Last reviewed · How we verify
Cyclophosphamide (drug)
Cyclophosphamide is an alkylating agent that cross-links DNA strands, preventing cell division and triggering apoptosis in rapidly dividing cells.
Cyclophosphamide is an alkylating agent that cross-links DNA strands, preventing cell division and triggering apoptosis in rapidly dividing cells. Used for Lymphomas (Hodgkin and non-Hodgkin), Breast cancer, Ovarian cancer.
At a glance
| Generic name | Cyclophosphamide (drug) |
|---|---|
| Sponsor | University Hospital, Bordeaux |
| Drug class | Alkylating agent |
| Target | DNA (non-specific alkylation) |
| Modality | Small molecule |
| Therapeutic area | Oncology, Immunology |
| Phase | Phase 3 |
Mechanism of action
Cyclophosphamide is a nitrogen mustard derivative that requires hepatic activation to form active metabolites. These metabolites covalently bind to DNA, creating inter- and intra-strand cross-links that impair DNA replication and transcription. This mechanism is effective against both malignant cells and rapidly proliferating immune cells, making it useful in oncology and autoimmune conditions.
Approved indications
- Lymphomas (Hodgkin and non-Hodgkin)
- Breast cancer
- Ovarian cancer
- Lung cancer
- Severe autoimmune diseases (systemic lupus erythematosus, vasculitis)
Common side effects
- Myelosuppression (neutropenia, thrombocytopenia)
- Hemorrhagic cystitis
- Nausea and vomiting
- Alopecia
- Infertility (gonadal dysfunction)
- Secondary malignancies
- Immunosuppression and infection risk
Key clinical trials
- Nivolumab in Combination With Chemo-Immunotherapy for the Treatment of Newly Diagnosed Primary Mediastinal B-Cell Lymphoma (PHASE3)
- Combination Chemotherapy With or Without Ganitumab in Treating Patients With Newly Diagnosed Metastatic Ewing Sarcoma (PHASE3)
- Novel Combination Therapy in the Treatment of Relapsed and Refractory Aggressive B-Cell Lymphoma (PHASE2)
- Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary Immunodeficiencies (PHASE2)
- Testing the Addition of the Anti-cancer Drug Venetoclax and/or the Anti-cancer Immunotherapy Blinatumomab to the Usual Chemotherapy Treatment for Infants With Newly Diagnosed KMT2A-rearranged or KMT2A-non-rearranged Leukemia (PHASE2)
- Metabolically Fit CD19 CAR T-cell Therapy With CD34 Selection in Patients With CD19+ Relapsed/Refractory NHL, CLL/SLL (PHASE1, PHASE2)
- A Clinical Study of Sacituzumab Tirumotecan (Sac-TMT, MK-2870) in People With Breast Cancer (MK-2870-032) (PHASE3)
- A Study to Investigate Blinatumomab in Combination With Chemotherapy in Patients With Newly Diagnosed B-Lymphoblastic Leukemia (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 |