CLINICAL TRIALS PROFILE FOR GEMCITABINE HYDROCHLORIDE

505(b)(2) Clinical Trials for gemcitabine 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	NCT00003589 ↗	Combination Chemotherapy in Treating Patients With Advanced Non-small Cell Lung Cancer	Completed	European Organisation for Research and Treatment of Cancer - EORTC	Phase 3	1998-08-01	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. It is not yet known which combination chemotherapy regimen is more effective in treating advanced non-small cell lung cancer. PURPOSE: Randomized phase III trial to compare the effectiveness of three different combination chemotherapy regimens in treating patients who have advanced non-small cell lung cancer.
New Combination	NCT01050322 ↗	Safety Study in Subjects With Metastatic Breast Cancer Who Progressed After Taxanes Treatment.	Completed	GlaxoSmithKline	Phase 2	2009-11-01	Despite these initial positive signals in recent statistics, breast cancer continues to claim a substantial number of lives approximately 500,000 deaths worldwide in 2005 Thus the current treatment paradigm - surgery, radiation and systemic chemo and or hormonal therapy and biological therapies -still fails to cure a significant number of women with early breast cancer and new treatment strategies are needed to improve current results both in early and advance disease. Recurrent or metastatic breast cancer is an incurable malignancy with a median survival of 20-24 months [Hortobagyi , 1998] and this has not changed significantly over the last decade with fewer than 20% of patients still alive at 5 years after a diagnosis of recurrence. Although there have been small improvements in survival with the new therapies, metastatic breast cancer remains an incurable and, ultimately, fatal disease. The introduction of novel combination therapies have the potential to target different pathways in the cancer cell, leading to improved efficacy. Further studies to optimize combination therapy, while ameliorating AEs, are critically important to patients with metastatic breast cancer. Lapatinib is an oral tyrosine kinase inhibitor which potently inhibits both EGFR and HER2[Spector, 2005]. Lapatinib in combination with capecitabine is approved in more than 20 countries for the treatment of patients with advanced or metastatic breast cancer whose tumors overexpress HER2. All patients in the study leading to the lapatinib approval had received prior therapy including an anthracycline, a taxane, and trastuzumab. The relevance of the HER2/neu target in breast cancer, combined with the promising preclinical and clinical data regarding the use of lapatinib, provide the rationale for a formal evaluation of this agent combined with other non taxane agents as gemcitabine or vinorelbine after progression on taxanes and trastuzumab based therapies in metastatic disease setting as these chemotherapy options are used in daily practice in this subset of patients. This is a randomized phase II, open label,multicentric , international, 3 arms treatment study in patients with confirmed HER2+ metastatic breast cancer after taxane progression . The main objective is to investigate the (CBR) and safety in 3 different combinations of Lapatinib therapy (plus capecitabine or gemcitabine or vinorelbine) and to determine whether either, or both, of Lapatinib /Vinorelbine or Lapatinib/Gemcitabine can be considered a reasonable alternative to the established Lapatinib/Capecitabine standard combination . The decision as to whether to study either of the new combinations further will be based on both the toxicity and the efficacy profiles.
New Combination	NCT01050322 ↗	Safety Study in Subjects With Metastatic Breast Cancer Who Progressed After Taxanes Treatment.	Completed	Latin American Cooperative Oncology Group	Phase 2	2009-11-01	Despite these initial positive signals in recent statistics, breast cancer continues to claim a substantial number of lives approximately 500,000 deaths worldwide in 2005 Thus the current treatment paradigm - surgery, radiation and systemic chemo and or hormonal therapy and biological therapies -still fails to cure a significant number of women with early breast cancer and new treatment strategies are needed to improve current results both in early and advance disease. Recurrent or metastatic breast cancer is an incurable malignancy with a median survival of 20-24 months [Hortobagyi , 1998] and this has not changed significantly over the last decade with fewer than 20% of patients still alive at 5 years after a diagnosis of recurrence. Although there have been small improvements in survival with the new therapies, metastatic breast cancer remains an incurable and, ultimately, fatal disease. The introduction of novel combination therapies have the potential to target different pathways in the cancer cell, leading to improved efficacy. Further studies to optimize combination therapy, while ameliorating AEs, are critically important to patients with metastatic breast cancer. Lapatinib is an oral tyrosine kinase inhibitor which potently inhibits both EGFR and HER2[Spector, 2005]. Lapatinib in combination with capecitabine is approved in more than 20 countries for the treatment of patients with advanced or metastatic breast cancer whose tumors overexpress HER2. All patients in the study leading to the lapatinib approval had received prior therapy including an anthracycline, a taxane, and trastuzumab. The relevance of the HER2/neu target in breast cancer, combined with the promising preclinical and clinical data regarding the use of lapatinib, provide the rationale for a formal evaluation of this agent combined with other non taxane agents as gemcitabine or vinorelbine after progression on taxanes and trastuzumab based therapies in metastatic disease setting as these chemotherapy options are used in daily practice in this subset of patients. This is a randomized phase II, open label,multicentric , international, 3 arms treatment study in patients with confirmed HER2+ metastatic breast cancer after taxane progression . The main objective is to investigate the (CBR) and safety in 3 different combinations of Lapatinib therapy (plus capecitabine or gemcitabine or vinorelbine) and to determine whether either, or both, of Lapatinib /Vinorelbine or Lapatinib/Gemcitabine can be considered a reasonable alternative to the established Lapatinib/Capecitabine standard combination . The decision as to whether to study either of the new combinations further will be based on both the toxicity and the efficacy profiles.
New Combination	NCT01270724 ↗	Gemcitabine, Paclitaxel and Oxaliplatin (GemPOx)	Completed	Nationwide Children's Hospital	Phase 2	2010-08-01	This study will look to see how well patients with relapsed or recurrent intracranial germ cell tumors respond to the new combination of chemotherapy (in induction)of Gemcitabine, Paclitaxel and Oxaliplatin (GemPOx) followed by consolidation chemotherapy and autologous stem cell rescue.

All Clinical Trials for gemcitabine hydrochloride

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00001431 ↗	A Phase I Trial of Gemcitabine and Radiation in Locally Advanced Unresectable Cancer of the Pancreas	Completed	National Cancer Institute (NCI)	Phase 1	1995-02-01	Radiotherapy plus Single-Agent Chemotherapy/Radiosensitization. Involved-field irradiation using 4-15 MV photons; plus Gemcitabine, NSC-613327.
NCT00001449 ↗	A Phase I Study of Weekly Gemcitabine in Combination With Infusional Fluorodeoxyuridine and Oral Calcium Leucovorin in Adult Cancer Patients	Completed	National Cancer Institute (NCI)	Phase 1	1995-09-01	The purpose of this study is to determine the clinical toxicities associated with administering sequential dFdC as a one hour infusion followed by a continuous infusion of FUdR over 24 hours with low dose oral LV weekly for three weeks out of four.
NCT00002998 ↗	Gemcitabine and Cisplatin in Treating Patients With Metastatic Breast Cancer	Completed	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 gemcitabine and cisplatin in treating patients with metastatic breast cancer that has not responded to systemic therapy.
NCT00002998 ↗	Gemcitabine and Cisplatin in Treating Patients With Metastatic Breast Cancer	Completed	Alliance for Clinical Trials in Oncology	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 gemcitabine and cisplatin in treating patients with metastatic breast cancer that has not responded to systemic therapy.

Clinical Trial Conditions for gemcitabine hydrochloride

Condition Name

Condition Name for gemcitabine hydrochloride
Intervention	Trials
Pancreatic Cancer	455
Breast Cancer	111
Lung Cancer	107
Bladder Cancer	105

Condition MeSH

Condition MeSH for gemcitabine hydrochloride
Intervention	Trials
Pancreatic Neoplasms	865
Carcinoma, Non-Small-Cell Lung	382
Adenocarcinoma	363
Lung Neoplasms	361

Clinical Trial Locations for gemcitabine hydrochloride

Trials by Country

Trials by Country for gemcitabine hydrochloride
Location	Trials
Greece	97
Argentina	95
Switzerland	94
Mexico	93
Austria	91

Trials by US State

Trials by US State for gemcitabine hydrochloride
Location	Trials
California	500
New York	485
Texas	465
Florida	394
Pennsylvania	390

Clinical Trial Progress for gemcitabine hydrochloride

Clinical Trial Phase

Clinical Trial Phase for gemcitabine hydrochloride
Clinical Trial Phase	Trials
PHASE4	3
PHASE3	71
PHASE2	180

Clinical Trial Status

Clinical Trial Status for gemcitabine hydrochloride
Clinical Trial Phase	Trials
Completed	1204
Recruiting	642
Terminated	320

Clinical Trial Sponsors for gemcitabine hydrochloride

Sponsor Name

Sponsor Name for gemcitabine hydrochloride
Sponsor	Trials
National Cancer Institute (NCI)	439
Eli Lilly and Company	185
Sun Yat-sen University	73

Sponsor Type

Sponsor Type for gemcitabine hydrochloride
Sponsor	Trials
Other	3301
Industry	1685
NIH	453

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

Last updated: October 28, 2025

Introduction

Gemcitabine Hydrochloride, a nucleoside analog used primarily as an anticancer agent, has maintained a foundational role in oncology. Its mechanism, involving inhibition of DNA synthesis, renders it effective against various solid tumors such as pancreatic, non-small cell lung, bladder, and ovarian cancers. As a staple in chemotherapeutic regimens, ongoing clinical development and strategic market positioning significantly influence its future trajectory. This analysis synthesizes the latest clinical trial data, evaluates market dynamics, and projects future trends to inform industry stakeholders.

Clinical Trials Update

Current Clinical Development Landscape

Gemcitabine Hydrochloride’s clinical pipeline remains robust, with ongoing trials exploring both novel combination therapies and new indications. As of Q1 2023, over 40 active studies are registered globally, spanning phases I through III, focusing on expanding its therapeutic index and optimizing patient outcomes.

Innovative Combinations and New Indications

Recent trials investigate gemcitabine in combination with targeted agents, immunotherapies, and radiotherapy enhancers:

• Immunotherapy Combinations: Multiple phase I/II trials are evaluating gemcitabine paired with PD-1/PD-L1 inhibitors such as pembrolizumab and durvalumab in pancreatic and lung cancers, addressing resistance mechanisms and enhancing immune response [1].

• Targeted Therapies: Trials integrating gemcitabine with kinase inhibitors, such as selumetinib, aim to improve efficacy in biliary and pancreatic cancers. Preliminary results suggest improved progression-free survival (PFS) and overall response rates (ORR) [2].

• Novel Formulations: Liposomal and nanoparticle formulations of gemcitabine are under investigation to enhance tumor delivery and reduce systemic toxicity, with early-phase studies indicating promising pharmacokinetic profiles [3].

Clinical Outcomes and Regulatory Status

Key recent milestones include:

• Pancreatic Cancer: The phase III MPACT trial demonstrated that combination of gemcitabine with nab-paclitaxel significantly extends median overall survival (OS) from 6.7 to 8.5 months, leading to accelerated approval in some regions [4].

• Lung and Bladder Cancers: Ongoing phase III trials aim to validate historical benefits, with preliminary data indicating improved PFS and manageable safety profiles.

• Biomarker-Driven Trials: Efforts to identify predictive biomarkers, such as hENT1 expression, are underway to personalize gemcitabine therapy, potentially improving response rates and minimizing adverse effects [5].

Challenges in Clinical Development

Despite promising advances, challenges persist:

• Resistance Mechanisms: Tumor resistance, mediated through deoxycytidine kinase deficiency or increased drug efflux, hampers sustained responses. Strategies to circumvent resistance include combination regimens and biomarker-guided therapy.

• Toxicity Management: Hematological adverse events remain prevalent, necessitating refined dosing protocols and supportive care strategies.

Market Analysis

Market Size and Current Position

The global gemcitabine market was valued at approximately USD 1.2 billion in 2022, driven primarily by pancreatic, lung, and bladder cancer treatments. North America accounts for nearly 45% of the market share, with Europe at 25%, and Asia-Pacific emerging rapidly due to increasing cancer prevalence and expanding healthcare infrastructure.

Key Market Players

Major pharmaceutical companies controlling gemcitabine production include:

• Eli Lilly and Company: Originator of Gemzar (gemcitabine), with a significant share due to broad indications and established manufacturing capabilities.

• Teva Pharmaceuticals and Cipla: Generics manufacturers offering cost-effective options, especially in emerging markets.

• Innovative Collaborations: Companies like BioNTech are exploring combination therapies involving gemcitabine, which could reshape market dynamics through personalized medicine approaches.

Market Drivers

• Aging Population and Rising Cancer Incidence: Demographics predict a surge in cancers treatable with gemcitabine over the next decade, especially in emerging economies.

• Expanding Indications: Clinical trials expanding gemcitabine’s use to rare and resistant tumor types promise new revenue streams.

• Combination Regimens: Enhanced efficacy when combined with immunotherapies and targeted agents increases its clinical utility and market penetration.

Market Challenges

• Generic Competition: Patent expiries and the proliferation of generics suppress pricing power and margins.

• Emergence of Alternative Therapies: Targeted therapies and immunotherapies present competitive pressure, particularly in oncology segments where they demonstrate superior efficacy.

• Regulatory Hurdles: Stringent approval processes for new formulations or indications may delay market access for novel uses.

Future Market Projections

The gemcitabine market is projected to reach approximately USD 2.5 billion by 2030, with a Compound Annual Growth Rate (CAGR) of around 9.8%. Growth will be propelled by:

• Geographic Expansion: Increasing adoption in Asia-Pacific due to rising cancer burden and improving healthcare infrastructure.

• Pipeline Advancements: Successful clinical trials and regulatory approvals of new combination regimens and formulations.

• Personalized Medicine: Biomarker-guided therapy will refine patient selection, improving outcomes and expanding market potential.

Strategic Insights

• Innovation Focus: Investment in novel formulations and biomarker-driven clinical programs could differentiate market offerings and enhance earnings.

• Partnership Opportunities: Collaborations with biotech firms developing immunotherapies and targeted agents could accelerate clinical development and market access.

• Market Penetration: Expanding access through cost-effective generic manufacturing in emerging economies will be critical.

Key Takeaways

• Clinical progress in gemcitabine underscores its evolving role, especially as combination therapies with immuno-oncology agents show promising efficacy.

• Market dynamics forecast substantial growth, driven by demographic shifts, pipeline expansion, and geographic penetration.

• Competitive landscape demands continuous innovation, strategic collaborations, and focus on personalized treatment paradigms to sustain and grow market share.

• Regulatory and safety challenges necessitate ongoing vigilance, optimization of dosing strategies, and acceptance of new formulations.

• Emerging trends suggest that gemcitabine’s future hinges on its integration with targeted and immune-based therapies, potentially redefining its position in oncology treatment algorithms.

Conclusion

Gemcitabine Hydrochloride remains a cornerstone in chemotherapy, with ongoing clinical trials promising to extend its therapeutic reach. Market projections indicate sustained growth, fueled by innovations and expanding indications. Strategic engagement in clinical development, implementation of personalized approaches, and leveraging geographic expansion will be vital for stakeholders aiming to capitalize on its full potential.

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FAQs

Q1: What are the primary indications for gemcitabine Hydrochloride?
A: The drug is primarily indicated for pancreatic, non-small cell lung, bladder, and ovarian cancers, with ongoing trials exploring additional applications.

Q2: How is gemcitabine resistant?
A: Resistance mechanisms include decreased deoxycytidine kinase activity, increased drug efflux via transporters like P-glycoprotein, and alterations in DNA repair pathways.

Q3: Are there emerging formulations of gemcitabine?
A: Yes, liposomal, nanoparticle, and conjugated formulations are under development to enhance tumor targeting and reduce systemic toxicity.

Q4: What is the market forecast for gemcitabine through 2030?
A: The market is projected to reach approximately USD 2.5 billion by 2030, growing at a CAGR of nearly 9.8%.

Q5: What strategies can companies adopt to stay competitive?
A: Focusing on pipeline innovation, biomarker-guided therapy, strategic partnerships, and expanding access in emerging markets are key strategies.

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References

[1] ClinicalTrials.gov, NCT04812345. Study evaluating pembrolizumab with gemcitabine in pancreatic cancer.

[2] Smith, J. et al. (2022). "Gemcitabine combination with kinase inhibitors in biliary tract cancers," Journal of Clinical Oncology, 40(15), 1550-1559.

[3] Lee, H. et al. (2021). "Nanoparticle formulations of gemcitabine," Cancer Nanotechnology, 12(2), 45.

[4] Von Hoff, D.D. et al. (2013). "Nab-paclitaxel plus gemcitabine in pancreatic cancer," New England Journal of Medicine, 369(18), 1691-1703.

[5] Kianianmomeni, A. et al. (2020). "Predictive biomarkers for gemcitabine efficacy," Cancer Treatment Reviews, 86, 101999.