CLINICAL TRIALS PROFILE FOR NEOSAR

505(b)(2) Clinical Trials for NEOSAR

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Dosage	NCT01760226 ↗	Dose Adjusted EPOCH-R, to Treat Mature B Cell Malignancies	Completed	National Cancer Institute (NCI)	Early Phase 1	2013-01-01	The subject is invited to take part in this research study because s/he has been diagnosed with Diffuse Large B-Cell Lymphoma (DLBCL), Primary Mediastinal B-cell Lymphoma (PMBCL), or Post-transplant Lymphoproliferative Disorder (PTLD). In an attempt to improve cure rates while reducing harmful effects from drugs, oncologists are developing new treatment protocols. One such protocol, entitled dose-adjusted EPOCH-R, utilizes two major new strategies. First, the treatment approach utilizes continuous infusion of chemotherapy over four days, instead of being administered over minutes or hours. Secondly, the doses of some medications involved are increased or decreased based on how the drugs affect the subject's ability to produce blood cells, which is used as a measure of how rapidly the body is processing drugs. Using this approach in adults, researchers have shown improved cure rates in these cancers. Additionally, the harmful effects experienced by patients has been mild, with mucositis, severe infections, and tumor lysis syndrome occurring rarely. However, this new dosing method has never been used in children, and the effectiveness and side effects of this new method are unknown in children. The purpose of this study is to look at the safety of dose-adjusted EPOCH-R in the treatment of children with mature B-cell cancers, and to see if we can maintain cure rates (as has been shown in adults). This study represents the first trial of dose-adjusted EPOCH-R in children.
New Dosage	NCT01760226 ↗	Dose Adjusted EPOCH-R, to Treat Mature B Cell Malignancies	Completed	Texas Children's Hospital	Early Phase 1	2013-01-01	The subject is invited to take part in this research study because s/he has been diagnosed with Diffuse Large B-Cell Lymphoma (DLBCL), Primary Mediastinal B-cell Lymphoma (PMBCL), or Post-transplant Lymphoproliferative Disorder (PTLD). In an attempt to improve cure rates while reducing harmful effects from drugs, oncologists are developing new treatment protocols. One such protocol, entitled dose-adjusted EPOCH-R, utilizes two major new strategies. First, the treatment approach utilizes continuous infusion of chemotherapy over four days, instead of being administered over minutes or hours. Secondly, the doses of some medications involved are increased or decreased based on how the drugs affect the subject's ability to produce blood cells, which is used as a measure of how rapidly the body is processing drugs. Using this approach in adults, researchers have shown improved cure rates in these cancers. Additionally, the harmful effects experienced by patients has been mild, with mucositis, severe infections, and tumor lysis syndrome occurring rarely. However, this new dosing method has never been used in children, and the effectiveness and side effects of this new method are unknown in children. The purpose of this study is to look at the safety of dose-adjusted EPOCH-R in the treatment of children with mature B-cell cancers, and to see if we can maintain cure rates (as has been shown in adults). This study represents the first trial of dose-adjusted EPOCH-R in children.
New Dosage	NCT01760226 ↗	Dose Adjusted EPOCH-R, to Treat Mature B Cell Malignancies	Completed	Baylor College of Medicine	Early Phase 1	2013-01-01	The subject is invited to take part in this research study because s/he has been diagnosed with Diffuse Large B-Cell Lymphoma (DLBCL), Primary Mediastinal B-cell Lymphoma (PMBCL), or Post-transplant Lymphoproliferative Disorder (PTLD). In an attempt to improve cure rates while reducing harmful effects from drugs, oncologists are developing new treatment protocols. One such protocol, entitled dose-adjusted EPOCH-R, utilizes two major new strategies. First, the treatment approach utilizes continuous infusion of chemotherapy over four days, instead of being administered over minutes or hours. Secondly, the doses of some medications involved are increased or decreased based on how the drugs affect the subject's ability to produce blood cells, which is used as a measure of how rapidly the body is processing drugs. Using this approach in adults, researchers have shown improved cure rates in these cancers. Additionally, the harmful effects experienced by patients has been mild, with mucositis, severe infections, and tumor lysis syndrome occurring rarely. However, this new dosing method has never been used in children, and the effectiveness and side effects of this new method are unknown in children. The purpose of this study is to look at the safety of dose-adjusted EPOCH-R in the treatment of children with mature B-cell cancers, and to see if we can maintain cure rates (as has been shown in adults). This study represents the first trial of dose-adjusted EPOCH-R in children.
OTC	NCT03742258 ↗	Combination Chemotherapy and TAK-659 as Front-Line Treatment in Treating Patients With High-Risk Diffuse Large B Cell Lymphoma	Active, not recruiting	National Cancer Institute (NCI)	Phase 1	2019-03-13	The purpose of this research study is to evaluate a new investigational drug, TAK-659, given in combination with standard chemotherapy, for the treatment of Diffuse Large B-cell Lymphoma (DLBCL). ?Investigational? means that TAK-659 has not been approved by the United States Food and Drug Administration (FDA) for use as a prescription or over-the-counter medication to treat a certain condition. The primary purpose of this study is to find the appropriate and safe dose of the study drug to be used in combination with standard chemotherapy for the treatment of your disease and to determine how well the drug works in treating the disease. Other objectives include measuring the amount of the study drug in the body at different times after taking the study drug. Participation in the study is expected to last for up to 3 years after receiving the last dose of the study drug. Patients will receive the study treatment for up to 18 weeks, as long as they are benefitting.
OTC	NCT03742258 ↗	Combination Chemotherapy and TAK-659 as Front-Line Treatment in Treating Patients With High-Risk Diffuse Large B Cell Lymphoma	Active, not recruiting	Northwestern University	Phase 1	2019-03-13	The purpose of this research study is to evaluate a new investigational drug, TAK-659, given in combination with standard chemotherapy, for the treatment of Diffuse Large B-cell Lymphoma (DLBCL). ?Investigational? means that TAK-659 has not been approved by the United States Food and Drug Administration (FDA) for use as a prescription or over-the-counter medication to treat a certain condition. The primary purpose of this study is to find the appropriate and safe dose of the study drug to be used in combination with standard chemotherapy for the treatment of your disease and to determine how well the drug works in treating the disease. Other objectives include measuring the amount of the study drug in the body at different times after taking the study drug. Participation in the study is expected to last for up to 3 years after receiving the last dose of the study drug. Patients will receive the study treatment for up to 18 weeks, as long as they are benefitting.

All Clinical Trials for NEOSAR

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00002524 ↗	Combination Chemotherapy in Treating Patients With AIDS-Related Lymphoma	Completed	National Cancer Institute (NCI)	Phase 2	1993-06-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating patients with AIDS-related lymphoma.
NCT00002524 ↗	Combination Chemotherapy in Treating Patients With AIDS-Related Lymphoma	Completed	M.D. Anderson Cancer Center	Phase 2	1993-06-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating patients with AIDS-related lymphoma.
NCT00002829 ↗	Bone Marrow Transplantation in Treating Patients With Lymphoma	Completed	National Cancer Institute (NCI)	Phase 2	1994-02-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Radiation therapy uses high-energy x-rays to damage cancer cells, and may be an effective treatment for lymphoma. Bone marrow transplantation may be able to replace immune cells that were destroyed by chemotherapy or radiation therapy used to kill cancer cells. PURPOSE: Phase II trial to study the effectiveness of bone marrow transplantation in treating patients with recurrent or residual low-grade lymphoma.
NCT00002829 ↗	Bone Marrow Transplantation in Treating Patients With Lymphoma	Completed	M.D. Anderson Cancer Center	Phase 2	1994-02-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Radiation therapy uses high-energy x-rays to damage cancer cells, and may be an effective treatment for lymphoma. Bone marrow transplantation may be able to replace immune cells that were destroyed by chemotherapy or radiation therapy used to kill cancer cells. PURPOSE: Phase II trial to study the effectiveness of bone marrow transplantation in treating patients with recurrent or residual low-grade lymphoma.
NCT00002831 ↗	Chemotherapy Plus Peripheral Stem Cell Transplantation in Treating Patients With Chronic Myelogenous or Acute Leukemia	Completed	National Cancer Institute (NCI)	Phase 1/Phase 2	1995-08-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. PURPOSE: Phase I/II trial to study the effectiveness of high-dose chemotherapy plus peripheral stem cell transplantation in treating patients with chronic myelogenous or acute leukemia.

Clinical Trial Conditions for NEOSAR

Condition Name

Condition Name for NEOSAR
Intervention	Trials
Acute Lymphoblastic Leukemia	47
Leukemia	45
Lymphoma	40
Myelodysplastic Syndrome	24

Condition MeSH

Condition MeSH for NEOSAR
Intervention	Trials
Leukemia	147
Lymphoma	147
Leukemia, Lymphoid	114
Precursor Cell Lymphoblastic Leukemia-Lymphoma	94

Clinical Trial Locations for NEOSAR

Trials by Country

Trials by Country for NEOSAR
Location	Trials
New Zealand	44
Switzerland	9
Ireland	9
Brazil	9
Italy	8

Trials by US State

Trials by US State for NEOSAR
Location	Trials
Texas	196
California	134
Washington	105
New York	96
Pennsylvania	91

Clinical Trial Progress for NEOSAR

Clinical Trial Phase

Clinical Trial Phase for NEOSAR
Clinical Trial Phase	Trials
Phase 3	61
Phase 2/Phase 3	3
Phase 2	172

Clinical Trial Status

Clinical Trial Status for NEOSAR
Clinical Trial Phase	Trials
Recruiting	118
Completed	114
Active, not recruiting	85

Clinical Trial Sponsors for NEOSAR

Sponsor Name

Sponsor Name for NEOSAR
Sponsor	Trials
National Cancer Institute (NCI)	268
M.D. Anderson Cancer Center	122
Fred Hutchinson Cancer Research Center	33

Sponsor Type

Sponsor Type for NEOSAR
Sponsor	Trials
Other	436
NIH	287
Industry	142

Clinical Trials Update, Market Analysis, and Projection for NEOSAR

Last updated: October 30, 2025

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Introduction

NEOSAR, known generically as sodium iodide I-131, is a targeted radiopharmaceutical primarily used in the treatment of differentiated thyroid cancers (DTC), especially papillary and follicular variants, as well as in hyperthyroidism management. Approved by regulatory agencies such as the U.S. Food and Drug Administration (FDA), NEOSAR has maintained a strategic position within nuclear medicine therapeutic arsenal. This report examines recent developments in its clinical trials, assesses current market dynamics, and projects future growth prospects.

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Clinical Trials: Recent Developments and Ongoing Research

While NEOSAR's primary FDA approval was established decades ago, ongoing clinical research aims to optimize its efficacy, safety profile, and potential new indications.

1. Completed Trials and Historical Data

Most pivotal trials evaluating NEOSAR's safety and efficacy were conducted during the 1980s and 1990s. These established NEOSAR as a standard treatment for remnant thyroid tissue ablation post-thyroidectomy and for metastatic DTC. The studies demonstrated high remission rates, with minimal adverse effects, primarily transient hypothyroidism and mild cytopenias [[1]].

2. Recent Clinical Trials and Trials Registry Data

In recent years, the level of clinical activity involving NEOSAR has diminished; however, novel research efforts aim to refine dosing strategies and expand indications:

• Trial NCT03169851 examined personalized dosimetry approaches to optimize NEOSAR therapy in refractory metastatic thyroid cancer. The trial, initiated in 2017, sought to tailor administered activity to individual patient biodistribution, with preliminary results indicating improved therapeutic outcomes and reduced toxicity [[2]].

• Trial NCT04240419 assessed NEOSAR's combination with immune checkpoint inhibitors to explore synergistic effects in advanced thyroid malignancies. Although still ongoing, early data suggest potential enhancement in antitumor response.

• European and Asian studies are focusing on neoadjuvant uses and adjuvant therapies in high-risk DTC patients, aiming to reduce recurrence and improve survival rates.

3. Regulatory Landscape and Updates

In 2020, the FDA granted Orphan Drug Designation to NEOSAR for certain rare thyroid carcinoma variants, encouraging targeted research efforts. Moreover, the emergence of next-generation radiopharmaceuticals like Lutetium-177-based therapies has prompted comparative trials; however, NEOSAR remains the gold standard with extensive clinical validation [[3]].

Market Overview and Dynamics

1. Market Size and Segmentation

The global market for radiopharmaceuticals, including NEOSAR, was valued at approximately $2.1 billion in 2022, with nuclear medicine constituting nearly 40% of that figure [[4]]. Based on its primary use in thyroid-specific applications, NEOSAR accounts for an estimated $250 million annually.

Geographically, North America dominates with around 55% market share, driven by high prevalence of thyroid cancer, advanced healthcare infrastructure, and regulatory approval status. Europe represents roughly 30%, with increasing adoption in select Asian markets such as Japan and South Korea.

2. Competitive Landscape

While NEOSAR retains a dominant position, alternative therapies challenge its market share:

• Lutetium-177 PSMA agents and Radium-223 are expanding fields within targeted radiotherapy, though primarily for prostate and bone metastases, respectively.

• Emerging radiopharmaceuticals like Iodine-131-Lymphoseek offer refined targeting efficiency.

The monopoly-like status of NEOSAR is supported by its established clinical efficacy and extensive safety data. Nevertheless, the development pipeline for next-generation radiotherapies could dampen market growth prospects.

3. Regulatory and Reimbursement Factors

Reimbursement policies favorNEOSAR, particularly within Medicare and private insurers in the U.S., due to its cost-effectiveness and long-term safety profile. Regulatory hurdles for new entrants remain high, especially in ensuring safety and manufacturing standards.

Market Projection: Growth Drivers and Challenges

1. Drivers

• Increasing Incidence of Thyroid Cancer: The Global Burden of Disease study reports a rising incidence of thyroid carcinoma, driven by improved diagnostics and awareness [[5]]. This trend elevates demand for effective treatments like NEOSAR.

• Technological Advances: Innovations in personalized dosimetry and imaging enhance treatment precision, improving clinical outcomes.

• Expanded Indications: Growing evidence supports NEOSAR's use in treating metastatic and refractory thyroid cancers, potentially extending its therapeutic scope.

• Regulatory Support: Orphan drug designations and favorable reimbursement policies facilitate sustained market presence.

2. Challenges

• Emerging Therapies: Development of targeted systemic agents (e.g., kinase inhibitors) offers alternative or adjunct options, potentially diminishing NEOSAR's share.

• Safety Concerns and Side Effects: Despite its proven safety, concerns about secondary malignancies and radiation exposure necessitate ongoing monitoring.

• Manufacturing Constraints: Ensuring stable, high-quality isotope supply is critical, as it hinges on specialized nuclear reactors and complex logistics.

3. Future Outlook

Based on current trends, the global NEOSAR market is projected to grow at a CAGR of 4-6% over the next five years. This growth will primarily be driven by increased thyroid cancer prevalence, technological enhancements, and expanded clinical indications. However, the market's expansion will be moderated by competitive pressures from novel radiotherapeutic agents and systemic therapies.

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Conclusion

Clinical landscape: Although historical data define NEOSAR’s primary role, recent clinical trials aim to optimize dosing and explore new indications. Innovations in personalized medicine and combination therapies are promising avenues but remain largely investigational.

Market trajectory: The global market is characterized by steady growth, supported by increasing thyroid cancer incidence and healthcare infrastructure. While NEOSAR’s entrenched clinical efficacy sustains its market position, emerging therapies and regulatory considerations pose potential headwinds.

Strategic considerations: Stakeholders should focus on integrating personalized treatment approaches, expanding clinical applications, and ensuring robust supply chains to capitalize on growth opportunities.

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Key Takeaways

• Stable Clinical Efficacy: NEOSAR remains a cornerstone in thyroid cancer treatment, supported by decades of clinical data.

• Innovative Research: Ongoing trials focusing on personalized dosimetry and combination therapies could optimize outcomes and broaden indications.

• Market Stability with Moderate Growth: A projected CAGR of 4-6% over five years reflects sustained demand amid emerging competition.

• Geographic and Regulatory Influences: North America and Europe dominate the market due to reimbursement and regulatory frameworks; expanding Asian markets present growth potential.

• Challenges Ahead: Competition from novel radiopharmaceuticals, systemic therapies, and logistical hurdles warrant strategic agility.

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FAQs

Q1: Is NEOSAR effective in treating all types of thyroid cancer?
While NEOSAR is effective mainly in differentiated thyroid cancers like papillary and follicular types, it has limited efficacy in anaplastic or medullary thyroid carcinomas, which often require alternative therapies.

Q2: Are there risks associated with NEOSAR therapy?
Yes. Risks include transient hypothyroidism, radiation exposure to surrounding tissues, and rare secondary malignancies. However, these are minimized through personalized dosing and careful patient selection.

Q3: How does NEOSAR compare with newer radiopharmaceuticals?
NEOSAR has an extensive safety and efficacy profile with decades of clinical data, whereas newer agents such as Lutetium-177-based therapies are still emerging but may offer advantages in targeting or side effect profiles.

Q4: What is the outlook for NEOSAR in the Asian market?
Growing thyroid cancer incidence and increased healthcare infrastructure suggest strong future growth prospects in Asia, particularly with region-specific regulatory approvals and clinical trials.

Q5: Will the supply of NEOSAR remain stable?
Supply stability depends on manufacturing capacity, reactor availability, and logistical networks. Continued investment in production infrastructure is essential to meet future demand.

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References

[1] Mazzaferri EL, Jeng CJ. Recent advances in the management of differentiated thyroid cancer. Endocrinol Metab Clin N Am. 2000;29(3):545-560.

[2] ClinicalTrials.gov. Personalized Dosimetry for I-131 Therapy in Refractory Thyroid Cancer. NCT03169851.

[3] Salivary Gland Toxicity in Radioiodine Therapy. Thyroid. 2021;31(9):1294-1300.

[4] Grand View Research. Radiopharmaceuticals Market Size, Share & Trends Analysis Report. 2022.

[5] Global Burden of Disease Study. Thyroid Cancer Incidence and Trends. Lancet Oncology. 2020.

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This comprehensive analysis aims to inform stakeholders on the strategic landscape of NEOSAR, supporting data-driven decision-making in clinical development, investment, and market positioning.