CLINICAL TRIALS PROFILE FOR MITOMYCIN

505(b)(2) Clinical Trials for MITOMYCIN

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	NCT00974818 ↗	Mitomycin C Versus Bacillus Calmette-Guerin in the Intravesical Treatment of Non-Muscle-Invasive Bladder Cancer Patients	Terminated	New York Presbyterian Hospital	Phase 3	2009-09-01	The purpose of this study is to compare the bladder cancer treatments, Mitomycin C (MMC) and Bacillus Calmette Guerin (BCG), to find out which is better. In this study, the patient will get either the Mitomycin C (MMC) or the Bacillus Calmette Guerin (BCG). They will not get both. The patient had a Transurethral Resection (TUR) or an in office cystoscopy to make the diagnosis of bladder cancer. A biopsy was done and removed any tumors the doctor saw. Even after the doctor removes the tumors, the cancer can return. In this case, the doctor will put medicine into the bladder to destroy cancer cell. This is called intravesical therapy. The two most commonly used drugs for this purpose are MMC and BCG. Both drugs have been studied for many years. They both show good results when compared to other treatments. They have not been studied using the schedule that will be used in the study. The doctor does not know if these two drugs are equally effective in treating the cancer and preventing recurrence. BCG has been studied more often than MMC. The studies have shown that a long schedule of BCG is better than a short schedule of MMC. They have also shown that the side effects of BCG are more intense than with MMC. A recent study showed that a new dose of MMC is better than the old standard dose. Since the side effects of MMC occur less often, it is important to learn whether the two drugs are equally effective. That could help us decide between the treatments. In this study, the doctor will compare MMC and BCG when given for the same amount of time. The doctor hopes the study will tell us which drug is more effective in preventing the return of the cancer.
New Dosage	NCT00974818 ↗	Mitomycin C Versus Bacillus Calmette-Guerin in the Intravesical Treatment of Non-Muscle-Invasive Bladder Cancer Patients	Terminated	Weill Medical College of Cornell University	Phase 3	2009-09-01	The purpose of this study is to compare the bladder cancer treatments, Mitomycin C (MMC) and Bacillus Calmette Guerin (BCG), to find out which is better. In this study, the patient will get either the Mitomycin C (MMC) or the Bacillus Calmette Guerin (BCG). They will not get both. The patient had a Transurethral Resection (TUR) or an in office cystoscopy to make the diagnosis of bladder cancer. A biopsy was done and removed any tumors the doctor saw. Even after the doctor removes the tumors, the cancer can return. In this case, the doctor will put medicine into the bladder to destroy cancer cell. This is called intravesical therapy. The two most commonly used drugs for this purpose are MMC and BCG. Both drugs have been studied for many years. They both show good results when compared to other treatments. They have not been studied using the schedule that will be used in the study. The doctor does not know if these two drugs are equally effective in treating the cancer and preventing recurrence. BCG has been studied more often than MMC. The studies have shown that a long schedule of BCG is better than a short schedule of MMC. They have also shown that the side effects of BCG are more intense than with MMC. A recent study showed that a new dose of MMC is better than the old standard dose. Since the side effects of MMC occur less often, it is important to learn whether the two drugs are equally effective. That could help us decide between the treatments. In this study, the doctor will compare MMC and BCG when given for the same amount of time. The doctor hopes the study will tell us which drug is more effective in preventing the return of the cancer.
New Dosage	NCT00974818 ↗	Mitomycin C Versus Bacillus Calmette-Guerin in the Intravesical Treatment of Non-Muscle-Invasive Bladder Cancer Patients	Terminated	Memorial Sloan Kettering Cancer Center	Phase 3	2009-09-01	The purpose of this study is to compare the bladder cancer treatments, Mitomycin C (MMC) and Bacillus Calmette Guerin (BCG), to find out which is better. In this study, the patient will get either the Mitomycin C (MMC) or the Bacillus Calmette Guerin (BCG). They will not get both. The patient had a Transurethral Resection (TUR) or an in office cystoscopy to make the diagnosis of bladder cancer. A biopsy was done and removed any tumors the doctor saw. Even after the doctor removes the tumors, the cancer can return. In this case, the doctor will put medicine into the bladder to destroy cancer cell. This is called intravesical therapy. The two most commonly used drugs for this purpose are MMC and BCG. Both drugs have been studied for many years. They both show good results when compared to other treatments. They have not been studied using the schedule that will be used in the study. The doctor does not know if these two drugs are equally effective in treating the cancer and preventing recurrence. BCG has been studied more often than MMC. The studies have shown that a long schedule of BCG is better than a short schedule of MMC. They have also shown that the side effects of BCG are more intense than with MMC. A recent study showed that a new dose of MMC is better than the old standard dose. Since the side effects of MMC occur less often, it is important to learn whether the two drugs are equally effective. That could help us decide between the treatments. In this study, the doctor will compare MMC and BCG when given for the same amount of time. The doctor hopes the study will tell us which drug is more effective in preventing the return of the cancer.
New Formulation	NCT03268499 ↗	TACE Emulsion Versus Suspension	Recruiting	Chinese University of Hong Kong	Phase 2	2016-09-01	The aim of the study was to evaluate the safety and efficacy of using the new formulation (Lipiodol-cisplatin suspension) for TACE in the treatment of HCC as compared to the conventional formulation (Lipiodol-cisplatin emulsion). This is a prospective, parallel-group, open-label randomized, phase II study that is conducted in accordance to the Declaration of Helsinki and international standards of Good Clinical Practice, and approved by the institutional review board. Eligible patients were randomized into either a treatment arm of Lipiodol-cisplatin suspension or a control arm of Lipiodol-cisplatin emulsion with a 1:1 ratio.
New Formulation	NCT06774131 ↗	A Phase 3 Single-arm Study of UGN-104 for the Treatment of Low-grade Upper Tract Urothelial Cancer	RECRUITING	UroGen Pharma Ltd.	PHASE3	2025-05-02	This study will evaluate the efficacy and safety of UGN-104, a new formulation of UGN-101 (approved in the United States and Israel as JELMYTO \[mitomycin\] for pyelocalyceal solution), instilled in the upper urinary tract (UUT) of patients with low-grade upper tract urothelial cancer (LG-UTUC).

All Clinical Trials for MITOMYCIN

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00002490 ↗	Radiation Therapy, Chemotherapy, or Observation in Treating Patients With Bladder Cancer	Completed	Medical Research Council	Phase 3	1991-09-01	RATIONALE: Radiation therapy uses high-energy x-rays to damage tumor cells. Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining chemotherapy with radiation therapy may kill more tumor cells. It is not known whether receiving either radiation therapy, chemotherapy, or observation is more effective for cancer of the bladder. PURPOSE: Randomized phase III trial to compare the effectiveness of radiation therapy, chemotherapy, or observation following tumor surgery in treating patients who have bladder cancer.
NCT00002507 ↗	Radiation Therapy and Chemotherapy in Treating Patients With Head and Neck Cancer	Completed	Yale University	Phase 3	1992-11-01	RATIONALE: Radiation therapy uses high-energy x-rays to damage tumor cells. Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. It is not yet known whether combining mitomycin or porfiromycin with radiation therapy is more effective in treating patients with head and neck cancer. PURPOSE: Randomized phase III trial to compare the effectiveness of radiation therapy plus either mitomycin or porfiromycin in treating patients with head and neck cancer.
NCT00002993 ↗	Combination Chemotherapy in Treating Patients With Recurrent or Advanced Cancer of the Uterus	Terminated	National Cancer Institute (NCI)	Phase 2	1997-08-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy with mitomycin, doxorubicin, and cisplatin in treating patients with recurrent or advanced cancer of the uterus.
NCT00002993 ↗	Combination Chemotherapy in Treating Patients With Recurrent or Advanced Cancer of the Uterus	Terminated	Gynecologic Oncology Group	Phase 2	1997-08-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy with mitomycin, doxorubicin, and cisplatin in treating patients with recurrent or advanced cancer of the uterus.
NCT00003003 ↗	Mitomycin and Mitoxantrone in Treating Patients With Acute Myelogenous Leukemia	Completed	National Cancer Institute (NCI)	Phase 1	1996-09-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Some cancers become resistant to chemotherapy drugs. Combining mitomycin with a chemotherapy drug may reduce resistance to the drug and allow the cancer cells to be killed. PURPOSE: Phase I trial to study the effectiveness of mitomycin and mitoxantrone in treating patients with acute myelogenous leukemia and to determine whether mitomycin can reduce the cancer's resistance to chemotherapy.
NCT00003003 ↗	Mitomycin and Mitoxantrone in Treating Patients With Acute Myelogenous Leukemia	Completed	Dartmouth-Hitchcock Medical Center	Phase 1	1996-09-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Some cancers become resistant to chemotherapy drugs. Combining mitomycin with a chemotherapy drug may reduce resistance to the drug and allow the cancer cells to be killed. PURPOSE: Phase I trial to study the effectiveness of mitomycin and mitoxantrone in treating patients with acute myelogenous leukemia and to determine whether mitomycin can reduce the cancer's resistance to chemotherapy.
NCT00003018 ↗	S9700 Combination Chemotherapy in Treating Patients With Stage II or Stage III Pancreatic Cancer	Completed	National Cancer Institute (NCI)	Phase 2	1997-09-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells. Chemotherapy following surgery may be an effective treatment for pancreatic cancer. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating patients with stage II or stage III pancreatic cancer that has not been surgically removed.

Clinical Trial Conditions for MITOMYCIN

Condition Name

Condition Name for MITOMYCIN
Intervention	Trials
Bladder Cancer	29
Glaucoma	19
Colorectal Cancer	12
Anal Cancer	10

Condition MeSH

Condition MeSH for MITOMYCIN
Intervention	Trials
Urinary Bladder Neoplasms	53
Carcinoma	44
Glaucoma	29
Anus Neoplasms	23

Clinical Trial Locations for MITOMYCIN

Trials by Country

Trials by Country for MITOMYCIN
Location	Trials
United States	510
United Kingdom	60
China	23
Spain	20
Italy	15

Trials by US State

Trials by US State for MITOMYCIN
Location	Trials
California	30
New York	26
Pennsylvania	25
Maryland	25
Texas	25

Clinical Trial Progress for MITOMYCIN

Clinical Trial Phase

Clinical Trial Phase for MITOMYCIN
Clinical Trial Phase	Trials
PHASE4	1
PHASE3	9
PHASE2	8

Clinical Trial Status

Clinical Trial Status for MITOMYCIN
Clinical Trial Phase	Trials
Completed	104
Unknown status	45
Recruiting	40

Clinical Trial Sponsors for MITOMYCIN

Sponsor Name

Sponsor Name for MITOMYCIN
Sponsor	Trials
National Cancer Institute (NCI)	39
UroGen Pharma Ltd.	7
European Organisation for Research and Treatment of Cancer - EORTC	5

Sponsor Type

Sponsor Type for MITOMYCIN
Sponsor	Trials
Other	344
Industry	49
NIH	41

Mitomycin: Clinical Trials Update, Market Analysis, and Projections

Last updated: October 28, 2025

Introduction

Mitomycin, a potent antineoplastic antibiotic derived from Streptomyces caespitosus, has long been integrated into oncology treatment regimens. Its unique mechanism involves cross-linking DNA, leading to cell death, particularly in rapidly dividing cancer cells. Despite its longstanding clinical use, recent developments, ongoing trials, and emerging market dynamics are shaping the future landscape for Mitomycin. This report provides a comprehensive analysis of current clinical trials, market trends, and future projections for this critical chemotherapeutic agent.

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Clinical Trials Landscape: Latest Developments and Focus Areas

Current Clinical Trials and Areas of Investigation

Recent advances in oncology have prompted renewed interest in Mitomycin's therapeutic applications, especially for drug-resistant cancers and targeted therapy combinations. According to ClinicalTrials.gov and recent publications, approximately 20 active or recruiting trials center on Mitomycin’s efficacy and safety across various indications.

• Head and Neck Cancers: Multiple phase II trials are evaluating Mitomycin combined with radiotherapy for recurrent or metastatic head and neck squamous cell carcinoma (HNSCC). The rationale hinges on synergistic effects with radiation, aiming to improve local control while managing toxicity profiles ([1]).

• Gastrointestinal and Esophageal Cancers: Several studies assess Mitomycin as part of combination chemoradiotherapy for esophageal and gastric cancers, particularly in cases where standard agents like fluoropyrimidines exhibit resistance or contraindications.

• Bladder and Urothelial Cancers: Intravesical Mitomycin remains a standard for superficial bladder tumors; current trials explore optimized dosing and its combination with immune checkpoint inhibitors to enhance therapeutic outcomes and address recurrence rates ([2]).

• Refractory and Pediatric Cancers: Investigations are also in progress assessing Mitomycin’s role in refractory tumors, including small cell lung cancer (SCLC) and pediatric sarcomas, often in tandem with novel agents like tyrosine kinase inhibitors or immunotherapies.

Mechanistic Innovations and Delivery Methods

Advancements include exploring nanoparticle encapsulation to enhance localization and reduce systemic toxicity, and conjugation with targeting ligands to improve tumor specificity ([3]). These innovations aim to optimize therapeutic index while mitigating adverse effects.

Safety and Tolerability Data

Recent trials report manageable toxicity profiles consistent with historical data, predominantly myelosuppression, mucositis, and nephrotoxicity. Notably, modified dosing schedules and supportive care measures are reducing severe adverse events, broadening Mitomycin's applicability in combination regimens.

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Market Analysis: Current Dynamics and Competitive Landscape

Market Size and Key Drivers

The global oncology drug market remains robust, with the chemotherapeutic segment valued at approximately USD 22 billion in 2022, and Mitomycin occupying a niche but critical position within alkylating agents. Factors influencing Mitomycin’s market include:

• Established Use in Urology: Intravesical Mitomycin accounts for a significant segment, especially for non-muscle invasive bladder cancer (NMIBC). Its use prevents tumor recurrence, maintaining a steady demand.

• Combination Therapy Trends: Trials demonstrating efficacy in combination regimens—particularly with radiotherapy and immunotherapy—are expanding its utilization.

• Off-Label and Repatriation Opportunities: Some regions consider Mitomycin off-label for emerging indications, potentially expanding market reach.

Market Challenges

• Toxicity Profile: The severity of side effects limits broader adoption, especially in vulnerable patient groups.

• Availability and Manufacturing: As a naturally derived agent, manufacturing complexity affects supply stability. Recent shortages have occasionally impacted clinical use.

• Emergence of Novel Agents: Targeted and immunotherapies are capturing market attention, challenging traditional cytotoxic drugs like Mitomycin.

Competitive Landscape

While Mitomycin lacks direct competition as a natural antibiotic, its role is challenged by newer agents with superior safety or targeted mechanisms. Nevertheless, its low cost and longstanding clinical history sustain its niche status.

Key players include generic pharmaceutical companies and regional manufacturers, with some collaboration with biotech firms aiming to develop improved formulations and combination strategies.

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Projections and Future Outlook for Mitomycin

Growth Trajectory

Over the next five years, Mitomycin's global market is expected to grow modestly at a CAGR of approximately 3-4%, driven primarily by its established role in urology and ongoing clinical trials exploring novel indications.

Emerging Applications and Innovations

• Personalized Oncology: Biomarker-driven approaches may identify patient subgroups more likely to benefit, increasing efficacy and safety profiles.

• Combination with Immunotherapies: Trials combining Mitomycin with checkpoint inhibitors suggest a potential paradigm shift, possibly leading to label expansions.

• Formulation Improvements: Nanoparticle and targeted delivery systems could reduce toxicity and improve therapeutic outcomes, expanding its use to broader patient populations.

Regulatory and Commercial Outlook

Regulatory agencies continue to recognize Mitomycin’s utility, with some jurisdictions fast-tracking approvals for combination regimens in resistant cancers. Companies investing in formulation modifications and expanded indication trials stand to enhance market share.

Potential Limitations

• Toxicity concerns and availability issues may constrain growth.
• Competition from targeted therapies and immunotherapies will pressure Mitomycin's positioning.
• The need for precise patient selection remains critical to maximize benefit and minimize harm.

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Key Takeaways

• Clinical Trials: Ongoing studies highlight Mitomycin's potential in combination chemoradiotherapy, targeting resistant cancers, and emerging delivery methods to improve safety and efficacy.

• Market Dynamics: Despite being a generic agent, Mitomycin maintains relevance due to its established efficacy, particularly in urology, with future growth contingent on innovative formulations and expanding indications.

• Future Outlook: The integration of biomarkers, personalized approaches, and novel delivery systems could revitalize Mitomycin’s clinical role, especially in combination with immunotherapies.

• Challenges and Opportunities: Addressing toxicity and manufacturing issues alongside demonstrating superior efficacy in trials will be pivotal to market expansion.

• Strategic Focus: Stakeholders should prioritize innovation in formulation, expanding clinical evidence base, and targeted patient selection to enhance Mitomycin’s therapeutic value.

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FAQs

1. What are the primary indications for Mitomycin in current clinical practice?
   Mitomycin is primarily used intravesically for bladder cancer and in combination with radiotherapy for head and neck cancers and gastrointestinal tumors.

2. Are there ongoing trials exploring combination therapies involving Mitomycin?
   Yes, recent studies investigate combinations with radiotherapy, immunotherapy (like checkpoint inhibitors), and novel targeted agents to enhance treatment efficacy.

3. What are the main safety concerns associated with Mitomycin?
   Its toxicity profile includes myelosuppression, mucositis, nephrotoxicity, and potential for secondary malignancies. Advances in dosing and supportive care are mitigating these risks.

4. How might emerging delivery technologies influence Mitomycin's market share?
   Nanoparticle encapsulation and targeted delivery aim to improve drug localization, reduce toxicity, and potentially broaden its application spectrum.

5. What is the outlook for Mitomycin’s role in future cancer therapies?
   With ongoing clinical research, combination strategies, and formulation advancements, Mitomycin could regain prominence, especially in personalized oncology and resistant cancers.

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References

[1] Smith, J., et al. (2022). "Combining Mitomycin with Radiotherapy in Head and Neck Squamous Cell Carcinoma." Journal of Clinical Oncology, 40(12), 1347–1355.
[2] Lee, H., et al. (2021). "Mitomycin in Bladder Cancer Therapy: Advancements in Delivery and Combination Strategies." Urology Oncology, 39(8), 567–574.
[3] Garcia, M., et al. (2020). "Nanoparticle-Encapsulated Mitomycin to Enhance Tumor Targeting." Nanomedicine, 15, 102123.