CLINICAL TRIALS PROFILE FOR CLOFARABINE

505(b)(2) Clinical Trials for Clofarabine

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 Combination	NCT01643668 ↗	Busulfan/Clofarabine + Allogeneic Stem Cell Transplantation	Completed	Massachusetts General Hospital	Phase 2	2012-07-01	This research is a phase II clinical trial. Phase II clinical trials test the effectiveness of an investigational intervention to learn whether it works in treating a specific cancer. "Investigational" means that the study intervention is still being studied and that research doctors are trying to find out more about it. It also means that the FDA has not yet approved this study intervention for your type of cancer. All participants on this study are treated in an identical manner. The investigators are doing this study because there continues to be a significant risk of relapse of disease after reduced intensity transplantation. In studies which have compared transplants using high-doses of chemotherapy and/or radiation versus reduced intensity transplants, patients undergoing reduced intensity transplants appear to have higher rates of relapse, but lower rates of toxicity and complication. This study attempts to utilize clofarabine, a newer chemotherapy agent shown to be quite active in AML, ALL, and MDS, to increase the anti-tumor effects of the conditioning regimen without accumulating unacceptable toxicity. The reduced intensity allogeneic stem cell transplantation procedure involves giving you chemotherapy in relatively less intense doses to suppress your immune system. This is followed by an infusion of healthy blood stem cells from a matched related donor or a matched unrelated volunteer donor. It is hoped that these donor cells can eventually then attack any cancer cells which remain. In this research study, the investigators are looking to see how well this new combination of busulfan and clofarabine works in reduced intensity allogeneic stem cell transplantation. By "works" the investigators mean to analyze safety, ability of donor cells to engraft (take hold), as well as measures of complications including toxicity, infections, graft-vs-host disease (GVHD), and relapse.

All Clinical Trials for Clofarabine

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00028418 ↗	Clofarabine in Chronic Lymphocytic Leukemia	Completed	M.D. Anderson Cancer Center	Phase 1	1999-02-01	This is a dose-escalation study to determine the maximum tolerated dose and toxic effects of clofarabine in patients with chronic lymphocytic leukemia and other acute leukemias. Clofarabine is a synthesized hybrid nucleoside analog, which is believed to possess the better qualities of fludarabine and chlorodeoxyadenosine, the 2 most active agents against lymphoproliferative disorders. Thus, it is hoped that this drug will be more active and less toxic than similar drugs.
NCT00042341 ↗	Phase II Study of Clofarabine in Pediatric Acute Lymphoblastic Leukemia (ALL)	Completed	Genzyme, a Sanofi Company	Phase 2	2002-05-01	Clofarabine (injection) is approved by the Food and Drug Administration (FDA) for the treatment of pediatric patients 1 to 21 years old with relapsed acute lymphoblastic leukemia (ALL) who have had at least 2 prior treatment regimens. The purpose of this study is to determine whether Clofarabine is safe and effective in the treatment of Acute Lymphoblastic Leukemia (ALL.)
NCT00042354 ↗	Phase II Study of Clofarabine in Pediatric Acute Myelogenous Leukemia (AML) Patients	Completed	Genzyme, a Sanofi Company	Phase 2	2002-05-01	Clofarabine (injection) is approved by the Food and Drug Administration (FDA) for the treatment of pediatric patients 1 to 21 years old with relapsed acute lymphoblastic leukemia (ALL) who have had at least 2 prior treatment regimens. The purpose of this study is to determine whether Clofarabine is safe and effective in the treatment of Acute Myelogenous Leukemia (AML.)
NCT00044889 ↗	Phase II Study of Clofarabine in Adult Patients With Refractory or Relapsed Acute Myelogenous Leukemia	Completed	Genzyme, a Sanofi Company	Phase 2	2002-05-01	Clofarabine (injection) is approved by the Food and Drug Administration (FDA) for the treatment of pediatric patients 1 to 21 years old with relapsed acute lymphoblastic leukemia (ALL) who have had at least 2 prior treatment regimens. This is a single arm, open-label, Phase II study of CLOFARABINE in adult patients with refractory or relapsed acute myelogenous leukemia (AML). Qualified patients must be refractory to one or two induction regimens, or have relapsed < one year from the date of confirmation of the initial complete remission (CR). There will be two phases in this study - an Induction phase and a Consolidation phase.
NCT00065143 ↗	Clofarabine Plus Cytarabine in Patients With Previously Untreated Acute Myeloid Leukemia and High-risk Myelodysplastic Syndrome	Completed	Genzyme, a Sanofi Company	Phase 2	2003-06-23	The goal of this clinical research study is to learn if clofarabine, when given in combination with ara-C (cytarabine), can help to improve the disease's response to therapy and to increase the duration of response in patients who are 50 years or older with leukemia. The safety of this combination treatment will also be studied.
NCT00065143 ↗	Clofarabine Plus Cytarabine in Patients With Previously Untreated Acute Myeloid Leukemia and High-risk Myelodysplastic Syndrome	Completed	M.D. Anderson Cancer Center	Phase 2	2003-06-23	The goal of this clinical research study is to learn if clofarabine, when given in combination with ara-C (cytarabine), can help to improve the disease's response to therapy and to increase the duration of response in patients who are 50 years or older with leukemia. The safety of this combination treatment will also be studied.
NCT00067028 ↗	Clofarabine Combinations in Relapsed/Refractory Acute Myeloid Leukemia (AML), Myelodysplastic Syndromes (MDS) and Myeloid Blast Phase Chronic Myeloid Leukemia (CML)	Completed	Genzyme, a Sanofi Company	Phase 2	2003-12-01	The goal is to compare the drug combinations clofarabine/idarubicin/ara-C, clofarabine/ara-C, and clofarabine/idarubicin in the treatment of patients with Acute Myeloid Leukemia, high-grade MDS, or myeloid blast phase of Chronic Myeloid Leukemia who have relapsed following their initial therapy.

Clinical Trial Conditions for Clofarabine

Condition Name

Condition Name for Clofarabine
Intervention	Trials
Leukemia	41
Acute Myeloid Leukemia	33
Myelodysplastic Syndrome	28
Acute Lymphoblastic Leukemia	24

Condition MeSH

Condition MeSH for Clofarabine
Intervention	Trials
Leukemia	120
Leukemia, Myeloid, Acute	84
Leukemia, Myeloid	80
Preleukemia	55

Clinical Trial Locations for Clofarabine

Trials by Country

Trials by Country for Clofarabine
Location	Trials
United States	482
Canada	21
France	12
United Kingdom	9
Italy	8

Trials by US State

Trials by US State for Clofarabine
Location	Trials
Texas	55
Tennessee	28
New York	28
California	26
Washington	20

Clinical Trial Progress for Clofarabine

Clinical Trial Phase

Clinical Trial Phase for Clofarabine
Clinical Trial Phase	Trials
Phase 3	8
Phase 2/Phase 3	4
Phase 2	82

Clinical Trial Status

Clinical Trial Status for Clofarabine
Clinical Trial Phase	Trials
Completed	94
Terminated	29
Recruiting	14

Clinical Trial Sponsors for Clofarabine

Sponsor Name

Sponsor Name for Clofarabine
Sponsor	Trials
Genzyme, a Sanofi Company	60
National Cancer Institute (NCI)	38
M.D. Anderson Cancer Center	31

Sponsor Type

Sponsor Type for Clofarabine
Sponsor	Trials
Other	192
Industry	89
NIH	41

Clofarabine: Clinical Trials, Market Analysis, and Projections

Introduction to Clofarabine

Clofarabine is a chemotherapy drug that has been approved by the FDA for the treatment of acute lymphoblastic leukemia (ALL) in children and adults who have not responded to other treatments. It works by inhibiting DNA synthesis, thereby halting the proliferation of cancerous cells.

Clinical Trials Update

Langerhans Cell Histiocytosis (LCH) and Other Histiocytic Disorders

A Phase II clinical trial is currently evaluating clofarabine as a potential treatment for Langerhans Cell Histiocytosis (LCH) and other histiocytic disorders. This trial aims to determine the effectiveness of clofarabine in treating these conditions, given its established use in leukemia[1].

Acute Myeloid Leukemia (AML)

A clinical trial, though no longer recruiting patients, has been conducted to assess the combination of clofarabine and liposomal daunorubicin for children and teenagers with acute myeloid leukemia (AML). This Phase 1 trial focused on finding the safest and most effective dose of clofarabine when combined with liposomal daunorubicin and evaluating the side effects and efficacy of this combination[4].

Market Analysis

Global Market Size and Growth

The global clofarabine market is projected to experience significant growth. As of 2023, the market size was estimated at USD 456 million and is expected to reach USD 489.45 million in 2024. By 2030, the market is forecasted to grow to USD 681.45 million at a compound annual growth rate (CAGR) of 5.90%[5].

Regional Market Dynamics

• North America: This region holds a major share of the global clofarabine market, with over 40% of the revenue. The growth in this region is driven by advanced healthcare infrastructure, robust research and development facilities, a strong pharmaceutical sector, and supportive regulatory frameworks[2].
• Asia Pacific: This region is expected to grow at a CAGR of 8.6% from 2024 to 2031, driven by the rapid expansion of the pharmaceutical sector, significant patient demographics, and investments in healthcare infrastructure[2].

Market Segments

• Hospital Segment: The hospital segment is expanding rapidly due to increasing patient admissions and the preference for inpatient therapies for acute leukemia. This segment is crucial for addressing the urgent medical needs of leukemia patients[2].
• Pharmacy Segment: The pharmacy segment is also growing significantly, fueled by the demand for outpatient therapies such as chemotherapy and the availability of clofarabine for home infusion, which enhances patient convenience[2].

Market Projections

Drivers of Growth

The growth of the clofarabine market is driven by several factors:

• Escalating Demand: Increasing incidence of pediatric leukemia and other hematological malignancies drives the demand for clofarabine.
• Advancements in Cancer Research: Ongoing research and clinical trials demonstrating the efficacy of clofarabine in various cancer treatments contribute to market growth.
• Innovative Treatment Methodologies: The exploration of combination therapies and novel formulations of clofarabine further expands its therapeutic scope[2][5].

Opportunities and Challenges

• Geographic Expansion: Emerging markets with rising cancer rates present opportunities for market expansion.
• Clinical Trials: Positive outcomes from clinical trials assessing clofarabine's efficacy in different cancers can foster widespread adoption and confidence within the healthcare community[2][5].

End-Use Scope

Clofarabine's end-use scope extends to:

• Healthcare Providers: Hospitals, specialized cancer clinics, and other medical facilities.
• Patients: Primarily pediatric patients suffering from acute lymphoblastic leukemia (ALL) and other hematological malignancies.
• Research Institutions: Continuous research and clinical trials are crucial for the development and expansion of clofarabine's applications[5].

Key Takeaways

• Clofarabine is being evaluated in clinical trials for its potential in treating Langerhans Cell Histiocytosis and other histiocytic disorders, as well as acute myeloid leukemia.
• The global clofarabine market is expected to grow significantly, driven by advancements in cancer research, increasing demand, and geographic expansion.
• North America and the Asia Pacific are key regions contributing to the market growth due to their advanced healthcare infrastructure and growing pharmaceutical sectors.
• The hospital and pharmacy segments are critical for the market's expansion, with a focus on both inpatient and outpatient treatments.

FAQs

What is clofarabine used for?

Clofarabine is primarily used to treat acute lymphoblastic leukemia (ALL) in children and adults who have not responded to other treatments. It is also being evaluated for its efficacy in other hematological malignancies such as Langerhans Cell Histiocytosis and acute myeloid leukemia.

What is the current market size of clofarabine?

As of 2023, the global clofarabine market size was estimated at USD 456 million and is expected to reach USD 489.45 million in 2024.

What is the projected growth rate of the clofarabine market?

The clofarabine market is forecasted to grow at a CAGR of 5.90% from 2023 to 2030, reaching USD 681.45 million by 2030.

Which regions are driving the growth of the clofarabine market?

North America and the Asia Pacific are the key regions driving the growth of the clofarabine market, due to their advanced healthcare infrastructure and growing pharmaceutical sectors.

What are the main segments of the clofarabine market?

The main segments include the hospital segment, which is expanding due to increasing patient admissions, and the pharmacy segment, which is growing due to the demand for outpatient therapies and home infusion options.

Sources

1. Boston Children's Hospital: NCT02425904 - Boston Children's Hospital.
2. Cognitive Market Research: Clofarabine Market Report 2024 (Global Edition).
3. Cognitive Market Research: Clofarabine Drugs Market Report 2024 (Global Edition).
4. Cancer Research UK: A trial of clofarabine and liposomal daunorubicin for children and teenagers with acute myeloid leukaemia (CLOUD).
5. 360iResearch: Clofarabine Market Size & Share 2025-2030.

Last updated: 2025-01-07