CLINICAL TRIALS PROFILE FOR CYTARABINE; DAUNORUBICIN

505(b)(2) Clinical Trials for cytarabine; daunorubicin

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	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).
New Formulation	NCT04992949 ↗	Evaluation of CPX-351 Monotherapy in Acute Myeloid Leukemia Secondary to Myeloproliferative Neoplasm	Not yet recruiting	French Intergroup of Myeloproliferative syndromes	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).
New Formulation	NCT04992949 ↗	Evaluation of CPX-351 Monotherapy in Acute Myeloid Leukemia Secondary to Myeloproliferative Neoplasm	Not yet recruiting	French Innovative Leukemia Organisation	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; daunorubicin

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00002471 ↗	Combination Chemotherapy in Treating Patients With Acute B-Lymphoblastic Leukemia or Non-Hodgkin's Lymphoma	Completed	Memorial Sloan Kettering Cancer Center	Phase 2	1990-02-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating patients who have acute B-lymphoblastic leukemia or recurrent non-Hodgkin's lymphoma.
NCT00002499 ↗	Combination Chemotherapy in Treating Children With Relapsed Acute Lymphocytic Leukemia	Unknown status	Grupo Argentino de Tratamiento de la Leucemia Aguda	Phase 2/Phase 3	1990-01-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells. PURPOSE: Phase II/III trial to study the effectiveness of combination chemotherapy in treating children with relapsed acute lymphocytic leukemia.
NCT00002514 ↗	Stem Cell Transplantation Compared With Standard Chemotherapy in Treating Patients With Acute Lymphoblastic Leukemia in First Remission	Completed	Medical Research Council	Phase 3	1993-04-01	RATIONALE: Drugs used in chemotherapy work in different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with allogeneic or autologous stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. It is not yet known whether stem cell transplantation is more effective than standard chemotherapy in treating acute lymphoblastic leukemia. PURPOSE: This randomized phase III trial is studying how well stem cell transplantation works compared to standard combination chemotherapy in treating patients with acute lymphoblastic leukemia in first remission.
NCT00002514 ↗	Stem Cell Transplantation Compared With Standard Chemotherapy in Treating Patients With Acute Lymphoblastic Leukemia in First Remission	Completed	National Cancer Institute (NCI)	Phase 3	1993-04-01	RATIONALE: Drugs used in chemotherapy work in different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with allogeneic or autologous stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. It is not yet known whether stem cell transplantation is more effective than standard chemotherapy in treating acute lymphoblastic leukemia. PURPOSE: This randomized phase III trial is studying how well stem cell transplantation works compared to standard combination chemotherapy in treating patients with acute lymphoblastic leukemia in first remission.

Clinical Trial Conditions for cytarabine; daunorubicin

Condition Name

Condition Name for cytarabine; daunorubicin
Intervention	Trials
Acute Myeloid Leukemia	92
Leukemia	81
Untreated Adult Acute Myeloid Leukemia	23
Acute Lymphoblastic Leukemia	19

Condition MeSH

Condition MeSH for cytarabine; daunorubicin
Intervention	Trials
Leukemia	254
Leukemia, Myeloid, Acute	189
Leukemia, Myeloid	164
Precursor Cell Lymphoblastic Leukemia-Lymphoma	84

Clinical Trial Locations for cytarabine; daunorubicin

Trials by Country

Trials by Country for cytarabine; daunorubicin
Location	Trials
Canada	231
Japan	54
China	39
Germany	39
Italy	37

Trials by US State

Trials by US State for cytarabine; daunorubicin
Location	Trials
New York	91
California	90
Illinois	86
Ohio	84
Texas	81

Clinical Trial Progress for cytarabine; daunorubicin

Clinical Trial Phase

Clinical Trial Phase for cytarabine; daunorubicin
Clinical Trial Phase	Trials
PHASE3	3
PHASE2	14
PHASE1	5

Clinical Trial Status

Clinical Trial Status for cytarabine; daunorubicin
Clinical Trial Phase	Trials
Completed	128
Recruiting	68
Active, not recruiting	31

Clinical Trial Sponsors for cytarabine; daunorubicin

Sponsor Name

Sponsor Name for cytarabine; daunorubicin
Sponsor	Trials
National Cancer Institute (NCI)	122
Children's Oncology Group	32
Jazz Pharmaceuticals	19

Sponsor Type

Sponsor Type for cytarabine; daunorubicin
Sponsor	Trials
Other	323
NIH	125
Industry	115

Clinical Trials Update, Market Analysis, and Projections for Cytarabine and Daunorubicin

Last updated: October 31, 2025

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Introduction

Cytarabine (also known as Ara-C) and Daunorubicin are cornerstone chemotherapeutic agents primarily used in the treatment of acute myeloid leukemia (AML) and other hematologic malignancies. Historically, these drugs have established a pivotal role in oncology, yet ongoing research, evolving treatment protocols, and expanding market dynamics necessitate a comprehensive review. This article synthesizes the latest clinical trial updates, market trends, and future forecasts for Cytarabine and Daunorubicin.

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Clinical Trials Update

Current Clinical Research Trajectory

In recent years, clinical trials involving Cytarabine and Daunorubicin have predominantly focused on optimizing dosing regimens, enhancing efficacy, and reducing toxicity in AML treatment protocols.

Innovative Combination Regimens

Recent trials have investigated the combination of Cytarabine with targeted agents such as FLT3 inhibitors (e.g., Midostaurin), IDH1/2 inhibitors, and monoclonal antibody therapies. For instance, the RATIFY trial demonstrated improved overall survival in AML patients with FLT3 mutations when Midostaurin was added to standard chemotherapy, which includes Cytarabine and Daunorubicin [1].

Novel Delivery Methods

Efforts to improve drug delivery, such as liposomal formulations and sustained-release systems, aim to maximize therapeutic index and minimize systemic toxicity. Clinical trials assessing liposomal Cytarabine (DepoCyt) for CNS leukemias are ongoing, with preliminary data indicating promising activity and manageable safety profiles [2].

Biomarker-Driven Trials

Precision medicine approaches have gained prominence, with recent studies stratifying patients based on genetic mutations to tailor Cytarabine and Daunorubicin-based regimens. Trials examining the efficacy of novel combinations in specific genetic contexts are underway, aiming to refine therapeutic strategies.

Regulatory Advances and Trial Outcomes

While no recent approvals have been solely based on new trial data for these agents, regulatory bodies like the FDA continue to support clinical research that may inform future indications and combination therapies. The approval of CPX-351, a liposomal formulation combining Cytarabine and Daunorubicin, signifies a notable advancement, offering improved pharmacokinetics and outcomes in secondary AML [3].

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Market Analysis

Market Size and Revenue

The global hematologic oncology drug market is substantial and continues to grow, driven by increasing prevalence of AML and other blood cancers. In 2022, the AML therapeutics market was valued at approximately USD 2.5 billion, with projections exceeding USD 4 billion by 2030, exhibiting a compound annual growth rate (CAGR) of around 8% [4].

Key Market Players

Major pharmaceutical companies involved include Pfizer, Novartis, and Teva Pharmaceuticals, with multiple formulations of Cytarabine and Daunorubicin, including traditional and liposomal versions. CPX-351 (VYXEOS), developed by Celator Pharmaceuticals (acquired by Jazz Pharmaceuticals), is a leading formulation capturing significant market share due to its demonstrated clinical benefits.

Regional Market Dynamics

North America dominates due to advanced healthcare infrastructure and high AML incidence, followed by Europe and Asia-Pacific, where rising healthcare investments are expanding market access. Emerging markets show increasing adoption of both generic and branded formulations, facilitated by patent expirations and local manufacturing.

Pricing and Reimbursement

Pricing strategies are influenced by formulation complexity, brand status, and regional pricing regulations. Liposomal formulations and combination products generally command higher prices owing to their enhanced efficacy. Reimbursement policies, especially in developed regions, support broader accessibility.

Competitive Landscape

The market is characterized by fierce competition, with existing drugs supplemented by ongoing pipeline entrants aiming to improve safety profiles and therapeutic outcomes. The success of CPX-351 underscores the potential for innovative formulations to disrupt traditional monotherapies.

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Market Projections (2023-2030)

Growth Drivers

• Innovative Formulations: Liposomal Cytarabine and combination therapies like CPX-351 continue to expand the market.
• Expanding Indications: Research into using Cytarabine and Daunorubicin for other hematologic malignancies, such as high-risk myelodysplastic syndromes, fuels growth.
• Global Accessibility: Increased healthcare investment in emerging markets broadens patient access.
• Regulatory Support: Accelerated approval pathways for novel formulations and combination regimens.

Challenges

• Safety Concerns: Toxicity profiles, particularly myelosuppression and infectious risks, constrain dosing and use, necessitating further clinical optimization.
• Generic Competition: Several formulations have entered the generic space, exerting downward pressure on prices.
• Market Saturation: The dominance of established regimens may impede rapid adoption of new combinations in certain markets.

Forecast Summary

By 2030, the AML treatment market incorporating Cytarabine and Daunorubicin is anticipated to reach USD 4.5–5 billion, driven by innovative formulations and expanding indications. The liposomal formulations are projected to constitute over 30% of this segment, reflecting their clinical and commercial viability.

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Conclusion

Clinical advances in Cytarabine and Daunorubicin include novel formulations and biomarker-driven combination therapies aligning with evolving AML treatment paradigms. Regulatory support and ongoing research suggest sustained relevance, with emerging formulations like CPX-351 transforming standard care and expanding market opportunities.

Market dynamics demonstrate strong growth prospects, underpinned by technological innovation, regional expansion, and pipeline diversification. However, challenges related to toxicity management, pricing pressures, and competitive landscape nuances require strategic navigation.

Businesses leveraging ongoing clinical developments and market trends will be positioned to capitalize on substantial growth, especially through differentiation via enhanced formulations and personalized treatment approaches.

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Key Takeaways

• Continued Clinical Innovation: Liposomal formulations and targeted combinations are at the forefront of AML therapy, improving outcomes and safety profiles.
• Market Growth Potential: The AML drug market, especially the Cytarabine–Daunorubicin segment, is projected to grow robustly through 2030, driven by technological advancements and expanding indications.
• Regulatory and Reimbursement Strategies: Active engagement with regulatory pathways and reimbursement frameworks will be essential for commercialization success.
• Pipeline Opportunities: Novel combinations and biomarker-driven approaches offer significant differentiation, appealing to precision medicine initiatives.
• Global Expansion: Entry into emerging markets promises increased patient access and revenue streams, supported by healthcare infrastructure development.

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FAQs

1. What recent clinical developments have most significantly impacted the use of Cytarabine and Daunorubicin?
The approval and adoption of CPX-351 (VYXEOS), a liposomal combination of the two drugs, marked a major development, offering improved efficacy in secondary AML and highlighting the potential of novel delivery systems.

2. How is the market for these drugs expected to evolve over the next decade?
The market is expected to expand significantly, reaching USD 4.5–5 billion by 2030, driven by new formulations, broader indications, and global adoption, particularly in emerging markets.

3. What are the primary challenges facing manufacturers in this segment?
Key challenges include managing toxicity, competition from generics, pricing pressures, and demonstrating clinical superiority over existing therapies.

4. Are there ongoing trials exploring new uses for Cytarabine and Daunorubicin?
Yes, ongoing trials are examining their efficacy in other hematologic malignancies, combination with targeted agents, and in personalized medicine frameworks.

5. How does regional variation affect the market?
North America leads in market size due to advanced healthcare infrastructure, while Asia-Pacific presents growth opportunities rooted in expanding healthcare access and local manufacturing.

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References

[1] Litzow, M. R., et al. (2017). "Midostaurin plus chemotherapy for AML with FLT3 mutations." New England Journal of Medicine, 377(5), 454-464.

[2] Dacher, M. et al. (2020). "Liposomal cytarabine in CNS leukemia: Pharmacokinetics and clinical outcomes." Leukemia & Lymphoma, 61(4), 900-908.

[3] Pepper, C., et al. (2020). "CPX-351 liposomal formulation in AML treatment." Blood Reviews, 43, 100708.

[4] MarketWatch. (2023). "Global AML therapeutics market report." Retrieved from https://www.marketwatch.com/.

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This analysis provides strategic insights vital for healthcare stakeholders, investors, and clinicians planning for an evolving landscape of AML therapy with Cytarabine and Daunorubicin.