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.
New Combination	NCT00230399 ↗	Combination Chemotherapy Treatments in Patients With Metastatic Colorectal Cancer	Completed	University of Michigan Rogel 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.
New Combination	NCT00876993 ↗	Study of Irinotecan and Bevacizumab With Temozolomide in Refractory/Relapsed Central Nervous System (CNS) Tumors	Completed	Brain Tumor Alliance	Phase 1	2008-09-01	Bevacizumab, irinotecan, and temozolomide are three agents shown to have promising activity in a variety of central nervous system tumors. No prospective studies have been published or are currently in progress within the major consortiums with this combination of drugs. Brain tumors are the second most common cause of cancer in pediatrics and the leading cause of cancer death in children. For children with High Grade Gliomas or with relapsed/refractory brain tumors, new agents in new combinations are needed. Historical data shows that newly diagnosed high grade gliomas 5 year progression free survival is 28-42%. Recurrent malignant gliomas median survival is 3-9 months. Recurrent medulloblastoma's 2 years survival is 9%. This study is a phase I study designed to provide an objective observation of toxicity and establish a maximum tolerated dose of this combination. In addition, this study will observe the response of children with relapsed or refractory central nervous system tumors.

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.
NCT00002933 ↗	Irinotecan in Treating Patients With Metastatic or Recurrent Colorectal Cancer	Completed	National Cancer Institute (NCI)	Phase 2	1996-09-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Phase II trial to study the effectiveness of irinotecan in treating patients who have metastatic or recurrent colorectal cancer.
NCT00002933 ↗	Irinotecan in Treating Patients With Metastatic or Recurrent Colorectal Cancer	Completed	Case Comprehensive Cancer Center	Phase 2	1996-09-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Phase II trial to study the effectiveness of irinotecan in treating patients who have metastatic or recurrent colorectal cancer.

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

IRINOTECAN HYDROCHLORIDE Market Analysis and Financial Projection

Last updated: April 25, 2026

Irinotecan Hydrochloride: Clinical Trials Update, Market Analysis, and Projection

What is the current clinical-trials status for irinotecan hydrochloride?

Irinotecan hydrochloride is a long-established oncology agent used across multiple regimens in colorectal cancer (CRC) and other solid tumors. Trial activity remains concentrated in: (1) combination regimens with fluoropyrimidines and oxaliplatin in CRC, (2) line-expansion studies in metastatic and advanced settings, and (3) reformulation or biologic/targeted combination strategies where irinotecan is the cytotoxic backbone.

Current-state signals (late-stage focus):

• The clinical pipeline emphasis stays on combinations that improve response and survival in CRC where irinotecan-based chemotherapy is standard of care.
• Trials also track sequencing questions in first-line vs second-line settings, including management of resistance to earlier fluoropyrimidine/oxaliplatin exposure.

Evidence base used in this analysis:

• FDA regulatory and label framework for irinotecan (as marketed under various brand and generic forms) supports ongoing combination use and population labeling in CRC.
• NCCN CRC guidance codifies irinotecan-containing systemic therapy pathways across lines, which drives continued trial enrollment and sponsor interest.

Regimen anchor in practice (guidance-derived):

• For metastatic CRC, NCCN-aligned systemic therapy typically includes irinotecan-based doublets or triplets (often with 5-FU/leucovorin and/or oxaliplatin) depending on prior therapy exposure and performance status. NCCN updates inform trial design and eligibility criteria across geographies and sponsors. [1], [2]

Which clinical trial themes are driving development?

The trial landscape for irinotecan hydrochloride is shaped by four recurring categories:

1. Combination chemotherapy intensification in CRC

   • Trials evaluate chemotherapy backbones with irinotecan plus standard partners (fluoropyrimidine and oxaliplatin) to improve objective response rate and survival.
   • Design logic targets first-line regimens and subsequent-line conversions based on prior exposure.

2. Sequencing and line-of-therapy strategies

   • Trials test the order of irinotecan-based regimens versus other cytotoxics after progression on earlier treatments.
   • Endpoints often include progression-free survival (PFS), overall survival (OS), and response durability.

3. Targeted and biologic combinations

   • Sponsors combine irinotecan with targeted agents used in mCRC.
   • These studies tend to recruit biomarker-enriched subsets or apply stratification for RAS/BRAF status, MSI/dMMR, and performance status.

4. Pharmacology and formulation optimization

   • Reformulation studies focus on dosing consistency, administration logistics, and tolerability management (e.g., diarrhea prophylaxis and supportive-care integration).
   • These efforts are important because irinotecan’s dose-limiting toxicities (notably delayed diarrhea) materially affect adherence and dose intensity.

How do labeling and safety requirements shape trial designs?

Irinotecan hydrochloride prescribing information and standards of care influence trial eligibility, supportive-care requirements, and dose modification rules. The main driver is toxicity management, particularly diarrhea and neutropenia, which are core protocol considerations.

Key label constraints relevant to development programs:

• Diarrhea risk: protocols require active monitoring and early intervention; supportive medication use is standard.
• Myelosuppression: dose modifications and growth-factor considerations are embedded into trial protocols where permitted.

These constraints are central to feasibility and enrollment targets because they affect protocol adherence and discontinuation rates in both global and regional sites. [1]

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What is the market size context for irinotecan hydrochloride and how is it changing?

How does the product fit the CRC market?

Irinotecan hydrochloride is entrenched in metastatic colorectal cancer chemotherapy. Market pull is supported by:

• guideline inclusion for systemic therapy pathways,
• broad historical use and established clinical positioning,
• continued demand for chemotherapy options where targeted therapy is not sufficient alone.

Market framing:

• In practice, the market value for irinotecan is driven by incidence of CRC, metastatic proportions, and second-line and later-line chemotherapy utilization rates.
• Price competition and generic penetration have compressive effects on revenue per unit, shifting value toward volume, procurement channels, and tender competitiveness.

What are the commercial headwinds?

The main commercial risks to revenue are structural:

• Generic competition: irinotecan’s patent landscape and multiple generic entrants typically reduce branded premium pricing.
• Substitution risk: other cytotoxics can substitute in later lines depending on toxicity profiles, access, and payer preferences.
• Toxicity-related treatment discontinuation: if a competitor’s supportive-care approach enables better adherence, it can shift market share even when efficacy is comparable.

What are the commercial tailwinds?

• Guideline persistence: irinotecan-containing regimens remain integrated in standard CRC workflows across multiple geographies.
• Combination demand: irinotecan is frequently used as a chemotherapy backbone in combination trials and real-world regimens.
• Global procurement scale: high-volume oncology chemotherapy tends to generate consistent purchasing even when unit pricing falls.

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What is the revenue/projection outlook for irinotecan hydrochloride?

How to project demand: a regimen-and-lines framework

Because irinotecan’s commercial demand correlates with CRC patient flow through lines of therapy, projections use a structured approach:

Projection drivers

• CRC incidence and stage distribution
• Metastatic conversion rate
• Proportion receiving systemic chemotherapy
• Average number of lines using irinotecan-containing regimens
• Dose intensity and treatment duration influenced by supportive care
• Market access and tendering dynamics

Projection shape

• Revenue is most likely to show stable-to-declining unit value driven by price compression and generic competition.
• Total market revenue can still expand modestly if volume growth outpaces unit price declines, which depends on regional CRC treatment coverage and adoption of multi-line chemotherapy.

Base-case market projection (directional)

Without a specific dataset of current global revenues and price levels for irinotecan hydrochloride by region, a numeric forecast cannot be credibly produced. What can be stated as decision-grade guidance is the expected direction:

• Volume trend: steady to gradually increasing where CRC screening and survivorship increase treatment prevalence, and where access expands.
• Price trend: downward due to generic competition and tender-driven pricing.
• Net revenue: likely flat to modestly declining in many markets, with regional variation driven by reimbursement systems and supply competition.

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Competitive and development implications for investors and R&D leaders

Where does development create differentiation?

For irinotecan hydrochloride, differentiation typically comes from one of three places:

1. Better tolerability via formulation or protocolized supportive care
2. Administration convenience (reduced infusion burden or improved stability)
3. Regimen-specific evidence that expands eligible patient subgroups

What is the “must-have” clinical evidence standard?

Sponsors typically need:

• demonstrated safety in populations aligned with label and CRC practice,
• non-inferior or improved efficacy in relevant endpoints (PFS/OS/ORR),
• manageability data that supports dose intensity and reduced discontinuation.

These standards align with regulatory and guideline expectations for chemotherapy combination development. [1], [2]

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Key Takeaways

• Clinical activity for irinotecan hydrochloride stays centered on colorectal cancer regimens, with development concentrated in combination chemotherapy strategies, sequencing, and biologic/targeted partner studies.
• Safety and dosing rules (notably diarrhea and myelosuppression management) materially shape trial design, eligibility, and discontinuation risk, which influences both endpoints and real-world adoption.
• Market revenue is likely to face persistent unit price pressure from generic competition, while volume demand is steadier, anchored by colorectal cancer incidence and multi-line chemotherapy usage.
• Projections for irinotecan hydrochloride should be modeled through CRC patient flow across lines of therapy, with adjustment for supportive-care impact on treatment duration and discontinuation.

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FAQs

1) What cancers drive irinotecan hydrochloride demand?

Colorectal cancer is the primary demand driver, because irinotecan is integrated into metastatic CRC chemotherapy pathways across multiple lines. [1], [2]

2) Why does toxicity management matter for both trials and sales?

Irinotecan’s diarrhea and neutropenia risk affects dose intensity and treatment continuation, which influences trial completion and real-world persistence, impacting both clinical outcomes and procurement demand. [1]

3) What clinical endpoints matter most in irinotecan combination trials?

Trials most commonly track PFS and OS, with ORR and duration of response used to contextualize efficacy in combination regimens. [2]

4) What is the main market risk for revenue?

Generic competition and tender-driven price compression are the dominant headwinds, reducing unit value even if volume stays stable.

5) What type of development is most likely to expand uptake?

Programs that improve tolerability and allow higher dose intensity or better adherence, or that generate regimen-specific evidence expanding labeled use in guideline pathways, are most likely to influence adoption. [1], [2]

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References

[1] U.S. Food and Drug Administration. (n.d.). Camptosar (irinotecan hydrochloride) prescribing information / label. FDA.
[2] National Comprehensive Cancer Network. (n.d.). Clinical Practice Guidelines in Oncology: Colon Cancer (and related updates). NCCN.