Data updated: May 26, 2026
TOPOTECAN (topotecan hydrochloride)
Trial Activity: Stable 65 active trials
Oncology
Approved 2011-02-25
Development Insights
National Cancer Institute (NCI) conducting 25 trials (17%)
328 indications explored (Broad Platform)
→ neuroblastoma (19 trials)
→ small cell lung cancer (16 trials)
→ ovarian cancer (14 trials)
1
Indication
--
Phase 3 Trials
15
Years on Market
Details
- Status
- Discontinued
- First Approved
- 2011-02-25
- Routes
- INTRAVENOUS
- Dosage Forms
- SOLUTION
TOPOTECAN Approval History
2012
2013
2014
2015
2016
2017
2018
2019
2020
2021
2022
2023
2024
2025
2026
Original
New Indication
New Form
Label Update
6 FDA actions from 2011 to 2014
What TOPOTECAN Treats
1 FDA approvalsOriginally approved for its first indication in 2011 .
- Other (1)
📋
Clinical Trial Registry
146 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT06172296 | NCI-2023-08530 NCI-2023-08530, ANBL2131 | Ph 3 | recruiting | Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma |
| NCT05353439 | NCI-2022-03215 NCI-2022-03215, 22-C-0005 | Ph 1 | active not recruiting | Testing of Tazemetostat in Combination With Topotecan and Pembrolizumab in Patients With Recurrent Small Cell Lung Cancer |
| NCT05153239 | PM1183-C-008-21 | Ph 3 | completed | Clinical Trial of Lurbinectedin as Single-agent or in Combination With Irinotecan Versus Topotecan or Irinotecan in Patients With Relapsed Small-cell Lung Cancer (LAGOON) |
| NCT06161025 | DS6000-109 REJOICE-Ovarian01, ENGOT-ov77 | Ph 2, Ph 3 | recruiting | A Study of Raludotatug Deruxtecan (R-DXd) in Subjects With Platinum-resistant, High-grade Ovarian, Primary Peritoneal, or Fallopian Tube Cancer |
| NCT02308527 BEACON | RG_11-087 2012-000072-42 | Ph 2 | completed | Activity Study of Bevacizumab With Temozolomide ± Irinotecan for Neuroblastoma in Children |
| NCT02354547 | SGT53-01-2 | Ph 1 | suspended | A Study of SGT-53 in Children With Refractory or Recurrent Solid Tumors |
| NCT06459180 | 2870-020 MK-2870-020, TroFuse-020 | Ph 3 | recruiting | A Study to Compare Sacituzumab Tirumotecan (MK-2870) Monotherapy Versus Treatment of Physician's Choice as Second-line Treatment for Participants With Recurrent or Metastatic Cervical Cancer (MK-2870-020/TroFuse-020/Gog-3101/ENGOT-cx20) |
| NCT06647953 | ARAR2331 NCI-2024-08232, ARAR2331 | Ph 3 | recruiting | Testing a Standardized Approach to Surgery and Chemotherapy for Type I Pleuropulmonary Blastoma or the Addition of an Anti-cancer Drug, Topotecan, to the Usual Treatment for Types II and III Pleuropulmonary Blastoma |
| NCT04322318 | AREN1921 NCI-2020-01561, AREN1921 | Ph 2 | recruiting | A Study of Combination Chemotherapy for Patients With Newly Diagnosed DAWT and Relapsed FHWT |
| NCT05613088 results posted | CA116-001 2021-004807-42 | Ph 2 | completed | A Study of MORAb-202 Versus Investigator's Choice Chemotherapy in Female Participants With Platinum-resistant High-grade Serous (HGS) Ovarian, Primary Peritoneal, or Fallopian Tube Cancer |
| NCT06801834 EVOKE-SCLC-04 | GS-US-600-6165 2024-515884-69 | Ph 3 | recruiting | Study of Sacituzumab Govitecan Versus Standard of Care in Participants With Previously Treated Extensive Stage Small Cell Lung Cancer |
| NCT02502266 results posted | NCI-2015-00651 NCI-2015-00651, NRG-GY005 | Ph 2, Ph 3 | active not recruiting | Testing the Combination of Cediranib and Olaparib in Comparison to Each Drug Alone or Other Chemotherapy in Recurrent Platinum-Resistant Ovarian Cancer |
| NCT06619236 RAINFOL-02 | GCT1184-02 jRCT2031250064, CTR20253987 | Ph 3 | active not recruiting | Study to Assess the Efficacy of Rina-S Compared to Treatment of Investigator's Choice in Participants With Platinum Resistant Ovarian Cancer |
| NCT01012817 results posted | NCI-2011-00312 NCI-2011-00312, 09-000742 | Ph 1, Ph 2 | active not recruiting | Veliparib and Topotecan Hydrochloride in Treating Patients With Solid Tumors, Relapsed or Refractory Ovarian Cancer, or Primary Peritoneal Cancer |
| NCT07545460 | BL-M07D1-308 | Ph 3 | not yet recruiting | A Study Comparing BL-M07D1 With Physician's Choice of Chemotherapy in Patients With HER2-Expressing Platinum-Resistant Recurrent Epithelial Ovarian Cancer, Fallopian Tube Cancer, and Primary Peritoneal Cancer |
| NCT03154996 | AAAQ9520 5R01CA161404-02 | Ph 1 | completed | Chronic Convection Enhanced Delivery of Topotecan |
| NCT07099898 | 223674 2025-521627-78-00 | Ph 3 | recruiting | A Study of GSK5764227 in Participants With Relapsed Small Cell Lung Cancer (SCLC) |
| NCT05101551 PARPAML | IRB-66573 PEDSHEMAML0008, POE22-01 | Ph 1 | recruiting | Study of Talazoparib in Combination With Chemotherapy in Relapsed Pediatric AML to Determine Safety and Efficacy |
| NCT04106219 | 17295 J1O-MC-JZHD, 2019-001042-18 | Ph 1 | active not recruiting | A Study of LY3295668 Erbumine in Participants With Relapsed/Refractory Neuroblastoma |
| NCT06449209 | BNT327-01 | Ph 2 | active not recruiting | Safety, Preliminary Effectiveness of BNT327, an Investigational Therapy for Patients With Small-cell Lung Cancer in Combination With Chemotherapy |
| NCT07213804 FRAmework-01 | 27727 2025-522255-25-00, J5E-MC-JZXB | Ph 3 | recruiting | A Two-Part Phase 3 Study of Sofetabart Mipitecan (LY4170156) in Participants With Platinum-Resistant (Part A) and Platinum-Sensitive (Part B) Ovarian Cancer |
| NCT06500026 | BL-B01D1-304 | Ph 3 | active not recruiting | A Study Comparing BL-B01D1 With Topotecan in Patients With Recurrent Small Cell Lung Cancer(PANKU-Lung03) |
| NCT01857934 results posted | NB2012 NCI-2013-00034 | Ph 2 | completed | Therapy for Children With Advanced Stage Neuroblastoma |
| NCT06666712 TPT CED rHGG | AAAV1710 | Ph 1 | recruiting | Chronic CED of TPT for Recurrent Malignant Glioma |
| NCT07437963 | NCI-2026-01050 NCI-2026-01050, ANBL2421 | Ph 1, Ph 2 | not yet recruiting | Testing the Addition of Iberdomide to Therapy in People With Neuroblastoma That Has Come Back, Not Responded to Treatment, or Gotten Worse |
| NCT03786783 results posted | NCI-2018-03732 NCI-2018-03732, ANBL17P1 | Ph 2 | active not recruiting | Dinutuximab, Sargramostim, and Combination Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma |
| NCT03289910 results posted | NCI-2017-01715 NCI-2017-01715, ETCTN10147 | Ph 2 | active not recruiting | Topotecan Hydrochloride and Carboplatin With or Without Veliparib in Treating Advanced Myeloproliferative Disorders and Acute Myeloid Leukemia or Chronic Myelomonocytic Leukemia |
| NCT03896503 results posted | NCI-2019-01769 NCI-2019-01769, 20-C-0009 | Ph 2 | active not recruiting | Randomized Trial of Topotecan With M6620, an ATR Kinase Inhibitor, in Small Cell Lung Cancers and Small Cell Cancers Outside of the Lungs |
| NCT04739800 results posted | NCI-2021-00615 NCI-2021-00615, NRG-GY023 | Ph 2 | active not recruiting | Comparison of Standard of Care Treatment With a Triplet Combination of Targeted Immunotherapeutic Agents |
| NCT05521984 | 202210013 | Ph 1 | recruiting | Targeting Pediatric Brain Tumors and Relapsed/Refractory Solid Tumors With Sodium Glucose Cotransporter 2 Inhibitors (SGLT2i) |
| NCT05990738 | 1438-0009 2023-506007-26-00, U1111-1293-4098 | Ph 1 | active not recruiting | DAREON™-9: A Study to Test How Well Different Doses of BI 764532 Are Tolerated by People With Small Cell Lung Cancer When Taken Together With a Single Agent Chemotherapy |
| NCT06930755 | PARPA-293-003 | Ph 1 | recruiting | Study of NMS-03305293 in Adult Patients With Relapsed Ovarian Cancer |
| NCT04994977 | 20-08022610 | Ph 1 | terminated | Intra-Arterial Chemotherapy for Newly Diagnosed, Residual, or Recurrent Atypical Choroid Plexus Papilloma and Choroid Plexus Carcinoma Prior to Second-Look Surgery |
| NCT02298348 N2013-02 | NANT 2013-02 | Ph 1 | active not recruiting | Sorafenib and Cyclophosphamide/Topotecan in Patients With Relapsed and Refractory Neuroblastoma |
| NCT07286266 | 224031 2025-523361-25-00 | Ph 3 | not yet recruiting | A Study to Investigate GSK5733584 Compared With Chemotherapy in Participants With Platinum-resistant Ovarian Cancer (BEHOLD-Ovarian01) |
| NCT02114229 SJATRT | ATRT NCI-2014-00901 | Ph 2 | active not recruiting | Phase 2 Study of Alisertib Therapy for Rhabdoid Tumors |
| NCT07194044 METTSEO | MCC-23281 | Ph 1 | recruiting | Metastatic Ewing's Trial Testing Schedule Enhancement to Improve Outcomes |
| NCT06203210 IDeate-Lung02 | DS7300-188 2023-509628-16-00, 2031230631 | Ph 3 | recruiting | A Study of Ifinatamab Deruxtecan Versus Treatment of Physician's Choice in Subjects With Relapsed Small Cell Lung Cancer |
| NCT03126916 | ANBL1531 NCI-2016-01734, ANBL1531 | Ph 3 | recruiting | Testing the Addition of 131I-MIBG or Lorlatinib to Intensive Therapy in People With High-Risk Neuroblastoma (NBL) |
| NCT04697628 innovaTV 301 results posted | SGNTV-003 C5721002, 2023-503813-31-01 | Ph 3 | completed | Tisotumab Vedotin vs Chemotherapy in Recurrent or Metastatic Cervical Cancer |
| NCT05535166 | SJiMB21 NCI-2022-07099, R21CA280187 | Ph 2 | recruiting | Molecular and Clinical Risk-Directed Therapy for Infants and Young Children With Newly Diagnosed Medulloblastoma |
| NCT06528496 | 24-144 | Ph 2 | recruiting | N10: A Study of Reduced Chemotherapy and Monoclonal Antibody (mAb)-Based Therapy in Children With Neuroblastoma |
| NCT07365241 | M23-384 2025-523819-11 | Ph 3 | not yet recruiting | A Study to Evaluate Adverse Events and Change in Disease Activity of Intravenous ABBV-706 Versus Standard of Care in Adult Participants With Relapsed/Refractory Small Cell Lung Cancer |
| NCT01175356 results posted | ANBL09P1 NCI-2011-01745, ANBL09P1 | Ph 1 | completed | Induction Therapy Including 131 I-MIBG and Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma Undergoing Stem Cell Transplant, Radiation Therapy, and Maintenance Therapy With Isotretinoin |
| NCT01231906 results posted | AEWS1031 NCI-2011-02611, S12-01231 | Ph 3 | completed | Combination Chemotherapy in Treating Patients With Non-Metastatic Extracranial Ewing Sarcoma |
| NCT02813135 ESMART | 2016-000133-40 2016/2396, 2024-514791-40-00 | Ph 1, Ph 2 | recruiting | European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory Tumors |
| NCT07218809 TREVI-OC-01 | D8991C00001 | Ph 3 | recruiting | AZD5335 vs. Mirvetuximab Soravtansine in FRα-high and AZD5335 vs. Chemotherapy in FRα-low Platinum-resistant Ovarian Cancer |
| NCT07334301 BEACON2 | RG_22-136 | Ph 1, Ph 2 | recruiting | A Multi-Arm, Platform Trial For Relapsed Neuroblastoma |
| NCT07321912 | BCC023 | Ph 2 | not yet recruiting | Eflornithine (DFMO) for Ewing Sarcoma and Osteosarcoma |
| NCT05740566 DeLLphi-304 results posted | 20210004 | Ph 3 | active not recruiting | Study Comparing Tarlatamab With Standard of Care Chemotherapy in Relapsed Small Cell Lung Cancer |
Showing 50 of 146 trials
🔬
Pro Active Pipeline
Ongoing clinical trials by development phase
Loading...
⭐
Pro Key Completed Trials
Completed studies with published results, ranked by significance
Loading...
📊
Trial Timeline
Full development history with FDA approval milestones
|
Loading...
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
TOPOTECAN FDA Label Details
Track TOPOTECAN with TheraRadar Pro
Watchlist alerts, full database access, CSV exports across 14,000+ drugs.
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment