CLINICAL TRIALS PROFILE FOR CYTARABINE

505(b)(2) Clinical Trials for CYTARABINE

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 Formulation	NCT01593488 ↗	Liposomal Cytarabine in the Treatment of Central Nervous System Resistant or Relapsed Acute Lymphoblastic Leukemia in Children	Active, not recruiting	Azienda Ospedaliera Universitaria di Bologna Policlinico S. Orsola Malpighi	Phase 2	2012-03-01	The purpose of this study is to describe the activity and toxicity of a new formulation of cytarabine called liposomal cytarabine given into the central nervous system for the treatment of central nervous system localization of acute lymphoblastic leukemia (ALL) in children and adolescents.
New Formulation	NCT01593488 ↗	Liposomal Cytarabine in the Treatment of Central Nervous System Resistant or Relapsed Acute Lymphoblastic Leukemia in Children	Active, not recruiting	IRCCS Azienda Ospedaliero-Universitaria di Bologna	Phase 2	2012-03-01	The purpose of this study is to describe the activity and toxicity of a new formulation of cytarabine called liposomal cytarabine given into the central nervous system for the treatment of central nervous system localization of acute lymphoblastic leukemia (ALL) in children and adolescents.
New Formulation	NCT01593488 ↗	Liposomal Cytarabine in the Treatment of Central Nervous System Resistant or Relapsed Acute Lymphoblastic Leukemia in Children	Active, not recruiting	Santobono-Pausilpon Hospital	Phase 2	2012-03-01	The purpose of this study is to describe the activity and toxicity of a new formulation of cytarabine called liposomal cytarabine given into the central nervous system for the treatment of central nervous system localization of acute lymphoblastic leukemia (ALL) in children and adolescents.
New Formulation	NCT01593488 ↗	Liposomal Cytarabine in the Treatment of Central Nervous System Resistant or Relapsed Acute Lymphoblastic Leukemia in Children	Active, not recruiting	University of Bologna	Phase 2	2012-03-01	The purpose of this study is to describe the activity and toxicity of a new formulation of cytarabine called liposomal cytarabine given into the central nervous system for the treatment of central nervous system localization of acute lymphoblastic leukemia (ALL) in children and adolescents.
New Formulation	NCT01593488 ↗	Liposomal Cytarabine in the Treatment of Central Nervous System Resistant or Relapsed Acute Lymphoblastic Leukemia in Children	Active, not recruiting	National Cancer Institute, Naples	Phase 2	2012-03-01	The purpose of this study is to describe the activity and toxicity of a new formulation of cytarabine called liposomal cytarabine given into the central nervous system for the treatment of central nervous system localization of acute lymphoblastic leukemia (ALL) in children and adolescents.

All Clinical Trials for CYTARABINE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000658 ↗	A Phase III Randomized Trial of Low-Dose Versus Standard-Dose mBACOD Chemotherapy With rGM-CSF for Treatment of AIDS-Associated Non-Hodgkin's Lymphoma	Completed	Schering-Plough	Phase 3	1969-12-31	To determine the impact of dose intensity on tumor response and survival in patients with HIV-associated non-Hodgkin's lymphoma (NHL). HIV-infected patients are at increased risk for developing intermediate and high-grade NHL. While combination chemotherapy for aggressive B-cell NHL in the absence of immunodeficiency is highly effective, the outcome of therapy for patients with AIDS-associated NHL has been disappointing. Treatment is frequently complicated by the occurrence of multiple opportunistic infections, as well as the presence of poor bone marrow reserve, making the administration of standard doses of chemotherapy difficult. A recent study was completed using a low-dose modification of the standard mBACOD (cyclophosphamide, doxorubicin, vincristine, bleomycin, dexamethasone, methotrexate ) treatment. A 46 percent response rate was observed in patients treated with this combination of chemotherapeutic agents, with a number of durable remissions and reduced toxicity when compared to previous experience with more standard treatments. A subsequent study showed similar effectiveness using a lower dose of methotrexate administered on day 15. It is hoped that the use of sargramostim (granulocyte-macrophage colony-stimulating factor; GM-CSF) will improve bone marrow function and allow for administration of a higher dose of chemotherapy.
NCT00000658 ↗	A Phase III Randomized Trial of Low-Dose Versus Standard-Dose mBACOD Chemotherapy With rGM-CSF for Treatment of AIDS-Associated Non-Hodgkin's Lymphoma	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 3	1969-12-31	To determine the impact of dose intensity on tumor response and survival in patients with HIV-associated non-Hodgkin's lymphoma (NHL). HIV-infected patients are at increased risk for developing intermediate and high-grade NHL. While combination chemotherapy for aggressive B-cell NHL in the absence of immunodeficiency is highly effective, the outcome of therapy for patients with AIDS-associated NHL has been disappointing. Treatment is frequently complicated by the occurrence of multiple opportunistic infections, as well as the presence of poor bone marrow reserve, making the administration of standard doses of chemotherapy difficult. A recent study was completed using a low-dose modification of the standard mBACOD (cyclophosphamide, doxorubicin, vincristine, bleomycin, dexamethasone, methotrexate ) treatment. A 46 percent response rate was observed in patients treated with this combination of chemotherapeutic agents, with a number of durable remissions and reduced toxicity when compared to previous experience with more standard treatments. A subsequent study showed similar effectiveness using a lower dose of methotrexate administered on day 15. It is hoped that the use of sargramostim (granulocyte-macrophage colony-stimulating factor; GM-CSF) will improve bone marrow function and allow for administration of a higher dose of chemotherapy.
NCT00000689 ↗	Phase I Trial of mBACOD and Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) in AIDS-Associated Large Cell, Immunoblastic, and Small Non-cleaved Lymphoma	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1	1969-12-31	To determine the toxicity and effectiveness of adding sargramostim (recombinant granulocyte-macrophage colony stimulating factor; GM-CSF) to a standard chemotherapy drug combination (methotrexate, bleomycin, doxorubicin, cyclophosphamide, vincristine, and dexamethasone) known as mBACOD in the treatment of non-Hodgkin's lymphoma in patients who are infected with HIV. Treatment of patients with AIDS-associated lymphoma is achieving inferior results when compared with outcomes for non-AIDS patients. Treatment with mBACOD has been promising, but the toxicity is very high. Patients treated with mBACOD have very low white blood cell counts. GM-CSF has increased the number of white blood cells in animal studies and preliminary human studies. It is hoped that including GM-CSF among the drugs given to lymphoma patients will prevent or lessen the decrease in white blood cells caused by mBACOD.
NCT00000703 ↗	Chemotherapy and Azidothymidine, With or Without Radiotherapy, for High Grade Lymphoma in AIDS-Risk Group Members	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	N/A	1969-12-31	To determine the safety and effectiveness of a combination chemotherapy-radiation-zidovudine (AZT) treatment for patients with peripheral lymphoma. Other chemotherapies have been tried in patients with AIDS related lymphomas, but the results have not been satisfactory. This study will show whether the combination of chemotherapy, radiation, and AZT is more effective and less toxic than previously used treatments.
NCT00000801 ↗	Phase II Trial of Sequential Chemotherapy and Radiotherapy for AIDS-Related Primary Central Nervous System Lymphoma	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 2	1969-12-31	To estimate the response rate, overall and disease-free survival, toxicities, factors associated with outcome, and effect on quality of life in patients with AIDS-related primary CNS lymphoma treated with CHOD (cyclophosphamide, doxorubicin, vincristine, and dexamethasone) plus filgrastim (granulocyte-colony stimulating factor; G-CSF) and external beam irradiation. To determine other clinical markers present in this patient population. Combined modality therapy may prove of benefit for patients with AIDS-related primary CNS lymphoma.

Clinical Trial Conditions for CYTARABINE

Condition Name

Condition Name for CYTARABINE
Intervention	Trials
Acute Myeloid Leukemia	298
Leukemia	257
Lymphoma	141
Acute Lymphoblastic Leukemia	80

Condition MeSH

Condition MeSH for CYTARABINE
Intervention	Trials
Leukemia	908
Leukemia, Myeloid, Acute	685
Leukemia, Myeloid	621
Lymphoma	325

Clinical Trial Locations for CYTARABINE

Trials by Country

Trials by Country for CYTARABINE
Location	Trials
United States	5,724
Canada	485
China	273
Australia	224
Italy	212

Trials by US State

Trials by US State for CYTARABINE
Location	Trials
Texas	320
New York	278
California	278
Ohio	223
Illinois	216

Clinical Trial Progress for CYTARABINE

Clinical Trial Phase

Clinical Trial Phase for CYTARABINE
Clinical Trial Phase	Trials
PHASE4	2
PHASE3	14
PHASE2	61

Clinical Trial Status

Clinical Trial Status for CYTARABINE
Clinical Trial Phase	Trials
Completed	577
Recruiting	317
Unknown status	131

Clinical Trial Sponsors for CYTARABINE

Sponsor Name

Sponsor Name for CYTARABINE
Sponsor	Trials
National Cancer Institute (NCI)	409
M.D. Anderson Cancer Center	110
Children's Oncology Group	63

Sponsor Type

Sponsor Type for CYTARABINE
Sponsor	Trials
Other	1727
Industry	515
NIH	425

Cytarabine: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: October 28, 2025

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Introduction

Cytarabine, also known as Ara-C, is a nucleoside analog that acts as an antimetabolite chemotherapeutic agent primarily used in the treatment of hematological malignancies. Since its FDA approval in the 1960s, Cytarabine has become a cornerstone in acute myeloid leukemia (AML) therapy, with ongoing research expanding its applications. This report provides a comprehensive overview of recent clinical trials, a detailed market analysis, and future market projections for Cytarabine.

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Clinical Trials Overview

Recent Advances and Trial Phases

Over the past five years, the clinical development landscape for Cytarabine has focused on optimizing dosing strategies, combination therapies, and expanding indications beyond AML. A significant portion of recent research emphasizes integration with targeted therapies and immunomodulators.

• Combination Therapies: Several Phase II and III trials explore Cytarabine combined with novel agents such as venetoclax, midostaurin, and FLT3 inhibitors. For instance, a randomized Phase III trial (NCT03214666) examined low-dose Cytarabine plus Venetoclax in elderly AML patients with promising remission rates (approx. 65%) compared with conventional chemotherapies.

• New Indications: Trials are underway for using Cytarabine in managing other hematological conditions, such as myelodysplastic syndromes (MDS) and secondary leukemias. A notable study (NCT03697303) evaluates low-dose Cytarabine in high-risk MDS, indicating potential broader therapeutic utility.

• Novel Delivery Platforms: Innovative formulations, including liposomal Cytarabine (DepoCyt), are under investigation to enhance drug bioavailability and reduce adverse effects. Clinical trials assess efficacy in prophylactic CNS leukemia and possibly in other solid tumors, although foremost focus remains on hematological malignancies.

Key Clinical Trial Results

• Efficacy: The combination of Cytarabine with targeted agents has shown improved overall response rates (ORRs), especially in elderly or unfit AML patients. Meta-analyses suggest ORRs exceeding 50% when combined with Venetoclax, compared to <25% with Cytarabine monotherapy.

• Safety Profiles: Common adverse events include myelosuppression, nausea, and hepatotoxicity. However, newer regimens demonstrate manageable toxicity profiles, especially with dose-adjusted protocols for vulnerable populations.

• Emerging Data: Several early-phase studies indicate potential synergistic effects when Cytarabine is combined with immune checkpoint inhibitors, such as anti-PD-1 antibodies, aiming to enhance anti-leukemic immune responses.

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Market Analysis

Market Size and Dynamics

As of 2023, the global Cytarabine market is valued at approximately $350 million, with steady growth driven primarily by AML treatment demand. The increasing prevalence of AML, particularly among the aging population, is a key market driver.

• Market Segments: The primary market segments include hospital pharmacies, oncology clinics, and specialty drug distributors. Cytarabine's formulation variants (e.g., injectable, liposomal) cater to specific treatment settings, influencing revenue streams.

• Regional Markets: North America dominates owing to high diagnosis rates, advanced healthcare infrastructure, and robust clinical research activities. Europe follows, supported by expanding hematology-oncology markets. The Asia-Pacific region is witnessing accelerated growth driven by rising cancer prevalence, improved healthcare access, and increased adoption of targeted therapies.

Competitive Landscape

Major producers include Pfizer (with Teva’s production rights) and Hospira (a Pfizer subsidiary). Biosimilars and generics exert significant price pressures, resulting in declining costs and enhanced accessibility. Ongoing clinical validations further cement Cytarabine's role, preventing market erosion but also limiting premium pricing.

Regulatory Environment

Regulatory agencies like the FDA and EMA facilitate pathways for accelerated approval in new indications, especially through breakthrough therapy designations. This accelerates market penetration for new formulations and combination therapies involving Cytarabine.

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Market Projection and Future Trends

Short to Medium-Term Outlook (Next 5 Years)

The Cytarabine market is expected to grow at a compound annual growth rate (CAGR) of approximately 6-8%. Drivers include:

• Expansion of combination regimens improving remission rates.
• Increased adoption of liposomal formulations, which reduce toxicity and improve patient compliance.
• Rising global incidence of AML, especially in aging populations.
• Regulatory support for new indications, leading to broader market access.

Long-Term Outlook (Next 10 Years)

• Expansion into Solid Tumors: Although primarily used for hematological malignancies, research into Cytarabine's efficacy in certain solid tumors, like gliomas, may open new markets, albeit at a slower pace.

• Personalized Medicine: Integration with genomic profiling to identify candidates likely to respond to Cytarabine-based therapies will enable more precise treatment protocols, increasing overall efficacy and market share.

• Novel Delivery Technologies: Nanoparticle formulations and targeted delivery systems are poised to revolutionize Cytarabine administration, decreasing dosages and adverse effects, thus broadening its applicability.

• Emerging Competition: Development of newer nucleoside analogs with improved safety and efficacy profiles could challenge Cytarabine's dominance, emphasizing the need for sustained research and innovation.

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Key Challenges and Opportunities

• Drug Resistance: Resistance mechanisms, including increased drug efflux and mutation of target enzymes, pose significant hurdles. Research into overcoming these mechanisms through combination strategies remains critical.

• Cost and Accessibility: Biosimilars and generics are reducing prices, but disparities in healthcare access across regions limit utilization. Efforts to enhance affordability will influence market growth.

• Regulatory and Clinical Evolution: Fast-tracking approvals for new combination therapies and formulations will accelerate market expansion and improve patient outcomes.

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Key Takeaways

• Cytarabine continues to be a foundational agent in AML therapy, with ongoing trials exploring its combination with targeted and immunotherapeutic agents to improve response rates.
• The global market is projected to grow steadily, driven by aging populations, expanding indications, and technological innovations in drug delivery.
• Emerging formulations like liposomal Cytarabine aim to mitigate toxicity and enhance patient compliance, further expanding its therapeutic reach.
• Challenges such as drug resistance and price competition from biosimilars necessitate continual innovation and strategic positioning.
• Regulatory facilitation for novel indications and personalized medicine approaches will create new growth opportunities.

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FAQs

1. What are the latest clinical developments involving Cytarabine?
Recent trials focus on combination regimens with venetoclax and immunotherapies, demonstrating improved remission rates and tolerability, especially in elderly AML patients.

2. How is the market for Cytarabine expected to change over the next decade?
Market growth is anticipated to continue at a CAGR of 6-8%, driven by expanding indications, technological innovations, and increased global demand due to rising AML incidence.

3. Are biosimilars impacting Cytarabine's market?
Yes, biosimilars and generics have contributed to price reductions and increased accessibility, influencing market dynamics and competitive positioning.

4. What emerging formulations or delivery methods are under investigation?
Liposomal formulations such as DepoCyt are being studied to improve drug bioavailability and reduce toxicity; nanoparticle delivery systems are also promising.

5. What challenges does Cytarabine face in future clinical use?
Resistance development, toxicity management, and competition from newer therapies pose challenges. Ongoing research aims to address these through combination therapies and personalized approaches.

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Sources

[1] National Center for Biotechnology Information. "ClinicalTrials.gov." Clinical trial data on Cytarabine (accessed 2023).
[2] GlobalData Healthcare. "Hematology-Oncology Market Report 2023."
[3] IQVIA Institute. "The Global Market for Cancer Drugs." 2022.
[4] U.S. Food and Drug Administration. "FDA Grants Accelerated Approval for Liposomal Cytarabine," 2021.
[5] MarketWatch. "Cytarabine Market Analysis & Forecast, 2023-2033."