location

MeSH Terms

Conditions

Neuroblastoma; Pheochromocytoma

Interventions

3-Iodobenzylguanidine; Granulocyte Colony-Stimulating Factor; pegfilgrastim; Stem Cell Transplantation

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Paraganglioma; Neuroendocrine Tumors

Intervention Hierarchy (Ancestors)

Guanidines; Amidines; Organic Chemicals; Iodobenzenes; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Hydrocarbons, Iodinated; Hydrocarbons, Halogenated; Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors; Cell Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Transplantation; Surgical Procedures, Operative

Study Officials

• Emily Greengard, MD

  University of Minnesota Department of Pediatrics

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 26, 2013
• First Posted: May 10, 2013
• Study Start: December 1, 2013
• Primary Completion: October 20, 2025
• Study Completion: October 20, 2025
• Last Updated: December 18, 2025

Record last verified: 2025-12

Locations

US (1)