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Cyclophosphamide and Topotecan
This combination uses cyclophosphamide as an alkylating agent to damage cancer cell DNA and topotecan as a topoisomerase I inhibitor to prevent DNA repair, working synergistically to kill cancer cells.
This combination uses cyclophosphamide as an alkylating agent to damage cancer cell DNA and topotecan as a topoisomerase I inhibitor to prevent DNA repair, working synergistically to kill cancer cells. Used for Pediatric solid tumors (Phase 2 investigational).
At a glance
| Generic name | Cyclophosphamide and Topotecan |
|---|---|
| Also known as | Cytoxan and Hycamptin |
| Sponsor | Children's Medical Center Dallas |
| Drug class | Combination chemotherapy (alkylating agent + topoisomerase I inhibitor) |
| Target | DNA (alkylation and topoisomerase I inhibition) |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 2 |
Mechanism of action
Cyclophosphamide is a nitrogen mustard alkylating agent that cross-links DNA strands, preventing replication and transcription. Topotecan inhibits topoisomerase I, an enzyme required to relieve DNA tension during replication, leading to DNA breaks. The combination exploits complementary mechanisms to enhance cytotoxicity in rapidly dividing cancer cells.
Approved indications
- Pediatric solid tumors (Phase 2 investigational)
Common side effects
- Myelosuppression (neutropenia, thrombocytopenia)
- Nausea and vomiting
- Alopecia
- Mucositis
- Hemorrhagic cystitis (cyclophosphamide-related)
Key clinical trials
- Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma (PHASE3)
- Testing the Addition of Iberdomide to Therapy in People With Neuroblastoma That Has Come Back, Not Responded to Treatment, or Gotten Worse (PHASE1, PHASE2)
- Sorafenib and Cyclophosphamide/Topotecan in Patients With Relapsed and Refractory Neuroblastoma (PHASE1)
- Phase 2 Study of Alisertib Therapy for Rhabdoid Tumors (PHASE2)
- Metastatic Ewing's Trial Testing Schedule Enhancement to Improve Outcomes (PHASE1)
- Testing the Addition of 131I-MIBG or Lorlatinib to Intensive Therapy in People With High-Risk Neuroblastoma (NBL) (PHASE3)
- Pilot Study of IT Topotecan and Maintenance Chemotherapy for HR-EBTs in Children < 6 Years, Post Consolidation (EARLY_PHASE1)
- 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 (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Cyclophosphamide and Topotecan CI brief — competitive landscape report
- Cyclophosphamide and Topotecan updates RSS · CI watch RSS
- Children's Medical Center Dallas portfolio CI