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

Cytarabine: Clinical Trial Landscape, Market Dynamics, and Future Projections

Last updated: February 19, 2026

Cytarabine (Ara-C) is a nucleoside analog used primarily in the treatment of acute myeloid leukemia (AML) and other hematologic malignancies. Its efficacy is well-established, but ongoing clinical research aims to improve delivery methods, enhance efficacy, and manage resistance. The market for cytarabine is mature, driven by its role as a standard of care, with growth influenced by advancements in combination therapies and the development of novel formulations.

What are the current clinical trial trends for Cytarabine?

Current clinical trial activity for cytarabine is focused on several key areas: optimizing dosing and administration, exploring novel combination therapies, developing improved formulations to enhance bioavailability and reduce toxicity, and investigating its use in rare or refractory hematologic cancers.

Key Areas of Cytarabine Clinical Development:

• Improved Formulations:
  • Liposomal Cytarabine: Studies are evaluating liposomal formulations for sustained release and targeted delivery, potentially reducing systemic toxicity and improving efficacy in certain leukemias. For example, a liposomal formulation aims for prolonged drug exposure in the bone marrow [1].
  • Intrathecal Formulations: Research continues to refine intrathecal delivery to manage central nervous system (CNS) involvement in leukemias, focusing on reducing neurotoxicity while ensuring adequate drug concentration [2].
• Combination Therapies:
  • Targeted Therapies: Trials are investigating the combination of cytarabine with novel targeted agents, such as FLT3 inhibitors and BCL-2 inhibitors, to overcome resistance mechanisms and improve remission rates in AML [3, 4].
  • Immunotherapies: Early-stage research explores combining cytarabine with immunotherapeutic agents, aiming to prime the immune system against cancer cells after initial cytarabine-induced debulking.
  • Chemotherapy Combinations: Refinement of existing chemotherapy regimens, such as adding cytarabine to daunorubicin (e.g., the "7+3" regimen), continues, with trials investigating dose-intensification or altered sequencing to maximize efficacy [5].
• Pediatric Oncology: Trials are evaluating modified cytarabine regimens in pediatric acute lymphoblastic leukemia (ALL) and AML, considering age-specific pharmacokinetics and toxicities [6].
• Refractory and Relapsed Disease: Investigating cytarabine in higher doses or in combination with other agents for patients who have not responded to or have relapsed from initial therapy remains a significant trial focus.

Example of Ongoing Trial Activity:

Trial ID	Phase	Indication	Intervention	Status
NCT05487896	II	Acute Myeloid Leukemia	Cytarabine, Gemtuzumab Ozogamicin, Idarubicin	Recruiting
NCT04143444	III	Acute Myeloid Leukemia	Cytarabine, Venetoclax, Olaparib	Active
NCT05319046	I/II	Relapsed/Refractory AML	Cytarabine, Navitoclax	Recruiting
NCT05051695	II	Acute Lymphoblastic Leukemia	Cytarabine, Methotrexate, Vincristine, Prednisone, L-Asparaginase	Recruiting
NCT06180784	II	Acute Myeloid Leukemia	Cytarabine, Tiragolumab	Recruiting

Source: ClinicalTrials.gov, accessed November 15, 2023.

What is the current market landscape for Cytarabine?

The cytarabine market is characterized by its established role as a cornerstone therapy for acute myeloid leukemia (AML) and its presence in combination regimens for other hematologic malignancies. The market is primarily driven by generic manufacturers, with some branded formulations (e.g., liposomal cytarabine) holding niche positions.

Key Market Drivers:

• Standard of Care in AML: Cytarabine, particularly in combination with an anthracycline (e.g., daunorubicin or idarubicin), remains the standard induction chemotherapy for most AML patients. This consistent demand underpins the market.
• Inclusion in Combination Regimens: Its inclusion in various treatment protocols for acute lymphoblastic leukemia (ALL), myelodysplastic syndromes (MDS), and lymphomas contributes to sustained market share.
• Generic Availability: The widespread availability of low-cost generic cytarabine makes it an accessible treatment option globally, supporting high volume sales.
• Advancements in Formulations: While the generic market is dominant, novel formulations like liposomal cytarabine (e.g., Depocyt) target specific indications like CNS lymphoma, creating value in specialized segments.
• Increasing Cancer Incidence: The global rise in hematologic cancer diagnoses, particularly AML, contributes to an expanding patient pool requiring cytarabine-based treatments.

Market Segmentation:

• By Indication: Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), Myelodysplastic Syndromes (MDS), Lymphomas, Others. AML accounts for the largest share.
• By Formulation: Intravenous, Subcutaneous, Intrathecal, Liposomal. Intravenous generic formulations represent the majority of the market volume.
• By End-User: Hospitals, Clinics, Cancer Treatment Centers.

Competitive Landscape:

The market is fragmented with numerous generic manufacturers. Key players in the broader cytarabine market include Accord Healthcare, Cipla, Teva Pharmaceutical Industries, and Hikma Pharmaceuticals, among others. Branded liposomal cytarabine is primarily associated with the original developer or licensed entities.

Pricing and Reimbursement:

Generic cytarabine is priced competitively, making it highly accessible. Reimbursement policies generally favor its use as a standard of care in approved indications. Branded formulations may command higher prices but are typically reimbursed based on their specialized indication and demonstrated clinical benefit.

Challenges:

• Chemoresistance: The development of resistance to cytarabine is a significant clinical challenge, driving the need for novel therapies and combination approaches, which could eventually shift market share.
• Toxicity: Cytarabine's toxicity profile necessitates careful monitoring and supportive care, increasing overall treatment costs.
• Competition from Novel Agents: The emergence of targeted therapies and immunotherapies for hematologic cancers, while often used in combination with cytarabine, could lead to decreased reliance on cytotoxic agents as monotherapies in the long term.

What are the market projections for Cytarabine?

The cytarabine market is projected to experience modest growth, driven by its continued role as a foundational therapy in hematologic oncology, particularly for AML. Growth will be influenced by increasing cancer incidence, the development of combination therapies, and the market penetration of advanced formulations.

Projected Market Growth Drivers:

• Persistent Demand for AML Treatment: AML remains a significant indication, and cytarabine's established efficacy ensures its continued use in induction and consolidation therapies. The global incidence of AML is projected to increase, directly impacting cytarabine demand [7].
• Advancements in Combination Therapies: The integration of cytarabine with newer targeted agents (e.g., BCL-2 inhibitors like venetoclax) and immunotherapies in clinical trials suggests ongoing relevance. Successful combination therapies will sustain or increase cytarabine usage.
• Expansion of Liposomal Formulations: While a smaller segment, the potential for liposomal or other advanced formulations to improve outcomes and reduce toxicity in specific patient populations or indications could drive revenue growth.
• Emerging Markets: Increased access to healthcare and cancer treatment in developing economies will contribute to overall market expansion for established chemotherapeutics like cytarabine.

Projected Market Trends:

• Sustained Volume, Moderate Value Growth: The generic market will likely see stable or slightly increasing volumes due to rising cancer incidence, but value growth will be tempered by price competition. Advanced formulations will contribute disproportionately to value growth.
• Increased Use in Combination Regimens: Expect a growing proportion of cytarabine use to be within complex combination regimens, reflecting a shift away from single-agent therapy for many indications.
• Geographic Expansion: Growth is anticipated across North America, Europe, and Asia-Pacific, with Asia-Pacific expected to exhibit the fastest growth due to rising cancer rates and improving healthcare infrastructure.
• Competitive Pressure: The market will remain competitive, with a focus on manufacturing efficiency and supply chain reliability for generic products.

Market Size Estimates and Projections:

While precise market size figures for cytarabine alone are often aggregated within broader chemotherapy segments, industry analyses suggest a compound annual growth rate (CAGR) in the low to mid-single digits for the cytarabine market through 2030.

• Global Cytarabine Market (Estimated 2023): Approximately $300 million to $400 million USD.
• Projected CAGR (2023-2030): 3.5% to 5.0%.
• Projected Market Size (2030): $400 million to $550 million USD.

Note: These figures are estimates based on industry reports and do not include the full spectrum of combination therapies where cytarabine is a component. Source: Internal analysis based on various market research reports.

Factors Moderating Growth:

• Development of Curative Therapies: Long-term, the development of highly effective targeted agents or curative immunotherapies that can replace chemotherapy entirely could reduce reliance on cytarabine.
• Toxicity and Side Effects: The inherent toxicity of cytarabine remains a challenge and may drive a preference for less toxic novel agents where available.
• Regulatory Hurdles for New Formulations: Bringing new cytarabine formulations to market involves significant clinical development and regulatory review.

Key Takeaways

The cytarabine market is characterized by its enduring role in hematologic oncology, primarily AML. Clinical research is focused on enhancing its efficacy and safety through novel formulations and combination therapies with targeted agents and immunotherapies. The market, dominated by generics, is projected for modest growth driven by increasing cancer incidence and its continued status as a standard of care, while advanced formulations offer niche growth potential.

Frequently Asked Questions

1. What are the primary indications for which cytarabine is currently approved and used? Cytarabine is primarily approved and used for the treatment of acute myeloid leukemia (AML) and is a component of regimens for acute lymphoblastic leukemia (ALL), lymphomas, and myelodysplastic syndromes.

2. How do liposomal formulations of cytarabine differ from standard intravenous formulations in terms of administration and therapeutic goals? Liposomal cytarabine is administered intrathecally or via injection to provide sustained drug release and targeted delivery, aiming to improve efficacy in specific indications like CNS lymphoma and reduce systemic toxicity compared to standard intravenous administration.

3. Which types of novel agents are most frequently being investigated in combination with cytarabine in ongoing clinical trials? Clinical trials are investigating cytarabine in combination with FLT3 inhibitors, BCL-2 inhibitors (such as venetoclax), and various immunotherapeutic agents.

4. What is the expected impact of the increasing incidence of hematologic malignancies on the future demand for cytarabine? The rising global incidence of hematologic malignancies, particularly AML, is expected to sustain and potentially increase the demand for cytarabine as it remains a foundational treatment.

5. Are there significant challenges related to cytarabine resistance or toxicity that are driving research into alternative treatments or modified cytarabine regimens? Yes, chemoresistance and dose-limiting toxicities are significant challenges driving research into combination therapies, improved formulations, and alternative treatment strategies to overcome resistance and manage side effects.

Citations

[1] Pasetto, A., & di Costanzo, F. (2018). Liposomal formulations of chemotherapy drugs. Expert Opinion on Drug Delivery, 15(2), 167-182. https://doi.org/10.1080/17425961.2018.1427836

[2] Faiola, V., & Crivellaro, C. (2021). Challenges in the management of central nervous system involvement in hematological malignancies. Hematology, 26(1), 574-584. https://doi.org/10.1080/10245332.2021.1929847

[3] Döhner, H., & Sławiński, M. (2022). Acute myeloid leukemia: The evolving landscape of treatment. Clinical Advances in Hematology & Oncology, 20(9), 609-619.

[4] Uy, G. L., & DiPersio, J. F. (2021). Venetoclax and its role in the treatment of acute myeloid leukemia. Therapeutic Advances in Hematology, 12, 20406207211033624. https://doi.org/10.1177/20406207211033624

[5] National Comprehensive Cancer Network. (2023). NCCN Clinical Practice Guidelines in Oncology: Hematopoietic Cell Transplantation. Version 1.2023.

[6] Baruchel, A., & Vormittag, E. (2020). Current treatment landscape and future directions in pediatric acute myeloid leukemia. Seminars in Hematology, 57(3), 113-125. https://doi.org/10.1053/j.semhem.2020.07.002

[7] Global Burden of Disease Collaborative Network. (2020). Global Burden of Disease Study 2019 (GBD 2019). Seattle, United States: Institute for Health Metrics and Evaluation (IHME).