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.

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.

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
Active, not recruiting	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 Clinical Trials Update, Market Outlook, and Competitive Projections

Last updated: July 26, 2026

Cyclophosphamide is an established, off-patent cytotoxic alkylating agent with long-running oncology and autoimmune use. Demand is driven by (1) continued chemotherapy standard-of-care regimens in hematologic malignancies and solid tumors, (2) transplant and conditioning protocols, and (3) sustained niche use in autoimmune disease. Near-to-medium-term clinical trial activity is concentrated in combination regimens (chemo-immunotherapy, targeted therapy pairings), dose/schedule optimizations, and conditioning strategies rather than new standalone “cyclophosphamide breakthrough” molecules.

What clinical trials are ongoing for cyclophosphamide in oncology and autoimmune disease?

Fast answer: Most active trials are combination studies using cyclophosphamide as an input variable within larger protocols, including lymphoid malignancies, breast and lung cancers, and autoimmune indications. Trial design emphasis is typically on response endpoints and safety when cyclophosphamide is paired with checkpoint inhibitors, CAR-T support strategies (where applicable), targeted agents, or other immunomodulators.

Oncology trial clusters

Common cyclophosphamide trial intent in oncology includes:

• Hematologic malignancies: regimens in lymphoma and multiple myeloma-adjacent workflows, often where cyclophosphamide remains a backbone in specific combinations.
• Solid tumors: combination studies where cyclophosphamide is added to immunotherapy platforms to improve immunogenicity and tumor control.
• Neoadjuvant/adjuvant optimization: trials evaluating schedule changes and sequencing with radiation or systemic therapy.

Clinical endpoints typically tracked

• Objective response rate and progression-free survival for combination efficacy
• Hematologic toxicity, infection rates, and hospitalization for tolerability
• Biomarkers tied to immunologic activation, when cyclophosphamide is used to shape the immune microenvironment

Autoimmune trial clusters

In autoimmune disease, cyclophosphamide trials largely target:

• Dose finding and safety in long-course use (e.g., limiting cumulative exposure while preserving remission induction)
• Combination regimens designed to reduce steroid burden or improve steroid-sparing outcomes
• Comparators vs other immunosuppressants where cyclophosphamide is positioned as induction therapy in refractory disease

Key execution reality for cyclophosphamide trials

Cyclophosphamide’s commercial and R&D posture is constrained by:

• Generic availability and substitution pressure
• Low incremental patentability for standard regimens and dosing schedules in most jurisdictions
• Clinical differentiation via formulation, delivery, dosing convenience, and companion use rather than new active ingredient IP

Which phases and trial designs dominate cyclophosphamide updates right now?

Fast answer: The “active trial” surface is dominated by Phase 1/2 and randomized Phase 2 studies focused on safety, tolerability, and early efficacy in specific combination contexts.

Common design patterns

• Phase 1 dose/schedule to define manageable hematologic and infection risk
• Phase 2 single-arm using response rate and time-to-event endpoints
• Randomized Phase 2 when cyclophosphamide is a constant component but sequencing or combination changes
• Post-market comparative tolerability studies, including supportive care optimization

Why Phase 1/2 is common for cyclophosphamide

Cyclophosphamide is rarely the novelty driver; it is often the “active backbone” added to a newer partner regimen. That leads to early-phase safety validation of the combination.

How is cyclophosphamide used clinically in key regimens and what drives demand?

Fast answer: Cyclophosphamide demand is tied to its role across multiple treatment categories rather than to a single blockbuster regimen.

Oncology demand drivers

• Hematologic malignancies: continued use in lymphoma- and leukemia-associated regimens and in certain maintenance or induction constructs.
• Breast and gynecologic oncology workflows: historical backbone use in combination chemo.
• Lung cancer and other solid tumors: use in specific chemo-immunotherapy protocols in selected settings.

Transplant and conditioning protocols

• Stem cell transplant conditioning and related mobilization-support regimens remain a durable demand driver.
• Hospitals use existing cytotoxic stocks and established dosing references, which supports consistent volume even as branded supply contracts shift.

Autoimmune demand drivers

• Induction therapy in severe autoimmune disease when first-line options are insufficient.
• Use patterns are influenced by guideline changes, steroid-sparing goals, and toxicity management.

What is the cyclophosphamide market size, segmentation, and pricing structure?

Fast answer: The cyclophosphamide market is a mature generic-heavy segment with pricing shaped by wholesale contract dynamics, tender-based hospital procurement, and availability of multiple approved manufacturers.

Segmentation by use

• Oncology (largest practical volume): chemo regimens across multiple tumor types
• Autoimmune (smaller but recurring niche): induction and refractory disease settings
• Transplant/conditioning (structural demand): hospital-based protocols

Product form and supply reality

• Injectables dominate institutional demand.
• Formulation and presentation (vial sizing, concentration, reconstitution usability) are material for procurement even when the active ingredient is interchangeable.

Pricing dynamics

• Generic substitution limits branded pricing power.
• Tender competition and manufacturing capacity swings typically drive short-cycle price movement.
• Shortages or manufacturing disruptions can temporarily lift realized pricing, but sustained price premiums are difficult without exclusive supply constraints.

How will cyclophosphamide market growth evolve from now through the next 5 to 10 years?

Fast answer: Volume growth is expected to track global oncology incidence and treatment mix, while revenue growth will likely be restrained by generics substitution and pricing compression.

Projection framework (directional)

• Units: grow with population aging, higher cancer screening detection, and increasing systemic therapy utilization
• Value: grows more slowly due to competitive generic tendering
• Geography: hospital procurement maturity and tender frequency affect revenue resilience

Base case (directional)

• Moderate market value expansion with low to mid-single-digit growth in most regions, driven by unit growth and partial price stabilization in periods of supply tightness.
• Higher risk of value softness where procurement shifts to lowest-cost bids and where additional manufacturers come online.

What competitive landscape does cyclophosphamide face from generic manufacturers?

Fast answer: Competitive pressure comes primarily from large-scale generic injectable manufacturers and regional suppliers that can win tender bids. Differentiation tends to be manufacturing reliability, packaging convenience, and supply continuity.

Key competitive vectors

• Supply continuity: delivery reliability for hospitals and group purchasing organizations
• Stability of supply allocations: ability to meet contracted demand
• Cost competitiveness: pricing aligned to tender schedules
• Regulatory compliance: facility inspections and quality systems affecting approved status

Does cyclophosphamide face biosimilar-type competition or is it purely generic?

Fast answer: Cyclophosphamide is a small-molecule cytotoxic. It is not a biologic, so “biosimilar” dynamics do not apply. Competition is primarily generic small-molecule supply and, in some contexts, alternative cytotoxic backbones within regimens.

What are the substitution risks to cyclophosphamide revenue?

Fast answer: The main substitution risk is regimen redesign over time, not a single direct replacement drug.

Substitution channels

• Regimen evolution toward targeted therapies and immunotherapy, potentially reducing cyclophosphamide exposure per patient in some subpopulations.
• Toxicity management shifts that favor different induction agents in autoimmune care.
• Clinical protocol updates that adjust dosing intensity, potentially lowering cumulative cyclophosphamide utilization.

What patent and regulatory landscape governs cyclophosphamide commercialization?

Fast answer: Cyclophosphamide is off-patent in most major markets for the active ingredient itself. Market differentiation is therefore driven by:

• Formulation and manufacturing process claims (limited, case-specific enforceability)
• Labeling and method-of-use distinctions that are harder to enforce across generic entries
• Regulatory exclusivities that, for this molecule, are not expected to create durable exclusivity windows for new entrants

Regulatory status and entry pathway implications

• Generics historically enter via ANDAs for small-molecule drugs under Orange Book frameworks.
• Any remaining exclusivity would be formulation- or labeling-linked and typically limited in scope and duration.

What generic entry risks exist for cyclophosphamide in different jurisdictions?

Fast answer: Generic entry risk is structurally high because cyclophosphamide is an established, widely manufactured cytotoxic. The more meaningful risk is market share churn via tender dynamics and supply disruptions rather than patent blocking.

Jurisdictional risk profile (directional)

• US: competitive ANDA landscape; barriers are more quality and supply chain than patents at molecule-level
• EU: similar generic substitution pressure, with reimbursement and tender structures driving winners
• Other markets: manufacturing maturity and regulatory approvals govern availability more than enforceable IP

How does cyclophosphamide compare with alternative alkylating agents in efficacy and safety profiles?

Fast answer: Cyclophosphamide’s role persists because it fits established regimen scaffolding. Alternatives (other alkylators) may be used in certain settings, but cyclophosphamide retains a broad fit across hematology and autoimmune indications.

Decision factors clinicians weigh

• Indication-specific guideline fit
• Expected hematologic toxicity profile
• Infection risk and supportive care protocols
• Cumulative exposure constraints and fertility considerations
• Administration compatibility with institutional protocols

What formulations and manufacturing IP barriers matter for cyclophosphamide?

Fast answer: While active ingredient IP is largely exhausted, barriers are increasingly practical:

• manufacturing controls and sterile injectable production capability
• stability and reconstitution usability
• packaging formats aligned to hospital workflows

Differentiation areas

• Concentration and vial size options
• Lyophilized vs solution presentation where applicable
• Shelf-life improvements and supply chain stability

Key Takeaways

• Cyclophosphamide clinical activity is concentrated in combination regimens and protocol optimizations, not new standalone innovations.
• Demand is anchored by onco backbone usage, transplant/conditioning protocols, and niche autoimmune induction.
• Market value growth is expected to be muted by generic substitution, with revenue resilience depending on supply continuity and tender pricing.
• Competitive advantage is mostly manufacturing reliability and cost, not IP-driven exclusivity.

FAQs

1) Are cyclophosphamide trials mostly single-agent studies or combination trials?
Combination trials dominate, typically using cyclophosphamide as a conditioning or backbone agent within larger chemo-immunotherapy or chemo-targeted regimens.

2) What are the main safety endpoints in cyclophosphamide combination trials?
Hematologic toxicity, infection risk, and dose/schedule tolerability are the core endpoints, with time-to-response and survival endpoints used to link regimen performance to clinical outcomes.

3) How does cyclophosphamide usage differ between oncology and autoimmune disease trials?
Oncology trials focus on response and disease control within tumor-specific protocols; autoimmune trials emphasize remission induction, steroid-sparing aims, and long-course toxicity management.

4) What drives market share for generic cyclophosphamide suppliers?
Tender wins, contracted pricing, and supply continuity are the dominant drivers; quality events and manufacturing downtime can quickly shift share.

5) Is cyclophosphamide exposed to “biosimilar” market dynamics?
No. It is a small-molecule and competes as a generic drug, not as a biologic biosimilar product.

References (APA)

1. No specific sources were provided in the prompt to cite.