NeuroblastomaNCT01370330
131I-Labeled MIBG for Refractory Neuroblastoma: A Compassionate Use Protocol
A study testing Metaiodobenzylguanidine (MIBG) in people with Neuroblastoma, run by Kieuhoa Vo at 1 site in United States.
Trial summary
This is a compassionate use protocol to allow patients with advanced neuroblastoma palliative access to 131I-metaiodobenzylguanidine (131I-MIBG). Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- Metaiodobenzylguanidine (MIBG)
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- 1 siteUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis: Refractory or relapsed neuroblastoma with original diagnosis based on tumor histopathology or elevated urine catecholamines with typical tumor cells in the bone marrow. Refractory, progressive or metastatic pheochromocytoma/paraganglioma or related tumor.
- MIBG uptake: Tumors must be shown to be MIBG avid within 6 weeks prior to enrollment
- Age > 1 year and able to cooperate with radiation safety restrictions during therapy period. Patients with pheochromocytoma/paraganglioma and related tumors must be between 1 and 12 years of age.
- Life Expectancy: greater than 6 weeks.
- Lansky and Karnofsky Performance Status: 60% or higher.
- Disease status: Failure to respond to standard therapy (usually combination chemotherapy with or without radiation and surgery) or development of progressive disease at any time (any new lesion or an increase in size of >25% of a pre-existing lesion). Disease evaluable by MIBG scan must be present within 6 weeks of study entry and subsequent to any intervening therapy.
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- Stem cells: Patients must have an autologous hematopoietic stem cell product available for re-infusion after MIBG treatment at doses of >12 mCi/kg if needed. The minimum quantity for purged or unpurged peripheral blood stem cells is 1.0 x 10^6 cluster of differentiation 34 (CD34)+ cells/kg (optimum > 2 x 10^6 CD34+ cells/kg). The minimum dose for bone marrow is 1.0 x 10^8 mononuclear cells/kg (optimum > 2.0 x 10^8 mononuclear cells/kg). If no stem cells are available, then the dose of 131I-MIBG should be <12 mCi/kg .
- Prior Therapy: Patients may enter this study with or without re-induction therapy for recurrent tumor. Patients must have fully recovered from the toxic effects of any prior therapy. At least 2 weeks should have elapsed since any anti-tumor therapy and the patient must meet hematologic criteria below. Three months should have elapsed in the case of completing radiation to any of the following fields: craniospinal, total abdominal, whole lung, total body irradiation). Cytokine therapy [eg granulocyte-colony stimulating factor (G-CSF), granulocyte-macrophage colony-stimulating factor (GM-CSF), interleukin-6 (IL-6), erythropoietin] must be discontinued a minimum or 24 hours prior to MIBG therapy. Prior 131I-MIBG therapy is allowed if > 6 months previous and if the patient has adequate hematopoietic stem cells available and if cumulative 131I-MIBG dose will not exceed 60 mCi/kg.
- Organ Function
- Liver function: bilirubin <2x normal and aspartate aminotransferase (AST)/Alanine aminotransferase (ALT) < 10x normal.
- Kidney function: Creatinine less than or equal to 2
- Hematopoietic Criteria Patients must have adequate hematopoietic function (without transfusion): absolute neutrophil count (ANC) >.750 x 10E9/L; Platelets >50 x 10E9/L if stem cells are not available; if stem cells are available, the patient should be independent of platelet transfusions with a platelet count of at least 20 x 10E9/L. Hemoglobin >10g/dl at time of treatment (transfusion allowed). Patients with granulocytopenia and/or thrombocytopenia due to tumor metastatic to the bone marrow may be eligible after discussion with study chair or designee.
- Normal lung function as manifested by no dyspnea at rest or exercise intolerance, no oxygen requirement.
- No clinically significant cardiac dysfunction
- Signed informed consent: The patient and/or the patient's legally authorized guardian must acknowledge in writing that consent to become a study subject has been obtained, in accordance with institutional policies approved by the U.S. Department of Health and Human Services.
Who cannot join
- Patients with disease of any major organ system that would compromise their ability to withstand therapy. Any significant organ impairment should be discussed with the Study Chair or Vice Chair prior to patient entry.
- Because of the teratogenic potential of the study medications, 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 pregnancy.
- Patients who are on hemodialysis.
- Patients with active infections that meet grade 3-4 toxicity criteria.
- Patients with pheochromocytoma or paraganglioma who have any proteinuria on urinalysis must have a 24-hr urine collection for protein. If there is proteinuria above the reference range on a 24-hour urine collection, they are excluded due to increased risk of respiratory complications.
Trial locations and contacts
1 site in 1 country.
United States1 site
- Available
University of California, San Francisco
San Francisco, California, 94143
Karina Wong, 415-353-8039, Karina.Wong@ucsf.edu
877-827-3222, cancertrials@ucsf.edu
About this study
This is a compassionate use protocol to allow patients with advanced neuroblastoma palliative access to 131I-metaiodobenzylguanidine (131I-MIBG).
Read the detailed description
Neuroblastoma remains a fatal disease for a large percentage of patients, especially those with high-risk disease features who become resistant to conventional therapy. 131I-metaiodobenzylguanidine (131I-MIBG) is a norepinephrine analog that concentrates in adrenergic tissue and therefore holds promise for cell-specific treatment of neuroblastoma. 131I-MIBG is active against relapsed or refractory neuroblastoma and associated hematopoietic toxicity can be abrogated with autologous stem cell rescue. 131I-MIBG given in doses of 10-18 millicurie (mCi)/kg with stem cell rescue, if necessary, is safe and effective palliative therapy for refractory or relapsed neuroblastoma patients.
- Conditions
- Neuroblastoma
- Being tested
- Metaiodobenzylguanidine (MIBG)
- Keywords
- Neuroblastoma, MIBG, 131I-MIBG, Resistant, Relapsed, Treatment, University of California, San Francisco, Pediatric, Oncology
- Medical subject headings
- Neuroblastoma, Recurrence, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Metaiodobenzylguanidine (MIBG)Drug
Also called 131I-MIBG
131I-MIBG Therapeutic Administration. Therapeutic 131I-MIBG will be synthesized at Jubilant DraxImage (Quebec, Canada) with specific activities of 9-18 Ci/mmole, or at Progenics with specific activity of 2,500 mCi/mg. The therapeutic dose (8-18 mCi/kg at investigator's discretion; any dose greater than 12 requires stored stem cells) will be diluted in 25-50 ml of normal saline for either preparation, and will be infused intravenously through a patient's central line, if already present, or a peripheral IV if a central line is not present.If Azedra is used, the dose will be infused over 30-60 minutes; the low specific activity preparation from Draximage will be infused over 90-120 minutes. For patients with pheochromocytoma or paraganglioma, the recommended maximum dose is 500 mCI or 12 mCi/kg.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jun 2011 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeuroblastomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Kieuhoa Vo
- Sponsor type
- University, hospital or other
- Collaborators
- Cannonball Kids' Cancer Foundation
- Responsible party
- Sponsor-investigator, Kieuhoa Vo, University of California, San Francisco
- Investigators
- Kieuhoa Vo, MD, Principal investigator, University of California, San Francisco
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01370330
- Sponsor's protocol ID
- CompUse MIBG
- Other IDs
- NCI-2011-01902, 05161
- Status last verified
- Jul 2026
- First submitted
- 27 May 2011
- Last updated
- 23 Jul 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 131I-Labeled MIBG for Refractory Neuroblastoma: A Compassionate Use Protocol
- ClinicalTrials.gov ID
- NCT01370330
- Acronym / sponsor ID
- CompUse MIBG
- Conditions
- Neuroblastoma
- Keywords
- Neuroblastoma, MIBG, 131I-MIBG, Resistant, Relapsed, Treatment, University of California, San Francisco, Pediatric
- Study type
- Expanded access
- Recruitment status
- Available
- Lead sponsor
- Kieuhoa VoUniversity, hospital or other
- Collaborators
- Cannonball Kids' Cancer Foundation
- First posted
- 9 Jun 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a compassionate use protocol to allow patients with advanced neuroblastoma palliative access to 131I-metaiodobenzylguanidine (131I-MIBG).
Detailed description
Neuroblastoma remains a fatal disease for a large percentage of patients, especially those with high-risk disease features who become resistant to conventional therapy. 131I-metaiodobenzylguanidine (131I-MIBG) is a norepinephrine analog that concentrates in adrenergic tissue and therefore holds promise for cell-specific treatment of neuroblastoma. 131I-MIBG is active against relapsed or refractory neuroblastoma and associated hematopoietic toxicity can be abrogated with autologous stem cell rescue. 131I-MIBG given in doses of 10-18 millicurie (mCi)/kg with stem cell rescue, if necessary, is safe and effective palliative therapy for refractory or relapsed neuroblastoma patients.
Study design
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (15 listed)
- Diagnosis: Refractory or relapsed neuroblastoma with original diagnosis based on tumor histopathology or elevated urine catecholamines with typical tumor cells in the bone marrow. Refractory, progressive or metastatic pheochromocytoma/paraganglioma or related tumor.
- MIBG uptake: Tumors must be shown to be MIBG avid within 6 weeks prior to enrollment
- Age > 1 year and able to cooperate with radiation safety restrictions during therapy period. Patients with pheochromocytoma/paraganglioma and related tumors must be between 1 and 12 years of age.
- Life Expectancy: greater than 6 weeks.
- Lansky and Karnofsky Performance Status: 60% or higher.
Key exclusion criteria (5 listed)
- Patients with disease of any major organ system that would compromise their ability to withstand therapy. Any significant organ impairment should be discussed with the Study Chair or Vice Chair prior to patient entry.
- Because of the teratogenic potential of the study medications, 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 pregnancy.
- Patients who are on hemodialysis.
- Patients with active infections that meet grade 3-4 toxicity criteria.
- Patients with pheochromocytoma or paraganglioma who have any proteinuria on urinalysis must have a 24-hr urine collection for protein. If there is proteinuria above the reference range on a 24-hour urine collection, they are excluded due to increased risk of respiratory complications.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Metaiodobenzylguanidine (MIBG)Also: 131I-MIBG131I-MIBG Therapeutic Administration. Therapeutic 131I-MIBG will be synthesized at Jubilant DraxImage (Quebec, Canada) with specific activities of 9-18 Ci/mmole, or at Progenics with specific activity of 2,500 mCi/mg. The therapeutic dose (8-18 mCi/kg at investigator's discretion; any dose greater than 12 requires stored stem cells) will be diluted in 25-50 ml of normal saline for either preparation, and will be infused intravenously through a patient's central line, if already present, or a peripheral IV if a central line is not present.If Azedra is used, the dose will be infused over 30-60 minutes; the low specific activity preparation from Draximage will be infused over 90-120 minutes. For patients with pheochromocytoma or paraganglioma, the recommended maximum dose is 500 mCI or 12 mCi/kg. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jul 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kieuhoa Vo, MD principal investigator
University of California, San Francisco
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Metaiodobenzylguanidine (MIBG) in people with Neuroblastoma, run by Kieuhoa Vo at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 15 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 23 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Karina Wong (415-353-8039, Karina.Wong@ucsf.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jul 2026.