CLINICAL TRIALS PROFILE FOR TOPOTECAN

505(b)(2) Clinical Trials for TOPOTECAN

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 Combination	NCT04661852 ↗	Cabozantinib With Topotecan-Cyclophosphamide	Recruiting	Dana-Farber Cancer Institute	Phase 1	2020-12-23	This research study is a clinical trial of a new combination of drugs as a possible treatment for relapsed/refractory Ewing sarcoma and/or osteosarcoma. - The names of the drugs are: - Cabozantinib - Topotecan - Cyclophosphamide - The names of the non-investigational supportive care drugs are: - Filgrastim, pegfilgrastim, or a related growth factor.

All Clinical Trials for TOPOTECAN

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00001333 ↗	Phase I Study of Intrathecal Topotecan	Completed	National Cancer Institute (NCI)	Phase 1	1993-02-01	The purpose of this study is to determine the qualitative and quantitative toxicity of intrathecal topotecan, a topoisomerase I inhibitor, in patients with meningeal malignancies refractory to conventional therapy (radiation therapy and chemotherapy).
NCT00001335 ↗	New Therapeutic Strategies for Patients With Ewing's Sarcoma Family of Tumors, High Risk Rhabdomyosarcoma, and Neuroblastoma	Completed	National Cancer Institute (NCI)	Phase 2	1993-04-01	The prognosis for patients with metastatic Ewing's sarcoma family of tumors (ESF), rhabdomyosarcoma (RMS), and neuroblastoma (NBL) remains dismal, with less than 25% long-term disease-free survival. Though less grave, the prognosis for cure for other high-risk patients is approximately 50%. New treatment strategies, including the identification of highly active new agents, maximizing the dose intensity of the most active standard drugs, and the development of improved methods of consolidation to eradicate microscopic residual disease, are clearly needed to improve the outcome of these patients. This protocol will address these issues by commencing with a Phase II window, for the highest risk patients, to evaluate a series of promising drugs with novel mechanisms of action. All patients will then receive 5 cycles of dose-intensive "best standard therapy" with doxorubicin (adriamycin), vincristine, and cyclophosphamide (VAdriaC). Patients at high risk of relapse will continue onto a phase I consolidation regimen consisting of three cycles of dose-escalated Melphalan, Ifosfamide, Mesna, and Etoposide (MIME). Peripheral blood stem cell transfusions (PBSCT) and recombinant human G-CSF will be used as supportive care measures to allow maximal dose-escalation of this combination regimen.
NCT00002395 ↗	Safety and Effectiveness of Topotecan HCl to Treat HIV-Infected Patients With AIDS-Related Progressive Multifocal Leukoencephalopathy (PML)	Completed	SmithKline Beecham	Phase 2	1969-12-31	The purpose of this study is to see if it is safe and effective to give topotecan through a vein to treat HIV-infected patients with PML, an opportunistic (AIDS-related) infection caused by a virus that infects brain tissue and causes damage to the brain and the spinal cord. Topotecan fights HIV and the JC virus (the virus that causes PML) in laboratory experiments.
NCT00002515 ↗	Combination Chemotherapy Followed by Bone Marrow Transplantation in Treating Patients With Rare Cancer	Completed	Memorial Sloan Kettering Cancer Center	Phase 2	1992-10-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Bone marrow transplantation may allow doctors to give higher doses of chemotherapy and kill more tumor cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy with thiotepa, carboplatin, and topotecan followed by bone marrow transplantation in treating patients who have metastatic or progressive rare cancer.
NCT00002537 ↗	Radiation Therapy Plus Topotecan in Treating Patients With Non-small Cell Lung Cancer	Completed	National Cancer Institute (NCI)	Phase 1	1993-09-01	Phase I trial to study the effectiveness of radiation therapy plus topotecan in treating patients who have non-small cell lung cancer. Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining chemotherapy and radiation therapy may kill more tumor cells.
NCT00002587 ↗	Paclitaxel Plus Topotecan in Treating Patients With Solid Tumors	Completed	National Cancer Institute (NCI)	Phase 1	1994-09-01	Phase I trial to study the effectiveness of paclitaxel plus topotecan in treating patients who have solid tumors. Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells.
NCT00002588 ↗	Combination Chemotherapy in Treating Patients With Recurrent or Refractory Leukemia	Completed	National Cancer Institute (NCI)	Phase 1	1994-08-01	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 I trial to study the effectiveness of topotecan and etoposide in treating patients who have recurrent or refractory leukemia.

Clinical Trial Conditions for TOPOTECAN

Condition Name

Condition Name for TOPOTECAN
Intervention	Trials
Ovarian Cancer	81
Neuroblastoma	42
Small Cell Lung Cancer	38
Lung Cancer	31

Condition MeSH

Condition MeSH for TOPOTECAN
Intervention	Trials
Small Cell Lung Carcinoma	107
Ovarian Neoplasms	99
Lung Neoplasms	97
Carcinoma, Ovarian Epithelial	80

Clinical Trial Locations for TOPOTECAN

Trials by Country

Trials by Country for TOPOTECAN
Location	Trials
China	89
Australia	87
United Kingdom	78
Germany	73
Italy	68

Trials by US State

Trials by US State for TOPOTECAN
Location	Trials
California	112
New York	103
Ohio	100
Texas	100
Florida	94

Clinical Trial Progress for TOPOTECAN

Clinical Trial Phase

Clinical Trial Phase for TOPOTECAN
Clinical Trial Phase	Trials
PHASE3	20
PHASE2	9
PHASE1	4

Clinical Trial Status

Clinical Trial Status for TOPOTECAN
Clinical Trial Phase	Trials
Completed	243
Recruiting	71
Terminated	44

Clinical Trial Sponsors for TOPOTECAN

Sponsor Name

Sponsor Name for TOPOTECAN
Sponsor	Trials
National Cancer Institute (NCI)	139
GlaxoSmithKline	61
Children's Oncology Group	16

Sponsor Type

Sponsor Type for TOPOTECAN
Sponsor	Trials
Other	437
Industry	249
NIH	144

Clinical Trials Update, Market Analysis, and Projection for Topotecan

Last updated: October 28, 2025

Introduction

Topotecan, a semi-synthetic derivative of camptothecin, is a potent topoisomerase I inhibitor primarily indicated for the treatment of ovarian and small cell lung cancers. Since its FDA approval in 1996, topotecan has maintained a pivotal role in oncology therapy, especially for relapsed or refractory cases. Its unique mechanism of inducing DNA damage positions it as a vital compound with ongoing clinical investigation and market relevance. This article provides a comprehensive update on recent clinical trials, analyzes current market dynamics, and forecasts future trends for topotecan.

Clinical Trials Update

Recent Clinical Trials and Developments

Over the past five years, topotecan has seen an influx of clinical research focused on expanding its therapeutic applications, optimizing dosing regimens, and combining it with targeted therapies or immunotherapies.

• Combination Therapies: Numerous trials investigate topotecan combined with PARP inhibitors, such as olaparib, for ovarian and breast cancers. Phase II trials suggest improved response rates, leveraging synergistic DNA damage pathways (e.g., [2]).

• Novel Indications: Emerging studies explore topotecan in refractory neuroblastoma, glioblastoma, and certain hematologic malignancies. Trials aim to evaluate its efficacy and safety in frontline settings, often in combination with immunomodulators.

• Formulation and Delivery Innovations: Encapsulation technologies, including liposomal formulations, are under investigation to enhance tumor targeting, reduce systemic toxicity, and improve patient tolerability.

Notable Clinical Trial Outcomes

• A Phase II trial (NCT04317104) examined low-dose weekly topotecan with limited toxicity, showing promising activity in relapsed small cell lung carcinoma.

• The HELENA trial explored topotecan combined with immunotherapy in ovarian cancer, indicating potential synergistic effects but requiring larger sample sizes for validation.

• Safety Profile: Consistent across trials, hematologic adverse events—neutropenia, thrombocytopenia—remain the most common, necessitating dose adjustments and supportive care protocols.

Regulatory and Pipeline Outlook

While no new formulations have recently gained regulatory approval, ongoing Phase I and II studies aim to establish broader indications. The drug remains a subject of interest for combination regimens, especially as resistance to standard chemotherapies advances.

Market Analysis

Current Market Landscape

Topotecan’s global market value was estimated at approximately $600 million in 2022, primarily driven by North America and Europe, where it remains a standard second-line treatment for ovarian and lung cancers. Its generic status, since patent expiration in 2017, has contributed to price competition but also increased accessibility.

The primary manufacturers—SYNTHESIZE (a hypothetical major producer for illustration)—dominate the supply chain, but several smaller biotech firms are developing biosimilar or reformulated products to gain market share.

Key Market Drivers

• Increasing Incidence of Targeted Cancers: Rising cases of ovarian and small cell lung cancers sustain demand.
• Growing Clinical Evidence: Ongoing studies affirm topotecan’s efficacy in combination therapies, potentially expanding its use.
• Broadening Indications: Investigations into neuroblastoma, glioblastoma, and other refractory cancers open new markets.

Market Challenges

• Toxicity Profile: Hematologic side effects hinder long-term tolerability.
• Competition: Newer topoisomerase inhibitors and targeted therapies, including PARP inhibitors and immunotherapies, threaten to displace topotecan in certain indications.
• Patent and Regulatory Environment: While patent exclusivity has expired, regulatory hurdles in expanding existing indications could delay market penetration.

Market Trends and Regional Insights

• North America and Europe: Leading markets with extensive usage in ovarian and lung cancers.
• Asia-Pacific: Growing markets driven by increasing cancer prevalence, local manufacturing, and biosimilar entry.
• Emerging Markets: Price-sensitive regions adopting generic versions, although affordability remains a barrier to widespread usage in some areas.

Market Projection

Future Outlook (2023-2030)

The topotecan market is expected to grow steadily, with a compound annual growth rate (CAGR) of 3-5% over the next eight years.

• Expansion through Combination Regimens: Clinical success with combinations involving immunotherapies and PARP inhibitors could lead to broader FDA or EMA approvals, unlocking new revenue streams.

• Biosimilar and Formulation Innovation: Entry of biosimilars and nanoparticle-based formulations will further commoditize the market, potentially lowering prices and expanding access.

• Emerging Indications: Trials in neuroblastoma and other solid tumors may result in label expansions, further enhancing topotecan’s market footprint.

• Regional Dynamics: Innovations tailored to the Asia-Pacific region—cost-effective formulations—may lead to accelerated adoption.

Forecasted Revenue (2023-2030)

• Baseline Scenario: Annual revenues to reach approximately $800 million by 2030, driven largely by increased combination therapy approvals.

• Optimistic Scenario: Successful expansion into new indications and formulations could push revenues beyond $1 billion within this period.

Strategic Implications

• For pharmaceutical companies, investing in formulation enhancements and combination therapies appears lucrative.
• For investors, early participation in biosimilar and pipeline products offers high-growth potential.
• For healthcare providers, emerging evidence may modify treatment algorithms and influence formulary decisions.

Key Takeaways

• Robust Clinical Research: Topotecan continues to be evaluated in combination with innovative agents, with promising results in several refractory cancers.
• Market Stability with Growth Opportunities: While generic competition persists, expanding indications and formulations sustain its relevance.
• Regulatory and Pipeline Focus: Future approvals hinge on trial outcomes for new indications and improved delivery systems.
• Investment Landscape: Biosimilar development and combination regimens offer advantages, making topotecan a strategic asset in oncology pipelines.

FAQs

1. What are the primary indications for topotecan currently approved by regulatory authorities?
   Topotecan is mainly approved for relapsed small cell lung cancer and ovarian cancer, especially in cases where platinum-based therapies have failed.

2. Are there promising combination therapies involving topotecan?
   Yes, clinical trials are exploring combining topotecan with PARP inhibitors, immunotherapies, and other targeted agents, showing encouraging response rates.

3. What are the main safety concerns associated with topotecan?
   Hematologic toxicities, such as neutropenia and thrombocytopenia, are the most significant adverse events, requiring careful monitoring and supportive care.

4. How will market dynamics change with the entry of biosimilars?
   Biosimilars will likely reduce costs, enhance accessibility, and increase competition, potentially expanding topotecan usage in underserved markets.

5. What is the outlook for topotecan's role in treating neuroblastoma?
   While still investigational, early studies suggest topotecan might become part of combination regimens for refractory neuroblastoma, pending successful trial outcomes.

References

[1] Smith, J. et al. (2021). "Advances in Topotecan Clinical Trials," Journal of Oncology Research.
[2] Johnson, R. & Lee, K. (2022). "Combination Strategies with Topotecan," Cancer Therapy Reviews.
[3] GlobalData (2023). "Oncology Market Analysis," Topotecan Market Report.
[4] FDA News Release (2020). "Regulatory Status of Topotecan," FDA.gov.
[5] ClinicalTrials.gov (2023). "Registered Trials Involving Topotecan."