CLINICAL TRIALS PROFILE FOR TOPOTECAN HYDROCHLORIDE

505(b)(2) Clinical Trials for topotecan hydrochloride

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 hydrochloride

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 hydrochloride

Condition Name

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

Condition MeSH

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

Clinical Trial Locations for topotecan hydrochloride

Trials by Country

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

Trials by US State

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

Clinical Trial Progress for topotecan hydrochloride

Clinical Trial Phase

Clinical Trial Phase for topotecan hydrochloride
Clinical Trial Phase	Trials
PHASE3	20
PHASE2	9
PHASE1	4

Clinical Trial Status

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

Clinical Trial Sponsors for topotecan hydrochloride

Sponsor Name

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

Sponsor Type

Sponsor Type for topotecan hydrochloride
Sponsor	Trials
Other	437
Industry	249
NIH	144

Topotecan Hydrochloride: Clinical Trials, Market Analysis, and Future Outlook

Last updated: October 28, 2025

Introduction

Topotecan hydrochloride, a topoisomerase I inhibitor, has established itself as a pivotal chemotherapeutic agent primarily used in the treatment of ovarian and small cell lung cancers. Since its FDA approval in 1996, the drug has undergone multiple clinical trials to expand its therapeutic indications, improve formulations, and evaluate combination therapies. The evolving landscape of oncology, coupled with advances in precision medicine, has spurred renewed interest in Topotecan Hydrochloride’s potential. This article provides a comprehensive update on ongoing clinical trials, analyzes its current market dynamics, forecasts future trends, and offers strategic insights for stakeholders.

Clinical Trials Landscape for Topotecan Hydrochloride

The clinical development of Topotecan Hydrochloride is characterized by both confirmatory studies and exploratory trials aimed at expanding its usage.

Ongoing Trials and Recent Updates

Recent registries, including ClinicalTrials.gov, list over 30 active or recruiting studies focusing on Topotecan Hydrochloride. The primary areas of investigation encompass:

• Combination regimens: Numerous trials are evaluating its efficacy when combined with immunotherapy agents, such as PD-1/PD-L1 inhibitors, to overcome resistance mechanisms in refractory ovarian and lung cancers. For example, a phase II trial (NCT04614536) assesses Topotecan in combination with pembrolizumab in relapsed ovarian carcinoma.

• New formulations: Liposomal and nanoparticle-based formulations are under investigation to enhance drug delivery and reduce toxicity. A notable phase I trial (NCT04593816) examines a liposomal Topotecan formulation in patients with advanced solid tumors.

• Alternative indications: Trials are exploring Topotecan for glioblastoma (NCT04509783), neuroendocrine tumors, and recurrent cervical cancer. These studies aim to identify new therapeutic niches beyond traditional indications.

• Biomarker-driven studies: Precision medicine approaches are prominent, where trials stratify patients based on TOP1 gene expression levels or DNA repair deficiency markers, aiming to optimize patient selection and response.

Challenges and Opportunities in Clinical Development

The patent expiry of original formulations and the advent of generic versions have diminished exclusivity, putting pressure on pharmaceutical companies to innovate. Nonetheless, ongoing trials highlight opportunities for niche indication expansion and combination therapies which could redefine Topotecan’s role in oncology. Developing biomarkers for responsiveness and integrating real-world data are key strategies to enhance clinical success.

Market Analysis of Topotecan Hydrochloride

Current Market Status

The global Oncology drug market is projected to reach approximately USD 233 billion by 2025, with topoisomerase inhibitors constituting a significant segment [1]. Topotecan Hydrochloride’s sales are concentrated in North America and Europe, driven by its approved indications and off-label uses. The drug’s current market value is estimated around USD 250 million annually, with steady growth fueled by new clinical evidence and strategic repurposing.

Competitive Landscape

While Topotecan faces competition from newer agents such as PARP inhibitors (e.g., Olaparib) and immunotherapies, it maintains a niche owing to its established efficacy in ovarian and lung cancers. Several generic versions have entered the market post-expiry of the original patent, impacting pricing and margins. However, branded formulations and combination protocols retain market share through clinical differentiation.

Market Drivers

• Growing prevalence of ovarian and lung cancers: According to GLOBOCAN 2020, ovarian cancer ranks as the sixth most common cancer among women, with approximately 314,000 new cases globally [2], driving demand for effective chemotherapeutics.
• Advances in combination therapy protocols: The integration of Topotecan in combination regimens has been shown to improve progression-free survival (PFS), thus broadening its utility.
• Development of targeted and biomarker-driven treatments: Personalized medicine strategies are increasing the specificity and efficacy of oncology therapeutics, supporting Topotecan’s repositioning efforts.
• Increasing focus on chemoresistant tumors: Clinical trials targeting resistant cancer types suggest potential for Topotecan to fill unmet needs.

Market Limitations

• Toxicity profile: Myelosuppression and gastrointestinal side effects limit dose optimization, impacting patient adherence.
• Generic competition: Price erosion reduces profitability, especially in developed markets with multiple generic options.
• Regulatory hurdles: Extended clinical trials are necessary for new indications and formulations, requiring substantial investment.

Future Market Projections

The Topotecan Hydrochloride market is projected to grow at a CAGR of approximately 4-6% over the next five years, driven predominantly by:

• Emerging combination therapies: Trials demonstrating superior efficacy could lead to expanded label indications, rejuvenating market interest.
• Formulation innovations: Liposomal and sustained-release formulations could minimize toxicity and improve patient outcomes, fostering market expansion.
• Geographic expansion: Growing adoption rates in Asian countries, especially China and India, promise new revenue streams.
• Pipeline integration: Incorporation into multi-agent regimens for resistant or refractory cancers enhances its therapeutic footprint.

Strategic Outlook

To capitalize on the growth opportunities, pharmaceutical companies should focus on clinical validation of combination therapies, biomarker-driven patient selection, and formulation enhancements. Regulatory pathways for new indications or innovative delivery systems remain critical considerations for market success. Moreover, strategic collaborations with academic institutions and biotech firms can Accelerate development and access to emerging therapies.

Conclusion

Topotecan Hydrochloride remains a cornerstone in chemotherapy for specific tumors, with ongoing research promising to diversify its clinical applications. The drug’s market trajectory is buoyed by advancements in personalized oncology and combination treatment paradigms, despite challenges posed by competition and toxicity concerns. Stakeholders that leverage emerging clinical data, invest in formulation chemistry, and adapt to evolving regulatory landscapes will be best positioned to maximize the drug’s therapeutic and commercial potential.

---

Key Takeaways:

• Continuous clinical trials are expanding Topotecan Hydrochloride’s therapeutic application, notably in combination with immunotherapies and targeted agents.
• Market growth is driven by rising cancer prevalence, innovation in formulations, and geographic expansion, especially into emerging markets.
• Challenges include toxicity management, generic competition, and the need for regulatory approval for new indications.
• Innovative strategies such as biomarker-driven patient selection and novel drug delivery systems are critical for future success.
• Collaboration and investment in research are essential to sustain market relevance and develop next-generation formulations.

---

FAQs

1. What are the main approved uses of Topotecan Hydrochloride?

Topotecan is FDA-approved for relapsed small cell lung cancer and ovarian cancer following failure of first-line chemotherapy.

2. Are there any promising combination therapies involving Topotecan?

Yes, ongoing trials are investigating its synergy with immune checkpoint inhibitors, particularly for refractory ovarian and lung cancers, showing promising preliminary results.

3. How does the toxicity profile impact clinical use?

Myelosuppression remains the primary concern, necessitating dose management and supportive care to maintain tolerability and adherence.

4. What emerging formulations could enhance Topotecan’s efficacy?

Liposomal and nanoparticle-based formulations aim to improve drug delivery, reduce toxicity, and enable higher dosing or sustained-release options.

5. What market strategies should stakeholders pursue?

Focusing on biomarker-driven indications, developing innovative formulations, and entering emerging markets will optimize commercial outcomes.

---

References

[1] Grand View Research. Oncology Drugs Market Size & Trends Analysis Report, 2020-2025.

[2] GLOBOCAN 2020. Global Cancer Statistics. International Agency for Research on Cancer.