MIBG Avid TumorsPhase 2MIBGNCT02378428
MIBG Therapy for Patients With MIBG Avid Tumors
A Phase 2 study testing MIBG in people with MIBG Avid Tumors, run by Nationwide Children's Hospital at 1 site in United States.
Trial summary
This is a Phase II study for patients with MIBG avid tumors. The study is to determine the response rate to <131>I-MIBG in patients with de novo, relapsed or refractory neuroblastoma or other MIBG avid malignant tumors 42 days post MIBG therapy. Read more about this study
- Who is it for?
- Ages 12 months to 65 years
- Conditions the study is for
- MIBG Avid Tumors
- What is being tested?
- MIBG
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 65 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Mar 2014to 31 Dec 2025
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 12 months to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients must be between 12 months and 65 years at the time of enrollment
- Diagnosis: diagnosis of neuroblastoma or at the time of relapse by histology and/or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites
- Disease Status:
- The presence of refractory or progressive disease (PD)
- For patients with neuroblastoma, the presence of mixed response (MR), or no response (NR) following the completion of A3973 or equivalent induction therapy, or the presence of a partial response (PR) with high Curie score (>2) following induction therapy.
- Patients with de novo high risk neuroblastoma who have completed standard induction therapy and do not achieve a CR, VGPR, or PR with low Curie score post induction.
Show all 28Show fewer
- Patients must have evidence of MIBG avid disease as determined by diagnostic MIBG scan obtained within 4 weeks of study entry.
- Patients who receive greater than 12 mCi/kg are required to have stem cell rescue products harvested prior to study treatment.
- Performance Level and Life Expectancy: Patients must have a Lansky Play Scale17 of 60% (<16 yrs old), Karnofsky score 60% (>16 yrs old), or ECOG score of < or equal to 2 and a life expectancy of 2 months.
- Patients may enter this study with or without salvage therapy for recurrent tumor. Patients must have fully recovered from the toxic effects of any prior therapy.
- Organ Function requirements:
- Hematopoietic Criteria:
- Hemoglobin- 10 gl/dl (transfusion allowed)
- ANC- 550 / cu mm (off myeloid growth factors)
- Platelets- > 50,000/cu mm. (transfusion allowed - however patients must not require more than two platelet transfusions per week).
- Renal Function:
- Serum Creatinine- < 2 x ULN for age.
- Hepatic Function:
- Total bilirubin <1.5 x ULN for age SGPT (ALT) and SGOT (AST) < 10 x ULN for age
- Cardiac Function:
- For children with NBL: Normal ejection fraction (>55%) documented by echocardiogram or radionuclide MUGA evaluation OR normal fractional shortening (>27%) documented by echocardiogram.
- For subjects with paraganglioma/ pheochromocytoma: No clinically significant cardiac dysfunction.
- Pulmonary Function:
- Patients must have clinically normal lung function as manifested by no dyspnea at rest and no oxygen requirement
- Reproductive Function:
- Females of childbearing potential must have a negative pregnancy test within 1 week prior to treatment with 131I-MIBG.
- Patients of childbearing potential must agree to use an effective birth control method.
- Female patients who are lactating must agree to stop breast feeding
Who cannot join
- Pregnancy or breast feeding
- Have undergone a prior allogeneic BMT.
- 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 prior to patient entry.
- Patients who are on hemodialysis.
- Hepatitis B surface antigen (+) or Hepatitis C positive in preceding six months.
- Patients with an active infection requiring intravenous antivirals, antibiotics or antifungals. Patients on prolonged antifungal therapy are still eligible if they are culture negative and biopsy negative in suspected residual radiographic lesions have stabilized or regressed and they meet other organ function criteria.
Show all 11Show fewer
- Prior total body irradiation, prior total abdominal or whole liver radiation
- Any medical or psychological condition or situation deemed by the PI to put the patient at increased risk of complications or noncompliance.
- Patients with curative treatment options.
- Patients for whom busulfan/ melphalan consolidation therapy following treatment with 131I-MIBG is planned.
- Patients for who CEM (carboplatin, etoposide, melphalan) therapy is administered within 30 days prior to 131I-MIBG therapy or for whom this therapy is planned within 30 days following administration of 131I-MIBG.
Trial locations and contacts
1 site in 1 country.
United States1 site
Nationwide Children's Hospital
Columbus, Ohio, 43205
About this study
Official title: A Phase II Study of <131>I-Metaiodobenzyguanidine (<131>I-MIBG) Therapy for Patients With MIBG Avid Tumors
This is a Phase II study for patients with MIBG avid tumors. The study is to determine the response rate to <131>I-MIBG in patients with de novo, relapsed or refractory neuroblastoma or other MIBG avid malignant tumors 42 days post MIBG therapy. It will also be evaluating the tolerability and safety of the study agent by evaluating the hematopoietic and non-hematopoietic toxicity of <131>I-MIBG therapy. Tumor response will be evaluated by comparing the patient's disease pre-treatment against the patient's day +42 post <131>I-MIBG treatment. The evaluations may include the following: <131>I-MIBG scan, CT or MRI, urine catecholamine, bone marrow analyses and any other tests considered standard of care for cancer evaluation. To be eligible for participation, patients must have tumors that are MIBG avid. Patients must also have a stem cell source for autologous rescue in the event of protracted therapy associated cytopenias. Peripheral stem cell collections are preferred as the hematopoietic cell source. Bone marrow harvests for a hematopoietic cell source is an alternative. This study will provide data for future clinical trials utilizing <131>I-MIBG therapies. A room on H12 has been prepared with lead lined walls, and many radiation safety components to accomodate this treatment. <131>I metaiodobenzlguanidine (<131>I-MIBG) is a radiopharmaceutical that concentrates within adrenomedullary tissue. The agent was initially used for tumor imaging due to its capability to locate pheochromocytomas, neuroblastomas and other neuroendocrine tumors. <131>I-MIBG was subsequently used as an therapeutic agent for these tumor types. Phase I and II therapeutic trials targeting neuroblastoma have reported response rates of 10-50%. Toxicities observed have been mainly hematopoietic, with ~50% of patients receiving 15mCi/kg requiring stem cell reinfusion. Observed non-hematopoietic toxicities have been mild. Most recently, trials have been conducted combining the study agent with myeloablative chemotherapy and stem cell reinfusion have been performed with initial response rates of ~50%.
- Conditions
- MIBG Avid Tumors
- Being tested
- MIBG
- Keywords
- Neuroblastoma
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MIBGDrug
Also called <131>I-Metaiodobenzylguanidine
| Study group | Role | What they receive |
|---|---|---|
| MIBG | Experimental | Research participants with MIBG avid tumors Receives: Drug: MIBG |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine the response rate to <131>I-MIBG treatment.
Responses will be defined by comparison to baseline studies obtained prior to study therapy. Disease status will be evaluated using CT/MRI scans, MIBG scans, plain films, as well as bone marrow aspiration and biopsy as dictated by the primary malignant diagnosis. Imaging modalities should be the same for pre and post MIBG assessments to facilitate response determination.
Measured: Response rate will be assessed at 42 days post therapy.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Mar 2015 (estimated)
- Study start1 Mar 2014
- Primary completion31 Dec 2025 (estimated)
- Study completion31 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MIBG Avid TumorsStudies of the same condition, open and under way.
- More trials run by Nationwide Children's HospitalThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 65 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Nationwide Children's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Mark A Ranalli, MD, Principal investigator, Nationwide Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02378428
- Sponsor's protocol ID
- IRB13-00656
- Other IDs
- NCH MIBG
- Acronym
- MIBG
- Status last verified
- May 2025
- First submitted
- 4 Mar 2014
- Last updated
- 13 May 2025
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 13 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of <131>I-Metaiodobenzyguanidine (<131>I-MIBG) Therapy for Patients With MIBG Avid Tumors
- ClinicalTrials.gov ID
- NCT02378428
- Acronym / sponsor ID
- MIBG / IRB13-00656
- Conditions
- MIBG Avid Tumors
- Keywords
- Neuroblastoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 65 participants (estimated)
- Lead sponsor
- Nationwide Children's HospitalUniversity, hospital or other
- Study start
- 1 Mar 2014
- Primary completion
- 31 Dec 2025 (estimated)
- Study completion
- 31 Dec 2025 (estimated)
- First posted
- 4 Mar 2015 (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 Phase II study for patients with MIBG avid tumors. The study is to determine the response rate to <131>I-MIBG in patients with de novo, relapsed or refractory neuroblastoma or other MIBG avid malignant tumors 42 days post MIBG therapy. It will also be evaluating the tolerability and safety of the study agent by evaluating the hematopoietic and non-hematopoietic toxicity of <131>I-MIBG therapy. Tumor response will be evaluated by comparing the patient's disease pre-treatment against the patient's day +42 post <131>I-MIBG treatment. The evaluations may include the following: <131>I-MIBG scan, CT or MRI, urine catecholamine, bone marrow analyses and any other tests considered standard of care for cancer evaluation. To be eligible for participation, patients must have tumors that are MIBG avid. Patients must also have a stem cell source for autologous rescue in the event of protracted therapy associated cytopenias. Peripheral stem cell collections are preferred as the hematopoietic cell source. Bone marrow harvests for a hematopoietic cell source is an alternative. This study will provide data for future clinical trials utilizing <131>I-MIBG therapies. A room on H12 has been prepared with lead lined walls, and many radiation safety components to accomodate this treatment. <131>I metaiodobenzlguanidine (<131>I-MIBG) is a radiopharmaceutical that concentrates within adrenomedullary tissue. The agent was initially used for tumor imaging due to its capability to locate pheochromocytomas, neuroblastomas and other neuroendocrine tumors. <131>I-MIBG was subsequently used as an therapeutic agent for these tumor types. Phase I and II therapeutic trials targeting neuroblastoma have reported response rates of 10-50%. Toxicities observed have been mainly hematopoietic, with ~50% of patients receiving 15mCi/kg requiring stem cell reinfusion. Observed non-hematopoietic toxicities have been mild. Most recently, trials have been conducted combining the study agent with myeloablative chemotherapy and stem cell reinfusion have been performed with initial response rates of ~50%.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| MIBG | Experimental | Drug: MIBGResearch participants with MIBG avid tumors |
Who may qualify
- Ages
- 12 months to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (28 listed)
- Patients must be between 12 months and 65 years at the time of enrollment
- Diagnosis: diagnosis of neuroblastoma or at the time of relapse by histology and/or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites
- Disease Status:
- Patients who receive greater than 12 mCi/kg are required to have stem cell rescue products harvested prior to study treatment.
- Performance Level and Life Expectancy: Patients must have a Lansky Play Scale17 of 60% (<16 yrs old), Karnofsky score 60% (>16 yrs old), or ECOG score of < or equal to 2 and a life expectancy of 2 months.
Key exclusion criteria (11 listed)
- Pregnancy or breast feeding
- Have undergone a prior allogeneic BMT.
- 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 prior to patient entry.
- Patients who are on hemodialysis.
- Hepatitis B surface antigen (+) or Hepatitis C positive in preceding six months.
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 |
|---|---|---|
| MIBGAlso: <131>I-Metaiodobenzylguanidine | Drug | MIBG |
Primary outcome
To determine the response rate to <131>I-MIBG treatment.
Time frame: Response rate will be assessed at 42 days post therapy.
Responses will be defined by comparison to baseline studies obtained prior to study therapy. Disease status will be evaluated using CT/MRI scans, MIBG scans, plain films, as well as bone marrow aspiration and biopsy as dictated by the primary malignant diagnosis. Imaging modalities should be the same for pre and post MIBG assessments to facilitate response determination.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 May 2025.
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.
Mark A Ranalli, MD principal investigator
Nationwide Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing MIBG in people with MIBG Avid Tumors, run by Nationwide Children's Hospital 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 28 inclusion and 11 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: under way, not looking for participants, as of the sponsor's last update on 13 May 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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?
To determine the response rate to <131>I-MIBG treatment. (time frame: Response rate will be assessed at 42 days post therapy.). 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 13 May 2025.