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	39
Lung Cancer	31

Condition MeSH

Condition MeSH for TOPOTECAN HYDROCHLORIDE
Intervention	Trials
Small Cell Lung Carcinoma	108
Ovarian Neoplasms	100
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	90
Australia	88
United Kingdom	79
Germany	73
Italy	68

Trials by US State

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

Clinical Trial Progress for TOPOTECAN HYDROCHLORIDE

Clinical Trial Phase

Clinical Trial Phase for TOPOTECAN HYDROCHLORIDE
Clinical Trial Phase	Trials
PHASE3	22
PHASE2	11
PHASE1	5

Clinical Trial Status

Clinical Trial Status for TOPOTECAN HYDROCHLORIDE
Clinical Trial Phase	Trials
Completed	243
Recruiting	72
Terminated	44

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

Sponsor Type

Sponsor Type for TOPOTECAN HYDROCHLORIDE
Sponsor	Trials
Other	441
Industry	252
NIH	144

Last updated: July 22, 2026

Topotecan Hydrochloride clinical trials update, market analysis, and exclusivity timeline (2026)

Topotecan hydrochloride is an established oncology cytotoxic with ongoing development focused on new combinations, schedules, and dosing strategies rather than a single “platform” breakthrough. Commercially, the drug remains a niche but defensible option in small-cell lung cancer (SCLC) and ovarian cancer, with demand constrained by limited first-line positioning, sensitivity-driven use patterns, and bioshare/generic pressure in many markets. From an IP and regulatory-exclusivity standpoint, topotecan’s key brand-era exclusivities are largely over; the primary remaining barriers are formulation, method-of-use, and jurisdiction-by-jurisdiction patent estates tied to specific presentations and dosing regimens.

---

What clinical trials are ongoing for topotecan hydrochloride in 2024–2026?

Answer: Most active activity is combination-focused (topotecan + PD-1/PD-L1 inhibitors, topotecan + anti-angiogenics, and topotecan + other cytotoxics), plus studies optimizing schedules to improve tolerability and response depth in heavily pretreated populations.

Which trial types dominate (and why)

1. Relapsed/refractory SCLC
   Topotecan remains a backbone option in later lines, so trials often test whether adding immunotherapy or targeted agents improves response rates and duration.
2. Recurrent ovarian cancer
   Trials tend to examine topotecan-based regimens when platinum sensitivity is limited or when recurrence occurs after prior lines.
3. Schedule and dose optimization
   Because topotecan toxicity is dose-limiting (especially myelosuppression), studies frequently compare standard vs altered schedules to reduce treatment interruptions.

What endpoints matter for decision-making

• ORR and DoR in SCLC and ovarian studies
• OS as the gating endpoint for payer and label expansion
• Safety/tolerability for regimen viability (dose intensity, grade 3–4 neutropenia, anemia, thrombocytopenia)
• Biomarker response signals (PD-L1, tumor mutational burden proxies, circulating tumor markers), typically exploratory

Trial update pattern likely to impact investment

• Earlier-stage studies usually focus on response improvements and safety to justify Phase 2 expansion.
• Later-stage programs tend to seek OS differentiation or to support a label claim tied to a combination partner.
• If a trial fails to show meaningful ORR/OS benefit, development typically exits quickly because topotecan lacks a proprietary delivery advantage in most markets.

Note: A precise, protocol-level “ongoing trials list” requires current registry pulls (ClinicalTrials.gov/EMA catalog) with drug presentation, sponsor, start/end status, and NCT numbers. Without that dataset in the prompt, this update is constrained to the development patterns that consistently characterize topotecan programs across major geographies.

---

How does topotecan hydrochloride compare with other SCLC and ovarian cytotoxics in efficacy?

Answer: In SCLC and recurrent ovarian cancer, topotecan competes with other later-line cytotoxics by balancing response rates, symptom control, and tolerability. Its differentiation is usually schedule- and combination-dependent, not an intrinsic superiority over all alternatives.

Key competitor clusters

• SCLC later lines: docetaxel, paclitaxel, vinorelbine, amrubicin (regional), gemcitabine (varies by market), and combination regimens
• Ovarian recurrent disease: pegylated liposomal doxorubicin, gemcitabine, weekly paclitaxel, etoposide-based regimens, and targeted/combination approaches depending on biomarker status

Practical positioning

• SCLC: topotecan is commonly used when prior regimens fail; outcomes are strongly influenced by prior exposure, performance status, and disease kinetics.
• Ovarian: topotecan use concentrates in refractory or recurrent settings with limited platinum options; prior PARP inhibitor exposure can shift sequencing decisions in practice.

---

What is the market size for topotecan hydrochloride and how is it projected to 2030?

Answer: The addressable market remains anchored in SCLC and recurrent ovarian cancer. Growth expectations are typically modest and come from geography-by-geography replacement of older presentations, uptake of combination protocols where supported by clinical data, and incremental demand from population and regimen sequencing. Structural decline risk exists from generic substitution, with any value uplift limited to branded combination utilization or patents that protect specific formulations.

Demand drivers

• Cancer incidence and treatment volumes in SCLC and ovarian cancer
• Line-of-therapy position (later-line use makes total volumes smaller but more stable)
• Hospital acquisition patterns favoring cost-effective cytotoxic generics
• Protocol adoption when combination trials show a workable benefit-to-toxicity ratio

Key headwinds

• Generic penetration for standard topotecan presentations in many jurisdictions
• Toxicity management costs (growth factor use, transfusion needs, monitoring)
• Guideline evolution that can shift cytotoxic choice when immunotherapy or targeted regimens become preferred in specific subgroups

Projection framing (business view)

• Base case: flat-to-low single-digit CAGR through 2030, with volume stability offset by price pressure from generics.
• Upside case: modest growth where combination partners expand adoption in SCLC/ovarian populations and where patent-protected presentations or local supply contracts sustain pricing.
• Downside case: faster erosion in price and substitution if more national markets fully transition to low-cost generic supply chains.

A quantified global market forecast (US/EU/ROW split, $ value CAGR) requires recent commercial datasets (IQVIA, GlobalData, company filings) not provided in the prompt.

---

Which companies sell topotecan hydrochloride globally and what is the competitive landscape?

Answer: Competition is dominated by generic manufacturers in mature markets, with brand-era or specialty supply sometimes persisting in select countries. Competitive intensity is driven by tender pricing in hospital oncology formularies.

How competition typically plays out

• US and EU: multiple approved generic versions; procurement tends to select lowest total cost including availability and contract terms.
• Emerging markets: fewer suppliers can exist per country, but price normalization trends persist as approvals expand.
• Presentation-specific competition: vial strength and administration form (IV vs oral, if applicable to a given approved presentation) can change local supplier landscapes.

What to monitor

• New ANDA/MAA approvals that lower price ceilings
• Drug shortages or supply disruptions that temporarily increase pricing power
• Replacement of originator stock with tender-led generic switches

---

What patents protect topotecan hydrochloride, and what is the strength of the patent estate?

Answer: For topotecan hydrochloride, patent protection in practice is usually concentrated in:

• Formulations (e.g., specific excipient systems, stability improvements, or manufacturing-related constraints)
• Methods of use (specific dosing regimens, combination therapies with defined sequences)
• Process/manufacturing patents (impurity profiles, salt/crystal form control where asserted)

Core active-ingredient composition protection from early development-era filings is typically expired or near-expired in most jurisdictions, making the remaining “estate” largely derivative and presentation- or jurisdiction-specific.

Where strength tends to concentrate

• Specific regimen patents can create “clinical use” barriers even after generic entry if the method claims match how clinicians treat.
• Combination method-of-use patents can matter most if a partner’s regimen becomes standard-of-care.
• Formulation patents can retain value if they map to commercially used presentations and if regulatory labeling ties to the protected formulation.

Litigation risk pattern

• Generic entry risk is highest when a jurisdiction’s remaining patents map cleanly to a common label indication and dosing schedule.
• Settlement often relies on agreed launch dates, supply constraints, or covenants that reduce risk while avoiding adjudication.

A complete, patent-number-level mapping across jurisdictions requires Orange Book and patent family extraction not included in the prompt.

---

When does topotecan hydrochloride lose exclusivity in key markets (US, EU, UK, Canada)?

Answer: Active-ingredient exclusivity for early topotecan introductions is largely over. Remaining exclusivity is typically driven by:

• Presentation-specific patents (formulations/process/method-of-use)
• Regulatory data exclusivity tied to specific filings
• Paediatric extensions or national SPC structures if applicable to later-protected presentations

Decision logic for launch planning

• Check whether the intended generic product is tied to a protected formulation/presentation.
• Verify whether the generic ANDA/MAA is designed to avoid infringement of the method-of-use claims (often via labeling carve-outs).

Exact dates require listing-specific regulatory and patent data. The prompt does not provide Orange Book/EP register numbers.

---

What is the Orange Book status of topotecan hydrochloride?

Answer: In the US, topotecan hydrochloride is not generally treated as an active “Orange Book blockbuster” case; most listings are associated with older brands or later formulation/process patents that may still be present for specific strengths/presentations.

A strict Orange Book status summary requires the drug’s NDC-specific Orange Book entry list, patent numbers, and expiration dates. No NDC list is provided in the prompt.

---

Which generic entry risks exist for topotecan hydrochloride (ANDA Paragraph IV)?

Answer: The entry risk exists where:

• Remaining patents cover a common dosing schedule or method-of-use claim that matches standard practice.
• A generic filer targets a label or indication that would infringe method claims.

In many cases, because older core claims are expired, litigation or Paragraph IV disputes trend toward resolving:

• Presentation/formulation infringement
• Method-of-use labeling boundaries

Paragraph IV incidence, suit dates, and court outcomes require the Orange Book litigation record or FDA Paragraph IV notices not included in the prompt.

---

What patent litigation affects topotecan hydrochloride?

Answer: Litigation risk typically involves infringement of:

• Method-of-use claims tied to dosing regimens or combinations
• Formulation/process claims tied to a particular manufacturing method or stability profile

Case-by-case outcomes determine whether later entrants face delayed launch or carve-out labeling.

A litigation “affected by” section requires named suits, venues, docket numbers, and settlement terms. None are provided in the prompt.

---

What formulations and dosing presentations are used commercially for topotecan hydrochloride?

Answer: Commercial practice generally uses IV topotecan for SCLC and ovarian indications, with dosing schedules that vary by protocol and line of therapy. Some markets also support oral topotecan presentations, but availability depends on local approvals and product launches.

What tends to matter for IP

• Patents may protect the specific formulation (excipients, stability, particle/crystal characteristics).
• IP may also protect dose scheduling when it maps to a method-of-use claim.

---

How does topotecan hydrochloride business performance differ by line of therapy and geography?

Answer: Topotecan’s business performance is best characterized as:

• Line-of-therapy constrained: later-line positioning limits total volume growth.
• Geography variable: tender systems and supplier count determine realized pricing more than clinical differentiation.
• Patient volume steadiness: incidence-related volumes can be stable, but regimen switching driven by new standards can shift demand away from topotecan.

What changes most performance in-year

• New combination uptake in SCLC and ovarian settings
• Generic pricing resets driven by competitive tender awards
• Shortage events or supply constraints that temporarily lift net price

---

Key Takeaways

• Topotecan hydrochloride development in 2024–2026 is primarily combination and schedule optimization rather than a new mechanism-led expansion.
• Market growth is typically modest and exposed to generic price erosion; upside depends on regimen adoption tied to partner-driven trials and label expansions.
• Remaining IP barriers are usually presentation- and regimen-specific (formulation, process, method-of-use) rather than active ingredient composition.
• A full exclusivity and litigation map requires NDC- and jurisdiction-specific Orange Book and patent-family extraction; core exclusivity from early launches is generally not the dominant constraint.

---

FAQs

1. Does topotecan hydrochloride have oral and IV exclusivity differences that affect generic launch timing?
2. What combination partners most frequently study topotecan in relapsed small-cell lung cancer?
3. How do method-of-use patents influence generic labeling for topotecan-based regimens?
4. What tolerability management strategies (G-CSF, transfusion thresholds) drive real-world dosing intensity for topotecan?
5. Where do drug shortages most affect topotecan pricing and hospital procurement behavior?

---

References

1. ClinicalTrials.gov. (accessed via ongoing registry review, not included in prompt dataset).
2. FDA Orange Book (topotecan hydrochloride NDC-specific listings, not included in prompt dataset).
3. EMA/UK national registers (SPC and patent term adjustments, not included in prompt dataset).