CLINICAL TRIALS PROFILE FOR VINCRISTINE SULFATE

505(b)(2) Clinical Trials for Vincristine Sulfate

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

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.

Clinical Trial Conditions for Vincristine Sulfate

Condition Name

Condition Name for Vincristine Sulfate
Intervention	Trials
Lymphoma	139
Leukemia	72
Neuroblastoma	33
Acute Lymphoblastic Leukemia	27

Condition MeSH

Condition MeSH for Vincristine Sulfate
Intervention	Trials
Lymphoma	216
Leukemia	137
Leukemia, Lymphoid	124
Precursor Cell Lymphoblastic Leukemia-Lymphoma	123

Clinical Trial Locations for Vincristine Sulfate

Trials by Country

Trials by Country for Vincristine Sulfate
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
Location	Trials
California	205
New York	199
Texas	177
Illinois	175
Ohio	172

Clinical Trial Progress for Vincristine Sulfate

Clinical Trial Phase

Clinical Trial Phase for Vincristine Sulfate
Clinical Trial Phase	Trials
Phase 4	1
Phase 3	162
Phase 2/Phase 3	9

Clinical Trial Status

Clinical Trial Status for Vincristine Sulfate
Clinical Trial Phase	Trials
Completed	258
Active, not recruiting	68
Unknown status	66

Clinical Trial Sponsors for Vincristine Sulfate

Sponsor Name

Sponsor Name for Vincristine Sulfate
Sponsor	Trials
National Cancer Institute (NCI)	286
Children's Oncology Group	95
Children's Cancer and Leukaemia Group	24

Sponsor Type

Sponsor Type for Vincristine Sulfate
Sponsor	Trials
Other	557
NIH	297
Industry	83

Clinical Trials Update, Market Analysis, and Projection for Vincristine Sulfate

Last updated: October 28, 2025

Introduction

Vincristine Sulfate, a cornerstone in oncology treatment, particularly for hematologic malignancies, remains a critical chemotherapeutic agent. Its role in treating conditions such as leukemia, lymphoma, and other cancers underscores the importance of ongoing clinical assessments, market dynamics, and future growth potential. This analysis synthesizes recent clinical trial developments, evaluates current market performance, and projects future trends to inform stakeholders and decision-makers.

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Clinical Trials Update

Recent Clinical Trials and Development Landscape

Vincristine Sulfate, a natural alkaloid derived from Catharanthus roseus, continues to be scrutinized through various clinical trials aimed at optimizing its efficacy and expanding its therapeutic scope. Recent developments focus on combination therapies, novel delivery methods, and managing adverse effects.

1. Combination Therapy Trials
   Several studies investigate Vincristine in conjunction with targeted agents and immunotherapies. For example, a Phase II trial exploring Vincristine combined with dasatinib in pediatric leukemia reported promising response rates, emphasizing synergistic benefits and manageable safety profiles (clinicaltrials.gov identifier: NCT04374778).

2. Formulation Innovations
   Researchers are exploring liposomal encapsulation and nanoparticle-based delivery to improve bioavailability and reduce neurotoxicity, a common adverse effect. Early preclinical trials demonstrate progress toward reducing peripheral neuropathy while maintaining antitumor activity.

3. Treatment of Solid Tumors
   Although traditionally used for hematological cancers, recent Phase I/II trials assess Vincristine’s efficacy in solid tumors such as neuroblastoma and sarcomas. Results suggest potential in combination regimens, though substantial clinical validation remains pending.

Safety and Efficacy Insights

• Ongoing trials reinforce Vincristine’s efficacy in standard dosing protocols; however, neurotoxicity remains a significant concern.
• Novel strategies aim to mitigate side effects through dose adjustments and adjunct therapies.
• Biomarker-guided therapy trials are in nascent phases, seeking to personalize dosing and improve tolerability.

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Market Analysis

Current Market Dynamics

Vincristine Sulfate remains a vital component of chemotherapy protocols globally. Despite its longstanding use, recent patent expirations, and generic competition have influenced market penetration and pricing strategies:

• Market Size and Revenue
  The global oncology drug market, valued at approximately USD 229 billion in 2022, includes a substantial subset dedicated to chemotherapeutic agents like Vincristine. The drug’s estimated global revenue is approximately USD 250-300 million annually, with North America accounting for over 65% due to high cancer prevalence and access to advanced healthcare (IQVIA).

• Major Players
  Most formulations are supplied by generic manufacturers such as Pfizer, Teva, and Sun Pharmaceutical Industries. Patent expirations and the advent of biosimilars are expected to further intensify price competition.

• Distribution Channels
  Hospital pharmacies dominate distribution, with chemotherapy regimens administered primarily within hospital settings. Access disparities exist in emerging markets due to cost and infrastructure limitations.

Market Challenges and Opportunities

• Challenges

  • Neurotoxicity and adverse effects limit dosing and patient adherence.
  • Regulatory constraints around manufacturing standards and quality control.
  • Emerging resistance mechanisms could compromise efficacy in certain tumor types.

• Opportunities

  • Development of improved formulations to enhance safety profiles.
  • Integration into combination therapies for personalized medicine approaches.
  • Expansion into treating solid tumors and rare cancers through clinical validation.

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Market Projection and Future Trends

Forecast for the Next 5-10 Years

Based on current clinical development trajectories, regulatory landscapes, and market dynamics, the Vincristine Sulfate market is projected to grow at a moderate CAGR of 3-4%, reaching USD 340-400 million by 2030.

Key Drivers

• Clinical Expansion
  Ongoing trials exploring combination therapies and new indications will broaden its therapeutic footprint.
• Biosimilar and Generic Competition
  Increased availability of cost-effective formulations will drive adoption in emerging markets, expanding access.
• Technological Innovations
  Advances in nanoparticle delivery are expected to reduce toxicity and improve patient outcomes, potentially enabling higher dosing and enhanced efficacy.

Potential Disruptors

• Development of novel targeted therapies and immunotherapies may reduce reliance on traditional chemotherapeutic agents.
• Regulatory pressures regarding side effect management may necessitate formulation reforms.
• Market entry of next-generation alkaloids with improved safety profiles.

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Regulatory and Commercial Outlook

Regulatory agencies like the FDA and EMA continue to approve combination regimens involving Vincristine, with evolving guidelines emphasizing personalized approaches. Commercially, investments in research to mitigate adverse effects and improve delivery are expected to sustain market relevance.

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Conclusion

Vincristine Sulfate remains a vital chemotherapeutic agent with ongoing clinical trials exploring expanded indications and improved formulations. The market, although challenged by generic competition and toxicity concerns, shows steady growth driven by clinical innovation and increasing global cancer prevalence. Strategic investments into formulation technology and combination therapies are essential for future market expansion.

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Key Takeaways

• Clinical Validation Continues: New trials focus on combination therapies, novel delivery methods, and expanding indications, promising improved efficacy and safety profiles.
• Market Stability with Growth Potential: Despite generic competition, the global demand remains strong, especially in emerging markets where access continues to improve.
• Innovation as a Catalyst: Formulation advancements, such as liposomal encapsulation and nanoparticles, are critical for mitigating toxicity and enabling higher dosing.
• Market Expansion Opportunities: Treating solid tumors and rare cancers offers significant growth avenues supported by clinical research.
• Regulatory Dynamics: Evolving guidelines favor personalized medicine approaches, emphasizing biomarker-driven therapies and safety management.

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FAQs

1. What are the latest clinical trial developments involving Vincristine Sulfate?
   Recent trials explore combination regimens with targeted therapies, novel delivery formats to reduce neurotoxicity, and expanding indications to solid tumors like neuroblastoma and sarcomas.

2. How does Vincristine Sulfate compare to newer oncology agents?
   As a longstanding chemotherapy drug, Vincristine remains essential but faces competition from targeted and immunotherapies that offer improved safety and specificity. Nonetheless, its cost-effectiveness and proven efficacy sustain its clinical relevance.

3. What are the main safety concerns associated with Vincristine Sulfate?
   Neurotoxicity, peripheral neuropathy, and myelosuppression are primary adverse effects. Research aims to refine dosing and develop formulations mitigating these issues.

4. What is the forecasted market growth for Vincristine Sulfate?
   The market is expected to grow at a CAGR of approximately 3-4% over the next decade, driven by clinical expansion and formulation improvements.

5. Are biosimilars or generics impacting Vincristine Sulfate’s market share?
   Yes. Multiple generics have entered the market post-patent expiry, increasing affordability and access, especially in emerging economies, thereby influencing overall market dynamics.

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Sources:

1. clinicaltrials.gov – Design and status of ongoing Vincristine studies.
2. IQVIA. Global Oncology Market Review, 2022.
3. Market Research Future. Chemotherapy Drugs Market Insights, 2022.
4. FDA and EMA regulatory publications on oncology drugs.