MIBG Therapy for Cancer

(MIBG Trial)

Overseen By Amy Yekisa

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Nationwide Children's Hospital

Must not be taking: Antivirals, Antibiotics, Antifungals

Disqualifiers: Pregnancy, Breastfeeding, Organ impairment, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 2

Duration: 6 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called <131>I-MIBG, a specialized medicine targeting certain cancer cells. It focuses on patients with specific tumors that appear on an MIBG scan, such as neuroblastoma, and aims to assess the tumors' response to the treatment. The study also evaluates the treatment's safety, particularly its side effects on the blood and other body parts. Suitable candidates for this trial have cancer that appears on MIBG scans and a backup plan for blood cell recovery, such as stored stem cells, in case the treatment affects their blood count. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group of people.

Do I have to stop taking my current medications for the MIBG Therapy trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss your specific medications with the study team to get a clear answer.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that the treatment \<131\>I-MIBG, used for tumors like neuroblastoma, has been tested for safety in other studies. These studies found that about half of the patients who received a dose of 15 mCi/kg required a stem cell reinfusion due to effects on their blood cells. This indicates that the treatment can lower blood cell counts, but stem cell support can manage this issue.

Side effects unrelated to blood cells have been mild, suggesting that most patients tolerate the treatment well. Some studies have also combined \<131\>I-MIBG with chemotherapy, and about half of the patients responded well to this combination. Overall, while some risks exist, the treatment has proven manageable with proper medical support. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

[^131I-MIBG](https://www.cancer.gov/about-cancer/treatment/clinical-trials/intervention/iobenguane-i-131) is unique because it uses a radioactive isotope, iodine-131, which specifically targets and destroys cancer cells with MIBG avid tumors, such as neuroblastoma. Unlike conventional treatments like chemotherapy and radiation, which can affect both healthy and cancerous cells, ^131I-MIBG zeroes in on cancer cells that take up the MIBG compound, sparing more healthy tissue. This targeted approach not only promises to be more effective but also potentially reduces side effects, making it a promising option for patients who have limited responses to standard treatments.

What evidence suggests that <131>I-MIBG might be an effective treatment for neuroblastoma?

Research shows that I-131 MIBG therapy, which participants in this trial will receive, can help treat neuroblastoma and other tumors that absorb MIBG. Studies indicate that the success of this treatment varies, with tumors shrinking or stopping growth in 10% to 50% of cases. When combined with chemotherapy and stem cell reinfusion, about 50% of patients initially respond well. However, the treatment can cause blood-related side effects, and around half of the patients might need stem cell reinfusion. Other side effects are usually mild. Overall, current evidence suggests a reasonable chance that this therapy will help reduce tumors. ^{2 3 4 6 7}

Who Is on the Research Team?

Mark Ranalli

Principal Investigator

Nationwide Children's Hospital

Are You a Good Fit for This Trial?

This trial is for people aged 1 to 65 with MIBG avid tumors, such as neuroblastoma. They should have recovered from previous treatments and not be pregnant or breastfeeding. Participants need a stem cell source ready for rescue in case of severe blood cell count drops due to treatment. Major organ function must be stable, and they can't have had certain prior radiation therapies or an active infection needing IV drugs.

Inclusion Criteria

I am between 1 year and 65 years old.

Evidence of MIBG avid disease as determined by diagnostic MIBG scan obtained within 4 weeks of study entry

My kidney function is normal based on my creatinine levels.

Exclusion Criteria

I am scheduled for busulfan/melphalan therapy after 131I-MIBG treatment.

I have had a bone marrow transplant from a donor.

There are treatments available that could potentially cure my condition.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive <131>I-MIBG therapy for MIBG avid tumors

6 weeks

1 visit (in-person) for treatment

Follow-up

Participants are monitored for safety and effectiveness after treatment, with evaluations at 42 days post therapy

6 weeks

1 visit (in-person) for evaluation

Extension

Participants may continue to be monitored for long-term safety and effectiveness

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• <131>I-MIBG

Trial Overview The study tests the effectiveness of <131>I-MIBG therapy on patients with new, relapsed, or resistant neuroendocrine tumors like neuroblastoma by measuring tumor response after 42 days. It also assesses safety regarding blood cell counts and other potential toxic effects. The process includes scans and various cancer evaluations before and after treatment.

How Is the Trial Designed?

Group I: MIBG Experimental Treatment 1 Intervention

Research participants with MIBG avid tumors

Who Is Running the Clinical Trial?

Nationwide Children's Hospital

Lead Sponsor

Trials

354

Recruited

5,228,000+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6066492/

Current Consensus on I-131 MIBG Therapy - PMC The objective response rate after auto-HCT was 57% of 16 patients, and the 10-year OS rate was only 6%. A similar European group evaluated the ...

2. jnm.snmjournals.org jnm.snmjournals.org/content/60/5/623

Efficacy and Safety of High-Specific-Activity 131I-MIBG ... HSA 131 I-MIBG offers multiple benefits, including sustained blood pressure control and tumor response in PPGL patients.

3. sciencedirect.com sciencedirect.com/science/article/pii/S2059702925004107

131I-mIBG therapy in relapsed/refractory neuroblastoma The 3-year overall survival was 55% (95% CI 33% to 73%) and event-free survival was 42% (95% CI 23% to 60%). Conclusion. The 131I-mIBG therapy plus melphalan is ...

4. frontiersin.org frontiersin.org/journals/oncology/articles/10.3389/fonc.2021.647361/full

Factors Modifying Outcome After MIBG Therapy in Children ... Despite the high number of neuroblastoma patients treated with MIBG-megatherapy, the mean tumor response rate in 25 studies was estimated at 32% ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC4581540/

Comparison of 123I-Metaiodobenzylguanidine (MIBG) and ... The 5-year event-free survival (EFS) and overall survival (OS) rates were 33.4 ± 3.6% and 45.6 ± 4.0% (N = 350). At post-induction, EFS (P = ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9150928/

A Safety and Feasibility Trial of 131I-MIBG in Newly ... MIBG labeled with iodine-131 (131I-MIBG) has activity against relapsed and newly diagnosed neuroblastoma. Early phase trials of 131I-MIBG in relapsed ...

7. jnm.snmjournals.org jnm.snmjournals.org/content/66/4/641

Tumor Dose–Response Relationship of [131I]MIBG Therapy ... This study demonstrated a clear correlation between tumor-absorbed dose and functional response in [ 131 I]MIBG therapy and proposes a target dose for response ...