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.
New Formulation	NCT04992949 ↗	Evaluation of CPX-351 Monotherapy in Acute Myeloid Leukemia Secondary to Myeloproliferative Neoplasm	Not yet recruiting	Acute Leukemia French Association	Phase 2	2021-10-01	The three classic myeloproliferative neoplasms (MPNs) include polycythemia Vera (PV), essential thrombocythemia (ET) and primary myelofibrosis (PMF). The natural history of these MPNs is the possible progression to acute myeloid leukemia (MPN-blast phase) at variable percentage depending the entity. Leukemic transformation of MPN occurs in 8% to 23% of primary myelofibrosis (PMF) patients in the first 10 years after diagnosis and in 4% to 8% of polycythemia vera (PV) and essential thrombocytosis (ET) patients within 18 years after diagnosis. The risk for leukemic transformation is increased by exposure to cytotoxic chemotherapy. The molecular pathogenesis of MPN-blast phase remains an area of active research. The prognosis of blast phase MPNs is very poor : approximately 50% of the patients are deemed eligible for intensive treatment (ie. conventional induction chemotherapy regimen with anthracyclines and cytarabine). The patients who are not fit for such intensive treatment approach due to age or comorbidities, are treated with Hypomethylating agents, low dose palliative chemotherapy, or supportive care. Nevertheless, there is a need for more effective and better tolerated treatment approaches in order to increase the response rate and hence, the transplant rates which should translate into improved survival. CPX-351 is a new formulation of cytarabine and daunorubicin encapsulated at a fixed 5:1 molar-ratio in liposomes that exploits molar ratio-dependent drug-drug synergy to enhance antileukemic efficacy. Based on similarities between post-myelodysplastic syndrome (MDS) and post-MPN secondary AML in terms of disease resistance to chemotherapy, of fragile patient profile, The hypotheses made is that CPX-351 may improve the results of induction chemotherapy without increasing its toxicity and therefore may increase the proportion of patients who could benefit from an allogeneic Stem Cell Transplantation (SCT).

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.
NCT00001048 ↗	Comparison of Anti HIV Drugs Used Alone or in Combination With Cytosine Arabinoside to Treat Progressive Multifocal Leukoencephalopathy (PML) in HIV-Infected Patients	Completed	Bristol-Myers Squibb	Phase 2	1969-12-31	To compare the safety and efficacy of antiretroviral therapy (zidovudine plus either didanosine or dideoxycytidine) versus antiretroviral therapy plus intravenous cytarabine (Ara-C) versus antiretroviral therapy plus intrathecal Ara-C in the maintenance or improvement of neurological function over 6 months in HIV-infected individuals who have developed progressive multifocal leukoencephalopathy (PML). To compare the effect of these three treatment regimens on Karnofsky score and MRI studies. The effectiveness of Ara-C in the treatment of PML, caused by a human DNA papovavirus (designated JC virus) infection, has not been determined, although the most encouraging results have occurred with intrathecal administration of the drug.

Clinical Trial Conditions for cytarabine

Condition Name

Condition Name for cytarabine
Intervention	Trials
Acute Myeloid Leukemia	300
Leukemia	258
Lymphoma	142
Acute Lymphoblastic Leukemia	81

Condition MeSH

Condition MeSH for cytarabine
Intervention	Trials
Leukemia	909
Leukemia, Myeloid, Acute	689
Leukemia, Myeloid	621
Lymphoma	326

Clinical Trial Locations for cytarabine

Trials by Country

Trials by Country for cytarabine
Location	Trials
United States	5,725
Canada	485
China	276
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	217

Clinical Trial Progress for cytarabine

Clinical Trial Phase

Clinical Trial Phase for cytarabine
Clinical Trial Phase	Trials
PHASE4	2
PHASE3	15
PHASE2	64

Clinical Trial Status

Clinical Trial Status for cytarabine
Clinical Trial Phase	Trials
Completed	577
Recruiting	319
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	1732
Industry	517
NIH	425

Last updated: May 20, 2026

Clinical Trials Update, Market Analysis, and Pricing/Exclusivity Outlook for Cytarabine

Cytarabine (Ara-C) is a cornerstone cytotoxic chemotherapy for acute myeloid leukemia (AML) and other hematologic malignancies. In the US, it is off-patent with long-running generic availability; the market is dominated by low-cost sterile injectables and intermittent supply constraints. The clinical pipeline is still active, but growth is driven mainly by formulation, scheduling, combination regimens, and survivorship in specific settings rather than new first-in-class molecular IP.

Key near-term market implications

• Pricing pressure and commoditization are persistent because cytarabine has broad generic substitution.
• Value migration is occurring toward higher-complexity delivery and regimen optimization (e.g., liposomal cytarabine in some use cases), but this does not eliminate the pricing drag from generic sterile products.
• Clinical trial activity is skewed toward niche populations and regimen combinations rather than monotherapy breakthroughs.

What is the current clinical trials landscape for cytarabine in AML and hematologic cancers?

The cytarabine clinical-trials landscape is dominated by:

• Combination studies in AML (often with venetoclax, FLT3 inhibitors, IDH inhibitors, hypomethylating agents, and targeted antibodies)
• Salvage/reinduction and older-fitness cohorts
• Post-remission and consolidation regimen optimization
• Comparisons between cytarabine schedules (high-dose vs intermediate-dose, continuous infusion vs intermittent dosing)

Which cytarabine regimens are most studied now

High-intensity schedules remain the focus, but trial design is moving toward:

• Lower-intensity or modified dosing strategies for older patients and comorbidity-limited populations
• Transplant-adjacent strategies that attempt to improve response depth and molecular clearance
• Reduced-toxification approaches using supportive-care optimization and modified infusion schedules

Where do trials tend to show differentiation

Differentiation is largely not from the active ingredient, but from:

• Dosing schedule and duration
• Combination partner selection
• Patient selection (cytogenetic/molecular risk strata)
• Adjunct supportive-care pathways and infection prophylaxis strategies
• Formulation and delivery (including liposomal cytarabine in relevant indications)

How does the cytarabine clinical pipeline differ by formulation: liposomal cytarabine vs conventional Ara-C?

Cytarabine exists in multiple commercially relevant presentations. Trial activity maps to the formulation because tolerability and exposure profiles differ.

Conventional cytarabine (Ara-C) trials

Conventional Ara-C supports broad use in AML induction, consolidation, salvage, and combination regimens. Trials with conventional cytarabine aim to:

• Identify optimal pairing partners for response rates and minimal residual disease (MRD)
• Improve outcomes in patients who are not candidates for the most intensive platforms

Liposomal cytarabine trials

Liposomal cytarabine is used for specific practice patterns and aims to:

• Improve cytarabine exposure and dosing convenience
• Potentially reduce peak-related toxicities while maintaining anti-leukemic activity
• Establish competitive positioning in relapse settings and in consolidation approaches depending on protocol and local standards

What is the latest FDA and regulatory status for cytarabine products in the US?

Cytarabine is approved for leukemia treatment. The practical regulatory picture is that:

• Multiple generic approvals sustain a stable baseline supply for conventional injectable cytarabine.
• Product-level regulatory relevance exists primarily for specific formulations, strengths, labeling expansions, and manufacturing changes rather than for new active-ingredient approvals.

What FDA labeling changes typically drive cytarabine utilization

Real-world utilization shifts tend to follow:

• Labeling updates that broaden indicated settings
• Post-approval safety or administration guidance updates
• Changes tied to manufacturing comparability or sterility assurance

What is the Orange Book status of cytarabine and how does it affect generic entry risk?

For a widely used cytotoxic active ingredient like cytarabine:

• The generic entry risk is low because active-ingredient exclusivity has long expired.
• Competitive risk is dominated by formulation-specific patents or manufacturing-process protections, which can delay individual products rather than the entire molecule.

What matters for market access and tendering

Market access is shaped by:

• WAC/net pricing dynamics and group purchasing organization (GPO) contracts
• Availability and sterility supply reliability
• Substitution rules and pharmacy benefit designs

Who are the key competitors in the cytarabine market and how do their products compare?

The cytarabine market is structurally commoditized. Competitive differences are usually:

• Presentation form and administration convenience (standard vs liposomal)
• Strengths and packaging that match protocols
• Supply continuity and quality record

Competitive set

• Generic sterile injectable cytarabine manufacturers dominate conventional cytarabine.
• Manufacturers of liposomal cytarabine occupy a narrower but higher-complexity segment.
• Brand competition is limited because cytarabine itself is no longer protected by meaningful active-ingredient exclusivity.

How big is the cytarabine market today and what drives revenue beyond volume?

Revenue drivers for cytarabine include:

• Volume tied to AML incidence and treatment protocols that use Ara-C as core therapy
• Intensity of treatment regimens (induction and consolidation cycles)
• Price per dose net of rebates under hospital contracting
• Shift between conventional and liposomal cytarabine based on local protocol and tolerability needs
• Supply events that temporarily raise market-clearing prices

Revenue model

Cytarabine revenue can be modeled as:

• Treated patient volume × doses per protocol × blended net price
  Where “doses per protocol” varies by schedule and consolidation approach.

When does cytarabine lose exclusivity and what does that imply for pricing?

Because cytarabine is an older active ingredient, the exclusivity landscape is no longer a single molecule-driven story. The pricing implications are:

• Sustained downward pressure on net prices for conventional sterile injectables
• Competition that is primarily supply and manufacturing driven
• Episodic price dislocations when certain SKUs face supply constraints

What market projections exist for cytarabine through 2029 and what scenario best fits a hospital budget outlook?

Given commoditization:

• Base-case growth typically tracks AML treatment volumes with modest price erosion.
• Upside case assumes regimen changes increase standardized cytarabine exposure and liposomal cytarabine adoption in relevant settings.
• Downside case assumes substitution and aggressive hospital formulary management keep net prices falling faster than volume growth.

Projected growth is more likely to be incremental and regional than a step-function change.

How do combination therapies (venetoclax, targeted agents, HMAs) influence cytarabine demand?

Cytarabine demand is sensitive to regimen architecture:

• When cytarabine is part of front-line or consolidation protocols, it remains a durable component.
• When regimens shift toward non-Ara-C platforms in certain patient groups, cytarabine exposure can decline.
• Combination partner selection drives which dosing platform is used, which affects whether clinicians choose conventional Ara-C or liposomal cytarabine.

MRD and response depth goals

Modern trials aim for deeper responses and MRD negativity. This affects cytarabine demand when:

• Protocols intensify cytarabine exposure to deepen response
• Protocols reduce cytarabine exposure due to synergy with targeted agents

What are the largest clinical and commercial risks for cytarabine supply and utilization?

Supply risk

• Sterile injectable supply is vulnerable to manufacturing disruptions.
• Even when multiple generics exist, constraints at particular plants or bottlenecks in sterile fill-finish can reduce availability.

Clinical risk

• Toxicity management and infection risk are central in AML induction and reinduction.
• Protocol-level changes can reduce cytarabine use if toxicity profiles are unacceptable for specific subsets.

How does cytarabine compare with alternative AML backbones (azacitidine, decitabine, cladribine, venetoclax-based regimens)?

Cytarabine remains a core cytotoxic backbone, but:

• Hypomethylating agents and venetoclax-based regimens can reduce the reliance on high-intensity cytarabine in older or frail patients.
• Cytarabine retains strength in fit-patient induction/consolidation strategies where intensive cytotoxic therapy is tolerated.

Net effect:

• Cytarabine demand is steady but not growing fast, with share influenced by patient fitness and platform preference.

Key takeaways

• Cytarabine’s clinical development is ongoing but is driven mainly by combinations, schedules, and formulation improvements rather than active-ingredient innovation.
• The market remains commoditized; generic availability is the baseline and pricing is dominated by contracting and supply reliability.
• Commercial upside is tied to protocol adherence and targeted uptake of higher-complexity formulations (where available) more than to new molecular exclusivity.
• Key business sensitivities are hospital formulary dynamics, sterile supply continuity, and AML regimen shifts by patient fitness and molecular risk.

FAQs

1) What cytarabine-based regimens are being tested in newly diagnosed AML?
Combination approaches with targeted agents and modern backbones, with ongoing evaluation of induction and consolidation scheduling.

2) Does liposomal cytarabine have a different clinical role than conventional Ara-C?
Yes. It is used where formulation exposure and administration profile fit specific protocol goals and tolerability targets.

3) What drives net price changes for cytarabine in US hospitals?
GPO contracting, competitive generic pricing, rebate structures, and episodic supply constraints at specific SKUs.

4) What supply factors most often affect cytarabine availability?
Sterile manufacturing capacity, fill-finish throughput, and site-specific quality or disruption events.

5) How are MRD endpoints shaping cytarabine clinical trial design?
Trials increasingly select dosing and scheduling strategies intended to deepen response and improve MRD-negativity rates.

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References (APA)

[No sources were provided in the prompt, and no verified, citable dataset was included to support a clinical-trials update, market sizing, or forecast figures for cytarabine.]