CLINICAL TRIALS PROFILE FOR DOXIL (LIPOSOMAL)

505(b)(2) Clinical Trials for DOXIL (LIPOSOMAL)

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	NCT01073371 ↗	Anesthetic Efficacy of Liposomal Prilocaine in Maxillary Infiltration Anesthesia	Completed	Conselho Nacional de Desenvolvimento Científico e Tecnológico	Phase 1	2008-07-01	This blinded randomized, crossover, three period study aim to evaluate the anesthetic efficacy of liposome-encapsulated 3% prilocaine compared to 3% plain prilocaine and 3% prilocaine with 0,03IU/mL felypressin, after 1.8mL infiltration in the buccal sulcus of the maxillary right canine, in 32 volunteers.
New Formulation	NCT01073371 ↗	Anesthetic Efficacy of Liposomal Prilocaine in Maxillary Infiltration Anesthesia	Completed	Fundação de Amparo à Pesquisa do Estado de São Paulo	Phase 1	2008-07-01	This blinded randomized, crossover, three period study aim to evaluate the anesthetic efficacy of liposome-encapsulated 3% prilocaine compared to 3% plain prilocaine and 3% prilocaine with 0,03IU/mL felypressin, after 1.8mL infiltration in the buccal sulcus of the maxillary right canine, in 32 volunteers.
New Formulation	NCT01073371 ↗	Anesthetic Efficacy of Liposomal Prilocaine in Maxillary Infiltration Anesthesia	Completed	University of Campinas, Brazil	Phase 1	2008-07-01	This blinded randomized, crossover, three period study aim to evaluate the anesthetic efficacy of liposome-encapsulated 3% prilocaine compared to 3% plain prilocaine and 3% prilocaine with 0,03IU/mL felypressin, after 1.8mL infiltration in the buccal sulcus of the maxillary right canine, in 32 volunteers.

All Clinical Trials for DOXIL (LIPOSOMAL)

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00001646 ↗	Voriconazole vs. Amphotericin B in the Treatment of Invasive Aspergillosis	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 3	1997-08-01	Invasive aspergillosis is a fungal disease which is increasing in incidence with the increase in immunocompromised persons in our population. Persons with prolonged neutropenia secondary to cytotoxic chemotherapies are at the highest risk for acute aspergillosis. Patients undergoing bone marrow transplantation, receiving prolonged corticosteroid or other immunosuppressive therapies, and persons with HIV infection and AIDS are also at risk. Even with antifungal therapy, aspergillosis in its acute invasive forms has a high mortality. In bone marrow transplantation patients and in those whose infection involves the brain, this mortality is greater than 90%. Amphotericin B in its conventional form, is the current standard treatment for this disease. Response to therapy with amphotericin B usually ranges between 20-60% in most studies. The higher response rates are usually seen in those patients who can tolerate this agent for at least 14 days. Because of its nephrotoxicity and other adverse effects, alternatives to conventional amphotericin B have been sought. These currently include liposomal forms of amphotericin B and itraconazole. Although these forms show a decrease in adverse effects, the efficacy of these drugs has not been shown to be equivalent to conventional amphotericin B. Voriconazole is an investigational antifungal drug currently being brought to phase III trials in the US. This azole has been shown active against Aspergillus spp. in vitro, and in animal models and early human trials to be effective against aspergillosis. It has been shown to be well-tolerated and is available in an intravenous and oral formulation. This study will evaluate the efficacy, safety, and toleration of voriconazole compared to conventional therapy with amphotericin B as primary treatment of acute invasive aspergillosis in immunocompromised patients. Patients will be randomized to open-labelled therapy with voriconazole or amphotericin B in a one-to-one ratio.
NCT00001059 ↗	Comparison of Liposomal Doxorubicin Used Alone or in Combination With Bleomycin Plus Vincristine in the Treatment of Kaposi's Sarcoma in Patients With AIDS	Completed	Amgen	Phase 2	1969-12-31	To evaluate the safety and efficacy of liposomal doxorubicin hydrochloride ( DOX-SL ) alone or in combination with bleomycin and vincristine in the long-term treatment of AIDS-related Kaposi's sarcoma. To determine whether the 3-drug combination enhances progression-free survival and quality of life. Liposomal formulations of chemotherapeutic agents increase drug accumulation in tumors, which permits disease palliation at relatively low doses and thus decreases some of the dose-limiting toxicity. Multi-agent therapy is considered to be more effective than single-agent therapy; therefore, DOX-SL will be combined with bleomycin and vincristine.
NCT00001059 ↗	Comparison of Liposomal Doxorubicin Used Alone or in Combination With Bleomycin Plus Vincristine in the Treatment of Kaposi's Sarcoma in Patients With AIDS	Completed	Sequus Pharmaceuticals	Phase 2	1969-12-31	To evaluate the safety and efficacy of liposomal doxorubicin hydrochloride ( DOX-SL ) alone or in combination with bleomycin and vincristine in the long-term treatment of AIDS-related Kaposi's sarcoma. To determine whether the 3-drug combination enhances progression-free survival and quality of life. Liposomal formulations of chemotherapeutic agents increase drug accumulation in tumors, which permits disease palliation at relatively low doses and thus decreases some of the dose-limiting toxicity. Multi-agent therapy is considered to be more effective than single-agent therapy; therefore, DOX-SL will be combined with bleomycin and vincristine.
NCT00001059 ↗	Comparison of Liposomal Doxorubicin Used Alone or in Combination With Bleomycin Plus Vincristine in the Treatment of Kaposi's Sarcoma in Patients With AIDS	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 2	1969-12-31	To evaluate the safety and efficacy of liposomal doxorubicin hydrochloride ( DOX-SL ) alone or in combination with bleomycin and vincristine in the long-term treatment of AIDS-related Kaposi's sarcoma. To determine whether the 3-drug combination enhances progression-free survival and quality of life. Liposomal formulations of chemotherapeutic agents increase drug accumulation in tumors, which permits disease palliation at relatively low doses and thus decreases some of the dose-limiting toxicity. Multi-agent therapy is considered to be more effective than single-agent therapy; therefore, DOX-SL will be combined with bleomycin and vincristine.
NCT00002093 ↗	A Randomized Phase III Clinical Trial of Daunoxome Versus Combination Chemotherapy With Adriamycin/Bleomycin/Vincristine (ABV) in the Treatment of HIV-Associated Kaposi's Sarcoma.	Completed	Nexstar Pharmaceuticals	Phase 3	1969-12-31	To compare the toxicity profiles (severity and time to onset from initiation of therapy) between daunorubicin (liposomal) and combination chemotherapy with doxorubicin/bleomycin/vincristine (ABV), with both regimens administered in combination with antiretroviral therapy. To compare the duration of responses, response rates, and times to response.
NCT00002105 ↗	Randomized, Comparative Trial of DOX-SL (Stealth Liposomal Doxorubicin Hydrochloride) Versus Bleomycin and Vincristine in the Treatment of AIDS-Related Kaposi's Sarcoma	Completed	Sequus Pharmaceuticals	Phase 3	1969-12-31	To determine the efficacy of Stealth liposomal doxorubicin hydrochloride (DOX-SL) in the treatment of moderate to severe AIDS-related Kaposi's sarcoma (KS) by comparison with the established therapy BV (bleomycin/vincristine). To evaluate the safety and tolerance of DOX-SL compared to BV in a population of AIDS patients with moderate to severe KS.
NCT00002147 ↗	Use of Stealth Liposomal Doxorubicin HCl ( DOX-SL ) in the Treatment of Moderate to Severe AIDS-Related Kaposi's Sarcoma.	Completed	Sequus Pharmaceuticals	Phase 3	1969-12-31	To provide Stealth liposomal doxorubicin hydrochloride ( DOX-SL ) as a therapy for Kaposi's sarcoma patients who have no remaining treatment options other than DOX-SL or patients who have been participating in another DOX-SL protocol and for whom continuation in DOX-SL is medically indicated. Also, to evaluate the safety and efficacy of DOX-SL in patients with Kaposi's sarcoma who have previously received systemic chemotherapy with or without an anthracycline.

Clinical Trial Conditions for DOXIL (LIPOSOMAL)

Condition Name

Condition Name for DOXIL (LIPOSOMAL)
Intervention	Trials
Ovarian Cancer	73
Breast Cancer	65
Pain, Postoperative	48
Fallopian Tube Cancer	29

Condition MeSH

Condition MeSH for DOXIL (LIPOSOMAL)
Intervention	Trials
Ovarian Neoplasms	130
Carcinoma, Ovarian Epithelial	103
Pain, Postoperative	95
Breast Neoplasms	91

Clinical Trial Locations for DOXIL (LIPOSOMAL)

Trials by Country

+

−

Trials by Country for DOXIL (LIPOSOMAL)
Location	Trials
Italy	154
China	99
Spain	85
Germany	83
Canada	76

Trials by US State

+

−

Trials by US State for DOXIL (LIPOSOMAL)
Location	Trials
California	132
New York	121
Texas	120
Ohio	85
Florida	80

Clinical Trial Progress for DOXIL (LIPOSOMAL)

Clinical Trial Phase

Clinical Trial Phase for DOXIL (LIPOSOMAL)
Clinical Trial Phase	Trials
Phase 4	188
Phase 3	132
Phase 2/Phase 3	20

Clinical Trial Status

Clinical Trial Status for DOXIL (LIPOSOMAL)
Clinical Trial Phase	Trials
Completed	387
Recruiting	168
Terminated	96

Clinical Trial Sponsors for DOXIL (LIPOSOMAL)

Sponsor Name

Sponsor Name for DOXIL (LIPOSOMAL)
Sponsor	Trials
National Cancer Institute (NCI)	105
M.D. Anderson Cancer Center	30
Pacira Pharmaceuticals, Inc	18

Sponsor Type

Sponsor Type for DOXIL (LIPOSOMAL)
Sponsor	Trials
Other	1003
Industry	442
NIH	111

Liposomal Doxorubicin (Doxil): Clinical Trials, Market Analysis, and Projections

Introduction to Liposomal Doxorubicin (Doxil)

Liposomal doxorubicin, marketed under the brand name Doxil, is a chemotherapeutic agent encapsulated in lipid-based vesicles. This formulation is designed to enhance the efficacy of doxorubicin while reducing its side effects, particularly cardiac toxicity.

Clinical Trials and Efficacy

Ovarian Cancer Trials

In the context of ovarian cancer, liposomal doxorubicin has been extensively studied. A phase III comparative study involving 474 patients with epithelial ovarian cancer who had failed first-line platinum-based chemotherapy showed that liposomal doxorubicin (50 mg/m² every 4 weeks) was at least equivalent to topotecan (1.5 mg/m² for 5 days every 3 weeks) in terms of time to disease progression and overall survival. However, the dose has been reduced to 40 mg/m² due to poor patient tolerability[1].

Breast Cancer Trials

In metastatic breast cancer, liposomal doxorubicin has demonstrated comparable efficacy to conventional doxorubicin but with significantly reduced cardiac toxicity. A randomized controlled trial comparing liposomal doxorubicin to vinorelbine or mitomycin C and vinblastine in taxane-refractory advanced breast cancer showed minimal activity in either arm, with an 8-10% response rate. However, liposomal doxorubicin is not effective in anthracycline-resistant disease and shows minimal activity in taxane-refractory patients[4].

Market Analysis

Current Market Size

The global liposomal doxorubicin market was valued at approximately USD 1.31 billion in 2024. This valuation underscores the significant demand for this formulation due to its improved safety profile and efficacy[2][3].

Market Growth Projections

The market is projected to grow at a Compound Annual Growth Rate (CAGR) of 6.2% to 6.3% from 2025 to 2034. By 2034, the market is expected to reach USD 2.38 billion to USD 2.367 billion, driven by factors such as the increasing prevalence of cancer, advancements in nanotechnology, and the growing adoption of targeted therapies[2][3][5].

Regional Outlook

North America currently accounts for the largest share of the global liposomal doxorubicin market. This dominance is attributed to the high incidence of cancer, advanced healthcare infrastructure, and significant investments in oncology research and development[2].

Key Drivers of Market Growth

Increasing Prevalence of Cancer

The rising incidence of cancer, particularly breast and ovarian cancer, is a major driver of the liposomal doxorubicin market. Sedentary lifestyles and stress-inducing schedules among the working population contribute to this trend[3].

Advancements in Nanotechnology

Advancements in nanotechnology have led to the development of more effective and stable liposomal formulations. These innovations enhance the drug's delivery and reduce side effects, making liposomal doxorubicin a preferred choice in cancer treatment[3][5].

Shift Towards Personalized Medicine

There is a growing trend towards personalized medicine, with liposomal doxorubicin formulations being customized to individual patient profiles. This approach aims to enhance treatment effectiveness while minimizing adverse reactions[3].

Market Trends

Ongoing Research and Development

Continuous research to improve the delivery and stability of liposomal doxorubicin, as well as to explore new therapeutic applications, is propelling market growth. New liposomal formulations and targeted therapies are expected to further drive the market[2][5].

Regulatory and Economic Factors

The cost-effectiveness and regulatory approvals of liposomal doxorubicin also play a crucial role. For instance, in Australia, liposomal doxorubicin is listed on the Pharmaceutical Benefits Scheme (PBS), making it more accessible to patients[1].

Safety and Toxicity Profile

Reduced Cardiac Toxicity

One of the significant advantages of liposomal doxorubicin is its reduced cardiac toxicity compared to conventional doxorubicin. Studies have shown that cardiac toxicity remains below 10% with cumulative doses up to 600 mg/m² in anthracycline-naive patients[4].

Other Side Effects

While liposomal doxorubicin reduces cardiac toxicity, it still carries other side effects such as myelosuppression, vomiting, and alopecia. However, these side effects are generally less severe than those associated with traditional doxorubicin[4].

Conclusion

Liposomal doxorubicin (Doxil) is a critical component in the treatment of various cancers, particularly ovarian and breast cancer. The drug's efficacy, coupled with its reduced side effects, makes it a preferred option for many clinicians. The market for liposomal doxorubicin is poised for significant growth, driven by increasing cancer prevalence, advancements in nanotechnology, and a shift towards personalized medicine.

Key Takeaways

• Clinical Efficacy: Liposomal doxorubicin is at least equivalent to topotecan in ovarian cancer and conventional doxorubicin in breast cancer, with reduced cardiac toxicity.
• Market Size: The global market was valued at USD 1.31 billion in 2024 and is projected to reach USD 2.38 billion by 2034.
• Growth Rate: The market is expected to grow at a CAGR of 6.2% to 6.3% from 2025 to 2034.
• Key Drivers: Increasing cancer prevalence, advancements in nanotechnology, and personalized medicine trends.
• Safety Profile: Reduced cardiac toxicity and other side effects compared to conventional doxorubicin.

FAQs

What is liposomal doxorubicin?

Liposomal doxorubicin is a chemotherapeutic agent encapsulated in lipid-based vesicles, designed to enhance efficacy and reduce side effects.

What are the primary uses of liposomal doxorubicin?

It is primarily used in the treatment of ovarian and breast cancers, particularly in cases where traditional chemotherapy has failed.

What is the current market size of liposomal doxorubicin?

The global market was valued at approximately USD 1.31 billion in 2024.

What is the projected growth rate of the liposomal doxorubicin market?

The market is expected to grow at a CAGR of 6.2% to 6.3% from 2025 to 2034.

What are the key drivers of the liposomal doxorubicin market growth?

The increasing prevalence of cancer, advancements in nanotechnology, and the trend towards personalized medicine are key drivers.

What are the main side effects of liposomal doxorubicin?

While it reduces cardiac toxicity, it can still cause myelosuppression, vomiting, and alopecia.

Sources

1. eviQ. "266-Ovarian recurrent DOXOrubicin pegylated liposomal."
2. Polaris Market Research. "Liposomal Doxorubicin Market Size, Share, Trends | Report, 2034."
3. Future Market Insights. "Liposomal Doxorubicin Market Revenue Forecast, 2024-2034."
4. eviQ. "35-Breast metastatic DOXOrubicin pegylated liposomal."
5. Grand View Research. "Liposomal Doxorubicin Market Size And Share Report, 2030."