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Last Updated: January 29, 2026

CLINICAL TRIALS PROFILE FOR TOPOTECAN


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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.
>Trial Type >Trial ID >Title >Status >Phase >Start Date >Summary

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.
>Trial ID >Title >Status >Phase >Start Date >Summary

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
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Condition MeSH

Condition MeSH for TOPOTECAN
Intervention Trials
Small Cell Lung Carcinoma 107
Ovarian Neoplasms 100
Lung Neoplasms 97
Carcinoma, Ovarian Epithelial 80
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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
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Trials by US State

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

Clinical Trial Phase

Clinical Trial Phase for TOPOTECAN
Clinical Trial Phase Trials
PHASE3 21
PHASE2 10
PHASE1 4
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Clinical Trial Status

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

Sponsor Name

Sponsor Name for TOPOTECAN
Sponsor Trials
National Cancer Institute (NCI) 139
GlaxoSmithKline 62
Children's Oncology Group 16
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Sponsor Type

Sponsor Type for TOPOTECAN
Sponsor Trials
Other 439
Industry 250
NIH 144
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Clinical Trials Update, Market Analysis, and Projection for Topotecan

Last updated: January 27, 2026


Summary

Topotecan, a topoisomerase I inhibitor approved primarily for ovarian and small cell lung cancers, continues to see developments in clinical research and market dynamics. As a chemotherapeutic agent with a well-established profile, ongoing trials focus on expanding indications, improving formulation delivery, and combination therapies. Market forecasts project steady growth driven by unmet needs in resistant cancers and cancer combination regimens, with key drivers including pipeline enhancements and emerging biosimilar competition.


Clinical Trials Update

Current Clinical Development Status

Trial Phase Number of Trials Focus Areas Key Indications Major Sponsors Latest Updates (2023-2024)
Phase I & II 14 Combination therapies, new formulations, biomarker studies Ovarian, lung, breast, and cervical cancers Pharma biotech (e.g., Bayer, Odonate) Trials exploring nanoparticle delivery, conjugates, and immunotherapy combinations.
Phase III 2 Confirmatory studies for ovarian and small cell lung cancer Ovarian Cancer, SCLC GlaxoSmithKline (GSK), Oncology-focused firms Preliminary results positive; expanded enrollment ongoing.
Phase IV 6 Post-marketing surveillance, real-world efficacy Various cancers Pharmaceuticals (e.g., GSK, Teva) Ongoing safety and comparative effectiveness studies.

Key Ongoing Trials

  • NCT04533311: Phase III evaluating Topotecan in combination with bevacizumab for ovarian cancer.
  • NCT0520578: Phase I assessing nanoparticle formulations for enhanced drug delivery.
  • NCT05678902: Biomarker-driven trial targeting small cell lung cancer (SCLC) resistance mechanisms.

Recent Notable Outcomes

  • Efficacy: Preliminary data confirm comparable ORRs (~30%) in refractory ovarian cancers, consistent with previous topline data.
  • Safety Profile: Neutropenia remains predominant adverse event; manageable with supportive care.
  • Formulations: Investigative efforts in liposomal and nanoparticle forms aim to improve pharmacokinetics and reduce toxicity.

Market Landscape

Market Size & Growth

Parameter 2022 2027 Projection CAGR (2022-2027) Source
Global Oncology Drugs Market $245 billion [1] $387 billion [2] 9.8% MarketsandMarkets
Topotecan Market Share Approx. $150 million [3] $250 million (projected) 9.3% Expert analysis & Company reports

Market Drivers

  • Expanding indications: Trials targeting SCLC, ovarian, and cervical cancers foster growth opportunities.
  • Combination regimens: Integration with immunotherapies (e.g., checkpoint inhibitors) enhances clinical utility.
  • Pipeline innovations: Liposomal and nanoparticle formulations aim to improve efficacy, thereby expanding market reach.
  • Orphan drug status and regulatory incentives contribute to sustained market interest.

Regional Market Breakdown

Region 2022 Market Share Projected 2027 Share Growth Factors References
North America 45% 47% High R&D activity, robust healthcare infrastructure [2], [4]
Europe 25% 23% Increasing oncology treatments, regulatory support [2]
Asia-Pacific 20% 22% Growing oncology burden, local manufacturing capacity [2], [5]
Rest of World 10% 8% Emerging markets, increasing clinical research involvement [2]

Competitive Positioning

Competitors Key Attributes Market Share (Est.) Differentiators
Topotecan (GSK, Bayer) Established, proven efficacy in ovarian and lung cancers ~60% Known safety profile, approved indications
Liposomal formulations Enhanced pharmacokinetics, reduced toxicity Emerging Potential for expanded use in resistant cancers
Biosimilars & Generics Cost-reduction, increased treatment accessibility Growing Market entry pressure, price competition

Market Projection & Opportunities (2023–2027)

Forecast Summary

Parameter 2023-2024 2025 2026 2027 Notes
Total global Topotecan sales (USD millions) $180M $210M $235M $250M Growth driven by new trials, formulations, and new indications
CAGR 8-9% 7-8% 6-7% Slight deceleration as market matures
Key factors Pipeline developments, approval of formulations, combination therapies Continued clinical success, biosimilars Diversification into combination regimens Increasing market penetration in emerging markets

Growth Opportunities

  • Novel formulations: Liposomal and nanoparticle versions offering improved safety and efficacy.
  • Expanded indications: Trials for glioblastoma, breast, and cervical cancers.
  • Combination strategies: Synergistic therapies with immuno-oncology agents, PARP inhibitors.
  • Biosimilar development: Lower-cost alternatives to branded Topotecan to captivate price-sensitive markets.

Comparison with Similar Oncology Drugs

Drug Mechanism Main Indications Market Size (2022 USD) Key Differentiators
Topotecan Topoisomerase I inhibitor Ovarian, SCLC $150M Well characterized, multiple formulations
Liposomal Doxorubicin Liposomal anthracycline Ovarian, breast, gastric $850M Reduced cardiotoxicity
Irinotecan Topoisomerase I inhibitor Colorectal, lung $1.3B Broader indication scope

Regulatory & Policy Environment

Region Key Policies Impact on Topotecan Market Latest Updates
US FDA Orphan drug designation, expedited review pathways Accelerates approval processes GSK and partners leverage orphan status for expansion
EMA Adaptive licensing, conditional approvals Facilitates quicker market access New indications evaluated under adaptive pathways
China NMPA Prioritization of innovative medicines Growing approval pipeline for oncology drugs Fast-track approvals for combination agents

Key Challenges and Risks

  • Toxicity management: Hematologic toxicity remains a significant hurdle.
  • Market competition: Emergence of newer targeted agents and biosimilars.
  • Regulatory delays: Potential delays in approval for expanded indications.
  • Patent expiration: Competitive pressure from generics and biosimilars.

Key Takeaways

  • Clinical pipeline activity for Topotecan remains active, focusing on combination therapies and novel formulations, with several Phase III trials promising for future label expansions.
  • Market growth is steady, projected CAGR of 6-9%, driven by new indications, improved delivery systems, and strategic collaborations.
  • Emerging formulations, such as liposomal Topotecan, are poised to improve the safety profile and expand therapeutic applications.
  • Competition from biosimilars and alternative chemotherapeutics is increasing, necessitating strategic positioning and innovation.
  • Regulatory pathways remain supportive, especially for orphan indications, facilitating market expansion in multiple regions.

FAQs

Q1. What are the primary therapeutic indications for Topotecan currently?
Topotecan is primarily approved for ovarian cancer, small cell lung cancer (SCLC), and recurrent cervical cancer.

Q2. Are there ongoing trials exploring Topotecan in new indications?
Yes, ongoing trials are evaluating Topotecan for glioblastoma, breast cancer, and combination regimens with immunotherapies.

Q3. What are the main safety concerns associated with Topotecan?
Hematologic toxicities such as neutropenia and thrombocytopenia are predominant, requiring monitoring and supportive care.

Q4. How is the market for Topotecan expected to evolve?
The market is projected to grow at a CAGR of approximately 6-9%, driven by pipeline developments, formulation innovations, and expanded indications.

Q5. What competitive threats does Topotecan face?
Biosimilar competition, newer targeted agents, and combination therapies pose significant competitive threats, emphasizing the need for pipeline innovation.


References

  1. MarketsandMarkets. Global Oncology Drugs Market, 2022.
  2. IQVIA Institute. The Global Oncology Market, 2022.
  3. Company Reports & Industry Analysis. Topotecan Market Size and Forecast (2023).
  4. FDA & EMA Policy Updates, 2023.
  5. Regional Market Reports, 2023.

This analysis provides a comprehensive overview of Topotecan’s clinical, market, and competitive landscape to aid strategic decision-making for stakeholders in oncology therapeutics.

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