Relapsed NeuroblastomaNCT01850888
MIBG for Refractory Neuroblastoma and Pheochromocytoma
A study testing 131 I-Metaiodobenzylguanidine (131I-MIBG), Potassium iodide solution and G-CSF in people with Relapsed Neuroblastoma and Metastatic Pheochromocytoma, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Relapsed Neuroblastoma, Metastatic Pheochromocytoma
- What is being tested?
- 131 I-Metaiodobenzylguanidine (131I-MIBG), Potassium iodide solution and G-CSF
- Type of treatment
- Pill or oral medicine, Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2013to 20 Oct 2025
- Phase
- Interventional
- 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:
- 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
Show all 22Show fewer
- 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.
- Prior Therapy: Patients must have recovered from all acute toxicities (defined as CTCAE 4.0 ≤ grade 1) associated with any prior therapy, and:
- Myelosuppressive chemotherapy: At least 2 weeks should have elapsed since any chemotherapy causing myelosuppression
- Biologic (anti-neoplastic agent): At least 7 days should have elapsed since the completion of therapy with a biologic agent.
- Monoclonal antibodies: At least 3 half-lives should have elapsed since therapy with a monoclonal antibody
- Radiation therapy: 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). For all other sites of radiation, at least 2 weeks should have relapsed.
- Cytokine therapy (e.g. G-CSF, GM-CSF, IL-6, IL-2): must be discontinued a minimum of 24 hours prior to MIBG therapy.
- Voluntary written informed consent
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Minnesota Masonic Cancer Center
Minneapolis, Minnesota, 55455
About this study
Official title: 131I-Metaiodobenzylguanidine (131I-MIBG) Therapy for Refractory Neuroblastoma and Pheochromocytoma
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.
Read the detailed description
Primary Objective is to provide access to therapy with 131I-MIBG for patients with relapsed/refractory neuroblastoma or metastatic pheochromocytoma.
Secondary Objective is to assess disease response to 131I-MIBG therapy for patients with relapsed/refractory neuroblastoma or metastatic pheochromocytoma.
Tertiary Objectives are to 1) gain more information about the toxicities of 131I-MIBG therapy; 2) assess improvement of symptoms, including pain and fatigue, for patients with relapsed/refractory neuroblastoma or metastatic pheochromocytoma who are receiving 131I-MIBG therapy.
- The therapeutic dose of 131I-MIBG will be based on the following:
- Minimum dose of 10 mCi/kg for patients without a stem cell source whose renal function is above the upper limit of normal but still meets eligibility criteria.
- Dose of 12 mCi/kg for patients without a stem cell source with normal renal function and meets other eligibility criteria.
- Dose of > 12 mCi/kg to 18 mCi/kg maximum at investigator's discretion for patients meeting eligibility criteria with stem cells available.
- A urinary catheter and intravenous fluids will be used for bladder protection, and potassium iodide solution for thyroid Protection.
- G-CSF is recommended for patients with ANC less than 750 after MIBG infusion.
- hematopoietic stem cell infusion is recommended for patients with grade 4 hematologic toxicity following 131I-MIBG therapy that continues to have an ANC <200 on G-CSF without signs of recovery for >2 weeks and any patient requiring platelet transfusion more than two times weekly for 4 consecutive weeks.
- Follow-up will be done until disease progression, death or other therapies are initiated.
- Being tested
- 131 I-Metaiodobenzylguanidine (131I-MIBG), Potassium iodide solution, G-CSF, hematopoietic stem cell infusion
- Keywords
- neuroblastoma, pheochromocytoma
- Medical subject headings
- Neuroblastoma, Pheochromocytoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
131 I-Metaiodobenzylguanidine (131I-MIBG)Drug
Also called 131I-MIBG
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.
Potassium iodide solutionDrug
Also called KI solution
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.
G-CSFDrug
Also called Neulasta
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).
hematopoietic stem cell infusionProcedure or surgery
Also called HSCT
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: 131 I-Metaiodobenzylguanidine (131I-MIBG); Drug: Potassium iodide solution; Drug: G-CSF; Procedure: hematopoietic stem cell infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 2 hours
Secondary outcomes (6)
Disease response
Disease response to 131 I-MIBG therapy in patients with relapsed/refractory neuroblastoma or metastatic pheochromocytoma. Disease response will be measured by Curie Score for patients with MIBG avid disease only or by both Currie Score and RECIST criteria for patients who have measureable disease in addition to MIBG avid disease. Disease response will be assessed at day 56 (+/- 14 days) and then every 3 months until 1-year post treatment, then every 6 months until progression, death or other therapy.
Measured: 1 year
Incidence of hematologic toxicities
Evaluation of hematologic toxicities of 131I MIBG therapy. CBC with differential and platelet count will be obtained prior to study enrollment, on day 0 and then twice weekly until ANC>500/mm3 and platelet count >20,000 x 3 days without transfusion. Once that is achieved CBC will be then obtained on day 56 and then every 3 months until 1 year post treatment, then every 6 months until progression, death or other therapy. In addition, we will be looking at the percent of patient that require infusion of stem cell product for cytopenias.
Measured: 1 year
Incidence of hepatic toxicities
ALT, AST, bilirubin will be obtained prior to study enrollment and then weekly until day 42, again on day 56 and then every 3 months until 1 year post treatment, then every 6 months until progression, death or other therapy
Measured: 1 year
Incidence of Thyroid Toxicity
T4 and TSH will be obtained prior to study enrollment and again on day 56 and then every 3 months until 1 year post treatment, then every 6 months until progression, death or other therapy.
Measured: 1 year
Improvement of pain symptoms
Assessment of pain will occur on day 0 of therapy, on the day of discharge and then weekly until day 42 and then again on day 56. The PedsQL Pediatric Pain Questionnaire will be used for assessment of pain in all patients. These questionnaires include both patient report and parent report, when appropriate.
Measured: 56 days
Improvement of fatigue
Assessment of fatigue will occur on day 0 of therapy, on the day of discharge and then weekly until day 42 and then again on day 56. The PedsQL Multidimensional Fatigue Scale will be used for assessment of fatigue in all patients. These scales include both patient report and parent report, when appropriate.
Measured: 56 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 May 2013 (estimated)
- Study startDec 2013
- Primary completion20 Oct 2025
- Study completion20 Oct 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Relapsed NeuroblastomaStudies of the same condition, open and under way.
- More trials run by Masonic Cancer Center, University of MinnesotaThe sponsor's other registered studies.
- 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Emily Greengard, MD, Principal investigator, University of Minnesota Department of Pediatrics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01850888
- Sponsor's protocol ID
- 2012LS107
- Status last verified
- Dec 2025
- First submitted
- 26 Mar 2013
- Last updated
- 18 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 131I-Metaiodobenzylguanidine (131I-MIBG) Therapy for Refractory Neuroblastoma and Pheochromocytoma
- ClinicalTrials.gov ID
- NCT01850888
- Acronym / sponsor ID
- 2012LS107
- Keywords
- neuroblastoma, pheochromocytoma
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- Dec 2013 (actual)
- Primary completion
- 20 Oct 2025 (actual)
- Study completion
- 20 Oct 2025 (actual)
- First posted
- 10 May 2013 (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 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.
Detailed description
Primary Objective is to provide access to therapy with 131I-MIBG for patients with relapsed/refractory neuroblastoma or metastatic pheochromocytoma.
Secondary Objective is to assess disease response to 131I-MIBG therapy for patients with relapsed/refractory neuroblastoma or metastatic pheochromocytoma.
Tertiary Objectives are to 1) gain more information about the toxicities of 131I-MIBG therapy; 2) assess improvement of symptoms, including pain and fatigue, for patients with relapsed/refractory neuroblastoma or metastatic pheochromocytoma who are receiving 131I-MIBG therapy.
- The therapeutic dose of 131I-MIBG will be based on the following:
- Minimum dose of 10 mCi/kg for patients without a stem cell source whose renal function is above the upper limit of normal but still meets eligibility criteria.
- Dose of 12 mCi/kg for patients without a stem cell source with normal renal function and meets other eligibility criteria.
- Dose of > 12 mCi/kg to 18 mCi/kg maximum at investigator's discretion for patients meeting eligibility criteria with stem cells available.
- A urinary catheter and intravenous fluids will be used for bladder protection, and potassium iodide solution for thyroid Protection.
- G-CSF is recommended for patients with ANC less than 750 after MIBG infusion.
- hematopoietic stem cell infusion is recommended for patients with grade 4 hematologic toxicity following 131I-MIBG therapy that continues to have an ANC <200 on G-CSF without signs of recovery for >2 weeks and any patient requiring platelet transfusion more than two times weekly for 4 consecutive weeks.
- Follow-up will be done until disease progression, death or other therapies are initiated.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 131 I-MIBG Treatment Arm | Experimental | Drug: 131 I-Metaiodobenzylguanidine (131I-MIBG), Drug: Potassium iodide solution, Drug: G-CSF, Procedure: hematopoietic stem cell infusionTherapeutic 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. |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (22 listed)
- Diagnosis:
- 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.
Key exclusion criteria (5 listed)
- 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
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 |
|---|---|---|
| 131 I-Metaiodobenzylguanidine (131I-MIBG)Also: 131I-MIBGMinimum 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. | Drug | 131 I-MIBG Treatment Arm |
| Potassium iodide solutionAlso: KI solutionFor 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. | Drug | 131 I-MIBG Treatment Arm |
| G-CSFAlso: NeulastaIt 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). | Drug | 131 I-MIBG Treatment Arm |
| hematopoietic stem cell infusionAlso: HSCTThe 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. | Procedure or surgery | 131 I-MIBG Treatment Arm |
Primary outcome
Number of patients who receive 131 I-MIBG.
Time frame: 2 hours
The number of patients with relapsed/refractory neuroblastoma or metastatic pheochromocytoma who receive access to 131 I-MIBG.
6 secondary outcomes
Disease response
Time frame: 1 year
Incidence of hematologic toxicities
Time frame: 1 year
Incidence of hepatic toxicities
Time frame: 1 year
Incidence of Thyroid Toxicity
Time frame: 1 year
Improvement of pain symptoms
Time frame: 56 days
Improvement of fatigue
Time frame: 56 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Dec 2025.
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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.
Emily Greengard, MD principal investigator
University of Minnesota Department of Pediatrics
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing 131 I-Metaiodobenzylguanidine (131I-MIBG), Potassium iodide solution and G-CSF in people with Relapsed Neuroblastoma and Metastatic Pheochromocytoma, run by Masonic Cancer Center, University of Minnesota 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 22 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: finished, as of the sponsor's last update on 18 Dec 2025. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of patients who receive 131 I-MIBG. (time frame: 2 hours). It is what the study was designed to measure, not a finding.
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 18 Dec 2025.