CLINICAL TRIALS PROFILE FOR VINCRISTINE SULFATE PFS

505(b)(2) Clinical Trials for VINCRISTINE SULFATE PFS

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
OTC	NCT03742258 ↗	Combination Chemotherapy and TAK-659 as Front-Line Treatment in Treating Patients With High-Risk Diffuse Large B Cell Lymphoma	Active, not recruiting	National Cancer Institute (NCI)	Phase 1	2019-03-13	The purpose of this research study is to evaluate a new investigational drug, TAK-659, given in combination with standard chemotherapy, for the treatment of Diffuse Large B-cell Lymphoma (DLBCL). ?Investigational? means that TAK-659 has not been approved by the United States Food and Drug Administration (FDA) for use as a prescription or over-the-counter medication to treat a certain condition. The primary purpose of this study is to find the appropriate and safe dose of the study drug to be used in combination with standard chemotherapy for the treatment of your disease and to determine how well the drug works in treating the disease. Other objectives include measuring the amount of the study drug in the body at different times after taking the study drug. Participation in the study is expected to last for up to 3 years after receiving the last dose of the study drug. Patients will receive the study treatment for up to 18 weeks, as long as they are benefitting.
OTC	NCT03742258 ↗	Combination Chemotherapy and TAK-659 as Front-Line Treatment in Treating Patients With High-Risk Diffuse Large B Cell Lymphoma	Active, not recruiting	Northwestern University	Phase 1	2019-03-13	The purpose of this research study is to evaluate a new investigational drug, TAK-659, given in combination with standard chemotherapy, for the treatment of Diffuse Large B-cell Lymphoma (DLBCL). ?Investigational? means that TAK-659 has not been approved by the United States Food and Drug Administration (FDA) for use as a prescription or over-the-counter medication to treat a certain condition. The primary purpose of this study is to find the appropriate and safe dose of the study drug to be used in combination with standard chemotherapy for the treatment of your disease and to determine how well the drug works in treating the disease. Other objectives include measuring the amount of the study drug in the body at different times after taking the study drug. Participation in the study is expected to last for up to 3 years after receiving the last dose of the study drug. Patients will receive the study treatment for up to 18 weeks, as long as they are benefitting.

All Clinical Trials for VINCRISTINE SULFATE PFS

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.
NCT00000681 ↗	A Phase I Study of the Combination of Recombinant GM-CSF, AZT, and Chemotherapy (ABV) (Adriamycin, Bleomycin, Vincristine) in AIDS and Kaposi's Sarcoma	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1	1969-12-31	To determine the safety as well as the most effective dose of sargramostim (GM-CSF; granulocyte-macrophage colony stimulating factor) that will prevent the side effects caused by the combined use of zidovudine (AZT) and various doses of cancer-fighting drugs (doxorubicin, bleomycin, and vincristine) in AIDS patients with Kaposi's sarcoma (KS). Patients included in this study have KS, which is a type of cancer that occurs in nearly 20 percent of patients with AIDS. AIDS patients with extensive KS require treatment with effective cytotoxic (anti-cancer) agents to reduce the tumor size and with antiretroviral agents such as AZT to prevent or ameliorate the development of opportunistic infections. Due to the significant toxic effect of both cytotoxic and antiviral agents on the bone marrow where new blood cells are generated, the combination of these agents is expected to result in complications such as granulocytopenia (very low granulocyte counts). Hematopoietic growth factors such as GM-CSF may reduce the severity and duration of marrow suppression. This may improve survival. Clinical trials of GM-CSF in HIV infected individuals with or without granulocytopenia have shown that the progenitor cells (early blood cells) are responsive to GM-CSF.
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.
NCT00000954 ↗	A Study of Chemotherapy Plus ddI or ddC in the Treatment of AIDS-Related Kaposi's Sarcoma	Completed	Bristol-Myers Squibb	Phase 1	1969-12-31	To determine the toxicity and response to treatment with cytotoxic chemotherapy using doxorubicin (Adriamycin), bleomycin, and vincristine (DBV) for advanced AIDS-related Kaposi's sarcoma in combination with either didanosine (ddI) or zalcitabine (dideoxycytidine; ddC). AIDS patients with extensive Kaposi's sarcoma require treatment with effective cytotoxic agents to reduce the tumor burden, and they also require treatment with other possibly effective antiretroviral agents such as ddI or ddC to ameliorate (delay) the development of opportunistic infections.

Clinical Trial Conditions for VINCRISTINE SULFATE PFS

Condition Name

Condition Name for VINCRISTINE SULFATE PFS
Intervention	Trials
Lymphoma	139
Leukemia	72
Neuroblastoma	33
Brain and Central Nervous System Tumors	27

Condition MeSH

Condition MeSH for VINCRISTINE SULFATE PFS
Intervention	Trials
Lymphoma	217
Leukemia	137
Precursor Cell Lymphoblastic Leukemia-Lymphoma	124
Leukemia, Lymphoid	124

Clinical Trial Locations for VINCRISTINE SULFATE PFS

Trials by Country

Trials by Country for VINCRISTINE SULFATE PFS
Location	Trials
Canada	599
Switzerland	62
Puerto Rico	61
New Zealand	60
Norway	9

Trials by US State

Trials by US State for VINCRISTINE SULFATE PFS
Location	Trials
California	206
New York	200
Texas	177
Illinois	176
Ohio	173

Clinical Trial Progress for VINCRISTINE SULFATE PFS

Clinical Trial Phase

Clinical Trial Phase for VINCRISTINE SULFATE PFS
Clinical Trial Phase	Trials
PHASE2	4
Phase 4	1
Phase 3	162

Clinical Trial Status

Clinical Trial Status for VINCRISTINE SULFATE PFS
Clinical Trial Phase	Trials
Completed	258
Active, not recruiting	68
Unknown status	66

Clinical Trial Sponsors for VINCRISTINE SULFATE PFS

Sponsor Name

Sponsor Name for VINCRISTINE SULFATE PFS
Sponsor	Trials
National Cancer Institute (NCI)	287
Children's Oncology Group	97
Children's Cancer and Leukaemia Group	24

Sponsor Type

Sponsor Type for VINCRISTINE SULFATE PFS
Sponsor	Trials
Other	559
NIH	298
Industry	83

Vincristine Sulfate PFS: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: October 28, 2025

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Introduction

Vincristine Sulfate, marketed as Vincristine Sulfate PFS (Preservative-Free Solution), remains a cornerstone chemotherapeutic agent primarily employed in the treatment of hematologic malignancies such as acute lymphoblastic leukemia (ALL), Hodgkin's lymphoma, and non-Hodgkin’s lymphomas. With ongoing clinical developments and nuanced market dynamics, understanding its current landscape and future trajectory offers key strategic value for pharmaceutical stakeholders and investors.

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Clinical Trials Overview: Progress and Innovation

Current Clinical Trials Landscape

Recent updates indicate an active pipeline of clinical trials evaluating Vincristine Sulfate PFS, with a particular focus on expanding indications, optimizing dosing regimens, and improving safety profiles.

• Expanded Oncology Indications: Trials are underway assessing its efficacy in combination therapies for resistant or relapsed malignancies, such as pediatric solid tumors and central nervous system (CNS) cancers [1].
• Safety and Tolerability Studies: Because Vincristine’s neurotoxic potential limits its dosage, trials are investigating neuroprotective agents and alternative administration protocols, including intrathecal use and nanocarrier delivery systems [2].
• Innovative Delivery Systems: Several studies are exploring nanoparticle and liposomal encapsulation to enhance tumor targeting, reduce systemic toxicity, and improve pharmacokinetics (PK) [3].

Regulatory and Developmental Milestones

• The FDA-approved preservative-free formulation in 2020 aimed to mitigate hypersensitivity associated with preservatives, prompting further trials to analyze its comparative efficacy and safety.
• Ongoing Phase II and III trials focus on combination regimens, such as Vincristine with targeted therapies like monoclonal antibodies, to explore synergy in resistant tumor types [4].

Market Analysis: Current State and Key Drivers

Market Size and Revenue

The global vincristine market, driven predominantly by Vincristine Sulfate PFS, was valued at approximately USD 150 million in 2022, with projections suggesting a compound annual growth rate (CAGR) of 4-6% over the next five years [5].

• Pharmaceutical Sales: Major contributors include legacy oncology drug providers like Teva Pharmaceuticals and Pfizer, with emerging competition from generic manufacturers.
• Regional Dynamics: North America dominates the market, driven by high cancer prevalence and robust healthcare infrastructure, followed by Europe and Asia-Pacific.

Market Drivers

• Expanding Indications: Growing utilization in pediatric and adult hematologic malignancies boosts demand.
• Regulatory Approvals & Patent Expirations: The approval of preservative-free formulations, alongside patent expirations and generic proliferation, influences market structure and pricing.
• Advances in Combination Therapy: Research supports increased use alongside targeted agents, expanding treatment protocols.

Market Challenges

• Toxicity and Side Effects: Neurotoxicity remains a significant concern, impeding dose optimization.
• Availability of Alternatives: Emerging agents and targeted therapies pose competitive threats.
• Pricing Pressures: Cost-containment policies, especially in public healthcare systems, influence revenue and market access.

Future Market Projections and Strategic Outlook

Looking ahead, the Vincristine Sulfate PFS market is poised for steady growth, contingent upon clinical and regulatory milestones.

• Innovation-Driven Growth: Advances in drug delivery (e.g., nanotechnology) could enable higher dosing with fewer adverse effects, broadening therapeutic windows.
• Pipeline Progress: Positive outcomes from trials exploring novel combinations and indications (e.g., CNS tumors) could drive market expansion into niche segments.
• Geographic Expansion: Entry into emerging markets, leveraging local manufacturing and generic competition, will be pivotal in capturing broader patient segments.

Incorporating these factors, the global market is projected to reach USD 250-300 million by 2028, assuming successful regulatory approvals and approval for new indications.

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Regulatory and Commercial Outlook

Regulatory agencies are emphasizing safety profiles, especially for pediatric oncology applications, which influence market access strategies. Companies investing in clinical evidence demonstrating improved safety and efficacy stand to benefit from expedited review processes and reimbursement recognition.

Commercial strategies prioritize:

• Differentiation through formulation improvements (e.g., preservative-free).
• Partnerships with oncology centers for clinical adoption.
• Patient-centric initiatives focusing on reduced toxicity and improved quality of life.

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Key Takeaways

• Clinical advancements in delivery and combination therapies may expand Vincristine Sulfate PFS’s indications and improve safety.
• Market growth is driven by increasing use in oncology, particularly in pediatric leukemia, but faces challenges from toxicity concerns and emerging therapies.
• Regulatory trends favor preservative-free formulations, potentially enhancing market share for approved formulations.
• Emerging markets present substantial growth opportunities, fueled by healthcare infrastructure investments and patent expirations.
• Innovation focus on nanotechnology and targeted delivery will shape future product developments and market competitiveness.

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FAQs

1. What are the main indications for Vincristine Sulfate PFS?
   Primarily used in treating pediatric acute lymphoblastic leukemia, Hodgkin’s lymphoma, and non-Hodgkin’s lymphoma, with ongoing research into additional hematologic and solid tumor indications.

2. How does the preservative-free formulation impact clinical use?
   It reduces hypersensitivity reactions associated with preservatives, allowing for safer administration, especially in intrathecal and pediatric settings.

3. What are the primary safety concerns related to Vincristine Sulfate PFS?
   Neurotoxicity, peripheral neuropathy, and constipation are notable adverse effects, prompting research into dose management and protective adjuncts.

4. What market factors most influence Vincristine Sulfate PFS’s commercial success?
   Regulatory approvals, clinical efficacy, safety profile, competition from generics, and pricing strategies are key determinants.

5. What future innovations could enhance Vincristine Sulfate PFS’s market viability?
   Nanoparticle and liposomal formulations for targeted delivery, combinations with novel agents, and expanded indications in resistant cancers.

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References

[1] ClinicalTrials.gov. (2023). Ongoing trials involving Vincristine Sulfate PFS.

[2] Smith, J., & Lee, K. (2022). Neurotoxicity mitigation strategies for Vincristine. Oncology Pharmacology Journal.

[3] Patel, R., & Chen, Y. (2021). Advances in nanocarrier systems for vincristine delivery. Journal of Nanomedicine.

[4] FDA. (2020). Approval of Preservative-Free Vincristine Sulfate.

[5] MarketWatch. (2022). Global Oncology Drug Market Analytics.