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

Sargramostim: Clinical Trials Update, Market Analysis, and Projection

Last updated: April 24, 2026

Where is sargramostim in clinical development?

Sargramostim (recombinant human granulocyte-macrophage colony-stimulating factor, GM-CSF) is a legacy biologic with no broadly documented, high-complexity late-stage pipeline expansions in the public domain during the most recent period of record. Public clinical activity centers on label-maintenance studies, real-world evidence frameworks, and country-specific or sponsor-specific protocol updates rather than large, globally pivotal phase programs.

Key clinical-trial data points (public registry)

The most consistently referenced public source for trial status is ClinicalTrials.gov. Trial counts and timelines change frequently; only registry-indexed, explicitly dated entries can be reliably mapped to “update” status. Current publicly surfaced activity is concentrated in:

• oncology support contexts (including neutrophil recovery frameworks around hematologic malignancies),
• hematologic support and infection-risk mitigation approaches,
• combination and dosing-regimen refinements in specific indications.

Actionable takeaway: For investment-grade pipeline visibility, sargramostim’s near-term value trajectory depends more on label positioning, payer access, and competitive substitution than on a clearly identifiable late-stage, global phase program.

What is the current competitive landscape?

Sargramostim competes in the “GM-CSF / myeloid growth factor” space and indirectly in supportive oncology where neutropenia prophylaxis and neutrophil recovery are managed. The practical competitive set is defined less by a single molecule and more by how clinicians and payers select agents based on:

• dosing convenience and administration patterns,
• clinical practice guidelines for neutropenia management,
• biosimilar penetration and WAC dynamics for GM-CSF analogs,
• formulary coverage and prior authorization thresholds.

Competitive pressure tends to concentrate on:

• formulary placement versus pegfilgrastim and filgrastim biosimilars in neutropenia prophylaxis and treatment support,
• localized retention policies for branded GM-CSF where historically adopted,
• price-to-value comparisons driven by administration and bundled care economics.

Where is sargramostim used in practice?

Sargramostim’s core value proposition historically ties to GM-CSF biology: it drives neutrophil and monocyte lineage support. In oncology supportive care and certain transplant-related or immune-compromised contexts, clinicians use GM-CSF to mitigate infection risk associated with neutropenia or immune suppression. Label scope varies by jurisdiction and product lineage (originator versus biosimilar where applicable).

Actionable takeaway: The most durable commercial driver is continued inclusion in supportive care pathways where GM-CSF use fits local guideline patterns and reimbursement rules.

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What does the market look like for sargramostim?

Market definition

For market modeling, sargramostim is typically assessed within:

• growth factors used for supportive cancer care,
• hematology supportive therapies used to manage neutropenia-related risk,
• transplant and immune recovery support in defined therapeutic areas.

Because sargramostim’s demand is highly sensitive to supportive oncology practice, the revenue pool is driven by:

• chemotherapy regimen intensity and guideline adherence,
• local stewardship rules for prophylaxis,
• substitution behavior among filgrastim/pegfilgrastim/biosimilars versus GM-CSF products.

Pricing and reimbursement dynamics that matter

Commercial performance for sargramostim is influenced by three variables that consistently impact biologic supportive care:

1. Biosimilar availability and competition in filgrastim/pegfilgrastim classes.
2. Formulary restriction: GM-CSFs often face narrower coverage than broader, high-volume biosimilar ecosystems.
3. Net price erosion: even for branded agents, WAC is not a good proxy for realized revenue; rebates and access programs determine outcomes.

Demand sensitivity

Sargramostim demand tends to be:

• lower-volume, higher-variability than pegfilgrastim-heavy prophylaxis markets,
• more guideline- and institution-dependent,
• more sensitive to hematology/oncology care volumes than to incremental adoption because it is not usually the default prophylactic option where biosimilars dominate.

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How should revenue be projected for sargramostim?

Without a current, auditable dataset for global sales by year and product line, a projection must be structured around scenario logic tied to observable levers. The projection framework below is designed for decision use in R&D and investing.

Projection drivers

Key levers that move sargramostim revenue:

• Formulary access (national and hospital level)
• Guideline positioning in neutropenia prophylaxis and treatment support
• Competition from biosimilars (especially filgrastim/pegfilgrastim ecosystems)
• Clinical adoption stability in the narrower GM-CSF-appropriate segments

Scenario model (semi-quantitative)

Use three scenarios for 3-year outlook post-base year. Each scenario includes the revenue direction expected based on typical supportive care substitution dynamics:

Scenario	Assumptions	Revenue trajectory (direction)	Primary risk
Downside	tighter formulary controls, stronger substitution to biosimilars	Decline	net price and access contraction
Base case	stable guideline fit in selected indications; modest net price pressure	Flat to slight decline	competitive substitution
Upside	improved access in targeted segments; stable prescribing behavior	Low growth	limited incremental patient pool

What breaks the model?

Sargramostim revenue would materially deviate upward if any of these occur:

• an approved new indication that expands usage beyond current supportive care patterns,
• a meaningful competitive discontinuation or supply interruption across key alternatives,
• a broad payer reversal that expands coverage from restricted to preferred status.

Actionable takeaway: For near-to-mid term forecasting, the dominant forecast signal is not “clinical novelty.” It is access, substitution, and guideline adherence.

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What do registries and regulatory touchpoints imply for near-term prospects?

Regulatory and registry visibility for sargramostim indicates a mature product with ongoing clinical reference activity. Mature products usually show:

• fewer high-impact phase transitions,
• more incremental studies (dose, population subgroups, safety/efficacy characterization),
• less global “pipeline-driven” value creation.

Actionable takeaway: Unless a new, clearly documented late-stage program or label expansion is present, sargramostim’s growth outlook is access-driven.

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Key Takeaways

• Sargramostim is a mature GM-CSF biologic with ongoing public clinical activity that is not clearly dominated by globally pivotal late-stage expansion in the most recent registry-visible record.
• The commercial future is primarily governed by supportive oncology access: formulary inclusion, payer coverage rules, and substitution dynamics versus biosimilar filgrastim/pegfilgrastim classes.
• Revenue projection should be modeled through scenario logic centered on net price and access rather than expecting pipeline-driven step changes.
• Upside outcomes require coverage expansion, guideline repositioning, or label expansion beyond current supportive care patterns.

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FAQs

1. Is sargramostim still being studied in clinical trials?
   Yes. Clinical activity persists, with registry-indexed studies typically focused on supportive care contexts and regimen or population refinement rather than clearly dominant late-stage, global pivotal programs.

2. What most affects sargramostim sales in the short term?
   Formulary access, net pricing after rebates, and prescribing substitution patterns versus biosimilar myeloid growth factor alternatives.

3. How does biosimilar competition impact sargramostim?
   Biosimilar ecosystems in filgrastim and pegfilgrastim frequently steer default neutropenia prophylaxis and treatment support away from narrower GM-CSF usage settings.

4. What would create an upside revenue deviation for sargramostim?
   A new label that expands usage beyond current supportive care pathways or a payer shift that moves sargramostim into preferred coverage tiers at scale.

5. How should investors model sargramostim given its maturity?
   Use base/downside/upside scenarios anchored on access and net pricing. Treat pipeline expansion as a secondary driver unless new late-stage visibility is demonstrable.

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References

[1] U.S. National Library of Medicine. ClinicalTrials.gov. https://clinicaltrials.gov/
[2] European Medicines Agency (EMA). Public assessment and product information resources for GM-CSF and related supportive care medicines. https://www.ema.europa.eu/
[3] U.S. Food and Drug Administration (FDA). Drugs@FDA database for product regulatory status and labeling. https://www.accessdata.fda.gov/scripts/cder/daf/