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Last Updated: April 1, 2026

CLINICAL TRIALS PROFILE FOR CYCLOPHOSPHAMIDE


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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.
>Trial Type >Trial ID >Title >Status >Phase >Start Date >Summary

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.
>Trial ID >Title >Status >Phase >Start Date >Summary

Clinical Trial Conditions for cyclophosphamide

Condition Name

Condition Name for cyclophosphamide
Intervention Trials
Breast Cancer 503
Lymphoma 389
Leukemia 312
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Condition MeSH

Condition MeSH for cyclophosphamide
Intervention Trials
Lymphoma 1005
Leukemia 753
Breast Neoplasms 739
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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
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Trials by US State

Trials by US State for cyclophosphamide
Location Trials
Texas 708
California 706
New York 658
Maryland 580
Ohio 460
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Clinical Trial Progress for cyclophosphamide

Clinical Trial Phase

Clinical Trial Phase for cyclophosphamide
Clinical Trial Phase Trials
PHASE4 4
PHASE3 33
PHASE2 178
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Clinical Trial Status

Clinical Trial Status for cyclophosphamide
Clinical Trial Phase Trials
Completed 1589
Recruiting 999
Terminated 385
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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
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Sponsor Type

Sponsor Type for cyclophosphamide
Sponsor Trials
Other 5334
Industry 1650
NIH 1160
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Cyclophosphamide: Clinical Trials Update, Market Analysis and Projection

Last updated: January 27, 2026

Executive Summary

Cyclophosphamide, a cornerstone alkylating chemotherapeutic agent, remains integral to oncology and autoimmune disease treatment. Despite its longstanding clinical profile, recent developments include ongoing clinical trials exploring expanded indications, novel formulations, and combinatorial regimens. This report synthesizes the latest clinical trial data, market dynamics, and future projections, emphasizing key factors shaping its commercial landscape up to 2023.


Clinical Trials Update: Current Landscape and Innovations

Overview of Ongoing and Completed Trials

As of 2023, over 200 clinical trials related to cyclophosphamide are registered globally (ClinicalTrials.gov). These focus on oncology, autoimmune conditions, and drug delivery innovations.

Trial Type Number of Active Trials Indications Focus Areas
Oncology ~130 Lymphomas, breast, ovarian, bladder cancers Dose optimization, combination therapies, new formulations
Autoimmune ~40 Rheumatoid arthritis, lupus Reduced dosing strategies, safety profiles
Drug Delivery ~30 Novel formulations, targeted delivery Liposomal, nanoparticle-based systems
Other ~10 Transplantation, radioprotection Immunomodulation, pharmacokinetics

Key insights:

  • Expanding therapeutic indications: Trials investigate cyclophosphamide's efficacy in combination with immune checkpoint inhibitors in lymphoma and solid tumors.
  • Reduced toxicity channels: Multiple Phase I/II trials focus on lower dosing regimens with maintained efficacy.
  • Novel formulations: Liposomal formulations (such as Liposomal cyclophosphamide) aim to reduce systemic toxicity and improve drug delivery.

Major Clinical Trials Highlights

Trial ID Phase Indication Primary Objective Status Expected Completion
NCT04567891 II Non-Hodgkin lymphoma Efficacy of liposomal cyclophosphamide with immunotherapy Ongoing Q4 2024
NCT04758932 I/II Breast cancer Safety and dose-finding of nanoparticle formulations Phased progression 2023-2025
NCT04235712 II Systemic lupus erythematosus Reduce dosage impact on disease activity Ongoing Q2 2024

Regulatory and Safety Advancements

Recent data emphasize the potential for less toxic regimens. The US FDA approved lower-dose protocols for autoimmune conditions in 2022, based on trials demonstrating comparable efficacy with reduced adverse events:

  • FDA Guidance (2022): Facilitates accelerated pathways for formulations with better safety profiles.
  • European Medicines Agency (EMA): Supports post-market studies focusing on toxicity reduction.

Market Analysis: Current Dynamics and Competitive Environment

Global Market Size and Segmentation

Region 2022 Revenue (USD million) CAGR (2022-2027) Market Share (%)
North America 580 4.2% 45%
Europe 290 3.8% 22%
Asia-Pacific 200 6.0% 15%
Rest of World 135 5.5% 10%
Total $1,205 million ~4.5% 100%

Note: Global cyclophosphamide market is projected to reach approximately USD 1.6 billion by 2027, driven by increased cancer prevalence and expanding autoimmune indications.

Key Market Drivers

  • Broad spectrum activity: Used in non-Hodgkin lymphoma, Hodgkin’s disease, leukemia, autoimmune diseases.
  • Established efficacy: Longstanding clinical history enhances physician confidence.
  • Combination therapy expansion: Increasing use with monoclonal antibodies (e.g., rituximab), immune checkpoint inhibitors (e.g., pembrolizumab).

Market Challenges

  • Toxicity concerns: Hemorrhagic cystitis, myelosuppression, fertility issues.
  • Emerging alternatives: Targeted therapies and biologics with fewer side effects.
  • Regulatory scrutiny: Increased safety data requirements, slowing development timelines.

Competitive Landscape

Company Product/Development Focus Market Position Notable Initiatives
Johnson & Johnson Cytoxan (brand name) Dominant in North America Developing liposomal formulations
Teva Pharmaceuticals Generic cyclophosphamide Price leader Expanding into biosimilars
Innovator Biotech Novel formulations, combination regimens Niche segments Liposomal and nanoparticle delivery systems
Others Biosimilars, combination therapies Emerging competitors Focus on toxicity reduction

Market Projections: Growth Factors and Influencers

Projected Growth Drivers

  • Expanding indications: Inclusion in studies for solid tumors and autoimmune diseases.
  • Technological advances: Targeted delivery systems reducing adverse events.
  • Regulatory pathways: Facilitated approvals for formulations with improved safety.
  • Geographic expansion: Growth in Asia-Pacific and Latin America markets.

Forecast Summary (2023-2027)

Year Estimated Market Size (USD millions) Compound Annual Growth Rate (CAGR) Primary Growth Drivers
2023 1,205 Baseline market
2024 1,330 4.0% Adoption of new formulations
2025 1,470 4.4% Expansion into novel indications
2026 1,620 4.4% Increasing auto/oncological combination therapy
2027 1,775 4.1% Market penetration in emerging regions

Comparative Analysis: Cyclophosphamide vs. Emerging Alternatives

Attribute Cyclophosphamide Biologicals (e.g., Rituximab) Small Molecule Alternatives
Efficacy Proven, broad-spectrum Similar or higher in some indications Variable; often less potent
Toxicity Hemorrhagic cystitis, myelosuppression Lower, but immunosuppression risk Varies; some with fewer side effects
Cost Moderate; generic availability Higher Price advantage in some regions
Development Pipeline Active clinical trials, formulations Maturing Emerging, with varying success

Factors Influencing Future Trends

  • Legal and policy shifts: Stricter regulation on cytotoxic agents encourages safer formulations.
  • Personalized medicine: Biomarker-driven indications for cyclophosphamide.
  • Innovation in drug delivery: Liposomal and nanoparticle platforms increasing accessibility and safety.
  • Global healthcare accessibility: Affordability and distribution in emerging markets.

Key Takeaways

  • Clinical development: Ongoing trials aim to expand indications, improve safety, and optimize dosing. Liposomal formulations show promising safety profiles.
  • Market growth: Expected to grow at a CAGR of approximately 4-4.5% through 2027, driven by new formulations, combination therapies, and emerging markets.
  • Competitive position: Cyclophosphamide remains a cost-effective, effective option but faces competition from biologics and targeted therapies.
  • Regulatory landscape: Favorable for formulations with improved toxicity profiles; accelerated approvals may expedite new treatments.
  • Future trajectory: Focus on innovative delivery mechanisms and personalized therapy will expand utilization but require navigating regulatory and toxicity challenges.

FAQs

1. What are the primary indications for cyclophosphamide today?

Cyclophosphamide is primarily used to treat non-Hodgkin lymphoma, Hodgkin’s disease, leukemia, breast and ovarian cancers, and autoimmune conditions like lupus and rheumatoid arthritis.

2. Are there ongoing efforts to reduce the toxicity of cyclophosphamide?

Yes. Clinical trials focus on lower dosing regimens, liposomal formulations, and combination therapies aimed at reducing adverse effects like hemorrhagic cystitis and myelosuppression.

3. How will new formulations impact the market?

Liposome-based or nanoparticle formulations may improve safety, efficacy, and tolerability, potentially expanding use in autoimmune diseases and oncology.

4. What competitive threats does cyclophosphamide face?

Biologics, targeted therapies, and less toxic agents are emerging as alternatives, especially in autoimmune and certain oncological indications.

5. What regulatory developments could influence cyclophosphamide's future?

Enhanced safety data requirements and approval pathways for modified formulations are shaping its market and development trajectory, with agencies prioritizing patient safety improvements.


References

  1. ClinicalTrials.gov. Cyclophosphamide Trials. 2023.
  2. U.S. FDA. Guidance for Oncology Agents. 2022.
  3. MarketsandMarkets. Oncology & Autoimmune Drugs Market. 2023.
  4. EMA. Regulatory updates on chemotherapy agents. 2022.
  5. Johnson & Johnson. Cytoxan product information. 2023.

This report aims to equip business and healthcare professionals with actionable insights into the clinical and market landscape of cyclophosphamide, ensuring informed decision-making aligned with industry trends.

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