CLINICAL TRIALS PROFILE FOR CYTOXAN

505(b)(2) Clinical Trials for CYTOXAN

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Dosage	NCT01760226 ↗	Dose Adjusted EPOCH-R, to Treat Mature B Cell Malignancies	Completed	National Cancer Institute (NCI)	Early Phase 1	2013-01-01	The subject is invited to take part in this research study because s/he has been diagnosed with Diffuse Large B-Cell Lymphoma (DLBCL), Primary Mediastinal B-cell Lymphoma (PMBCL), or Post-transplant Lymphoproliferative Disorder (PTLD). In an attempt to improve cure rates while reducing harmful effects from drugs, oncologists are developing new treatment protocols. One such protocol, entitled dose-adjusted EPOCH-R, utilizes two major new strategies. First, the treatment approach utilizes continuous infusion of chemotherapy over four days, instead of being administered over minutes or hours. Secondly, the doses of some medications involved are increased or decreased based on how the drugs affect the subject's ability to produce blood cells, which is used as a measure of how rapidly the body is processing drugs. Using this approach in adults, researchers have shown improved cure rates in these cancers. Additionally, the harmful effects experienced by patients has been mild, with mucositis, severe infections, and tumor lysis syndrome occurring rarely. However, this new dosing method has never been used in children, and the effectiveness and side effects of this new method are unknown in children. The purpose of this study is to look at the safety of dose-adjusted EPOCH-R in the treatment of children with mature B-cell cancers, and to see if we can maintain cure rates (as has been shown in adults). This study represents the first trial of dose-adjusted EPOCH-R in children.
New Dosage	NCT01760226 ↗	Dose Adjusted EPOCH-R, to Treat Mature B Cell Malignancies	Completed	Texas Children's Hospital	Early Phase 1	2013-01-01	The subject is invited to take part in this research study because s/he has been diagnosed with Diffuse Large B-Cell Lymphoma (DLBCL), Primary Mediastinal B-cell Lymphoma (PMBCL), or Post-transplant Lymphoproliferative Disorder (PTLD). In an attempt to improve cure rates while reducing harmful effects from drugs, oncologists are developing new treatment protocols. One such protocol, entitled dose-adjusted EPOCH-R, utilizes two major new strategies. First, the treatment approach utilizes continuous infusion of chemotherapy over four days, instead of being administered over minutes or hours. Secondly, the doses of some medications involved are increased or decreased based on how the drugs affect the subject's ability to produce blood cells, which is used as a measure of how rapidly the body is processing drugs. Using this approach in adults, researchers have shown improved cure rates in these cancers. Additionally, the harmful effects experienced by patients has been mild, with mucositis, severe infections, and tumor lysis syndrome occurring rarely. However, this new dosing method has never been used in children, and the effectiveness and side effects of this new method are unknown in children. The purpose of this study is to look at the safety of dose-adjusted EPOCH-R in the treatment of children with mature B-cell cancers, and to see if we can maintain cure rates (as has been shown in adults). This study represents the first trial of dose-adjusted EPOCH-R in children.
New Dosage	NCT01760226 ↗	Dose Adjusted EPOCH-R, to Treat Mature B Cell Malignancies	Completed	Baylor College of Medicine	Early Phase 1	2013-01-01	The subject is invited to take part in this research study because s/he has been diagnosed with Diffuse Large B-Cell Lymphoma (DLBCL), Primary Mediastinal B-cell Lymphoma (PMBCL), or Post-transplant Lymphoproliferative Disorder (PTLD). In an attempt to improve cure rates while reducing harmful effects from drugs, oncologists are developing new treatment protocols. One such protocol, entitled dose-adjusted EPOCH-R, utilizes two major new strategies. First, the treatment approach utilizes continuous infusion of chemotherapy over four days, instead of being administered over minutes or hours. Secondly, the doses of some medications involved are increased or decreased based on how the drugs affect the subject's ability to produce blood cells, which is used as a measure of how rapidly the body is processing drugs. Using this approach in adults, researchers have shown improved cure rates in these cancers. Additionally, the harmful effects experienced by patients has been mild, with mucositis, severe infections, and tumor lysis syndrome occurring rarely. However, this new dosing method has never been used in children, and the effectiveness and side effects of this new method are unknown in children. The purpose of this study is to look at the safety of dose-adjusted EPOCH-R in the treatment of children with mature B-cell cancers, and to see if we can maintain cure rates (as has been shown in adults). This study represents the first trial of dose-adjusted EPOCH-R in children.

All Clinical Trials for CYTOXAN

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00001209 ↗	A Pilot Study for the Treatment of Patients With Metastatic and High Risk Sarcomas and Primitive Neuroectodermal Tumors	Completed	National Cancer Institute (NCI)	Phase 1	1986-10-01	This protocol is designed to test the feasibility of the administration of vincristine, adriamycin and cytoxan, alternating with the newly developed regimen ifosfamide VP-16 as well as the efficacy of this therapy in addition to radiotherapy in producing complete responses and disease-free survival in patients with Ewing's sarcoma, primitive sarcoma of bone, peripheral neuroepithelioma, and soft tissue sarcoma. This will not be a randomized study but will be comparable to the large data base of similar patients treated on successive Pediatric Branch studies.
NCT00001239 ↗	Combination Chemotherapy (FLAC) Combined With Granulocyte-Macrophage Colony Stimulating Factor in Locally Advanced and Metastatic Breast Cancer	Completed	National Cancer Institute (NCI)	Phase 2	1989-07-01	To evaluate a dose intensive chemotherapy regimen for the treatment of locally advanced and metastatic breast cancer using granulocyte-macrophage colony-stimulating factor (GM-CSF) to ameliorate chemotherapy-induced toxicity. Combination chemotherapy consists of Flurouricil, Leucovorin, Adriamycin, and Cytoxan (FLAC) which will be given every 21 days for 10 cycles. This protocol will replace the phase I study of this regimen (MB-232/88-C-0207) which found the MTD of this regimen to be at the first dose level. This is a phase II study to determine response rates of this regimen in advanced breast cancer.
NCT00001250 ↗	Effect of Preoperative Chemotherapy on Axillary Lymph Node Metastases in Stage II Breast Cancer: A Prospective Randomized Trial	Completed	National Cancer Institute (NCI)	Phase 2	1989-12-01	Patients with untreated clinical stage II breast cancer are eligible. An excisional biopsy of the primary tumor is acceptable, but without definitive local therapy or prior chemotherapy. Histologic confirmation of invasive carcinoma is required. Patients are prospectively randomized to receive five 21-day cycles of dose-intense (5-fluorouracil, adriamycin, leucovorin, cytoxan, granuloctye-colony stimulating factor [FLAC/G-CSF]) chemotherapy either before (preoperative) or after (postoperative) local therapy. Chemotherapy is given as an outpatient. For patients receiving preoperative chemotherapy, local therapy (modified radical mastectomy, or breast segmentectomy/axillary dissection/breast radiotherapy according to patient preference) is performed 3-4 weeks after last chemotherapy. For patients receiving postoperative chemotherapy, chemotherapy will begin 2-3 weeks after local therapy. Immediate reconstruction for mastectomy is acceptable. Upon completion of local therapy and chemotherapy in either treatment group, all estrogen receptor positive patients receive tamoxifen for 5 years. Follow-up consists of history and physical examination each 3 months for first 3 years, each six months for years 4 and 5, and yearly thereafter. Mammogram, bone scan, chest x-ray and blood work are performed yearly.

Clinical Trial Conditions for CYTOXAN

Condition Name

Condition Name for CYTOXAN
Intervention	Trials
Leukemia	85
Breast Cancer	83
Lymphoma	81

Condition MeSH

Condition MeSH for CYTOXAN
Intervention	Trials
Leukemia	270
Lymphoma	268
Leukemia, Lymphoid	196

Clinical Trial Locations for CYTOXAN

Trials by Country

Trials by Country for CYTOXAN
Location	Trials
Canada	513
Spain	67
New Zealand	57
Puerto Rico	50
Japan	8

Trials by US State

Trials by US State for CYTOXAN
Location	Trials
Texas	325
California	267
Maryland	222
New York	210
Washington	206

Clinical Trial Progress for CYTOXAN

Clinical Trial Phase

Clinical Trial Phase for CYTOXAN
Clinical Trial Phase	Trials
PHASE2	5
Phase 4	5
Phase 3	122

Clinical Trial Status

Clinical Trial Status for CYTOXAN
Clinical Trial Phase	Trials
Completed	395
Recruiting	212
Terminated	147

Clinical Trial Sponsors for CYTOXAN

Sponsor Name

Sponsor Name for CYTOXAN
Sponsor	Trials
National Cancer Institute (NCI)	484
M.D. Anderson Cancer Center	125
Children's Oncology Group	74

Sponsor Type

Sponsor Type for CYTOXAN
Sponsor	Trials
Other	1250
NIH	538
Industry	324

Last updated: April 23, 2026

Cytoxan (cyclophosphamide): Clinical-Development Update, Market Read-Through, and Supply-Side Projection

What is Cytoxan and where does it sit clinically?

Cytoxan is cyclophosphamide, an alkylating agent used broadly across oncology and some immunology indications. Clinically, it is a long-established product with extensive label history and generics that dominate real-world prescribing in most geographies.

Implication for a “clinical trials update”: Cytoxan itself is rarely the primary sponsor-led development asset in modern portfolios. Ongoing clinical activity typically clusters around:

• Combination regimens (cyclophosphamide used as backbone in newer protocols rather than as an investigational single agent)
• New formulation or dosing strategies (often executed by generic/formulation companies or as part of comparative trials)
• New indications using cyclophosphamide within transplant, lymphoproliferative, and immune-mediated frameworks

Because “Cytoxan” is a branded reference to an older molecule with widespread generic access, a sponsor-by-sponsor, trial-by-trial update depends on the specific national reference (FDA label lineages, sponsor names, trial registries, and whether the trials are explicitly for “cyclophosphamide/Cytoxan” vs protocol-level use). That said, the molecule’s clinical footprint is mature and stable.

What do regulatory and label facts indicate about current clinical demand?

Cyclophosphamide has long-standing regulatory standing and widespread use. The FDA label for cyclophosphamide (brand-specific where applicable) anchors safety monitoring expectations (myelosuppression, hemorrhagic cystitis risk, infection risk) and standard supportive care practice. That label stability drives predictable prescribing patterns and limits the probability of abrupt demand shifts from new clinical evidence.

Demand stability mechanism: clinicians rely on established response rates and the drug’s role in time-tested regimens (for example, conditioning regimens in transplant settings and combination chemotherapy in multiple cancers). Market demand thus tracks incidence and regimen preference more than on-cycle clinical trial readouts.

What is the current “market reality” for Cytoxan?

Cytoxan competes in a crowded and price-pressured generic alkylating agent market.

Key structural features that govern market performance:

• Generic penetration: cyclophosphamide is widely available in multiple dosage forms.
• Switching economics: formularies and procurement systems prefer lowest net cost for an older small molecule when therapeutic equivalence is assured.
• Bottleneck is supply and quality, not innovation: for an off-patent molecule, supply reliability, manufacturing capacity, and compliance track more strongly with revenue than new clinical differentiation.

Net result: branded Cytoxan revenue growth is constrained; total category demand is driven by oncology volume, hematologic malignancies incidence, and transplant activity.

Market analysis: how to project the category, not the brand

A realistic projection for Cytoxan should be built from:

1. Therapy volumes: chemotherapy and conditioning regimens that use cyclophosphamide as a backbone
2. Utilization intensity: cycles per patient and dose intensity, influenced by treatment protocols
3. Substitution: branded share vs generics, which will drift with pricing and payer policy
4. Supply-side constraints: manufacturing capacity, raw material availability, and quality events

Because cyclophosphamide is off-patent in many markets, the “brand” curve diverges from the “category” curve. For investment-grade read-through, the correct focus is often:

• Category demand stability plus
• Share capture by manufacturers with reliable supply and competitive pricing

What do market drivers imply for near-term demand (12 to 36 months)?

Demand drivers likely to be durable

• Cancer incidence growth and high baseline use in hematology and oncology protocols
• Transplant and conditioning-related use that is less sensitive to payer preference than new-oncology uptake
• Entrenched prescriber experience and routine supportive-care workflows

Demand headwinds likely to be persistent

• Price compression from generic competition
• Procurement-driven substitution across equivalent formulations
• Clinical protocol shifts toward regimens that may reduce cyclophosphamide exposure in select niches, offset by growth in other niches

Projection direction

• Category volume: likely stable to mildly up, tied to patient volume and protocol mix.
• Branded Cytoxan net revenue: likely flat to declining unless a payer/formulary shock or supply constraint temporarily lifts branded share.

Supply-side projection: what determines whether volumes translate into revenue?

For off-patent oncology generics, revenue and allocation are commonly governed by:

• Manufacturing uptime and batch release reliability
• Inspection outcomes and quality system stability
• Working-capital and procurement terms that determine whether supply is available when tenders clear
• Global sourcing of intermediates and finished dosage forms

In practice, when supply tightens, net pricing can improve even for generics. When supply normalizes, price reverts quickly.

How should investors and R&D teams treat “clinical trial updates” for Cytoxan?

A molecule like cyclophosphamide rarely generates step-change demand through new pivotal trials at the brand level. Clinical activity matters when it changes:

• patient selection (who gets cyclophosphamide),
• dose and schedule (how much),
• or protocol positioning (backbone vs salvage vs conditioning).

For a mature alkylator, most “trial updates” that move the needle are comparative protocol studies that alter regimen construction. Those studies are typically oncology/hematology protocol-driven rather than cyclophosphamide-centric investigational development.

What is the actionable read-through for business planning?

1) Expect protocol-driven usage, not molecule-driven innovation

• Cytoxan’s role will continue where regimens include cyclophosphamide backbone.
• The highest-value analytics focus on protocol adoption in key tumor types and transplant/conditioning pathways.

2) Treat revenue forecasting as a supply-and-share exercise

• Revenue depends on net pricing, tender outcomes, and allocation stability.
• Category growth is not the same thing as branded growth.

3) If you are running R&D

• Strategy should focus on formulation, lifecycle management, or combination protocol positioning where endpoints and safety profiles can matter.
• Trial design should anticipate payer and formulary scrutiny around supportive care and toxicity management, since cyclophosphamide has well-characterized risks.

Clinical activity map (how to structure monitoring for Cytoxan)

Monitor clinical evidence for cyclophosphamide use under the following buckets:

• Hematology regimens: lymphomas, leukemias, and immune-mediated conditions where cyclophosphamide is used in combination or conditioning
• Transplant/conditioning: conditioning regimens that use alkylators, where standard-of-care shifts can change dose exposure and utilization windows
• Immunology protocols: trials where cyclophosphamide is part of escalation or induction strategies

This monitoring approach is more decision-relevant than tracking “Cytoxan-only” trials, because modern evidence is typically protocol-level.

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Key Takeaways

• Cytoxan is cyclophosphamide, a mature off-patent alkylating agent with stable label-based safety and broad regimen usage.
• Clinical-trial impact on demand is typically protocol-driven, not molecule-brand driven; brand revenue is constrained by generic penetration.
• Market performance is primarily supply-and-share driven for off-patent oncology generics, with pricing sensitive to availability and procurement cycles.
• For forecasting, project category demand via regimen and patient-volume drivers, then apply brand share and net pricing assumptions tied to tender and manufacturing reliability.

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FAQs

1) Is Cytoxan currently a novel development target?

No. Cyclophosphamide is long-established; most current evidence tends to be protocol-level combination or regimen positioning rather than new molecule approvals.

2) What most affects Cytoxan pricing in the near term?

Generic supply conditions, batch release reliability, and tender-driven substitution versus brand availability.

3) Does new oncology trial data routinely change cyclophosphamide demand?

Only when the trial changes regimen construction (patient selection, dosing, or protocol position). Otherwise, impact is incremental.

4) What tumor types are most relevant for cyclophosphamide utilization trends?

Hematologic malignancies and transplant/conditioning-related settings are consistently important due to cyclophosphamide’s backbone role.

5) How should a brand vs category projection be handled?

Treat category as tied to patient volumes and regimen mix; treat brand as tied to net pricing, payer formulary allocation, and manufacturing/supply-driven share.

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References

[1] FDA. (n.d.). Cyclophosphamide prescribing information (label information for cyclophosphamide products). U.S. Food and Drug Administration. https://www.accessdata.fda.gov/