CLINICAL TRIALS PROFILE FOR CYCLOPHOSPHAMIDE

505(b)(2) Clinical Trials for CYCLOPHOSPHAMIDE

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.
New Combination	NCT02188368 ↗	Pomalidomide for Lenalidomide for Relapsed or Refractory Multiple Myeloma Patients	Active, not recruiting	Celgene Corporation	Phase 2	2014-08-01	The purpose of this clinical research study is to evaluate the safety and effectiveness (good and bad effects) of pomalidomide given as part of a combination therapy that include more than just steroids to treat subjects with relapsed (subjects whose disease came back) or refractory (subjects whose disease did not respond to past treatment) multiple myeloma (MM). Pomalidomide (alone or in combination with dexamethasone) has been approved by the United States Food and Drug Administration (FDA) for the treatment of MM patients who have received at least two prior therapies, including lenalidomide and bortezomib, and have demonstrated disease progression on or within 60 days of completion of their last therapy. However, the use of pomalidomide in combination with other drugs used to treat MM, such as chemotherapeutic agents and proteasome inhibitors, is currently being tested and is not approved. Pomalidomide is in the same drug class as thalidomide and lenalidomide. Like lenalidomide, pomalidomide is a drug that alters the immune system and it may also interfere with the development of small blood vessels that help support tumor growth. Therefore, in theory, it may reduce or prevent the growth of cancer cells. The testing done with pomalidomide thus far has shown that it is well-tolerated and effective for subjects with MM both on its own and in combination with dexamethasone. Using another drug class, namely proteasome inhibitors, we have demonstrated that simply replacing a proteasome inhibitor with another in an established anti-myeloma treatment regimen can frequently overcome resistance regardless of the other agents that are part of the anti-myeloma regimen. Importantly, the toxicity profile of the new combinations closely resembled that of the proteasome inhibitor administered as a single agent. Based on this experience, we hypothesize that the replacement of lenalidomide with pomalidomide will yield similar results in a similar relapsed/refractory MM patient population.
New Combination	NCT02188368 ↗	Pomalidomide for Lenalidomide for Relapsed or Refractory Multiple Myeloma Patients	Active, not recruiting	Oncotherapeutics	Phase 2	2014-08-01	The purpose of this clinical research study is to evaluate the safety and effectiveness (good and bad effects) of pomalidomide given as part of a combination therapy that include more than just steroids to treat subjects with relapsed (subjects whose disease came back) or refractory (subjects whose disease did not respond to past treatment) multiple myeloma (MM). Pomalidomide (alone or in combination with dexamethasone) has been approved by the United States Food and Drug Administration (FDA) for the treatment of MM patients who have received at least two prior therapies, including lenalidomide and bortezomib, and have demonstrated disease progression on or within 60 days of completion of their last therapy. However, the use of pomalidomide in combination with other drugs used to treat MM, such as chemotherapeutic agents and proteasome inhibitors, is currently being tested and is not approved. Pomalidomide is in the same drug class as thalidomide and lenalidomide. Like lenalidomide, pomalidomide is a drug that alters the immune system and it may also interfere with the development of small blood vessels that help support tumor growth. Therefore, in theory, it may reduce or prevent the growth of cancer cells. The testing done with pomalidomide thus far has shown that it is well-tolerated and effective for subjects with MM both on its own and in combination with dexamethasone. Using another drug class, namely proteasome inhibitors, we have demonstrated that simply replacing a proteasome inhibitor with another in an established anti-myeloma treatment regimen can frequently overcome resistance regardless of the other agents that are part of the anti-myeloma regimen. Importantly, the toxicity profile of the new combinations closely resembled that of the proteasome inhibitor administered as a single agent. Based on this experience, we hypothesize that the replacement of lenalidomide with pomalidomide will yield similar results in a similar relapsed/refractory MM patient population.
New Combination	NCT02436707 ↗	Novel Combination Therapy in the Treatment of Relapsed and Refractory Aggressive B-Cell Lymphoma	Recruiting	Janssen, LP	Phase 2	2015-05-05	The purpose of this study is to find out what effects new combinations of treatment will have this disease. New promising treatment strategies will be added to this study as they are available to be compared against the standard treatment.
New Combination	NCT02436707 ↗	Novel Combination Therapy in the Treatment of Relapsed and Refractory Aggressive B-Cell Lymphoma	Recruiting	Karyopharm Therapeutics Inc	Phase 2	2015-05-05	The purpose of this study is to find out what effects new combinations of treatment will have this disease. New promising treatment strategies will be added to this study as they are available to be compared against the standard treatment.

All Clinical Trials for CYCLOPHOSPHAMIDE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000361 ↗	Autoimmunity in Inner Ear Disease	Terminated	National Institute on Deafness and Other Communication Disorders (NIDCD)	Phase 3	1998-03-01	The purpose of this study is to determine whether prednisone, methotrexate, and cyclophosphamide are effective in the treatment of rapidly progressive sensorineural hearing loss in both ears. This condition is called autoimmune inner ear disease (AIED), because it is thought that the hearing loss is triggered by an autoimmune process. Treatment attempts to suppress or control this process with powerful anti-inflammatory drugs. This is a Phase III, outpatient study. All study participants will be assigned to one of four different groups testing the experimental use of drugs. The study is scheduled to run for 18 months, with a minimum of 11 visits per participant.
NCT00000420 ↗	Safety of Estrogens in Lupus: Birth Control Pills	Completed	National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)	Phase 3	1997-06-01	Safety of Estrogens in Lupus Erythematosus - National Assessment (SELENA) is a study to test whether women with systemic lupus erythematosus (SLE or lupus) can safely use estrogen. We will determine this by looking at the effects of oral contraceptives (birth control pills, also known as "the pill") on disease activity and severity in women with SLE. The results of the study will show whether it is safe for women with SLE to use the pill.
NCT00000420 ↗	Safety of Estrogens in Lupus: Birth Control Pills	Completed	Office of Research on Women's Health (ORWH)	Phase 3	1997-06-01	Safety of Estrogens in Lupus Erythematosus - National Assessment (SELENA) is a study to test whether women with systemic lupus erythematosus (SLE or lupus) can safely use estrogen. We will determine this by looking at the effects of oral contraceptives (birth control pills, also known as "the pill") on disease activity and severity in women with SLE. The results of the study will show whether it is safe for women with SLE to use the pill.
NCT00000420 ↗	Safety of Estrogens in Lupus: Birth Control Pills	Completed	New York University School of Medicine	Phase 3	1997-06-01	Safety of Estrogens in Lupus Erythematosus - National Assessment (SELENA) is a study to test whether women with systemic lupus erythematosus (SLE or lupus) can safely use estrogen. We will determine this by looking at the effects of oral contraceptives (birth control pills, also known as "the pill") on disease activity and severity in women with SLE. The results of the study will show whether it is safe for women with SLE to use the pill.
NCT00000420 ↗	Safety of Estrogens in Lupus: Birth Control Pills	Completed	NYU Langone Health	Phase 3	1997-06-01	Safety of Estrogens in Lupus Erythematosus - National Assessment (SELENA) is a study to test whether women with systemic lupus erythematosus (SLE or lupus) can safely use estrogen. We will determine this by looking at the effects of oral contraceptives (birth control pills, also known as "the pill") on disease activity and severity in women with SLE. The results of the study will show whether it is safe for women with SLE to use the pill.
NCT00000596 ↗	Diffuse Fibrotic Lung Disease	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 2	1978-06-01	To determine the effects of cyclophosphamide compared with prednisone, dapsone, or high-dose intermittent 'pulse' therapy with methylprednisolone in patients with idiopathic pulmonary fibrosis. Also, to evaluate the use of intermittent, short-term, high-dose intravenous corticosteroids in patients with sarcoidosis. There were actually four separate clinical trials.
NCT00000658 ↗	A Phase III Randomized Trial of Low-Dose Versus Standard-Dose mBACOD Chemotherapy With rGM-CSF for Treatment of AIDS-Associated Non-Hodgkin's Lymphoma	Completed	Schering-Plough	Phase 3	1969-12-31	To determine the impact of dose intensity on tumor response and survival in patients with HIV-associated non-Hodgkin's lymphoma (NHL). HIV-infected patients are at increased risk for developing intermediate and high-grade NHL. While combination chemotherapy for aggressive B-cell NHL in the absence of immunodeficiency is highly effective, the outcome of therapy for patients with AIDS-associated NHL has been disappointing. Treatment is frequently complicated by the occurrence of multiple opportunistic infections, as well as the presence of poor bone marrow reserve, making the administration of standard doses of chemotherapy difficult. A recent study was completed using a low-dose modification of the standard mBACOD (cyclophosphamide, doxorubicin, vincristine, bleomycin, dexamethasone, methotrexate ) treatment. A 46 percent response rate was observed in patients treated with this combination of chemotherapeutic agents, with a number of durable remissions and reduced toxicity when compared to previous experience with more standard treatments. A subsequent study showed similar effectiveness using a lower dose of methotrexate administered on day 15. It is hoped that the use of sargramostim (granulocyte-macrophage colony-stimulating factor; GM-CSF) will improve bone marrow function and allow for administration of a higher dose of chemotherapy.

Clinical Trial Conditions for CYCLOPHOSPHAMIDE

Condition Name

Condition Name for CYCLOPHOSPHAMIDE
Intervention	Trials
Breast Cancer	503
Lymphoma	389
Leukemia	312
Multiple Myeloma	211

Condition MeSH

Condition MeSH for CYCLOPHOSPHAMIDE
Intervention	Trials
Lymphoma	1005
Leukemia	753
Breast Neoplasms	739
Leukemia, Lymphoid	484

Clinical Trial Locations for CYCLOPHOSPHAMIDE

Trials by Country

Trials by Country for CYCLOPHOSPHAMIDE
Location	Trials
China	977
Italy	602
Australia	570
Spain	521
France	454

Trials by US State

Trials by US State for CYCLOPHOSPHAMIDE
Location	Trials
Texas	708
California	706
New York	658
Maryland	580
Ohio	460

Clinical Trial Progress for CYCLOPHOSPHAMIDE

Clinical Trial Phase

Clinical Trial Phase for CYCLOPHOSPHAMIDE
Clinical Trial Phase	Trials
PHASE4	4
PHASE3	33
PHASE2	178

Clinical Trial Status

Clinical Trial Status for CYCLOPHOSPHAMIDE
Clinical Trial Phase	Trials
Completed	1589
Recruiting	999
Terminated	385

Clinical Trial Sponsors for CYCLOPHOSPHAMIDE

Sponsor Name

Sponsor Name for CYCLOPHOSPHAMIDE
Sponsor	Trials
National Cancer Institute (NCI)	999
M.D. Anderson Cancer Center	191
Children's Oncology Group	101

Sponsor Type

Sponsor Type for CYCLOPHOSPHAMIDE
Sponsor	Trials
Other	5334
Industry	1650
NIH	1160

CYCLOPHOSPHAMIDE Market Analysis and Financial Projection

Last updated: April 27, 2026

Cyclophosphamide Clinical Trials Update, Market Analysis and 2026+ Projection

Cyclophosphamide is an established alkylating agent with a long commercial history. The current clinical-trials signal is dominated by (1) dose-optimized and regimen-optimized oncology studies using existing formulations and (2) comparative or combination trials in hematologic malignancies. Commercial market growth is constrained by genericization and by a maturing oncology treatment landscape in which cyclophosphamide is a backbone component rather than a sole-driver therapy.

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What clinical-trial activity maps to cyclophosphamide right now?

Cyclophosphamide’s modern trial footprint is concentrated in oncology and transplant conditioning, with high enrollment stemming from combination regimens. Trial structures typically use cyclophosphamide as part of standard-of-care backbones, so the “innovation” is usually in the partner drug, schedule, or route rather than in the cyclophosphamide molecule itself.

Clinical-trial activity pattern (high-level)

• Disease focus: hematologic malignancies (notably lymphomas and myeloma contexts), breast cancer combinations, and hematopoietic stem cell transplant (conditioning) settings.
• Trial focus: regimen comparison, combination therapy sequencing, and schedule/dose optimization.
• Interventions: cyclophosphamide used with other systemic anti-cancer agents; common endpoints include response rate, progression-free survival (PFS), overall survival (OS), and toxicity profiles (myelosuppression, hemorrhagic cystitis risk management).

What this means for development strategy

• Cyclophosphamide is rarely the primary differentiator in late-stage programs, so clinical differentiation typically depends on how it is used (timing, dosing, combination partner, and supportive-care strategy).
• New entrants are more likely to compete on formulation/administration and supply/price than on novel MoA claims.

Evidence base used for the clinical-trial landscape

• Cyclophosphamide is a widely used alkylating agent in oncology regimens and transplant conditioning, with established clinical use documented in clinical pharmacology and safety labeling sources. [1], [2]

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What is the market structure for cyclophosphamide today?

The market is structurally mature: cyclophosphamide is available as a generic drug in most major markets, and pricing dynamics are driven by manufacturing scale, product availability, distribution contracts, and regulatory inspection outcomes.

Market structure

• Category: generic oncology chemotherapy agent.
• Customer base: oncology hospitals, infusion centers, and pharmacy benefit programs serving hematology and oncology.
• Buying behavior: volume-driven procurement; tenders and formulary inclusion determine share more than brand differentiation.
• Competitive set: multiple generic manufacturers and authorized generics.

Key demand drivers

• Ongoing incidence of hematologic malignancies and breast cancer where cyclophosphamide-based regimens persist.
• Continued use in conditioning regimens and salvage/combination contexts.
• Clinical conservatism: cyclophosphamide remains a familiar backbone with predictable toxicity management.

Key constraints

• Price erosion from generic competition.
• Substitution by regimens that do not require cyclophosphamide in some niches (agent selection varies by tumor subtype and line of therapy).
• Safety management costs (hydration, monitoring) that are handled operationally but can affect procurement economics at the margin.

Regulatory and safety positioning Cyclophosphamide labeling emphasizes boxed warnings and safety considerations that shape hospital protocols and procurement requirements. [1], [2]

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How should investors model 2026+ growth for cyclophosphamide?

For an established generic cytotoxic, growth is best modeled as volume plus modest price/mix effects, not as sustained premium adoption.

Base-case drivers

• Volume stability: stable patient throughput in oncology and conditioning indications.
• Share shifts: contract-based share movement across suppliers.
• Portfolio effects: demand remains resilient due to entrenched use as part of established regimen templates.

Bear-case risks

• Further price compression.
• Manufacturing disruption (API supply concentration and sterile fill-finish constraints).
• Safety scares tied to specific lots or manufacturer-specific quality events (impact is episodic but can be material).

Bull-case scenarios

• Formulary tightening that favors reliable supply and cost per dose.
• Replacement of intermittent shortages by compliant supply chains (temporary share gains).
• Mix toward regimens or settings with higher cyclophosphamide intensity.

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Market projection: 2026+ outlook (practical modeling framework)

Because cyclophosphamide is generic, “growth” in public forecasting is often reported as market value changes that reflect both unit volumes and pricing. A defensible model uses three levers:

1. Treatment volume index
   Track oncology and transplant conditioning patient counts by major indication categories in the geography set (US/EU5 plus Japan if covered).
2. Net price per dose
   Use historical tender and reimbursement trends, and assume continued erosion with periodic stabilization after supplier shakeouts.
3. Product availability and lot stability factor
   Treat shortages as step functions that can create temporary demand pull-through for suppliers with uninterrupted supply.

This framework fits the way healthcare systems source low-differentiation generics, where procurement outcomes dominate.

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What do patents and exclusivity imply for future supply and pricing?

Cyclophosphamide’s molecule is not patent-led in the way novel biologics are. Commercial dynamics are therefore governed by:

• expiration of molecule-related and formulation-specific exclusivities (where applicable),
• ANDA approvals and manufacturing capacity,
• and quality/inspection outcomes affecting effective supply.

In practice, for cyclophosphamide, long-run pricing is mainly a function of competitive generic intensity and supply reliability rather than patent fences.

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What are the main clinical safety and handling issues that shape real-world use?

Cyclophosphamide requires robust toxicity monitoring and supportive-care protocols. These operational requirements affect dosing schedules, infusion workflows, and patient selection.

Core safety themes in labeling

• Hemorrhagic cystitis risk and bladder protection strategies.
• Myelosuppression with infection risk.
• Gonadal toxicity and teratogenic risk.
• Secondary malignancy risk consistent with alkylating chemotherapy class effects. [1], [2]

These safety considerations are baked into standard clinical pathways, which supports demand continuity but also reinforces why procurement and supply reliability matter.

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Competitive positioning: where can cyclophosphamide create measurable commercial edge?

With no meaningful molecular differentiation, competitive edge tends to be commercial execution:

• Supply reliability: continuous availability reduces hospital substitution friction.
• Cost per dose: tender performance wins.
• Packaging and administration fit: consistent concentration/volume, reconstitution and handling ease.
• Stability and lot consistency: fewer disruptions and returns.

This is the basis for winning share in a commodity-like oncology chemotherapy segment.

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Key Takeaways

• Cyclophosphamide’s clinical-trials landscape is dominated by combination and regimen-optimization studies rather than molecule innovation.
• Market growth is constrained by genericization; value movement is driven by volume stability and pricing compression.
• 2026+ projection should be modeled with procurement realities: net price per dose, patient throughput, and supply reliability are the primary levers.
• Competitive advantage is operational, not patent-driven, and hinges on stable manufacturing and tender economics.
• Safety and handling requirements are stable operational determinants that reinforce standardized, ongoing demand.

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FAQs

1) Is cyclophosphamide still entering new clinical trials?

Yes. Trial activity continues, but cyclophosphamide typically appears as a component of combination regimens or conditioning strategies rather than as a novel stand-alone investigational mechanism. [1], [2]

2) What drives demand more: new indications or core oncology use?

Core oncology use and conditioning regimens drive demand. New studies tend to refine how cyclophosphamide is used in established pathways. [1], [2]

3) How does generic competition affect pricing?

It creates persistent price pressure and shifts competition to tenders, supply reliability, and cost-per-dose economics rather than differentiation. (Derived from generic market structure principles.) [1], [2]

4) What clinical safety elements matter most for real-world adoption?

Myelosuppression, hemorrhagic cystitis risk management, and teratogenicity are key protocol drivers that standardize care and patient monitoring. [1], [2]

5) Do patent expirations meaningfully change 2026+ cyclophosphamide supply?

Cyclophosphamide’s future availability is primarily governed by generic manufacturing capacity and regulatory quality outcomes, not by patent fences typical of novel drugs. (Consistent with long-established generic status.) [1], [2]

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References

[1] U.S. Food and Drug Administration. Cytoxan (cyclophosphamide) prescribing information.
[2] European Medicines Agency. Cyclophosphamide: product information and assessment documents.