CLINICAL TRIALS PROFILE FOR IRINOTECAN HYDROCHLORIDE

505(b)(2) Clinical Trials for irinotecan hydrochloride

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 Combination	NCT00177853 ↗	Celecoxib, Irinotecan and Concurrent Radiotherapy in Preoperative Pancreatic Cancer	Terminated	Pharmacia and Upjohn	Phase 1	2006-12-01	The purposes of this study are to examine the effects of a new combination of drugs, celecoxib (Celebrex®) and irinotecan (CPT-11), with standard radiation therapy on people before they undergo surgery; to determine what effects this combination has on pancreatic cancer; and to determine the highest dose of celecoxib and irinotecan that can be given safely without causing severe side effects. While not an endpoint, it is hoped that this combination will also shrink tumors enough for excision.
New Combination	NCT00177853 ↗	Celecoxib, Irinotecan and Concurrent Radiotherapy in Preoperative Pancreatic Cancer	Terminated	University of Pittsburgh	Phase 1	2006-12-01	The purposes of this study are to examine the effects of a new combination of drugs, celecoxib (Celebrex®) and irinotecan (CPT-11), with standard radiation therapy on people before they undergo surgery; to determine what effects this combination has on pancreatic cancer; and to determine the highest dose of celecoxib and irinotecan that can be given safely without causing severe side effects. While not an endpoint, it is hoped that this combination will also shrink tumors enough for excision.
New Combination	NCT00215982 ↗	Study of Capecitabine With Irinotecan and Oxaliplatin (Eloxatin) in Advanced Colorectal Cancer	Completed	Roche Pharma AG	Phase 2	2004-12-01	The purpose of this study is to find out how effective the new combination of the drugs Capecitabine (Xeloda), Oxaliplatin (Eloxatin), and Irinotecan (Camptosar) are against colon and rectal cancer. All three of these drugs are approved by the Food and Drug Administration (FDA) for the treatment of colon or rectal cancer. This however is the first time that these three drugs have been combined in this schedule for the treatment of colon/rectal cancer.
New Combination	NCT00215982 ↗	Study of Capecitabine With Irinotecan and Oxaliplatin (Eloxatin) in Advanced Colorectal Cancer	Completed	H. Lee Moffitt Cancer Center and Research Institute	Phase 2	2004-12-01	The purpose of this study is to find out how effective the new combination of the drugs Capecitabine (Xeloda), Oxaliplatin (Eloxatin), and Irinotecan (Camptosar) are against colon and rectal cancer. All three of these drugs are approved by the Food and Drug Administration (FDA) for the treatment of colon or rectal cancer. This however is the first time that these three drugs have been combined in this schedule for the treatment of colon/rectal cancer.
New Combination	NCT00230399 ↗	Combination Chemotherapy Treatments in Patients With Metastatic Colorectal Cancer	Completed	University of Michigan Cancer Center	Phase 2	2003-06-01	This study will examine a new combination of drugs: celecoxib, capecitabine and irinotecan, for the treatment of metastatic colorectal cancer. Capecitabine and irinotecan, individually, are approved by the Food and Drug Administration (FDA) for use in colorectal cancer. The combination of these two drugs is experimental (not approved by the FDA as standard treatment), but is a widely used treatment option and preliminary studies have shown that treatment with the combination of capecitabine and irinotecan has a positive effect on metastatic colorectal cancer. Likewise, previous research in animals has shown that celecoxib, a drug approved for arthritis therapy, also has activity against this tumor type and may improve the anti-cancer activity of the combination of capecitabine and irinotecan.

All Clinical Trials for irinotecan hydrochloride

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00001495 ↗	A Phase I Study of Irinotecan (CPT-11) Administered as a Prolonged Infusion in Adult Patients With Solid Tumors	Completed	National Cancer Institute (NCI)	Phase 1	1995-11-01	This study examines a 96 hour infusion schedule of irinotecan alternating with 72 hour drug-free intervals in patients with solid tumors in order to determine the maximum tolerated dose of this regimen.
NCT00002759 ↗	Irinotecan Plus Cyclosporine and Phenobarbital in Treating Patients With Solid Tumors or Lymphoma	Completed	National Cancer Institute (NCI)	Phase 1	1996-06-01	Phase I trial to study the effectiveness of irinotecan plus cyclosporine and phenobarbital in treating patients who have solid tumors or lymphoma that is refractory to standard therapy. Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Cyclosporine and phenobarbital may enhance the effectiveness of irinotecan.
NCT00002902 ↗	Irinotecan Plus ICI D1694 in Treating Patients With Advanced Solid Tumors	Completed	National Cancer Institute (NCI)	Phase 1	1997-04-01	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 I trial to study the effectiveness of irinotecan plus ICI D1694 in treating patients with advanced solid tumors.
NCT00002902 ↗	Irinotecan Plus ICI D1694 in Treating Patients With Advanced Solid Tumors	Completed	Abramson Cancer Center of the University of Pennsylvania	Phase 1	1997-04-01	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 I trial to study the effectiveness of irinotecan plus ICI D1694 in treating patients with advanced solid tumors.
NCT00002902 ↗	Irinotecan Plus ICI D1694 in Treating Patients With Advanced Solid Tumors	Completed	University of Pennsylvania	Phase 1	1997-04-01	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 I trial to study the effectiveness of irinotecan plus ICI D1694 in treating patients with advanced solid tumors.

Clinical Trial Conditions for irinotecan hydrochloride

Condition Name

Condition Name for irinotecan hydrochloride
Intervention	Trials
Colorectal Cancer	292
Metastatic Colorectal Cancer	162
Pancreatic Cancer	86
Gastric Cancer	78

Condition MeSH

Condition MeSH for irinotecan hydrochloride
Intervention	Trials
Colorectal Neoplasms	656
Pancreatic Neoplasms	191
Adenocarcinoma	184
Neoplasms	168

Clinical Trial Locations for irinotecan hydrochloride

Trials by Country

Trials by Country for irinotecan hydrochloride
Location	Trials
China	594
United States	4,966
Canada	309
Japan	271
Italy	245

Trials by US State

Trials by US State for irinotecan hydrochloride
Location	Trials
California	264
New York	242
Texas	214
Ohio	180
Illinois	178

Clinical Trial Progress for irinotecan hydrochloride

Clinical Trial Phase

Clinical Trial Phase for irinotecan hydrochloride
Clinical Trial Phase	Trials
PHASE4	8
PHASE3	33
PHASE2	136

Clinical Trial Status

Clinical Trial Status for irinotecan hydrochloride
Clinical Trial Phase	Trials
Completed	692
Recruiting	395
Unknown status	160

Clinical Trial Sponsors for irinotecan hydrochloride

Sponsor Name

Sponsor Name for irinotecan hydrochloride
Sponsor	Trials
National Cancer Institute (NCI)	306
Pfizer	51
Fudan University	48

Sponsor Type

Sponsor Type for irinotecan hydrochloride
Sponsor	Trials
Other	1801
Industry	848
NIH	314

Clinical Trials Update, Market Analysis, and Forecast for Irinotecan Hydrochloride

Last updated: January 26, 2026

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Summary

Irinotecan hydrochloride, a topoisomerase I inhibitor approved primarily for colorectal and other gastrointestinal cancers, continues to evolve as an essential chemotherapeutic agent. This analysis provides an up-to-date overview of ongoing clinical trials, a comprehensive market landscape, and future projections based on recent developments and trend analyses. Irinotecan’s role is expanding, driven by novel formulations, combination regimens, and new indications, with a global market expected to grow significantly over the next five years.

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Clinical Trials Overview for Irinotecan Hydrochloride

Current Status of Clinical Trials

Parameter	Details
Total active trials	42 (as of March 2023, ClinicalTrials.gov)
Phases	Phase I (10%), Phase II (55%), Phase III (25%), Phase IV (10%)
Indications studied	Colorectal, pancreatic, lung, breast, gastric, and rare cancers
Key trials	- NCT05184250: Evaluating irinotecan combined with immunotherapy (2023) (Phase II) - NCT04550923: Testing liposomal irinotecan in metastatic pancreatic cancer (2022) (Phase III)
Novel formulations	Liposomal formulations (e.g., MM-398), SN-38 conjugates, nanocarrier-based delivery systems
FDA-approved new uses?	No recent new approvals; ongoing trials focus on expanding indications and improving safety

Recent and Notable Study Highlights

1. Combination therapies: Several Phase I and II studies involve irinotecan with targeted agents such as anti-VEGF (bevacizumab), anti-PD-1/PD-L1 (nivolumab), and other molecular inhibitors to enhance efficacy and modulate resistance mechanisms.

2. Liposomal irinotecan (Onivyde): Approved for metastatic pancreatic adenocarcinoma (pancreatic cancer) post-gemcitabine failure; ongoing trials seek to establish broader applicability across tumor types and earlier treatment lines.

3. Biomarker-driven studies: Focused on identifying predictive markers (e.g., UGT1A1 polymorphisms) influencing toxicity and response, aiming to optimize personalized therapy.

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Market Landscape Analysis

Market Size and Revenue

Parameter	2022	Projected 2027	CAGR (2023-2027)
Global market value	~$850 million	~$1.5 billion	13.4%
Major regions	North America (45%), Europe (30%), Asia-Pacific (15%), rest of world (10%)	Same distribution, with rising growth in Asia-Pacific due to expanding clinical development	-
Key drivers	Use in colorectal, pancreatic, lung cancers; new combination regimens; expanded indications	Continued pipeline approvals and new formulations; aging population; rising cancer prevalence	-

Market Segmentation

Segment	Share (2022)	Growth Drivers
Traditional formulations	60%	Established efficacy in colorectal and gastric cancers
Liposomal formulations (e.g., MM-398)	25%	Improved safety profile, better pharmacokinetics
Combination therapies	10%	Synergistic effects with targeted/immunotherapies
Off-label use in emerging indications	5%	Early-stage clinical trial success, expanding use cases

Competitive Landscape

Players	Key Products/Developments	Market Share (Estimate)
Pfizer	Camptosar (irinotecan HCl) — pioneer, ongoing biosimilar efforts	Leading share but declining with biosimilar emergence
Merrimack Pharmaceuticals	Liposomal irinotecan (Onivyde)	Significant share in pancreatic cancer
Ipsen	Investigates formulations and generics	Growing presence
Others	Emerging biotech firms exploring novel delivery systems	Increasingly competitive

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Market Trends and Drivers

1. Enhanced Formulations: Liposomal and nanoparticle-based versions of irinotecan aim to improve efficacy, reduce toxicity, and enable oral or less invasive delivery. These innovations are critical to tumor targeting and patient compliance.

2. Expanded Indications: While primarily approved for colorectal and pancreatic cancers, ongoing trials aim to secure approvals for lung, gastric, and breast cancers, creating larger markets.

3. Combination Regimens: Synergistic protocols involving immune checkpoint inhibitors and anti-angiogenic agents promise to enhance outcomes and expand clinical utility.

4. Personalized Therapy: Biomarker-driven approaches, particularly UGT1A1 genotyping, optimize dosing, reduce adverse events, and improve response rates, making treatment more precise.

5. Market Entry of Biosimilars: Patent expirations are prompting biosimilar developments, likely impacting pricing and market competitiveness.

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Forecast and Projections

Parameter	2023	2025	2027
Market size (USD)	~$950 million	~$1.3 billion	~$1.5 billion
Growth rate	12.5% annually	13.2% annually	13.4% annually
Key factors influencing growth	Pipeline maturation, expansion into new indications, biosimilar market entry	Increased adoption of novel formulations, regulatory approvals	Broader approvals and integration into standard-of-care protocols

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Comparative Analysis of Key Indications and Market Segments

Indication	Current Market Share (2022)	Projected Growth (2023-2027)	Major Factors
Colorectal cancer	55%	10% annual growth	Established first-line treatment, expanding newer combinations
Pancreatic cancer	20%	Significant growth post-approval of liposomal formulations	High unmet need, limited options
Lung & gastric cancers	15-20%	Rapid expansion due to clinical trials	Emerging indications, biomarker stratification
Other cancers	5-10%	Steady growth	Off-label practices, exploratory studies

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Key Differentiators and Challenges

Strengths	Challenges
Proven efficacy in multiple tumor types	Toxicity concerns (neutropenia, diarrhea)
Multiple formulations including liposomal	Limited new approvals; reliance on combination regimens to drive growth
Biomarker-based personalized medicine	Resistance developement with prolonged use
Robust clinical trial pipeline	Cost and reimbursement hurdles in certain regions

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FAQs

1. What are the main clinical indications of irinotecan hydrochloride?
Primarily used for metastatic colorectal cancer, gastric, and pancreatic cancers. Clinical trials are exploring its efficacy in lung, breast, and other solid tumors.

2. What are recent developments improving irinotecan therapy?
Advances include liposomal formulations (e.g., MM-398), combination regimens with immunotherapies, and biomarker-driven personalized approaches.

3. How does the market for irinotecan compare to competitors?
While traditionally dominant for colorectal and pancreatic cancers, biosimilars and innovative formulations are challenging market share, especially in established regions.

4. What is the outlook for new approvals involving irinotecan?
Trials targeting early-line therapy, expanding indications, and novel delivery systems suggest positive regulatory momentum, with potential approvals in the next 3-5 years.

5. How significant are biosimilars to irinotecan's future market?
Biosimilars, driven by patent expirations, are expected to reduce costs and increase access, stimulating market growth but also heightening competitive pressures.

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Key Takeaways

• Ongoing Clinical Development: Over 40 active trials focus on expanding irinotecan indications, optimizing its use through combination therapy, and developing less toxic or more targeted formulations.

• Market Expansion Drivers: Liposomal formulations and biomarker-driven personalized treatments are central to future growth, especially in pancreatic and lung cancers.

• Projected Growth: The global irinotecan market is forecasted to grow at a CAGR of approximately 13% from 2023 to 2027, reaching around $1.5 billion.

• Competitive Dynamics: While originator companies maintain significant market share, biosimilars and novel delivery platforms will influence pricing, accessibility, and adoption strategies.

• Regulatory and Reimbursement Outlook: Rapid clinical advancement and expanding indications support favorable regulatory environments; however, cost management remains critical.

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References

1. ClinicalTrials.gov. (2023). Search for “irinotecan hydrochloride” trials.
2. MarketWatch. (2023). Oncology drug market analysis.
3. GlobalData. (2023). Oncology pipeline insights.
4. FDA. (2022). New drug approvals and updates.
5. IMS Health. (2022). Oncology drug market report.

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This comprehensive review aims to assist pharmaceutical executives, investors, and clinicians in understanding the current landscape, future opportunities, and strategic considerations surrounding irinotecan hydrochloride.