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.

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.

Clinical Trial Conditions for Cytarabine

Condition Name

Condition Name for Cytarabine
Intervention	Trials
Acute Myeloid Leukemia	267
Leukemia	254
Lymphoma	139

Condition MeSH

Condition MeSH for Cytarabine
Intervention	Trials
Leukemia	904
Leukemia, Myeloid, Acute	632
Leukemia, Myeloid	620

Clinical Trial Locations for Cytarabine

Trials by Country

Trials by Country for Cytarabine
Location	Trials
United States	5,619
Canada	484
Australia	219
Italy	206
Germany	180

Trials by US State

Trials by US State for Cytarabine
Location	Trials
Texas	311
New York	273
California	269
Ohio	215
Illinois	212

Clinical Trial Progress for Cytarabine

Clinical Trial Phase

Clinical Trial Phase for Cytarabine
Clinical Trial Phase	Trials
Phase 4	34
Phase 3	228
Phase 2/Phase 3	30

Clinical Trial Status

Clinical Trial Status for Cytarabine
Clinical Trial Phase	Trials
Completed	575
Recruiting	240
Unknown status	131

Clinical Trial Sponsors for Cytarabine

Sponsor Name

Sponsor Name for Cytarabine
Sponsor	Trials
National Cancer Institute (NCI)	403
M.D. Anderson Cancer Center	106
Children's Oncology Group	60

Sponsor Type

Sponsor Type for Cytarabine
Sponsor	Trials
Other	1524
Industry	489
NIH	419

Cytarabine: Clinical Trials, Market Analysis, and Projections

Introduction to Cytarabine

Cytarabine, also known as cytosine arabinoside or Ara-C, is a chemotherapy medication used to treat various forms of leukemia, including acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), and acute promyelocytic leukemia (APL). It is also used in the treatment of Hodgkin's lymphoma and other types of lymphoma.

Current Clinical Trials

MIRACLE Trial: Annamycin and Cytarabine Combination

One of the most significant ongoing clinical trials involving cytarabine is the MIRACLE trial conducted by Moleculin. This pivotal, adaptive Phase 3 trial (MB-108) is designed to evaluate the efficacy and safety of Annamycin in combination with cytarabine (referred to as AnnAraC) for the treatment of relapsed or refractory AML.

• Trial Design: The trial will initially randomize 75 to 90 subjects to receive high-dose cytarabine (HiDAC) combined with either placebo, 190 mg/m² of Annamycin, or 230 mg/m² of Annamycin. After selecting the optimum dose based on safety, pharmacokinetics, and efficacy, approximately 240 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin[1][4].
• Expected Outcomes: The trial aims for possible accelerated approval of Annamycin in combination with cytarabine. The positive feedback from clinicians and alignment with the FDA on the trial design have given Moleculin a high level of confidence in the potential success of this combination.

Market Analysis

Market Size and Growth

The market for cytarabine is part of the broader acute myeloid leukemia therapeutics market, which is expected to grow significantly.

• Forecast: The AML therapeutics market is projected to reach USD 1.83 billion in 2024 and grow at a CAGR of 10.15% to reach USD 2.97 billion by 2029[2].
• Cytarabine Segment: Cytarabine is expected to witness high growth due to its effectiveness in treating AML. The drug's wide use in chemotherapy and recent regulatory approvals, such as the revised label for Vyxeos (daunorubicin and cytarabine) by the FDA, are driving this growth[2].

Key Drivers

Several factors are driving the growth of the cytarabine market:

• Increasing Incidence of Cancer: The rising percentage of geriatrics in the global population and the increasing prevalence of target diseases like leukemia are significant drivers[3].
• Regulatory Approvals: Recent approvals by regulatory authorities, such as the FDA, for new indications and formulations of cytarabine, are boosting market growth[2].
• Advances in Drug Delivery: Innovations in liposomal drug delivery technology are enhancing the efficacy and reducing the systemic toxicity of cytarabine, making it a more attractive treatment option[5].

Market Challenges

Despite the growth potential, the market faces several challenges:

• High Treatment Costs: The cost of chemotherapy treatments, including those involving cytarabine, is a significant barrier to wider adoption[5].
• Complex Manufacturing Processes: The production of liposomal formulations of cytarabine is complex and costly, which can limit market expansion[5].
• Regulatory Framework: The absence of a predefined regulatory framework can hinder the demand for cytarabine and other chemotherapy agents[3].

Market Projections

Geographical Outlook

The global cytarabine market is expected to see significant growth across various regions:

• North America: This region is likely to continue to account for a significant share of the revenue due to the presence of several pharmaceutical and biosciences companies and the high demand for advanced therapeutics[3].

Competitive Landscape

The market for cytarabine is highly competitive, with several key players:

• Major Players: Companies like Jazz Pharmaceuticals PLC, Celator Pharmaceuticals, and others are prominent in the market. These companies are investing heavily in research and development and forming strategic partnerships to stay ahead[3].

Technological Advancements

Technological advancements in drug delivery systems, particularly liposomal formulations, are expected to drive the market forward:

• Liposomal Formulations: These formulations enhance the delivery and efficacy of cytarabine while reducing systemic toxicity. Research is ongoing to explore their use in various types of leukemia and other cancers[5].

Key Takeaways

• Clinical Trials: The MIRACLE trial is a significant ongoing clinical trial evaluating the combination of Annamycin and cytarabine for relapsed or refractory AML.
• Market Growth: The AML therapeutics market, including cytarabine, is expected to grow at a CAGR of 10.15% to reach USD 2.97 billion by 2029.
• Drivers: Increasing cancer incidence, regulatory approvals, and advances in drug delivery technology are key drivers.
• Challenges: High treatment costs, complex manufacturing processes, and regulatory framework issues are significant challenges.
• Geographical Outlook: North America is expected to remain a leading region in the market.

FAQs

What is the current status of the MIRACLE trial involving cytarabine?

The MIRACLE trial, conducted by Moleculin, has received Institutional Review Board (IRB) approval and is on track to commence enrollment in the first quarter of 2025. The trial aims to evaluate the efficacy and safety of Annamycin in combination with cytarabine for the treatment of relapsed or refractory AML[1][4].

How is the market for cytarabine expected to grow?

The market for cytarabine is expected to grow significantly, with the AML therapeutics market projected to reach USD 2.97 billion by 2029 at a CAGR of 10.15%[2].

What are the key drivers of the cytarabine market?

Key drivers include the increasing incidence of cancer, recent regulatory approvals, and advances in liposomal drug delivery technology[2][3][5].

What challenges does the cytarabine market face?

The market faces challenges such as high treatment costs, complex manufacturing processes, and the absence of a predefined regulatory framework[3][5].

Which region is expected to lead the cytarabine market?

North America is expected to continue to account for a significant share of the revenue due to the presence of several pharmaceutical and biosciences companies and the high demand for advanced therapeutics[3].

How are technological advancements impacting the cytarabine market?

Technological advancements, particularly in liposomal formulations, are enhancing the delivery and efficacy of cytarabine while reducing systemic toxicity. This is driving interest and adoption in the global healthcare landscape[5].

Sources

1. Moleculin Receives Institutional Review Board Approval for MIRACLE Phase 3 Pivotal Trial of Annamycin in Combination with Cytarabine for the Treatment of R/R Acute Myeloid Leukemia (AML) - Moleculin.
2. Acute Myeloid Leukemia Market Size & Share Analysis - Mordor Intelligence.
3. Cytarabine and Daunorubicin Market Key Players, Sales, Demand, Dynamic Forces and Forecast 2029 - Biospace.
4. Moleculin Receives Institutional Review Board Approval for MIRACLE Phase 3 Pivotal Trial of Annamycin in Combination with Cytarabine for the Treatment of R/R Acute Myeloid Leukemia (AML) - PR Newswire.
5. Liposomal Daunorubicin & Cytarabine Market Size 2025-2030 - 360iResearch.