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.
New Formulation	NCT01839487 ↗	PEGPH20 Plus Nab-Paclitaxel Plus Gemcitabine Compared With Nab-Paclitaxel Plus Gemcitabine in Participants With Stage IV Untreated Pancreatic Cancer	Completed	Halozyme Therapeutics	Phase 2	2013-05-14	This study is designed to compare the treatment effect of PEGPH20 combined with nab-paclitaxel (NAB) and gemcitabine (GEM) [PAG] to NAB and GEM [AG] in participants with Stage IV previously untreated pancreatic ductal adenocarcinoma (PDA). The study will have 2 run-in phases, one for each formulation of PEGPH20 (original and new formulations), and a Phase 2 portion. The 2 run-in phases will evaluate the safety and tolerability of the PAG treatment using the original and new succinic acid PEGPH20 formulation, respectively, compared with AG treatment. Phase 2 will have 2 stages due to a partial clinical hold that occurred from April through July 2014. The participants will be randomized in 3:1 for the run-in phases. The first stage will randomize participants in a 1:1 ratio. The second stage will randomize participants in a 2:1 ratio (PAG:AG). This is an open-label study. To minimize bias to the progression-free survival endpoint, disease progression will be based on the assessment of the Central Imaging Reader (CIR). Determination of clinical progression by the Investigator without corresponding CIR confirmation will be documented with the relevant signs and symptoms.
New Combination	NCT01884428 ↗	Study of Combination of PIGEV Before Autologous Stem Cell Transplant in Patients With Hodgkin's Lymphoma	Unknown status	Armando Santoro, MD	Phase 1	2011-07-01	study to assess maximum tolerated dose (MTD), safety, tolerability and activity of IGEV (Ifosfamide, Gemcitabine,Vinorelbine, Prednisolone) + Panobinostat new combination in order to determine the recommended phase II dose

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.
NCT00003001 ↗	Combination Chemotherapy in Treating Patients With Recurrent or Metastatic Colorectal Cancer	Unknown status	Stony Brook University	Phase 1/Phase 2	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/II trial to study the effectiveness of combination chemotherapy consisting of fluorouracil, leucovorin, and gemcitabine in treating patients with recurrent or metastatic colorectal cancer.
NCT00003037 ↗	Combination Chemotherapy as Induction Therapy in Treating Patients With Stage III Non-small Cell Lung Cancer	Completed	National Cancer Institute (NCI)	Phase 1/Phase 2	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/II trial to study the effectiveness of combination chemotherapy with docetaxel, gemcitabine, and cisplatin as induction therapy in treating patients with stage III non-small cell lung cancer.

Clinical Trial Conditions for gemcitabine hydrochloride

Condition Name

Condition Name for gemcitabine hydrochloride
Intervention	Trials
Pancreatic Cancer	456
Breast Cancer	111
Lung Cancer	107
Bladder Cancer	107

Condition MeSH

Condition MeSH for gemcitabine hydrochloride
Intervention	Trials
Pancreatic Neoplasms	869
Carcinoma, Non-Small-Cell Lung	384
Adenocarcinoma	364
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	502
New York	485
Texas	466
Florida	394
Pennsylvania	390

Clinical Trial Progress for gemcitabine hydrochloride

Clinical Trial Phase

Clinical Trial Phase for gemcitabine hydrochloride
Clinical Trial Phase	Trials
PHASE4	4
PHASE3	78
PHASE2	201

Clinical Trial Status

Clinical Trial Status for gemcitabine hydrochloride
Clinical Trial Phase	Trials
Completed	1205
Recruiting	651
Terminated	320

Clinical Trial Sponsors for gemcitabine hydrochloride

Sponsor Name

Sponsor Name for gemcitabine hydrochloride
Sponsor	Trials
National Cancer Institute (NCI)	441
Eli Lilly and Company	185
Sun Yat-sen University	75

Sponsor Type

Sponsor Type for gemcitabine hydrochloride
Sponsor	Trials
Other	3337
Industry	1698
NIH	455

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

Last updated: April 24, 2026

What is gemcitabine hydrochloride’s clinical status in 2024–2026?

Gemcitabine (hydrochloride) is an established oncology cytidine nucleoside analog. Trial activity in recent years concentrates on combination regimens, line- and biomarker-specific strategies, formulation variants, and metastatic solid tumors. The most commercially relevant development pattern is not label expansion into novel tumor types, but incremental gains through pairing with chemotherapy backbones, immuno-oncology agents, targeted therapies, and radiotherapy.

Clinical trial activity: what the current landscape looks like

• Trial design focus: combination therapy (gemcitabine plus agents targeting EGFR/VEGF pathways, immune checkpoints, DNA damage repair, or cell cycle pathways)
• Setting focus: advanced/metastatic disease; neoadjuvant/adjuvant studies appear but remain secondary to combination and maintenance strategies
• Endpoints used: ORR, PFS, OS; in some programs MRD or response-adapted endpoints are used, but gemcitabine has long-standing historical OS-driven assessment in pancreatic, biliary tract, and non-small cell lung cancer

Practical read-through for investors

• Near-term incremental upside comes from trials designed to win specific subpopulations: biomarker-defined cohorts, prior-treatment stratification, and regimen selection.
• Regulatory risk remains driven by the evidentiary standard for comparative superiority versus gemcitabine-based reference regimens, especially where OS is difficult to move.

Which clinical areas drive gemcitabine’s ongoing trial demand?

The bulk of current trial enrollment and sponsor attention clusters in tumor domains where gemcitabine is already a backbone or a comparator.

Key tumor categories with active development

• Pancreatic cancer
• Biliary tract cancers
• Non-small cell lung cancer (NSCLC)
• Ovarian cancer and other gynecologic malignancies in limited pockets of late-stage combination research
• Urothelial and other solid tumors where gemcitabine is used as a chemotherapy backbone in combination settings

Common combination partners seen across trial programs

• Immune checkpoint inhibitors (PD-1/PD-L1 axis)
• Anti-angiogenic agents (VEGF pathway)
• Targeted agents tied to EGFR/HER2 or DNA damage response logic
• Radiotherapy and radiosensitizing schedules in regionally advanced disease

What does gemcitabine’s market look like today?

Gemcitabine is a high-volume, mature oncology product with broad global availability and competitive generic penetration in many markets. Revenue performance is driven by:

1. Usage durability in pancreatic and biliary tract regimens
2. Indexing to oncology spending rather than new molecular entity dynamics
3. Patent and exclusivity structure across geographies (originator and multiple generics)
4. Formulation and access mechanisms (tenders, hospital formularies, reimbursement)

Market segmentation that matters for projections

• By geography: US, EU5, UK, Japan, rest of world
• By oncology indication mix: pancreatic, biliary, NSCLC dominate
• By product form: single-agent gemcitabine vials plus combination products (where marketed in specific territories)

Competitive structure

• Originator competition vs. generics: gemcitabine faces sustained price competition where patents and exclusivity have expired.
• Therapy competition: even in off-patent settings, clinicians select regimens based on local guideline adoption and tolerability, which can shift demand between gemcitabine-based and non-gemcitabine regimens.

What market projections are realistic for gemcitabine hydrochloride?

A defensible projection for a mature oncology cytotoxic depends on two forces:

• Unit demand stability (patients remain treated using gemcitabine backbone regimens)
• Unit price erosion (ongoing generic pricing pressure)
• Policy and access (tender systems can accelerate price compression or sustain volume for cost-effective suppliers)

Projection framework (base case)

• Global growth: low single-digit CAGR or flat-to-low positive growth in revenue terms in the absence of new major label expansions.
• Volume: relatively stable with possible modest growth in some regions driven by incidence trends and guideline adherence.
• Price: continued downward pressure in many markets due to generic competition.

Drivers that can move the base case

• Positive driver: successful trials that translate into stronger guideline preference for gemcitabine-based combination standards in defined subgroups
• Negative driver: regimen substitution by non-gemcitabine chemotherapy or targeted/immune strategies that reduce reliance on gemcitabine backbone therapy

Time horizons

• 2024–2026: price pressure remains the dominant factor; clinical updates can affect prescribing patterns but rarely reverse structural pricing dynamics for off-patent brands.
• 2027–2030: growth becomes mostly access- and guideline-dependent, with incremental clinical wins translating into modest mix shifts rather than major market expansion.

How do clinical trial outcomes map to commercial impact?

The translation path from trial results to commercial demand follows a predictable chain:

1. Guideline adoption
2. Hospital formulary inclusion
3. Oncologist regimen preference
4. Tender and contracting outcomes
5. Net price negotiation and reimbursement consistency

Commercially meaningful trial “wins”

• OS benefit or clear clinically meaningful PFS benefit over standard gemcitabine-based comparators
• Tolerability advantage that changes sequencing or reduces discontinuation
• Biomarker-defined response enabling targeted reimbursement and narrower clinician decision trees
• Regimen simplicity for pharmacy workflow and administration

Why many trials do not move markets

• In off-patent settings, even improved efficacy may not produce proportional revenue lift because the market clears on cost, not branded differentiation.
• Trial wins can still drive volume retention and protect market share against competing regimens, even when revenue growth is limited.

What are the key risks to market growth?

• Price compression from generic oversupply
• Regimen substitution where immuno-oncology or targeted therapy schedules reduce gemcitabine usage
• Failure to secure guideline adoption for new combination sequences
• Manufacturing and supply risk affecting tender competitiveness

Key Takeaways

• Gemcitabine is in a mature, off-patent phase where trial value concentrates on combination sequencing, biomarker stratification, and regimen standard-of-care reinforcement rather than major label changes.
• Market performance is shaped mainly by volume stability and ongoing price erosion driven by generic competition and procurement systems.
• Projections for 2024–2030 are most realistic as flat-to-low growth in revenue with modest mix shifts if combination data supports guideline uptake.
• Commercially meaningful clinical wins typically require OS or robust PFS gains with tolerability advantages that translate into formulary and tender adoption.

FAQs

1) Does gemcitabine still attract new clinical trial enrollment?

Yes, trial activity persists, largely in combination regimens and treatment sequencing across pancreatic, biliary tract, and NSCLC settings.

2) What endpoints matter most commercially for gemcitabine-based combinations?

OS and clinically meaningful PFS improvements versus gemcitabine-based standards, plus tolerability or operational advantages that change prescribing behavior.

3) Why might trial efficacy not lift revenue proportionally?

Most markets price gemcitabine through procurement and generic competition, so efficacy gains often shift practice patterns more than price levels.

4) What tumor indications carry the most market weight?

Pancreatic cancer and biliary tract cancers drive much of the durable demand; NSCLC is another recurring backbone area.

5) What is the dominant market risk over the next 2–3 years?

Continued price compression from generics and contracting dynamics, which can offset modest volume gains.

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References (APA)

[1] National Cancer Institute. (n.d.). Gemcitabine. NCI Drug Dictionary. https://www.cancer.gov/about-cancer/treatment/drugs/gemcitabine
[2] Drugs@FDA. (n.d.). Gemcitabine hydrochloride (FDA product information and labeling records). U.S. Food and Drug Administration. https://www.accessdata.fda.gov/scripts/cder/daf/
[3] U.S. National Library of Medicine. (n.d.). ClinicalTrials.gov (Search results for gemcitabine combinations and solid tumor trials). https://clinicaltrials.gov/