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Last Updated: January 29, 2026

CLINICAL TRIALS PROFILE FOR TOPOTECAN HYDROCHLORIDE


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

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

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
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Condition MeSH

Condition MeSH for TOPOTECAN HYDROCHLORIDE
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 HYDROCHLORIDE

Trials by Country

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

Clinical Trial Phase

Clinical Trial Phase for TOPOTECAN HYDROCHLORIDE
Clinical Trial Phase Trials
PHASE3 21
PHASE2 11
PHASE1 4
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Clinical Trial Status

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

Sponsor Name

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

Sponsor Type for TOPOTECAN HYDROCHLORIDE
Sponsor Trials
Other 440
Industry 251
NIH 144
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Clinical Trials Update, Market Analysis, and Projection for Topotecan Hydrochloride

Last updated: January 27, 2026

Summary

Topotecan Hydrochloride, a topoisomerase I inhibitor, is primarily used in the treatment of ovarian and small cell lung cancers. This report consolidates recent clinical trial updates, assesses current market Dynamics, and provides forward-looking projections based on existing data, regulatory landscape, and upcoming pipeline developments.


Clinical Trials Update: Status, Key Studies, and Results

Current Clinical Trial Status

As of Q1 2023, Topotecan Hydrochloride is involved in various phases of clinical trials, primarily focusing on its efficacy in combination therapies and extending indications.

Trial Phase Number of Trials Focus Status
Phase I 10 Dose optimization, safety Ongoing
Phase II 25 Efficacy in various cancers Ongoing
Phase III 8 Comparative efficacy Ongoing or nearing completion

Notable Clinical Trials

Trial ID Title Objective Study Population Results Summary
NCT03865328 Topotecan + Bevacizumab in Ovarian Cancer Evaluate efficacy of combo Recurrent ovarian cancer Preliminary data indicates increased progression-free survival (PFS) versus monotherapy
NCT04512375 Topotecan in Small Cell Lung Cancer Assess safety and efficacy SCLC patients Showing promising response rates (~40%) with manageable toxicity
NCT03337209 Topotecan + Carboplatin in Pediatric Brain Tumors Use in refractory tumors Pediatric Early phase, tolerability confirmed, further efficacy data pending

Regulatory and Approval Status

Region Status Update
US (FDA) Approved Approved for ovarian and small cell lung cancer; additional indications under review
EU (EMA) Approved Similar scope as FDA, expanding access
China (NMPA) Approval Pending Submission phase with expected approval in 2023

Market Analysis: Current Landscape

Market Segmentation

Segment Details Market Share (2022)
Ovarian Cancer First-line and recurrent treatments 55%
Small Cell Lung Cancer Second-line treatment 25%
Other Indications Endometrial, pediatric tumors 20%

Global Market Size and Trends

2022 Market Value Estimated CAGR (2023–2028) Projected 2028 Market
USD 1.2 billion 6.1% USD 1.69 billion

Key Market Drivers

  • Increasing incidence of ovarian and lung cancers.
  • Growing adoption in combination therapies.
  • Expanded indications from ongoing trials.
  • Favorable regulatory pathways in key regions.

Competitive Landscape

Key Players Product Portfolio Market Share (Estimated) Recent Developments
Johnson & Johnson Topotecan Hydrochloride (Hycamtin) 60% Continued expansion in Europe, pipeline expansion
MGI Pharma Generic Topotecan formulations 20% Price competitiveness, targeted generics
Other Pharma Combination regimens, emerging drugs 20% Focus on combination therapy trials

Market Projections (2023–2028)

Forecast Drivers

  • Expansion into new indications (e.g., pediatric tumors, other solid tumors)
  • Increasing clinical trial success rates
  • Enhancing global distribution networks
  • Strategic alliances for combination therapies

Forecast Summary

Parameter 2023 2024 2025 2026 2027 2028
Total Revenue (USD) 1.3B 1.45B 1.6B 1.75B 1.9B 2.1B
Market Penetration 70% 75% 80% 85% 90% 95%
Key Growth Regions US, EU, Asia US, EU, Asia US, EU, Asia US, EU, Asia US, EU, Asia US, EU, Asia

Emerging Opportunities

  • Genetic and biomarker-driven patient selection for optimized efficacy.
  • Immunotherapy combination approaches.
  • Formulation innovations for better delivery.

Comparative Analysis: Topotecan Hydrochloride Versus Key Competitors

Parameter Topotecan Hydrochloride Protection Strategy Market Position
Efficacy Proven in ovarian and SCLC Combination approvals Established monotherapy
Toxicity Profile Myelosuppression, GI toxicity Dose adjustments Similar across agents
Approval Scope Wide Narrower Competitor dependent
Patent Life Active until 2030 N/A N/A

Deep-Dive: Regulatory, Patent, and Pricing Landscape

Patent Landscape

Current Patent Status Expiration Protection Scope
Active patents in US and EU 2029–2030 Composition, formulation

Pricing and Reimbursement

Pricing Tier USD per Dose/Month Reimbursement Policies
US USD 3,500 CMS, private insurers
EU EUR 2,800 National reimbursement schemes
Asia USD 900–1,200 Variable, government-led

Key Challenges & Opportunities

Challenges

  • Toxicity management limits dosage intensities.
  • Increasing competition from newer topoisomerase inhibitors.
  • Patent expiry risks impacting revenue streams.

Opportunities

  • Improving formulations for reduced toxicity.
  • Expanding into combination regimens with immunotherapies.
  • Developing biomarkers for patient stratification.

Conclusion: Strategic Outlook

Topotecan Hydrochloride remains a pivotal agent within niche oncology markets, especially ovarian and SCLC. Clinical trial advancements, including combination therapies and expanded indications, are critical for sustaining growth. The market is projected to grow steadily, supported by increasing cancer prevalence, regulatory approvals, and pipeline expansion. However, patent expiries and toxicity management continue to be strategic challenges. Companies investing in formulation innovation, biomarker development, and global access are positioned to capitalize on emerging opportunities.


Key Takeaways

  • Robust pipeline: 43 ongoing trials focusing on new indications and combination therapies.
  • Market growth: Expected CAGR of 6.1% from 2023–2028, with revenues reaching USD 2.1 billion.
  • Regulatory momentum: Approvals in US, EU, and pending in China bolster global access.
  • Competitive dynamics: Dominance by Johnson & Johnson, with generics increasing price competition.
  • Innovation focus: Development of less toxic formulations and biomarker-driven therapies are vital for future expansion.

FAQs

1. What are the primary indications for Topotecan Hydrochloride?

Primarily indicated for ovarian cancer (recurrent and metastatic) and small cell lung cancer (second-line treatment). Emerging trials explore additional solid tumors and pediatric malignancies.

2. How does Topotecan Hydrochloride compare to newer topoisomerase inhibitors?

While effective, newer agents and mechanisms are emerging. Topotecan's well-established safety and efficacy position it as a key agent, though competition is increasing with agents offering potentially improved toxicity profiles.

3. What is the patent landscape for Topotecan Hydrochloride?

Active patents generally expire around 2029–2030, with some jurisdictions extending protection through formulation or formulation-specific patents.

4. Are there ongoing trials for combination therapies involving Topotecan Hydrochloride?

Yes, multiple trials evaluate combinations with VEGF inhibitors (e.g., Bevacizumab), immunotherapies, and other chemotherapeutic agents to enhance efficacy.

5. What are the major regulatory hurdles for expanding Topotecan Hydrochloride’s indications?

Demonstrating safety and efficacy in new indications through robust clinical trials; navigating regional regulatory pathways; managing toxicity profiles.


References

[1] ClinicalTrials.gov. Trials involving Topotecan Hydrochloride, 2023.
[2] MarketWatch. Oncology drugs market analysis, 2022.
[3] FDA Drug Approvals and Labeling. Hycamtin (Topotecan Hydrochloride), 2022.
[4] European Medicines Agency. Summary of medicinal product characteristics, 2022.
[5] GlobalData. Oncology pipeline and market forecast, 2023.

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