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Compound · clinical trials

Topotecan clinical trials

Source: ClinicalTrials.gov, U.S. National Library of Medicine — the 31 trials in our China, USA, UK, Ireland and India registers that name Topotecan as an intervention. Synced 25 Sep 2026
31Current trials
18Recruiting now
25Lead sponsors
5Country registers
Maintained by Sreepriya Prasannan
MSc Digital Transformation of Life Sciences (Innopharma Education / Griffith College); MSc & BSc Botany HSE Spark Ignite 2026 โ€” Top 14 finalist
Compiled from ClinicalTrials.gov registry records ยท Last verified ยท Editorial standards
At a glance

Topotecan is named as an intervention in 31 current clinical trials in our China, USA, UK, Ireland and India registers. 18 are recruiting patients now. The most common phase is Phase 3. The sponsor with the most trials is Sichuan Baili Pharmaceutical Co., Ltd (3). The most-studied condition is Small Cell Lung Cancer.

Trials by status

  • Recruiting1858%
  • Not yet open413%
  • Active, closed929%

Trials by phase

  • Phase 32168%
  • Phase 2413%
  • Phase 1310%
  • Phase 2/313%
  • Phase 1/213%
  • Phase EARLY_PHASE113%
01

Clinical trials of Topotecan

31 trials
StudyPhaseStatusLead sponsorRegisters
High-dose Topotecan for Retinoblastoma With Recurrent or Refractory Vitreous Seed
NCT06972602 · ClinicalTrials.gov ↗ · Retinoblastoma, Retinoblastoma, Recurrent
Phase EARLY_PHASE1 Recruiting Eye & ENT Hospital of Fudan University China
Topotecan and Melphalan for Retinoblastoma
NCT04799002 · ClinicalTrials.gov ↗ · Retinoblastoma, Chemotherapy Effect
Phase 3 Recruiting Sun Yat-sen University China
A Study of Ifinatamab Deruxtecan Versus Treatment of Physician's Choice in Subjects With Relapsed Small Cell Lung Cancer
NCT06203210 · ClinicalTrials.gov ↗ · Small Cell Lung Cancer
Phase 3 Recruiting Daiichi Sankyo USA, China
SHR-A1921 for Injection in Patients With Platinum-Resistant Recurrent Epithelial Ovarian Cancer
NCT06394492 · ClinicalTrials.gov ↗ · Ovarian Cancer
Phase 3 Recruiting Suzhou Suncadia Biopharmaceuticals Co., Ltd. China
Lurbinectedin or in Combination with Irinotecan Versus Topotecan in Patients with Relapsed SCLC
NCT06496048 · ClinicalTrials.gov ↗ · Relapsed Small Cell Lung Cancer
Phase 3 Recruiting Luye Pharma Group Ltd. China
ARTEMIS-008๏ผšHS-20093 Compared With Topotecan in Subjects With Relapsed Small Cell Lung Cancer
NCT06498479 · ClinicalTrials.gov ↗ · Small Cell Lung Cancer
Phase 3 Recruiting Hansoh BioMedical R&D Company China
Study of Sacituzumab Govitecan Versus Standard of Care in Participants With Previously Treated Extensive Stage Small Cell Lung Cancer
NCT06801834 · ClinicalTrials.gov ↗ · Extensive Stage Small Cell Lung Cancer (ES-SCLC)
Phase 3 Recruiting Gilead Sciences UK, USA, China
HS-20089 for Injection in Patients With Platinum-Resistant Recurrent Epithelial Ovarian Cancer
NCT06855069 · ClinicalTrials.gov ↗ · Ovarian Cancer
Phase 3 Recruiting Hansoh BioMedical R&D Company China
A Study of MHB088C Injection Versus Treatment of Physician's Choice in Subjects With Relapsed Small Cell Lung Cancer
NCT06954246 · ClinicalTrials.gov ↗ · Small Cell Lung Cancer Extensive Stage
Phase 3 Recruiting Qilu Pharmaceutical Co., Ltd. China
A Study Comparing BL-B01D1 With the Investigator's Choice of Chemotherapy in Patients With Platinum-resistant Recurrent Epithelial Ovarian Cancer(PANKU-GYN01)
NCT06994195 · ClinicalTrials.gov ↗ · Epithelial Ovarian Cancer, Fallopian Tube Cancer, Primary Peritoneal Cancer
Phase 3 Recruiting Sichuan Baili Pharmaceutical Co., Ltd. China
A Study of GSK5764227 in Participants With Relapsed Small Cell Lung Cancer (SCLC)
NCT07099898 · ClinicalTrials.gov ↗ · Neoplasms, Lung
Phase 3 Recruiting GlaxoSmithKline Ireland, UK, USA
A Three-Part Phase 3 Study of Sofetabart Mipitecan in Participants With Platinum-Resistant (Part A) and Platinum-Sensitive (Parts B and C) Ovarian Cancer
NCT07213804 · ClinicalTrials.gov ↗ · Ovarian Neoplasms, Fallopian Tube Neoplasms, Peritoneal Neoplasms
Phase 3 Recruiting Eli Lilly and Company Ireland, UK, USA, China
AZD5335 vs. Mirvetuximab Soravtansine in FRฮฑ-high and AZD5335 vs. Chemotherapy in FRฮฑ-low Platinum-resistant Ovarian Cancer
NCT07218809 · Epithelial Ovarian Cancer
Phase 3 Recruiting AstraZeneca Ireland, UK, USA, India, China
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
NCT07365241 · Small Cell Lung Cancer
Phase 3 Recruiting AbbVie USA
An Extension Study to Assess the Efficacy of Rina-S Compared to Treatment of Investigator's Choice in Participants With Platinum Resistant Ovarian Cancer in China
NCT07604766 · ClinicalTrials.gov ↗ · Platinum-resistant Ovarian Cancer
Phase 3 Recruiting Genmab China
A Study of Raludotatug Deruxtecan (R-DXd) in Subjects With Platinum-resistant, High-grade Ovarian, Primary Peritoneal, or Fallopian Tube Cancer
NCT06161025 · ClinicalTrials.gov ↗ · Solid Cancer
Phase 2/3 Recruiting Daiichi Sankyo UK, USA, China
A Study of Peluntamig (PT217) in Patients With Neuroendocrine Carcinomas Expressing DLL3 (the SKYBRIDGE Study)
NCT05652686 · ClinicalTrials.gov ↗ · Small Cell Lung Cancer (SCLC), Large Cell Neuroendocrine Cancer (LCNEC), Neuroendocrine Prostate Cancer (NEPC)
Phase 1/2 Recruiting Phanes Therapeutics USA
Study of NMS-03305293 in Adult Patients With Relapsed Ovarian Cancer
NCT06930755 · Ovarian Cancer
Phase 1 Recruiting Nerviano Medical Sciences USA
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
NCT07545460 · ClinicalTrials.gov ↗ · Epithelial Ovarian Cancer, Fallopian Tube Cancer, Primary Peritoneal Cancer
Phase 3 Not yet open Sichuan Baili Pharmaceutical Co., Ltd. China
JSKN033 Versus Investigator's Choice of Chemotherapy in Recurrent or Metastatic Cervical Cancer
NCT07797153 · ClinicalTrials.gov ↗ · Recurrent or Metastatic Cervical Cancer
Phase 3 Not yet open Jiangsu Alphamab Biopharmaceuticals Co., Ltd China
Allogeneic Hematopoietic Stem Cell Transplantation for 4/M Neuroblastoma
NCT05303727 · ClinicalTrials.gov ↗ · Neuroblastoma
Phase 2 Not yet open Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University China
Oral Topotecan With Toripalimab for Patients With Endometrial Cancer
NCT06548763 · ClinicalTrials.gov ↗ · Endometrial Cancer
Phase 2 Not yet open Fudan University China
Study Comparing Tarlatamab With Standard of Care Chemotherapy in Relapsed Small Cell Lung Cancer
NCT05740566 · Small Cell Lung Cancer (SCLC)
Phase 3 Active, closed Amgen Ireland, UK, USA, China
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)
NCT06459180 · ClinicalTrials.gov ↗ · Cervical Cancer
Phase 3 Active, closed Merck Sharp & Dohme LLC Ireland, UK, USA, China
A Study Comparing BL-B01D1 With Topotecan in Patients With Recurrent Small Cell Lung Cancer(PANKU-Lung03)
NCT06500026 · ClinicalTrials.gov ↗ · Small Cell Lung Cancer
Phase 3 Active, closed Sichuan Baili Pharmaceutical Co., Ltd. China
PM8002 in Combination With Paclitaxel Compared With Chemotherapy as Second-line Treatment in Small Cell Lung Cancer
NCT06616532 · ClinicalTrials.gov ↗ · SCLC
Phase 3 Active, closed Biotheus Inc. China
Study to Assess the Efficacy of Rina-S Compared to Treatment of Investigator's Choice in Participants With Platinum Resistant Ovarian Cancer
NCT06619236 · ClinicalTrials.gov ↗ · Platinum-resistant Ovarian Cancer
Phase 3 Active, closed Genmab USA, China
Study Of Palbociclib Combined With Chemotherapy In Pediatric Patients With Recurrent/Refractory Solid Tumors
NCT03709680 · ClinicalTrials.gov ↗ · Ewing Sarcoma, Solid Tumors, Rhabdoid Tumor
Phase 2 Active, closed Pfizer UK, USA, India
Safety, Preliminary Effectiveness of BNT327, an Investigational Therapy for Patients With Small-cell Lung Cancer in Combination With Chemotherapy
NCT06449209 · ClinicalTrials.gov ↗ · Extensive-stage Small-cell Lung Cancer, Small-cell Lung Cancer
Phase 2 Active, closed BioNTech SE UK, USA
A Study of LY3295668 Erbumine in Participants With Relapsed/Refractory Neuroblastoma
NCT04106219 · ClinicalTrials.gov ↗ · Neuroblastoma
Phase 1 Active, closed Eli Lilly and Company UK, USA
DAREONโ„ข-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
NCT05990738 · Small Cell Lung Carcinoma (SCLC)
Phase 1 Active, closed Boehringer Ingelheim UK, USA

Registers list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Trial titles and statuses are copied from the registry as published.

02

Conditions studied with Topotecan

34 conditions
Small Cell Lung Cancer4Epithelial Ovarian Cancer3Ovarian Cancer3Small Cell Lung Cancer (SCLC)2Neuroblastoma2Platinum-resistant Ovarian Cancer2Retinoblastoma2Fallopian Tube Cancer2Primary Peritoneal Cancer2Ovarian Neoplasms1Fallopian Tube Neoplasms1Peritoneal Neoplasms1
All 34 conditions
Condition (as registered)Trials
Small Cell Lung Cancer4
Epithelial Ovarian Cancer3
Ovarian Cancer3
Small Cell Lung Cancer (SCLC)2
Neuroblastoma2
Platinum-resistant Ovarian Cancer2
Retinoblastoma2
Fallopian Tube Cancer2
Primary Peritoneal Cancer2
Ovarian Neoplasms1
Fallopian Tube Neoplasms1
Peritoneal Neoplasms1
Neoplasm Metastasis1
Neoplasms, Lung1
Cervical Cancer1
Small Cell Lung Carcinoma (SCLC)1
Solid Cancer1
Extensive-stage Small-cell Lung Cancer1
Small-cell Lung Cancer1
Extensive Stage Small Cell Lung Cancer (ES-SCLC)1
Ewing Sarcoma1
Solid Tumors1
Rhabdoid Tumor1
Rhabdomyosarcoma1
Large Cell Neuroendocrine Cancer (LCNEC)1
Neuroendocrine Prostate Cancer (NEPC)1
Gastroenteropancreatic Neuroendocrine Carcinoma (GEP-NEC)1
Recurrent or Metastatic Cervical Cancer1
Retinoblastoma, Recurrent1
SCLC1
Small Cell Lung Cancer Extensive Stage1
Chemotherapy Effect1
Relapsed Small Cell Lung Cancer1
Endometrial Cancer1
03

Sponsors

lead sponsor per trial
Lead sponsorTrials
Sichuan Baili Pharmaceutical Co., Ltd.3
Eli Lilly and Company2
Daiichi Sankyo2
Genmab2
Hansoh BioMedical R&D Company2
AstraZeneca1
Amgen1
GlaxoSmithKline1
Merck Sharp & Dohme LLC1
Boehringer Ingelheim1
BioNTech SE1
Gilead Sciences1
Pfizer1
Nerviano Medical Sciences1
AbbVie1

+ 10 more sponsors in the trial list above.

04

By country register

RegisterTrialsRecruiting
Clinical trials in China2314
Clinical trials in USA169
Clinical trials in UK115
Clinical trials in Ireland53
Clinical trials in India21

A trial with sites in more than one country appears in each of those registers.

05

Related compounds

06

Questions about Topotecan trials

How many clinical trials are testing Topotecan?

31 current trials in our China, USA, UK, Ireland and India registers name Topotecan as a drug or biological intervention, as of the registry sync on 25 September 2026. Completed and withdrawn studies are not counted.

Are Topotecan trials recruiting patients?

Yes. 18 of the 31 trials are recruiting: 14 with sites in China, 9 with sites in USA, 5 with sites in UK, 3 with sites in Ireland and 1 with sites in India. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.

Who sponsors Topotecan clinical trials?

Sichuan Baili Pharmaceutical Co., Ltd (3), Eli Lilly and Company (2), Daiichi Sankyo (2) and Genmab (2), among 25 lead sponsors in total. The number in brackets is how many trials each organisation leads.

Where does this information come from?

Every trial, status, phase and sponsor on this page is copied from public ClinicalTrials.gov records by a daily sync. Nothing here describes how Topotecan works or whether it is effective, and it is not medical advice.

How this page is built

Everything on this page is copied from public ClinicalTrials.gov registry records โ€” it is not written from memory, and nothing here describes how Topotecan works or whether it is effective. Each trial links to its own record so you can check it yourself.

This page is built automatically: a daily sync job reads the registry records behind our country trial registers, collects every trial that lists Topotecan (or a registered synonym) as a drug or biological intervention, and tabulates the registry fields directly. Placebos and generic comparators are excluded. There is no AI drafting and no per-compound editorial pass, and the page has not been clinically reviewed. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The ClinicalTrials.gov data on this page was last retrieved on 25 Sep 2026.

Please verify before you rely on this. This page is general information for life-science and pharmaceutical professionals. It is not medical advice, and it has not been reviewed by a clinician โ€” our editorial team holds life-science qualifications, not clinical ones. It is not exhaustive and may not reflect the most recent label change. Always check the official prescribing information (US Prescribing Information or EU SmPC) and speak to your doctor or pharmacist before acting on anything here. Drugs in the same class are not automatically interchangeable, and approvals, brand names and indications differ between the US, the EU/Ireland (EMA/HPRA) and other regions. Spotted an error? Tell us โ€” we correct promptly and log it.