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Standard of Care: Cyclophosphamide
Cyclophosphamide is an alkylating agent that cross-links DNA strands, preventing cell division and causing cancer cell death.
Cyclophosphamide is an alkylating agent that cross-links DNA strands, preventing cell division and causing cancer cell death. Used for Lymphomas (Hodgkin and non-Hodgkin), Leukemias, Multiple myeloma.
At a glance
| Generic name | Standard of Care: Cyclophosphamide |
|---|---|
| Also known as | Cytoxan® |
| Sponsor | H. Lee Moffitt Cancer Center and Research Institute |
| Drug class | Alkylating agent |
| Target | DNA |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | FDA-approved |
Mechanism of action
Cyclophosphamide is a nitrogen mustard derivative that requires hepatic activation to form active metabolites. These metabolites bind to DNA and form inter- and intra-strand cross-links, disrupting DNA replication and transcription. This leads to apoptosis of rapidly dividing cells, particularly cancer cells, though it also affects immune cells and has immunosuppressive properties at lower doses.
Approved indications
- Lymphomas (Hodgkin and non-Hodgkin)
- Leukemias
- Multiple myeloma
- Breast cancer
- Ovarian cancer
- Lung cancer
- Autoimmune diseases (systemic lupus erythematosus, vasculitis)
Common side effects
- Myelosuppression (neutropenia, thrombocytopenia, anemia)
- Nausea and vomiting
- Alopecia
- Hemorrhagic cystitis
- Infertility
- Secondary malignancies
- Immunosuppression and infection risk
- Cardiotoxicity (at high doses)
Key clinical trials
- A Study Comparing Anitocabtagene Autoleucel to Standard of Care Therapy in Participants With Relapsed/ Refractory Multiple Myeloma (PHASE3)
- A Study to Compare Standard Therapy to Treat Hodgkin Lymphoma to the Use of Two Drugs, Brentuximab Vedotin and Nivolumab (PHASE3)
- A Phase III Trial Comparing Tisagenlecleucel to Standard of Care (SoC) in Adult Participants With r/r Follicular Lymphoma (PHASE3)
- US Study of ECT-001-CB in Pediatric and Young Adult Patients With High-Risk Myeloid Malignancies (PHASE1, PHASE2)
- Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide (EARLY_PHASE1)
- Impact of Vitamin D Supplementation on the Rate of Pathologic Complete Response in Vitamin D Deficient Patients (PHASE2)
- US Study of UM171-Expanded CB in Patients With High Risk Leukemia/Myelodysplasia (PHASE2)
- Cyclophosphamide, Paclitaxel, and Trastuzumab in Treating Stage I-II HER2/Neu Positive Breast Cancer After Surgery (PHASE2)
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 |
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