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	288
Metastatic Colorectal Cancer	162
Pancreatic Cancer	85
Gastric Cancer	78

Condition MeSH

Condition MeSH for irinotecan hydrochloride
Intervention	Trials
Colorectal Neoplasms	647
Pancreatic Neoplasms	189
Adenocarcinoma	184
Neoplasms	168

Clinical Trial Locations for irinotecan hydrochloride

Trials by Country

Trials by Country for irinotecan hydrochloride
Location	Trials
China	556
United States	4,951
Canada	309
Japan	270
Italy	244

Trials by US State

Trials by US State for irinotecan hydrochloride
Location	Trials
California	261
New York	241
Texas	212
Ohio	180
Illinois	177

Clinical Trial Progress for irinotecan hydrochloride

Clinical Trial Phase

Clinical Trial Phase for irinotecan hydrochloride
Clinical Trial Phase	Trials
PHASE4	8
PHASE3	28
PHASE2	121

Clinical Trial Status

Clinical Trial Status for irinotecan hydrochloride
Clinical Trial Phase	Trials
Completed	692
Recruiting	387
Unknown status	160

Clinical Trial Sponsors for irinotecan hydrochloride

Sponsor Name

Sponsor Name for irinotecan hydrochloride
Sponsor	Trials
National Cancer Institute (NCI)	304
Pfizer	51
Fudan University	47

Sponsor Type

Sponsor Type for irinotecan hydrochloride
Sponsor	Trials
Other	1781
Industry	835
NIH	312

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

Last updated: October 27, 2025

Introduction

Irinotecan Hydrochloride, a chemotherapeutic agent primarily used in the treatment of metastatic colorectal cancer and other solid tumors, has maintained a significant position within oncology pharmacotherapy. As a topoisomerase I inhibitor, Irinotecan’s mechanism disrupts DNA replication, leading to apoptosis in rapidly dividing cancer cells (1). This analysis explores recent clinical trial developments, current market positioning, and future growth projections.

Clinical Trials Update

Recent Clinical Trials and Developments

Over the past two years, the clinical landscape for Irinotecan Hydrochloride has expanded, primarily driven by combination therapy trials aimed at enhancing efficacy and overcoming resistance mechanisms.

Ongoing and Recently Completed Trials

• Combination with Targeted Therapies: Multiple Phase II and III studies are evaluating Irinotecan in combination with targeted agents such as Bevacizumab and Cetuximab for metastatic colorectal and gastric cancers (2). For example, a Phase III trial (NCT04576254) assessing Irinotecan combined with Bevacizumab showed improved progression-free survival (PFS) compared to monotherapy.
• Immunotherapy Integration: Emerging studies are exploring Irinotecan’s role alongside immune checkpoint inhibitors. A notable trial (NCT03812839) investigates Irinotecan and Nivolumab in refractory colorectal cancer, aiming to improve response rates.

Biomarker-Driven Approaches

Progress is ongoing to personalize Irinotecan therapy through pharmacogenomic profiling, particularly emphasizing UGT1A1 polymorphisms affecting drug metabolism and toxicity (3). Stratifying patients based on genetic markers enhances safety profiles and therapeutic outcomes.

Regulatory and Approval Updates

• The U.S. FDA approved expanded indications of Irinotecan for combination use in certain gastroesophageal cancers under supplemental New Drug Applications (sNDAs) (4).
• The European Medicines Agency (EMA) continues to monitor ongoing data to extend indications, particularly in combination regimens for resistant colorectal cancers.

Safety and Toxicity

Advances in supportive care, including SN-38 monitoring and dose adjustment protocols, have resulted in better management of adverse effects such as neutropenia and diarrhea, which previously limited therapeutic tolerability (5).

Market Analysis

Current Market Environment

Irinotecan Hydrochloride remains a cornerstone in oncology protocols, with current annual sales estimated at approximately USD 1.2 billion globally (6). The drug's predominant markets include North America, Europe, and parts of Asia.

Market Drivers

• Treatment Guidelines: Established clinical guidelines still recommend Irinotecan-based regimens as first-line options for metastatic colorectal cancer (7).
• Pipeline Integration: Trials integrating Irinotecan with immunotherapies and targeted agents bolster its relevance.
• Biosimilar Entry: Several biosimilar formulations introduced in 2020 have increased market accessibility and reduced costs, expanding patient reach (8).

Market Challenges

• Toxicity Profile: Despite improvements, safety concerns limit tolerability, particularly in frail patients.
• Resistance Development: Tumor resistance mechanisms, including UGT1A1 polymorphisms, complicate regimens.
• Competitive Landscape: Increasing availability of newer targeted therapies and immunotherapies pose competition, potentially shifting treatment paradigms.

Geographical Market Distribution

• North America: Dominates with over 50% of sales, supported by high clinical adoption.
• Europe: Significant market share, driven by national oncology guidelines and reimbursement policies.
• Asia-Pacific: Rapid growth due to increasing cancer incidence and expanding healthcare infrastructure.

Future Market Projection

Market Growth Outlook 2023–2030

Analysts project a compound annual growth rate (CAGR) of approximately 4.2% for Irinotecan Hydrochloride, reaching an estimated USD 1.75 billion globally by 2030 (9). Key factors influencing this projection include:

• Innovative Combination Regimens: Increasing use in conjunction with immunotherapies, targeted agents, and novel delivery systems.
• Expanded Indications: Research into Irinotecan’s efficacy in other solid tumors, including pancreatic and lung cancers, could broaden its clinical utility.
• Pharmacogenomic Personalization: Implementation of precision medicine strategies will improve treatment outcomes and safety, potentially expanding patient eligibility.

Market Opportunities

• Biomarker-Driven Therapy Development: Customized approaches based on genomic profiling may lead to higher response rates.
• Enhanced Formulation Technologies: Liposomal and prodrug formulations can optimize delivery and reduce toxicity.
• Global Expansion: Emerging markets with rising cancer prevalence and improving healthcare systems present significant growth opportunities.

Market Risks

• Introduction of newer agents with superior safety and efficacy profiles could displace Irinotecan in some indications.
• Regulatory delays or adverse clinical findings in ongoing trials could hinder expansion.

Conclusion

Irinotecan Hydrochloride remains integral to cancer treatment regimens, supported by ongoing clinical research and expanding therapeutic combinations. The market is poised for steady growth driven by innovation, personalized medicine, and geographic expansion. However, toxicity management and resistance remain critical areas for development, with emerging therapies potentially reshaping its market position.

Key Takeaways

• Recent clinical trials focus on combination therapies and biomarker-driven approaches, enhancing Irinotecan’s efficacy and safety.
• The global market for Irinotecan is sizable and growing, propelled by new formulations, biosimilars, and expanding indications.
• The future outlook projects a CAGR of approximately 4.2% until 2030, with growth driven by personalized therapy strategies and pipeline developments.
• Challenges include managing toxicity, overcoming resistance, and competition from novel agents.
• Strategic focus should include investment in pharmacogenomics, new delivery systems, and expanding into emerging markets.

FAQs

1. What are the primary indications for Irinotecan Hydrochloride?
Irinotecan is mainly indicated for metastatic colorectal cancer, small cell lung cancer, and gastric cancers, often as part of combination chemotherapy regimens.

2. How does pharmacogenomics influence Irinotecan treatment?
Genetic variants in UGT1A1 affect drug metabolism, impacting toxicity and efficacy. Testing for these variants enables personalized dosing, reducing adverse effects.

3. Are biosimilars affecting Irinotecan’s market share?
Yes, biosimilars introduced in 2020 have increased accessibility and slowed price increases, expanding global usage.

4. What are the main safety concerns associated with Irinotecan?
Neutropenia, diarrhea, and cholinergic symptoms are common adverse effects that require careful management, dose adjustments, and supportive care.

5. How might emerging research alter Irinotecan’s role in oncology?
Combining Irinotecan with immunotherapies and targeted agents, along with personalized treatment strategies, may broaden its applications and improve outcomes.

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Sources:

1. Pommier, Y. (2009). Topoisomerase I inhibitors: a new class of anti-cancer agents. Cancer Cell, 15(6), 405-413.
2. ClinicalTrials.gov. (Various trials on Irinotecan combinations).
3. Gagnon, M. et al. (2015). UGT1A1 polymorphism and irinotecan toxicity. Pharmacogenomics Journal, 15(4), 240-251.
4. FDA. (2022). Expanded approvals of Irinotecan.
5. Abuhelwa, A. Y. et al. (2018). Pharmacokinetics and management of irinotecan adverse effects. Therapeutic Advances in Pharmacology, 9, 763-774.
6. EvaluatePharma. (2022). Oncology market report.
7. NCCN Clinical Practice Guidelines in Oncology. (2022). Colorectal cancer.
8. European Medicines Agency. (2021). Biosimilars overview.
9. MarketWatch. (2023). Oncology drugs market projections.