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Last Updated: February 8, 2025

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

Clinical Trial Conditions for Topotecan

Condition Name

Condition Name for Topotecan
Intervention Trials
Ovarian Cancer 76
Neuroblastoma 40
Small Cell Lung Cancer 33
Lung Cancer 31
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Condition MeSH

Condition MeSH for Topotecan
Intervention Trials
Lung Neoplasms 96
Small Cell Lung Carcinoma 95
Ovarian Neoplasms 92
Carcinoma, Ovarian Epithelial 79
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Clinical Trial Locations for Topotecan

Trials by Country

Trials by Country for Topotecan
Location Trials
Australia 74
United Kingdom 72
Germany 69
Italy 61
Spain 57
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Trials by US State

Trials by US State for Topotecan
Location Trials
California 106
New York 95
Texas 93
Ohio 92
Florida 88
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Clinical Trial Progress for Topotecan

Clinical Trial Phase

Clinical Trial Phase for Topotecan
Clinical Trial Phase Trials
Phase 4 4
Phase 3 66
Phase 2/Phase 3 6
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Clinical Trial Status

Clinical Trial Status for Topotecan
Clinical Trial Phase Trials
Completed 243
Recruiting 50
Terminated 44
[disabled in preview] 32
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Clinical Trial Sponsors for Topotecan

Sponsor Name

Sponsor Name for Topotecan
Sponsor Trials
National Cancer Institute (NCI) 139
GlaxoSmithKline 60
Gynecologic Oncology Group 15
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Sponsor Type

Sponsor Type for Topotecan
Sponsor Trials
Other 421
Industry 225
NIH 144
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Topotecan: Clinical Trials, Market Analysis, and Projections

Introduction to Topotecan

Topotecan, marketed under the brand name Hycamtin, is a chemotherapeutic agent that functions as a topoisomerase I inhibitor. It disrupts DNA replication, leading to the death of cancer cells. This drug is primarily used in the treatment of various cancers, including ovarian cancer, small cell lung cancer (SCLC), refractory leukemia, non-Hodgkin's lymphoma, and pediatric solid tumors[3].

Clinical Trials and Efficacy

Randomized Comparative Trial

In a significant randomized comparative trial, topotecan was compared to the CAV regimen (cyclophosphamide, doxorubicin, vincristine) in patients with recurrent or progressive SCLC. The trial involved 211 patients who were randomized to receive either topotecan or CAV. The results showed that while topotecan had similar efficacy outcomes (overall response rate, response duration, time to progression, and overall survival) to CAV, there were no statistically significant improvements in these parameters[1].

Single-Arm Trials

Topotecan was also evaluated in three single-arm trials involving 319 patients with recurrent or progressive SCLC. These trials stratified patients as either sensitive or refractory to first-line chemotherapy. The response rates ranged from 11% to 31% for sensitive patients and 2% to 7% for refractory patients. The median time to progression and median survival were similar across all trials[1].

Ongoing and Future Evaluations

There is ongoing research to optimize topotecan dosing, scheduling, and its incorporation into combination regimens. For instance, the development of an oral formulation of topotecan is being assessed for its convenience, quality of life, toxicity, and overall therapeutic indices. This could potentially expand the clinical utility of topotecan, especially in settings where the agent had previously shown marginal activity[3].

Market Analysis

Global Market Size and Growth

The global Topotecan Hydrochloride Sales Market is projected to be USD 572.6 million in 2024 and is expected to grow at a compound annual growth rate (CAGR) of 5.00% from 2024 to 2031, reaching USD 805.70 million by 2031. This growth is driven by the increasing prevalence of cancer and advancements in drug formulations[2].

Regional Market Dynamics

  • North America: This region holds the largest market share, with a market size of USD 229.04 million in 2024, expected to grow at a CAGR of 3.2% from 2024 to 2031.
  • Europe: Europe accounts for more than 30% of the global revenue, with a market size of USD 171.78 million in 2024, growing at a CAGR of 3.5% from 2024 to 2031.
  • Asia-Pacific: This region is projected to expand at the fastest CAGR, driven by increasing cancer prevalence, improving healthcare infrastructure, and rising investments in medical research. The market size here was USD 131.70 million in 2024, expected to grow at a CAGR of 7.0% from 2024 to 2031.
  • Latin America and Middle East & Africa: These regions also show growth potential, with market sizes of USD 28.63 million and USD 11.45 million in 2024, respectively, and expected CAGRs of 4.4% and 4.7% from 2024 to 2031[2].

Factors Influencing Market Growth

Increasing Cancer Prevalence

The rising incidence of cancers such as ovarian and small cell lung cancer is a significant driver of the topotecan hydrochloride market. As the global population ages and lifestyle factors contribute to higher cancer rates, the demand for effective chemotherapeutic agents like topotecan is expected to increase[2].

Advancements in Drug Formulations

The development of new drug delivery systems and the introduction of oral formulations are enhancing the market for topotecan. These advancements improve the convenience, quality of life, and therapeutic indices associated with topotecan treatment[3].

Increased Investments in Oncology Research

Ongoing clinical trials and increased investments in oncology research are further boosting the market. Novel combination regimens and the integration of topotecan into personalized medicine and targeted therapies are key areas of focus[2].

Clinical Utility and Future Perspectives

Current Clinical Use

Topotecan has shown notable activity against a wide range of tumors, including recurrent and refractory ovarian cancer, SCLC, refractory leukemia, non-Hodgkin's lymphoma, and pediatric solid tumors. Its unique distributive characteristics across the blood-brain barrier make it particularly useful for treating neoplasms involving the central nervous system[3].

Potential for Combination Regimens

There is significant potential for topotecan in combination regimens. For example, the LAGOON trial is evaluating lurbinectedin alone or in combination with irinotecan versus investigator's choice (topotecan or irinotecan) in patients with relapsed SCLC. Preliminary results from such trials suggest that combination therapies could enhance the therapeutic index and overall applicability of topotecan[4].

Oral Formulation and Quality of Life

The availability of an oral formulation of topotecan is expected to improve patient convenience and quality of life. This formulation may also lead to reevaluations of topotecan's utility in clinical situations where its potential benefit was previously underestimated[3].

Key Takeaways

  • Topotecan is a chemotherapeutic agent with significant efficacy in treating various cancers, including ovarian and small cell lung cancer.
  • Clinical trials have shown comparable efficacy to other chemotherapy regimens, though optimization of dosing and scheduling is ongoing.
  • The global market for topotecan hydrochloride is projected to grow at a CAGR of 5.00% from 2024 to 2031, driven by increasing cancer prevalence and advancements in drug formulations.
  • Regional markets, particularly in Asia-Pacific, are expected to show rapid growth due to improving healthcare infrastructure and rising investments in oncology research.
  • The development of oral formulations and combination regimens is likely to enhance the clinical utility and market expansion of topotecan.

FAQs

What is topotecan used for?

Topotecan is used primarily in the treatment of various cancers, including ovarian cancer, small cell lung cancer, refractory leukemia, non-Hodgkin's lymphoma, and pediatric solid tumors.

How does topotecan work?

Topotecan functions as a topoisomerase I inhibitor, disrupting DNA replication and leading to the death of cancer cells.

What are the key clinical trials for topotecan?

Significant clinical trials include a randomized comparative trial against the CAV regimen and single-arm trials in patients with recurrent or progressive SCLC. Ongoing trials are also evaluating the optimal use of topotecan in combination regimens.

What is the market size and growth projection for topotecan hydrochloride?

The global Topotecan Hydrochloride Sales Market size is estimated to be USD 572.6 million in 2024 and is expected to grow at a CAGR of 5.00% from 2024 to 2031, reaching USD 805.70 million by 2031.

Which regions are expected to drive the growth of the topotecan hydrochloride market?

The Asia-Pacific region is projected to expand at the fastest CAGR, driven by increasing cancer prevalence, improving healthcare infrastructure, and rising investments in medical research.

What are the factors influencing the growth of the topotecan hydrochloride market?

Key factors include the increasing prevalence of cancer, advancements in drug formulations, increased investments in oncology research, and the growing demand for personalized medicine and targeted therapies.

Sources

  1. Pfizer Medical Information: Topotecan injection Clinical Studies.
  2. Cognitive Market Research: Topotecan Hydrochloride Sales Market Report 2024 (Global Edition).
  3. Cancer Network: Clinical Status and Optimal Use of Topotecan.
  4. Journal of Clinical Oncology: A phase III study of lurbinectedin alone or in combination with irinotecan vs investigator's choice (topotecan or irinotecan) in patients with relapsed small cell lung cancer (SCLC; LAGOON trial).

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