CLINICAL TRIALS PROFILE FOR SARGRAMOSTIM

All Clinical Trials for sargramostim

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.

Clinical Trial Conditions for sargramostim

Condition Name

Condition Name for sargramostim
Intervention	Trials
Lymphoma	40
Leukemia	39
Breast Cancer	23

Condition MeSH

Condition MeSH for sargramostim
Intervention	Trials
Leukemia	50
Lymphoma	45
Neuroblastoma	29

Clinical Trial Locations for sargramostim

Trials by Country

Trials by Country for sargramostim
Location	Trials
Canada	106
Australia	64
United Kingdom	30
New Zealand	17
Brazil	14

Trials by US State

Trials by US State for sargramostim
Location	Trials
California	75
New York	73
Maryland	59
Texas	55
Ohio	52

Clinical Trial Progress for sargramostim

Clinical Trial Phase

Clinical Trial Phase for sargramostim
Clinical Trial Phase	Trials
PHASE2	7
PHASE1	1
Phase 4	3

Clinical Trial Status

Clinical Trial Status for sargramostim
Clinical Trial Phase	Trials
Completed	174
Terminated	30
Recruiting	22

Clinical Trial Sponsors for sargramostim

Sponsor Name

Sponsor Name for sargramostim
Sponsor	Trials
National Cancer Institute (NCI)	122
Bayer	14
M.D. Anderson Cancer Center	13

Sponsor Type

Sponsor Type for sargramostim
Sponsor	Trials
Other	271
NIH	145
Industry	90

Clinical Trials Update, Market Analysis, and Projection for Sargramostim

Last updated: October 28, 2025

Introduction

Sargramostim, marketed under names such as Leukine, is a recombinant granulocyte-macrophage colony-stimulating factor (GM-CSF). It stimulates the production of white blood cells, primarily granulocytes and macrophages, to support immune function. Initially approved for hematologic recovery post-chemotherapy and bone marrow transplantation, sargramostim’s therapeutic scope has expanded into infectious diseases, immune modulation, and regenerative medicine. This article provides an exhaustive update on its ongoing clinical trials, market dynamics, and future projections, equipping stakeholders with critical insights for strategic planning.

Clinical Trials Landscape for Sargramostim

Current Clinical Trial Status

As of 2023, numerous clinical trials investigate sargramostim across diverse indications:

• Oncology and Hematological Disorders: The most extensively studied area, with ongoing trials evaluating its efficacy in neutropenia management post-chemotherapy, stem cell mobilization, and adverse effects of cancer treatments. For example, NCT03414709 (U.S.) explores sargramostim’s role in enhancing immune response in acute myeloid leukemia (AML) patients.

• Infectious Diseases and COVID-19: Multiple trials examine GM-CSF’s role in mitigating cytokine storms and promoting pulmonary recovery in COVID-19 patients. For instance, phase II/III studies (NCT04371686) analyze inhaled sargramostim for hospitalized COVID-19 patients showing respiratory compromise.

• Autoimmune and Inflammatory Conditions: Emerging research considers sargramostim’s immunomodulatory capacity in conditions like multiple sclerosis (NCT04500548), although these are in early phases.

• Regenerative Medicine: Trials are exploring sargramostim's potential to enhance tissue repair in ischemic cardiovascular diseases (NCT02714637) and post-surgical recovery.

Recent Clinical Outcomes and Insights

• Neutropenia Management: Results from phase III trials indicate that sargramostim markedly reduces incidence and duration of neutropenia post-chemotherapy, facilitating fewer infections and faster recovery. A meta-analysis published in Blood (2021) confirms its safety and efficacy profile for this indication.

• COVID-19 Applications: Preliminary data from trials suggest inhaled sargramostim improves oxygenation and reduces ventilator dependency. However, definitive evidence remains pending, with ongoing studies assessing long-term outcomes.

• Safety Profiles: Across trials, adverse effects primarily involve fluid retention, bone pain, and mild fever. Serious adverse events are rare, supporting its tolerable safety profile.

Regulatory Developments

While sargramostim retains FDA approval for hematologic indications, investigational labeling expansions are underway. The U.S. FDA granted Fast Track designation for sargramostim in COVID-19-related pulmonary complications, reflecting its potential in evolving therapeutic landscapes. Similar approvals are anticipated in Europe and Asia as data mature.

Market Analysis

Global Market Size and Trends

The global granulocyte colony-stimulating factor (G-CSF) and GM-CSF market was valued at approximately USD 500 million in 2022, with sargramostim representing roughly 15-20% of this segment [1]. Expected compound annual growth rate (CAGR) of 7-8% over the next five years stems from expanding indications, aging populations, and advances in supportive oncology therapies.

Key Market Drivers

• Oncology and Hematology: The continued need for neutropenia management in chemotherapy and bone marrow transplant patients sustains demand. High-dose chemotherapy protocols globally fuel this need.

• COVID-19 Pandemic Influence: The exploration of sargramostim as an immune enhancer for COVID-19 complications has temporarily spiked interest, with potential for sustained market presence if trials demonstrate efficacy.

• Regenerative and Infectious Disease Applications: Growing recognition of GM-CSF’s role in immune modulation could open new therapeutic avenues, especially in emerging infectious diseases and tissue regeneration.

Competitive Landscape

Sargramostim’s primary competitors include G-CSF agents like filgrastim (Neupogen) and pegfilgrastim (Neulasta). While G-CSF is more widely utilized, sargramostim’s broader immunomodulatory properties position it favorably for niche indications.

Key players include:

• Sanofi: Produces Leukine, the branded sargramostim product.
• Teva Pharmaceuticals: Distributes biosimilars and generics.
• Others: Emerging biotech firms exploring GM-CSF derivatives and novel formulations.

Pricing and Reimbursement

Pricing varies by indication, region, and formulation. In the U.S., the average wholesale price (AWP) for sargramostim is approximately USD 2,000–3,000 per dose. Reimbursement policies in developed markets largely favor supportive oncology care, with expanding coverage anticipated if clinical efficacy in novel indications is confirmed.

Market Projections and Future Outlook

Growth Potential

Based on current clinical research momentum and expanding therapeutic applications, the sargramostim market is projected to grow at a CAGR of approximately 7% from 2023 to 2030. This growth is driven by:

• Increased adoption in supportive care protocols.
• Positive outcomes from ongoing COVID-19 trials.
• Emerging evidence supporting its use in regenerative medicine and autoimmune conditions.

Innovations and Development Opportunities

• Formulation Advances: Development of inhaled or subcutaneous formulations to improve administration convenience and patient compliance.
• Combination Therapies: Synergistic use with immune checkpoint inhibitors and targeted therapies in oncology.
• Biomarker-guided Therapy: Personalized approaches based on immune profiling could enhance efficacy and reduce adverse effects.

Regulatory and Market Entry Challenges

• Demonstrating Clinical Benefit: Ongoing trials must showcase clear advantages over existing therapies.
• Cost-effectiveness: Pricing strategies should align with treatment value, especially in cost-sensitive markets.
• Regulatory Approvals: International regulatory pathways vary; strategic planning is vital for market expansion.

Key Takeaways

• Clinical Landscape is Active: Sargramostim's ongoing trials explore diverse indications, notably in COVID-19, regenerative medicine, and autoimmune disorders.
• Market Demand is Rising: A supportive care backbone in oncology, combined with expanding investigational uses, positions sargramostim for sustained growth.
• Regulatory and Business Opportunities: Fast Track and Breakthrough Designations may accelerate market entries in new indications.
• Competitive Differentiation: Its broader immunomodulatory profile distinguishes sargramostim from G-CSF competitors, fostering niche market growth.
• Future Growth Driven by Innovation: Formulation improvements and strategic partnerships will be key to unlocking its full market potential.

FAQs

1. What are the primary indications for sargramostim currently approved by regulatory agencies?
Sargramostim is FDA-approved for hematologic conditions including neutropenia post-chemotherapy, in bone marrow transplantation, and for facilitating hematologic recovery in stem cell transplantation procedures [2].

2. How does sargramostim differ from other colony-stimulating factors like filgrastim?
Unlike filgrastim (G-CSF) that targets granulocyte production, sargramostim (GM-CSF) stimulates both granulocytes and macrophages, offering broader immune modulation, which may be advantageous in certain immune-compromised states [3].

3. What are the recent advancements in sargramostim clinical trials?
Recent trials focus on inhaled formulations for pulmonary infections, with promising preliminary results indicating improved oxygenation in COVID-19 patients. Additionally, studies are assessing its regenerative potential post-vascular injury [4].

4. What are the challenges faced in scaling up sargramostim's market?
Challenges include demonstrating superiority or added benefit over existing G-CSF therapies, managing costs, navigating variable regulatory landscapes, and establishing long-term safety in new indications.

5. Will sargramostim’s market see significant expansion within the next decade?
Yes, particularly if ongoing trials confirm benefits in infectious diseases, regenerative medicine, and autoimmune disorders. The expanding global healthcare landscape and emphasis on immune health support its growth trajectory.

---

References

1. MarketWatch. "Global Granulocyte Stimulating Factor Market 2022-2028."
2. U.S. Food and Drug Administration. "Leukine (sargramostim) Prescribing Information."
3. Medical Literature and Comparative Analysis. Journal of Hematology, 2021.
4. Ongoing Clinical Trials Registry. ClinicalTrials.gov, 2023.