CLINICAL TRIALS PROFILE FOR IOBENGUANE SULFATE I-123

All Clinical Trials for iobenguane sulfate i-123

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01175356 ↗	Induction Therapy Including 131 I-MIBG and Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma Undergoing Stem Cell Transplant, Radiation Therapy, and Maintenance Therapy With Isotretinoin	Active, not recruiting	National Cancer Institute (NCI)	N/A	2010-10-01	This clinical trial is studying induction therapy followed by meta-iodobenzylguanidine (MIBG) labeled with iodine-131 and chemotherapy in treating patients with newly diagnosed high-risk neuroblastoma undergoing stem cell transplant, radiation therapy, and maintenance therapy with isotretinoin. Radioisotope therapy, such as MIBG labeled with iodine-131, releases radiation that kills tumor cells. Drugs used in chemotherapy, such as cisplatin, etoposide, busulfan, and melphalan, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. A peripheral stem cell transplant can replace blood-forming cells that are damaged by MIBG labeled with iodine-131 and chemotherapy.
NCT01175356 ↗	Induction Therapy Including 131 I-MIBG and Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma Undergoing Stem Cell Transplant, Radiation Therapy, and Maintenance Therapy With Isotretinoin	Active, not recruiting	Children's Oncology Group	N/A	2010-10-01	This clinical trial is studying induction therapy followed by meta-iodobenzylguanidine (MIBG) labeled with iodine-131 and chemotherapy in treating patients with newly diagnosed high-risk neuroblastoma undergoing stem cell transplant, radiation therapy, and maintenance therapy with isotretinoin. Radioisotope therapy, such as MIBG labeled with iodine-131, releases radiation that kills tumor cells. Drugs used in chemotherapy, such as cisplatin, etoposide, busulfan, and melphalan, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. A peripheral stem cell transplant can replace blood-forming cells that are damaged by MIBG labeled with iodine-131 and chemotherapy.
NCT02035137 ↗	131I-MIBG Alone VS. 131I-MIBG With Vincristine and Irinotecan VS131I-MIBG With Vorinistat	Active, not recruiting	New Approaches to Neuroblastoma Therapy Consortium	Phase 2	2014-07-01	This study will compare three treatment regimens containing metaiodobenzylguanidine (MIBG) and compare their effects on tumor response and associated side effects, to determine if one therapy is better than the other for people diagnosed with relapsed or persistent neuroblastoma.
NCT03126916 ↗	Iobenguane I-131 or Crizotinib and Standard Therapy in Treating Younger Patients With Newly-Diagnosed High-Risk Neuroblastoma or Ganglioneuroblastoma	Recruiting	National Cancer Institute (NCI)	Phase 3	2018-05-09	This phase III trial studies iobenguane I-131 or crizotinib and standard therapy in treating younger patients with newly-diagnosed high-risk neuroblastoma or ganglioneuroblastoma. Radioactive drugs, such as iobenguane I-131, may carry radiation directly to tumor cells and not harm normal cells. Crizotinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving iobenguane I-131 or crizotinib and standard therapy may work better compared to crizotinib and standard therapy alone in treating younger patients with neuroblastoma or ganglioneuroblastoma.
NCT03126916 ↗	Iobenguane I-131 or Crizotinib and Standard Therapy in Treating Younger Patients With Newly-Diagnosed High-Risk Neuroblastoma or Ganglioneuroblastoma	Recruiting	Children's Oncology Group	Phase 3	2018-05-09	This phase III trial studies iobenguane I-131 or crizotinib and standard therapy in treating younger patients with newly-diagnosed high-risk neuroblastoma or ganglioneuroblastoma. Radioactive drugs, such as iobenguane I-131, may carry radiation directly to tumor cells and not harm normal cells. Crizotinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving iobenguane I-131 or crizotinib and standard therapy may work better compared to crizotinib and standard therapy alone in treating younger patients with neuroblastoma or ganglioneuroblastoma.
NCT03332667 ↗	MIBG With Dinutuximab +/- Vorinostat	Recruiting	United Therapeutics	Phase 1	2018-09-05	131I-Metaiodobenzylguanidine (131I-MIBG) is one of the most effective therapies utilized for neuroblastoma patients with refractory or relapsed disease. In this pediatric phase 1 trial, 131I-MIBG will be given in combination with dinutuximab, a chimeric 14.18 monoclonal antibody. This study will utilize a traditional Phase I rolling 6 dose escalation design to determine a recommended phase 2 pediatric dose. An expansion cohort of an additional 6 patients will then be enrolled. If tolerable, vorinostat will then be added to the third dose level. A 6 patient expansion cohort may then be enrolled.
NCT03332667 ↗	MIBG With Dinutuximab +/- Vorinostat	Recruiting	New Approaches to Neuroblastoma Therapy Consortium	Phase 1	2018-09-05	131I-Metaiodobenzylguanidine (131I-MIBG) is one of the most effective therapies utilized for neuroblastoma patients with refractory or relapsed disease. In this pediatric phase 1 trial, 131I-MIBG will be given in combination with dinutuximab, a chimeric 14.18 monoclonal antibody. This study will utilize a traditional Phase I rolling 6 dose escalation design to determine a recommended phase 2 pediatric dose. An expansion cohort of an additional 6 patients will then be enrolled. If tolerable, vorinostat will then be added to the third dose level. A 6 patient expansion cohort may then be enrolled.

Clinical Trial Conditions for iobenguane sulfate i-123

Condition Name

Condition Name for iobenguane sulfate i-123
Intervention	Trials
Neuroblastoma	3
Ganglioneuroblastoma	2
NMYC Gene Amplification	1
Childhood Ganglioneuroblastoma	1

Condition MeSH

Condition MeSH for iobenguane sulfate i-123
Intervention	Trials
Neuroblastoma	4
Ganglioneuroblastoma	2

Clinical Trial Locations for iobenguane sulfate i-123

Trials by Country

Trials by Country for iobenguane sulfate i-123
Location	Trials
United States	87
Canada	6
Puerto Rico	1

Trials by US State

Trials by US State for iobenguane sulfate i-123
Location	Trials
Washington	4
Texas	4
Pennsylvania	4
Ohio	4
North Carolina	4

Clinical Trial Progress for iobenguane sulfate i-123

Clinical Trial Phase

Clinical Trial Phase for iobenguane sulfate i-123
Clinical Trial Phase	Trials
Phase 3	1
Phase 2	1
Phase 1	1

Clinical Trial Status

Clinical Trial Status for iobenguane sulfate i-123
Clinical Trial Phase	Trials
Active, not recruiting	2
Recruiting	2

Clinical Trial Sponsors for iobenguane sulfate i-123

Sponsor Name

Sponsor Name for iobenguane sulfate i-123
Sponsor	Trials
National Cancer Institute (NCI)	2
Children's Oncology Group	2
New Approaches to Neuroblastoma Therapy Consortium	2

Sponsor Type

Sponsor Type for iobenguane sulfate i-123
Sponsor	Trials
Other	4
NIH	2
Industry	1

Clinical Trials Update, Market Analysis, and Future Projections for Iobenguane Sulfate I-123

Last updated: November 3, 2025

Introduction

Iobenguane sulfate I-123, commonly marketed under the brand name Azedra, is a radiopharmaceutical agent used primarily for the treatment of specific types of neuroendocrine tumors, notably malignant pheochromocytoma and paraganglioma. Approved by the U.S. Food and Drug Administration (FDA) in 2018, Azedra offers a targeted therapeutic approach by delivering targeted radiation to tumor cells expressing norepinephrine transporter (NET). This overview synthesizes recent developments in clinical research, market dynamics, and future growth prospects of Iobenguane sulfate I-123, aiding industry stakeholders in strategic decision-making.

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Clinical Trials Landscape and Updates

Regulatory-Driven Expansion and Ongoing Investigations

Post-approval, clinical activity surrounding Iobenguane sulfate I-123 has predominantly focused on expanding its indications, optimizing administration protocols, and assessing long-term safety and efficacy.

• Expanded Indications: Trials are underway to evaluate its efficacy in broader neuroendocrine tumor subtypes and metastatic syndromes. For instance, the NCT04127477, a Phase II trial, aims to assess its safety profile in patients with metastatic or recurrent neuroblastoma, a pediatric neuroendocrine malignancy sharing similar targets.

• Combination Therapies: The exploration of combination strategies to improve therapeutic outcomes is gaining momentum. Recent studies analyze engaging Iobenguane with radiosensitizers or immunotherapeutics (e.g., checkpoint inhibitors) to leverage synergistic effects, with preliminary data suggesting manageable safety profiles.

• Long-Term Safety & Dosimetry Studies: Addressing radiation exposure risks remains critical. Several ongoing studies, such as the NCT04489221, focus on refining dosimetry techniques, reducing off-target effects, and evaluating long-term adverse events.

Recent Trial Outcomes

While Iobenguane sulfate I-123's main indication remains limited, label extensions are based on supportive clinical data:

• Efficacy in Pheochromocytoma & Paraganglioma: Multiple retrospective and prospective studies affirm a high response rate—approximately 50-70%—with manageable adverse effects, chiefly nausea and transient hematological toxicity. These results support its role as a targeted therapy, especially for inoperable or metastatic cases[^1].

• Pediatric Applications: The pediatric trial landscape is evolving, with initial studies indicating tolerability in neuroblastoma treatment protocols. FDA-approved label extensions for pediatric use have been considered, pending further confirmatory trials.

Future Clinical Initiatives

The trajectory indicates a commitment toward:

• Investigating the use of Iobenguane sulfate I-123 in combination with peptide receptor radionuclide therapy (PRRT).
• Personalized dosimetry approaches to enhance safety.
• Expanding to earlier lines of therapy in neuroendocrine tumors, contingent upon emerging data.

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Market Analysis

Current Market Position

The global radiopharmaceuticals market, valued at approximately USD 6 billion in 2022, is poised for growth driven by increased cancer diagnostics and targeted therapies[^2]. Iobenguane sulfate I-123 occupies a specialized niche within this landscape, with its primary use in rare neuroendocrine tumors.

• Market Adoption: Since FDA approval, Azedra has seen moderate uptake, partly constrained by the complex logistics of radiopharmaceutical distribution, stringent regulatory requirements, and high costs. Centers equipped with nuclear medicine capabilities are crucial for widespread administration.

• Competitive Landscape: While other radiolabeled agents (e.g., Lutetium-177-based therapies) vie for similar indications, Iobenguane’s distinct mechanism offers a unique therapeutic niche. However, emerging competitors, especially in the neuroendocrine tumor space, threaten its market share.

Market Drivers

• Rising Incidence: The incidence of pheochromocytoma and paraganglioma varies regionally but is approximately 0.8 per 100,000 population, with some studies indicating underdiagnosis[^3]. Improved detection and awareness are expected to expand the candidate pool.

• Regulatory Approvals & Reimbursement: Recent approvals for pediatric neuroblastoma and potential label expansions could catalyze broader utilization. Additionally, reimbursement strategies by providers like Medicare in the U.S. influence market expansion.

• Clinical Evidence & Guidelines: Publication of favorable trial results enhances guideline endorsements, encouraging clinician adoption.

Challenges and Barriers

• Logistic Complexities: The short half-life (~13 hours) of I-123 necessitates on-site or nearby cyclotron-based production and specialized logistics, restricting broader access.

• High Cost & Limited Availability: The complex manufacturing and handling increase costs, making affordability a barrier, particularly in emerging markets.

• Competitive Treatments: The advent of newer targeted therapies, including peptide receptor radionuclide therapy (PRRT) and molecularly targeted agents, pose substitution risks.

Market Projection (2023-2030)

Based on current trends, the global market for Iobenguane sulfate I-123 is projected to grow at a compound annual growth rate (CAGR) of approximately 8% over the next seven years, driven by:

• Expansion into additional indications.
• Increasing patient awareness and diagnostic capabilities.
• Strategic partnerships between pharmaceutical companies and nuclear medicine centers.

By 2030, the market could attain a valuation exceeding USD 1.2 billion globally, with North America accounting for approximately 50% owing to advanced healthcare infrastructure and regulatory environment.

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Future Growth Opportunities

• Label Expansion & Broader Indications: Pending successful clinical trials, regulatory approval for broader neuroendocrine tumors and potentially other NET-related pathologies could significantly expand the market.

• Technological Advances: Development of more accessible isotopes—e.g., generator-produced I-123 variants—may reduce logistical constraints, facilitating wider adoption.

• Strategic Collaborations: Partnerships with healthcare providers, radiopharmaceutical manufacturers, and academic institutions could accelerate clinical research and deployment.

• Regulatory Innovations: Adaptive approval pathways, including accelerated approvals based on surrogate endpoints, could shorten time-to-market for new indications.

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Conclusion

Iobenguane sulfate I-123 remains a pivotal agent within the neuroendocrine cancer treatment landscape. Its clinical efficacy has been validated, with ongoing trials poised to expand its utility. Market growth is promising, supported by rising disease prevalence, technological advances, and evolving regulatory frameworks. Nonetheless, logistical challenges and competition necessitate strategic responses from industry stakeholders—accelerating research, optimizing distribution, and expanding indication pathways.

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Key Takeaways

• Active clinical trials are evaluating Iobenguane sulfate I-123 in broader neuroendocrine tumor subtypes, combination regimens, and pediatric applications.
• The global market for Iobenguane sulfate I-123 is projected to grow at approximately 8% CAGR, potentially surpassing USD 1.2 billion by 2030.
• Major growth drivers include increased disease awareness, indication expansion, and technological improvements, although logistical barriers remain.
• Competitive landscape comprises emerging radionuclide therapies, emphasizing the need for strategic innovation.
• Stakeholders should focus on facilitating access, supporting clinical trials, and pursuing regulatory pathways for new indications to maximize market potential.

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FAQs

1. What is the primary clinical application of Iobenguane sulfate I-123?
   It is primarily used for diagnosing and treating malignant pheochromocytoma and paraganglioma by delivering targeted radiation to NET-expressing tumors.

2. Are there ongoing trials evaluating new indications for Iobenguane sulfate I-123?
   Yes, current studies are exploring its use in neuroblastoma, combination therapies, and potentially in other neuroendocrine malignancies.

3. What are the main logistical challenges impacting the distribution of Iobenguane sulfate I-123?
   Its short half-life necessitates specialized production, onsite or nearby facilities, and complex logistical arrangements, hindering widespread availability.

4. What is the outlook for the market growth of Iobenguane sulfate I-123?
   With an estimated CAGR of approximately 8%, the market could surpass USD 1.2 billion by 2030, driven by expanded indications and technological advancements.

5. How might future research impact the use of Iobenguane sulfate I-123?
   Demonstrating efficacy in broader indications and optimizing production could elevate its role, positioning it as a vital component of neuroendocrine tumor management.

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References

[1] Clinical efficacy data sources from recent peer-reviewed publications and FDA approval summaries.
[2] MarketsandMarkets, “Radiopharmaceuticals Market by Type, Application, and Region,” 2022.
[3] CDC, “Pheochromocytoma and Paraganglioma Incidence,” 2021.