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.
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.
NCT00000694 ↗	A Phase I Trial of Recombinant Human Granulocyte-Macrophage Colony Stimulating Factor (rHuGM-CSF), Recombinant Alpha Interferon and Azidothymidine (AZT) in AIDS-Associated Kaposi's Sarcoma	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1	1969-12-31	To define the best doses of sargramostim ( granulocyte-macrophage colony-stimulating factor; GM-CSF ), interferon alfa-2b ( IFN-A2b ), and zidovudine ( AZT ) to give together in patients with AIDS-associated Kaposi's sarcoma ( KS ), to learn about the side effects of these drugs when they are given together for 8 weeks, and to find out whether the combination of GM-CSF, IFN-A2b, and AZT has any effect on KS, HIV, or the immune system. Studies show that IFN-A2b can cause KS tumors to shrink or disappear in about 30 percent of patients. IFN-A2b can greatly reduce the growth of the HIV virus in test tube experiments and perhaps in patients. AZT has also been shown to reduce the growth of HIV and show improvements in the immune system with fewer infections. Test tube experiments show that when IFN-A2b and AZT are used together, they reduce the growth of the HIV virus much more effectively than when either drug is used alone. In recent studies of the combination of interferon alpha and AZT in patients with KS, more than 40 percent of the patients showed shrinkage of their tumors, and some showed evidence for suppression of HIV growth in the body. However, the combination of IFN-A2b with AZT often caused a marked lowering of the white blood cell (WBC) count, especially a type of WBC called the granulocyte (or neutrophil) which is important in the body's defense against infection. Recombinant human GM-CSF is a human protein which is produced in bacteria. It has been shown to cause an increase in the WBC count.
NCT00000711 ↗	Granulocyte-Macrophage Colony-Stimulating Factor and Zidovudine: A Phase I Study of Concurrent Administration in Patients With AIDS and Severe ARC	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1	1969-12-31	To administer colony-stimulating factor (GM-CSF) for 4 weeks to AIDS and advanced AIDS related complex (ARC) patients who have been receiving zidovudine (AZT) therapy, in order to obtain data on short-term effectiveness, safety, toxicity, pharmacokinetics, and tolerance of combined treatment with the two drugs. Persons infected with HIV virus may undergo a long latency or persistent virus blood levels which may be present before any symptomatic illness. These individuals could, therefore, benefit from therapy with an effective antiretroviral agent. AZT, which is a powerful inhibitor of human retrovirus, has been approved for management of patients with symptomatic HIV infection. GM-CSF not only stimulates the bone marrow, it enhances the function of mature blood cells and has been found to enhance the ability of AZT to suppress HIV replication in vitro (test tube). Combination therapy with GM-CSF and AZT may lower complications as well as the morbidity and mortality associated with HIV infection.

Clinical Trial Conditions for sargramostim

Condition Name

Condition Name for sargramostim
Intervention	Trials
Lymphoma	40
Leukemia	39
Breast Cancer	23
Multiple Myeloma and Plasma Cell Neoplasm	18

Condition MeSH

Condition MeSH for sargramostim
Intervention	Trials
Leukemia	50
Lymphoma	45
Neuroblastoma	29
Leukemia, Myeloid	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 and market projection: development pipeline, regulatory status, and commercial outlook

Last updated: July 25, 2026

Sargramostim is a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) used to improve white blood cell recovery and related hematologic parameters in defined settings. Commercially, the key market questions are (1) what new indications or formulations are actively in late-stage development, (2) what biosimilar or competitive product pressure exists in GM-CSF, and (3) whether any new labeling expansion changes revenue durability.

Market projection hinges on how sargramostim is positioned versus shorter-acting G-CSFs for neutropenia and versus alternative GM-CSF regimens, as well as on whether upcoming trials broaden use beyond current practice patterns.

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Sargramostim clinical trials update: what studies are active, recruiting, or in late-stage?

Featured answer: A current, complete “active/recruiting/late-stage” sargramostim trial map cannot be produced from the information available in this chat. A precise update requires the exact trial list and statuses from registries (e.g., ClinicalTrials.gov) and the protocol-level endpoints.

What trial types typically drive sargramostim label expansion?

Sargramostim’s clinical strategy historically concentrates on hematologic recovery and survival-adjacent endpoints in oncology- and transplant-associated settings:

• Neutropenia and delayed immune reconstitution
• Bone marrow transplant recovery
• Chemotherapy- or radiation-induced myelosuppression
• Immunologic recovery post–hematopoietic stem cell transplant (HSCT)

What endpoints would matter for market impact?

For commercial sensitivity, endpoints tend to include:

• Time to neutrophil recovery (ANC)
• Incidence of severe infections and/or febrile neutropenia
• Duration of hospitalization
• Transplant engraftment metrics
• Overall survival and relapse-free survival in curative-intent oncology when feasible

Practical signal: late-stage recruitment beats early exploratory work

Even modest label expansions can be meaningful for a specialized hospital-administered biologic like sargramostim, but only if trials:

• Use outcomes aligned to FDA labeling (hematologic recovery, clinically meaningful infection outcomes)
• Enroll in populations with clear unmet need or high current treatment failure rates
• Demonstrate consistent effect in subgroup analyses that map to prescribing cohorts (HSCT centers, oncology networks)

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What is the FDA regulatory status of sargramostim (brand, labels, and Orange Book listings)?

Featured answer: A definitive Orange Book status cannot be stated without access to the current FDA listing set in this chat context. A full regulatory posture requires the approved product name(s), strength/dosage forms, listed patents, and exclusivity expirations.

Label and use profile that typically supports durability

Sargramostim’s regulatory and commercial durability generally depends on:

• Whether the approved indications remain narrow but consistent in high-utilization care settings (transplant conditioning, infection-risk cohorts)
• Hospital formulary adoption cycles and procurement patterns for GM-CSF biosupplies
• Competition dynamics in the GM-CSF space

Orange Book and exclusivity questions that drive generic/biosimilar risk

For market planning, the decisive regulatory items are:

• Patent-protected processes or formulations
• Method-of-use claims tied to dosing schedules
• Exclusivity periods (if any) that block approval pathways
• Whether the product is treated as a biologic with biosimilar routes versus a small-molecule generics framework (sargramostim is generally regulated as a biologic)

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How strong is the patent estate for sargramostim, and when do patents expire?

Featured answer: A complete, accurate sargramostim patent-expiration timeline cannot be generated here without the specific FDA/Orange Book patent numbers, application identifiers, and assignees.

What to map for infringement and entry risk

For sargramostim, the patent estate analysis should separate:

• Composition and formulation patents (including stabilizers, reconstitution formats, concentration-specific claims)
• Manufacturing/process patents (cell line, fermentation, purification, refolding if relevant)
• Method-of-use patents (indications, dosing, timing around chemo or transplant)
• Device or kit-related patents (where applicable for administration convenience)

How expiration timing affects market share

Even when clinical evidence is stable, market share shifts around entry windows:

• Biosimilar launch lead time (CMC, analytics, comparability, bridging studies)
• Patent litigation timing and settlement “carve-outs” for specific presentations
• Payer formulary changes after biosimilar pricing becomes available

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What generic or biosimilar entry risks exist for sargramostim?

Featured answer: An entry-risk call cannot be made without the current list of biosimilar/generic challengers, their FDA application statuses, and any litigations or stays.

How biosimilar entry would typically play out for GM-CSF

Key risk factors:

• Ability to demonstrate high similarity for sequence, glycosylation patterns, and potency assays
• Manufacturing robustness for consistent biological activity
• Whether method-of-use protections constrain substitution even after product approval
• Interchangeability designation relevance in hospital procurement

Market reaction to first biosimilar

When a biosimilar lands in hospital biosupply chains:

• Procurement shifts tend to be fastest where contracts include automatic replacement clauses
• Clinical switching barriers depend on physician experience and inventory overlap windows
• Pricing discounts often start modest and widen after subsequent competition

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How does sargramostim compare with competing GM-CSF and neutropenia agents on efficacy and adoption?

Featured answer: A defensible comparative analysis requires specific trial comparisons and guideline positions that are not available in this chat context.

Competitive set to consider in market modeling

Sargramostim competes indirectly and sometimes directly with:

• G-CSFs (filgrastim, pegfilgrastim, biosimilar variants)
• Other hematopoietic growth factors depending on country formularies and transplant protocols
• Alternative supportive care pathways (antibiotic prophylaxis strategies, dose modifications)

Adoption drivers that matter more than head-to-head potency

Market uptake often correlates with:

• Guideline inclusion in specific clinical pathways (transplant protocols, chemo intensity strata)
• Ease of administration and supply reliability
• Costs under DRG/packaging rules and hospital budgeting
• Prescriber familiarity and center-level protocols

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What formulations and dosing schedules are protected for sargramostim, and do they block substitution?

Featured answer: A substitution-blocking assessment cannot be produced without a listing of specific protected presentations, dosage strengths, and method-of-use claim scope.

Formulation-specific commercialization implications

For a biologic like sargramostim, formulation matters commercially:

• Reconstitution constraints and stability windows affect pharmacy operations
• Dose titration and vial size affect wastage and pharmacy purchasing economics
• Storage requirements drive inventory management costs

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What patent litigation affects sargramostim, and what settlements or injunctions matter for entry timing?

Featured answer: A litigation and settlement chronology cannot be listed without the case docket identifiers and outcomes.

What to track for a litigation-based timeline

For a biologic patent landscape, timelines are usually driven by:

• Rule 4 and early motions
• Claim construction outcomes for “core” method-of-use claims
• Settlement effective dates and “design-around” approvals
• Any appellate stay or post-judgment injunction posture

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Clinical and commercial market projection for sargramostim: how big is the revenue opportunity?

Featured answer: A quantified market projection cannot be generated without baseline product-level revenue, unit volume, and forecast methodology inputs that are not available in this chat.

What typically determines revenue trajectory for sargramostim

A robust forecast model for sargramostim would be anchored to:

• Indication incidence: HSCT volume, chemo regimens tied to GM-CSF protocols, infection-risk prevalence
• Treatment rates: percentage of eligible patients receiving sargramostim versus alternative G-CSFs or supportive protocols
• Pricing: net price after rebates and hospital contract discounts
• Competitive erosion: biosimilar or alternative therapy penetration
• Supply continuity: any manufacturing disruptions affecting hospital purchasing

Scenario framework used in market planning

For investment and licensing, projection is usually scenario-based:

• Base case: stable label use with modest substitution toward competing agents in neutropenia
• Downside case: faster guideline shifts and biosimilar pressure in adjacent supportive care classes
• Upside case: label expansion or compelling trial outcomes in HSCT or high-risk chemo populations

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Geographic market analysis: where will sargramostim gain or lose share?

Featured answer: A geographic outlook cannot be provided precisely without regional approval/usage data and payer or formulary adoption metrics.

Geographic drivers

Market share variance typically comes from:

• Regulatory label breadth differences across jurisdictions
• HSCT center density and transplant incidence trends
• National tenders and hospital procurement rules for biologics
• Biosimilar regulatory environment and substitution policies

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Key takeaways

• Sargramostim’s commercial trajectory is primarily driven by hospital-centered adoption in specific hematologic recovery pathways rather than broad outpatient use.
• A complete “clinical trials update,” including recruiting/late-stage status, requires trial registry data not present in this chat context.
• A definitive regulatory and patent-expiration assessment, including Orange Book listings and entry/Paragraph IV-type risk, also requires FDA listing and litigation datasets not present here.
• Market projection depends on baseline product revenue/units, competition penetration, and indication incidence trends; those inputs are not available in this chat context.
• The highest-leverage business question for sargramostim is whether near-term trial outcomes will expand use beyond current practice patterns enough to offset competitive substitution by G-CSF and other supportive care options.

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FAQs

1. Is sargramostim used as first-line therapy for febrile neutropenia in current guidelines?
2. What hematopoietic recovery endpoints best predict FDA-label expansion for sargramostim-type GM-CSF therapies?
3. How do method-of-use patents affect biosimilar substitution for sargramostim in hospital formularies?
4. What supply and CMC comparability risks most commonly delay biosimilar launches for GM-CSF biologics?
5. How should investors model sargramostim revenue under scenarios of G-CSF biosimilar penetration?

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References

1. [No sources were cited because registry, FDA listing, patent, and litigation datasets were not provided in the prompt or chat context.]