CLINICAL TRIALS PROFILE FOR ETOPOSIDE

505(b)(2) Clinical Trials for ETOPOSIDE

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 Combination	NCT00186888 ↗	Study of Treatment for Patients With Cancer of the Eye -Retinoblastoma	Active, not recruiting	National Cancer Institute (NCI)	Phase 3	2005-04-07	Retinoblastoma is a childhood cancer which affects the retina of the eye. The retina is the light sensitive layer of tissue that lines the back of the eyeball; sends visual messages through the optic nerve to the brain. When only one eye is affected, this is known as unilateral retinoblastoma and when both eyes are affected, it is called bilateral retinoblastoma. Treatment for retinoblastoma is individualized for each patient and is based on the form and the stage of the disease (inside the eye or has moved outside). The main goal is always to cure the cancer, and save the life of the child. Treatments are also designed with the hope of saving the vision, while completely destroying the tumor. Therapies may involve surgery, chemotherapy, radiation, and other treatments called focal treatments. Focal treatments may be laser therapy, freezing, or heat treatments meant to shrink and kill the tumor. In this study, researchers want to investigate how different participants respond to different therapies that are individualized specifically for them. Participants will be divided into three main groups, depending on whether the disease is unilateral or bilateral, and the stage of the disease. One of the main objectives of the study is to investigate how advanced tumors in children with bilateral disease respond to a new combination of chemotherapy with topotecan and vincristine, with G-CSF support. In order to improve results, some children with very advanced disease may receive carboplatin chemotherapy given around the eye at the same time that they receive topotecan by vein. Also, because children with retinoblastoma are diagnosed so early in life and the vision may be significantly impaired, this study will investigate how children develop and how the brain adjusts and compensates for the visual deficits. Finally, this study also investigates the biology of retinoblastoma, in order to understand better how this cancer develops.
New Combination	NCT00186888 ↗	Study of Treatment for Patients With Cancer of the Eye -Retinoblastoma	Active, not recruiting	St. Jude Children's Research Hospital	Phase 3	2005-04-07	Retinoblastoma is a childhood cancer which affects the retina of the eye. The retina is the light sensitive layer of tissue that lines the back of the eyeball; sends visual messages through the optic nerve to the brain. When only one eye is affected, this is known as unilateral retinoblastoma and when both eyes are affected, it is called bilateral retinoblastoma. Treatment for retinoblastoma is individualized for each patient and is based on the form and the stage of the disease (inside the eye or has moved outside). The main goal is always to cure the cancer, and save the life of the child. Treatments are also designed with the hope of saving the vision, while completely destroying the tumor. Therapies may involve surgery, chemotherapy, radiation, and other treatments called focal treatments. Focal treatments may be laser therapy, freezing, or heat treatments meant to shrink and kill the tumor. In this study, researchers want to investigate how different participants respond to different therapies that are individualized specifically for them. Participants will be divided into three main groups, depending on whether the disease is unilateral or bilateral, and the stage of the disease. One of the main objectives of the study is to investigate how advanced tumors in children with bilateral disease respond to a new combination of chemotherapy with topotecan and vincristine, with G-CSF support. In order to improve results, some children with very advanced disease may receive carboplatin chemotherapy given around the eye at the same time that they receive topotecan by vein. Also, because children with retinoblastoma are diagnosed so early in life and the vision may be significantly impaired, this study will investigate how children develop and how the brain adjusts and compensates for the visual deficits. Finally, this study also investigates the biology of retinoblastoma, in order to understand better how this cancer develops.
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.

All Clinical Trials for ETOPOSIDE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000660 ↗	Phase I Study of Weekly Oral VP-16 for AIDS-Associated Kaposi's Sarcoma	Completed	Bristol-Myers Squibb	Phase 1	1969-12-31	To define the toxicity and maximum-tolerated dose of weekly oral etoposide (VP-16) in patients with AIDS-related Kaposi's sarcoma; to determine the clinical pharmacology of orally administered VP-16 in AIDS patients. A secondary objective is to obtain preliminary data for determining the effect of oral VP-16 on Kaposi's sarcoma. VP-16 is an antitumor agent. Previous problems with VP-16 include the route of administration and the toxicities. VP-16 has been given intravenously for 3 consecutive days in a 21-day cycle for lung cancer and testicular cancer. VP-16 has also been used in lymphoma therapy. Oral VP-16 would eliminate the need for an intravenous catheter and so a patient could avoid the pain, inconvenience, and potential complications associated with medications administered intravenously. The relative ease of outpatient administration and the potentially significant antitumor activity of oral VP-16 motivates this study. The possibility of weekly drug administration is the other focus of this study.
NCT00000660 ↗	Phase I Study of Weekly Oral VP-16 for AIDS-Associated Kaposi's Sarcoma	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1	1969-12-31	To define the toxicity and maximum-tolerated dose of weekly oral etoposide (VP-16) in patients with AIDS-related Kaposi's sarcoma; to determine the clinical pharmacology of orally administered VP-16 in AIDS patients. A secondary objective is to obtain preliminary data for determining the effect of oral VP-16 on Kaposi's sarcoma. VP-16 is an antitumor agent. Previous problems with VP-16 include the route of administration and the toxicities. VP-16 has been given intravenously for 3 consecutive days in a 21-day cycle for lung cancer and testicular cancer. VP-16 has also been used in lymphoma therapy. Oral VP-16 would eliminate the need for an intravenous catheter and so a patient could avoid the pain, inconvenience, and potential complications associated with medications administered intravenously. The relative ease of outpatient administration and the potentially significant antitumor activity of oral VP-16 motivates this study. The possibility of weekly drug administration is the other focus of this study.
NCT00000807 ↗	Phase II Evaluation of Low-Dose Oral Etoposide for the Treatment of Relapsed or Progressed AIDS-Related Kaposi's Sarcoma After Systemic Chemotherapy	Completed	Bristol-Myers Squibb	Phase 2	1969-12-31	To assess the toxicity, tumor response rate, and effect on quality of life of daily low-dose etoposide administered for 7 consecutive days every other week in patients with AIDS-related Kaposi's sarcoma that has relapsed or progressed after systemic chemotherapy. Etoposide may be at least as, or even more, effective and less myelotoxic when given in low doses over prolonged periods of time.
NCT00000807 ↗	Phase II Evaluation of Low-Dose Oral Etoposide for the Treatment of Relapsed or Progressed AIDS-Related Kaposi's Sarcoma After Systemic Chemotherapy	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 2	1969-12-31	To assess the toxicity, tumor response rate, and effect on quality of life of daily low-dose etoposide administered for 7 consecutive days every other week in patients with AIDS-related Kaposi's sarcoma that has relapsed or progressed after systemic chemotherapy. Etoposide may be at least as, or even more, effective and less myelotoxic when given in low doses over prolonged periods of time.
NCT00001270 ↗	Feasibility Study of Interleukin 1-Alpha With Ifosfamide, CBDCA, and Etoposide With Autologous Bone Marrow Transplant in Metastatic Carcinoma and Lymphoma	Completed	National Cancer Institute (NCI)	Phase 1	1991-06-01	This is a phase I/II study of interleukin-1, G-CSF and high dose ICE chemotherapy with autologous bone marrow transplant in patients with relapsed breast, testicular and lymphoid cancers. The initial goal of this study was to define the toxicity of interleukin-1 administered for 7 days prior to ICE chemotherapy. A total of 22 patients have been treated with IL-1 and ICE and results showed a more rapid engraftment (4.5 days) with IL-1. A second cohort of 18 patients also received G-CSF and engraftment was further shortened in some subgroups. Overall, the median time to engraftment was 16 days with both IL-1 and G-CSF. Accrual will continue to further define the toxicity and efficacy of this regimen.

Clinical Trial Conditions for ETOPOSIDE

Condition Name

Condition Name for ETOPOSIDE
Intervention	Trials
Lymphoma	211
Leukemia	128
Small Cell Lung Cancer	80
Lung Cancer	72

Condition MeSH

Condition MeSH for ETOPOSIDE
Intervention	Trials
Lymphoma	507
Leukemia	295
Small Cell Lung Carcinoma	293
Lung Neoplasms	293

Clinical Trial Locations for ETOPOSIDE

Trials by Country

Trials by Country for ETOPOSIDE
Location	Trials
Canada	701
Korea, Republic of	92
Switzerland	88
Netherlands	81
Belgium	81

Trials by US State

Trials by US State for ETOPOSIDE
Location	Trials
California	339
New York	318
Texas	300
Ohio	271
Illinois	267

Clinical Trial Progress for ETOPOSIDE

Clinical Trial Phase

Clinical Trial Phase for ETOPOSIDE
Clinical Trial Phase	Trials
PHASE4	3
PHASE3	15
PHASE2	90

Clinical Trial Status

Clinical Trial Status for ETOPOSIDE
Clinical Trial Phase	Trials
Completed	685
Recruiting	357
Unknown status	176

Clinical Trial Sponsors for ETOPOSIDE

Sponsor Name

Sponsor Name for ETOPOSIDE
Sponsor	Trials
National Cancer Institute (NCI)	505
Children's Oncology Group	86
M.D. Anderson Cancer Center	47

Sponsor Type

Sponsor Type for ETOPOSIDE
Sponsor	Trials
Other	2241
NIH	527
Industry	511

Clinical Trials Update, Market Analysis, and Projection for Etoposide

Last updated: October 28, 2025

Introduction

Etoposide (also known as VP-16) remains a cornerstone chemotherapeutic agent in the treatment of various malignancies, notably small cell lung cancer, testicular cancer, and certain leukemias. Originally approved by the U.S. Food and Drug Administration (FDA) in the 1980s, its clinical profile continues to evolve amid ongoing research, regulatory updates, and market dynamics. This report synthesizes recent clinical trial developments, current market trends, and future projections for Etoposide, offering strategic insights for stakeholders within the pharmaceutical and biotech sectors.

Clinical Trials Landscape: Recent Developments

Phase and Scope of Ongoing Trials

As of late 2023, Etoposide is primarily evaluated within combination regimens in phase II and III trials. Notably, clinical investigations focus on refractory or relapsed cancers, leveraging Etoposide’s known efficacy in combination therapies. There are approximately 20 active clinical trials registered globally, predominantly sponsored by academic institutions and pharmaceutical companies seeking to optimize its therapeutic index.

Key Clinical Trial Highlights

1. Combination Therapies in Lung and Ovarian Cancers: Several studies examine Etoposide combined with immune checkpoint inhibitors (e.g., Pembrolizumab) to enhance antitumor responses in non-small cell lung cancer (NSCLC) and ovarian carcinoma. Preliminary data suggest improved progression-free survival but warrant further validation [2].

2. Biomarker-Driven Approaches: Trials are increasingly incorporating molecular profiling to identify patient populations most likely to benefit from Etoposide-based regimens, aligning with personalized medicine trends.

3. Novel Formulations and Delivery Mechanisms: Investigations into nanoparticle and liposomal formulations aim to improve drug targeting, reduce systemic toxicity, and enhance bioavailability—potentially extending Etoposide’s clinical utility.

Regulatory and Safety Updates

While Etoposide’s patent status has long expired, recent regulatory discussions involve revisiting label indications, notably for pediatric cancers and combination protocols. Post-market surveillance continues to affirm its safety profile; however, risk factors such as secondary malignancies and myelosuppression necessitate ongoing monitoring.

Market Analysis: Current Dynamics and Trends

Market Size and Segmentation

Globally, Etoposide is supplied predominantly as injectable and capsule formulations, with regional markets in North America, Europe, and Asia-Pacific. In 2022, the estimated global Etoposide market was valued at approximately USD 500 million, with a compound annual growth rate (CAGR) of around 4.2% projected over the next five years [1]. The key segments include:

• Hospital-based chemotherapy departments (primary consumer)
• Oncology specialty clinics (growing segment owing to targeted therapies)
• Generic pharmaceutical manufacturers (main suppliers post-patent expiry)

Competitive Landscape

The market is highly consolidated among generic manufacturers such as Teva, Sagent, and Pfizer. The absence of patent exclusivity fosters price competition and limits premium pricing strategies. Nonetheless, ongoing trials exploring novel combinations could bolster demand, especially if Etoposide gains approval for new indications.

Market Drivers and Constraints

• Drivers: Rising cancer incidence, expanding indications for combination regimens, increased adoption in developing regions, and ongoing clinical research.
• Constraints: Competition from newer targeted agents and immunotherapies, toxicity concerns limiting use in some patient groups, and manufacturing challenges in ensuring consistent bioavailability of formulations.

Market Projection and Future Outlook

Forecast for 2023-2028

The Etoposide market is expected to grow modestly, driven by its extensive use in combination therapy and expanding clinical trials. The CAGR is projected at approximately 4% to 5%, reaching an estimated USD 650-700 million by 2028. This growth will be moderated by the increasing availability of targeted molecular agents and immunotherapies that are replacing traditional chemotherapies in certain indications.

Potential Growth Opportunities

• Combination with Immunotherapies: Positive preliminary data suggest potential for Etoposide in enhancing immunotherapy efficacy, which could catalyze new combination approvals.
• Developments in Formulation Technologies: Liposomal and nanoparticle formulations may permit safer, more effective delivery, expanding market reach.
• Emerging Markets: Rising cancer burdens in Asia-Pacific countries present significant growth opportunity, especially as healthcare infrastructure improves.

Risks and Challenges

• Slow adoption in markets favoring targeted therapies.
• Regulatory hurdles for new formulations or expanded indications.
• Patent expirations limiting pricing leverage and R&D investment incentives.

Strategic Insights for Stakeholders

• Pharmaceutical companies should monitor ongoing trials for opportunity to reposition Etoposide in novel therapeutic regimens or formulations.
• Investors should weigh the potential upside from combination therapies and niche indications against competitive pressures.
• Regulatory entities may consider pathways to optimize access, especially for pediatric or refractory patient populations.

Key Takeaways

• Etoposide remains a foundational chemotherapeutic agent, with ongoing clinical research refining its role in combination therapies, notably with immuno-oncology agents.
• The global market, valued at approximately USD 500 million in 2022, is poised for modest growth driven by expanding indications and technological innovations.
• Innovations in drug delivery and precision medicine approaches are potential catalysts for market expansion.
• Competitive pressures from targeted therapies necessitate strategic positioning, including exploring new combinations and formulations.
• The evolving regulatory landscape and regional market dynamics define potential growth pathways and challenges.

FAQs

1. What are the main indications for Etoposide today?
   Etoposide is primarily indicated for small cell lung cancer, testicular cancer, and certain leukemias, often as part of combination chemotherapy protocols.

2. Are there ongoing efforts to improve Etoposide formulations?
   Yes, research into liposomal and nanoparticle formulations aims to enhance targeting, reduce toxicity, and improve therapeutic efficacy.

3. How does the combination of Etoposide with immunotherapy agents like Pembrolizumab impact clinical outcomes?
   Early studies suggest potential improvements in response rates and progression-free survival, though conclusive evidence from large-scale trials is pending.

4. What are the key challenges facing Etoposide’s market growth?
   Competition from newer targeted therapies, toxicity concerns, and patent expiration leading to generics are primary challenges.

5. What is the outlook for Etoposide in emerging markets?
   Growing cancer prevalence and improved healthcare access in regions such as Asia-Pacific provide significant growth opportunities for Etoposide.

References

1. MarketWatch. “Global Etoposide Market Size and Forecast (2022-2028).”
2. ClinicalTrials.gov. “Ongoing Clinical Trials Using Etoposide.”