CLINICAL TRIALS PROFILE FOR LEUKINE

All Clinical Trials for LEUKINE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00002594 ↗	Combination Chemotherapy Followed by Bone Marrow and/or Peripheral Stem Cell Transplantation in Treating Patients With Recurrent Medulloblastoma or CNS Germ Cell Tumors	Completed	National Cancer Institute (NCI)	Phase 2	1994-09-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so that they stop growing or die. bone marrow transplantation and peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy followed by bone marrow transplantation and/or peripheral stem cell transplantation in treating patients who have recurrent medulloblastoma or CNS germ cell tumors.
NCT00002594 ↗	Combination Chemotherapy Followed by Bone Marrow and/or Peripheral Stem Cell Transplantation in Treating Patients With Recurrent Medulloblastoma or CNS Germ Cell Tumors	Completed	Children's Oncology Group	Phase 2	1994-09-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so that they stop growing or die. bone marrow transplantation and peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy followed by bone marrow transplantation and/or peripheral stem cell transplantation in treating patients who have recurrent medulloblastoma or CNS germ cell tumors.
NCT00003093 ↗	Combination Chemotherapy in Treating Children With Neuroblastoma	Completed	National Cancer Institute (NCI)	Phase 3	1988-03-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells. Combination chemotherapy plus surgery may be an effective treatment for neuroblastoma. PURPOSE: This phase III trial is studying how well combination chemotherapy followed by surgery works in treating young patients with neuroblastoma.
NCT00003093 ↗	Combination Chemotherapy in Treating Children With Neuroblastoma	Completed	Children's Oncology Group	Phase 3	1988-03-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells. Combination chemotherapy plus surgery may be an effective treatment for neuroblastoma. PURPOSE: This phase III trial is studying how well combination chemotherapy followed by surgery works in treating young patients with neuroblastoma.
NCT00003199 ↗	Combination Chemotherapy and Peripheral Blood Stem Cell Transplant Followed By Aldesleukin and Sargramostim in Treating Patients With Inflammatory Stage IIIB or Metastatic Stage IV Breast Cancer	Completed	National Cancer Institute (NCI)	Phase 2	1997-11-01	This phase II trial studies how well giving combination chemotherapy and peripheral blood stem cell transplant followed by aldesleukin and sargramostim works in treating patients with inflammatory stage IIIB or metastatic stage IV breast cancer. Drugs used in chemotherapy, such as busulfan, melphalan, and thiotepa, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. A peripheral stem cell transplant may be able to replace blood-forming cells that were destroyed by chemotherapy. This may allow more chemotherapy to be given so that more tumor cells are killed. Aldesleukin may stimulate the white blood cells to kill breast cancer cells. Giving aldesleukin together with sargramostim may kill more tumor cells
NCT00003199 ↗	Combination Chemotherapy and Peripheral Blood Stem Cell Transplant Followed By Aldesleukin and Sargramostim in Treating Patients With Inflammatory Stage IIIB or Metastatic Stage IV Breast Cancer	Completed	Fred Hutchinson Cancer Research Center	Phase 2	1997-11-01	This phase II trial studies how well giving combination chemotherapy and peripheral blood stem cell transplant followed by aldesleukin and sargramostim works in treating patients with inflammatory stage IIIB or metastatic stage IV breast cancer. Drugs used in chemotherapy, such as busulfan, melphalan, and thiotepa, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. A peripheral stem cell transplant may be able to replace blood-forming cells that were destroyed by chemotherapy. This may allow more chemotherapy to be given so that more tumor cells are killed. Aldesleukin may stimulate the white blood cells to kill breast cancer cells. Giving aldesleukin together with sargramostim may kill more tumor cells

Clinical Trial Conditions for LEUKINE

Condition Name

Condition Name for LEUKINE
Intervention	Trials
Lymphoma	7
Breast Cancer	6
Crohn Disease	6
Recurrent Neuroblastoma	6

Condition MeSH

Condition MeSH for LEUKINE
Intervention	Trials
Leukemia	13
Neuroblastoma	13
Breast Neoplasms	11
Lymphoma	9

Clinical Trial Locations for LEUKINE

Trials by Country

Trials by Country for LEUKINE
Location	Trials
United States	636
Canada	61
Australia	48
United Kingdom	22
New Zealand	14

Trials by US State

Trials by US State for LEUKINE
Location	Trials
Texas	38
California	30
New York	29
Pennsylvania	22
Ohio	21

Clinical Trial Progress for LEUKINE

Clinical Trial Phase

Clinical Trial Phase for LEUKINE
Clinical Trial Phase	Trials
Phase 4	4
Phase 3	11
Phase 2/Phase 3	1

Clinical Trial Status

Clinical Trial Status for LEUKINE
Clinical Trial Phase	Trials
Completed	55
Terminated	16
Active, not recruiting	13

Clinical Trial Sponsors for LEUKINE

Sponsor Name

Sponsor Name for LEUKINE
Sponsor	Trials
National Cancer Institute (NCI)	40
Bayer	14
M.D. Anderson Cancer Center	11

Sponsor Type

Sponsor Type for LEUKINE
Sponsor	Trials
Other	128
Industry	62
NIH	51

Last updated: July 30, 2026

LEUKINE (sargramostim) clinical trials update, market analysis, and exclusivity-to-revenue projection

Executive summary: LEUKINE (sargramostim, GM-CSF) remains an established oncology and immunology biologic with a concentrated U.S. commercial footprint and episodic demand tied to specific indications, hospital acquisition cycles, and off-cycle procurement patterns. The near-term market path is driven by (1) current indication labels and competing GM-CSF biosimilars and branded analogs, (2) payer coverage and hospital formulary position, and (3) biologic lifecycle constraints including manufacturing continuity and any ongoing regulatory actions affecting specific lots/labeling. A complete “clinical trials update” and defensible “exclusivity-to-projection” requires current trial identifiers and U.S. regulatory exclusivity/patent status that cannot be produced from the information provided.

What is LEUKINE (sargramostim) and what indications drive demand?

Featured snippet: LEUKINE is sargramostim, a recombinant human GM-CSF used to stimulate myeloid cell recovery and immune function in defined clinical contexts under its FDA labels.

Which labeled uses most directly affect sales?

Sales of sargramostim products typically track demand from:

• Hematology/oncology supportive care workflows (myeloid recovery, infection risk management).
• Stem cell transplant and conditioning-related supportive protocols where clinicians use GM-CSF to shorten neutropenia duration.
• Other label-specific use cases that vary with guideline uptake and institutional practice.

How do hospital procurement and contracting shape quarterly revenue?

• GM-CSF utilization is protocol-driven and depends on facility standard of care.
• Group purchasing organization (GPO) contracts can shift share quickly even without new clinical evidence.
• Demand is often lumpy because transplant conditioning schedules are calendared.

What is the current clinical trials status for LEUKINE?

Featured snippet: No trial identifiers or up-to-date listing details were provided, so a concrete “clinical trials update” listing phases, endpoints, enrollment, and expected readouts cannot be assembled.

Which trial types typically matter for sargramostim strategy?

• New indications where GM-CSF is positioned for immune recovery or peri-therapy support.
• Comparative trials versus other GM-CSFs or supportive regimens to support guideline adoption.
• Formulation or administration route studies (less common for mature biologics but used for access expansion).

What would a high-value clinical update look like?

A decision-grade update would contain, at minimum:

• Trial registry IDs (e.g., NCT numbers), phase, sponsor, and enrollment status.
• Primary/secondary endpoints tied to FDA label expansion or competitive switching.
• Data cut dates and regulatory next steps (PDUFA-type milestones, sBLA supplements).

How large is the LEUKINE market and what is the competitive landscape?

Featured snippet: Sargramostim’s market is niche versus broader oncology biologics, with competition coming from other GM-CSF therapies and supportive care alternatives used in similar settings.

Competitor set that typically pressures sargramostim share

• Other GM-CSF products used for similar supportive outcomes (neutrophil recovery, transplant support).
• Growth factor substitution in practice when guideline or payer protocols favor another agent.
• Biosimilar dynamics in adjacent classes can indirectly affect GM-CSF mix through contracting.

Market share drivers

• Formulary placement and automatic substitution policies where available.
• Acquisition price and rebate structure in hospital channel.
• Clinical differentiation perceived by institutions: dosing schedules, response duration, adverse event profile.

When does LEUKINE lose exclusivity and when can generics or biosimilars enter?

Featured snippet: A definitive exclusivity-to-launch timetable requires current U.S. regulatory and patent/Orange Book or FDA reference product data for sargramostim that is not included in the request inputs.

What typically determines biosimilar entry timing for GM-CSFs?

• Patent term end for composition and manufacturing claims.
• Patent term adjustments or extensions.
• Any regulatory exclusivity periods tied to the reference product’s approval history.
• Procedural timelines from any biosimilar application route to licensure.

What patents protect LEUKINE and how strong is the estate?

Featured snippet: Patent estate mapping cannot be produced without the underlying patent list (publication numbers/assignees/claims) for sargramostim and the specific branded product listings.

What to look for in a sargramostim patent portfolio

• Composition and sequence/protein engineering claims.
• Formulation and stabilization claims.
• Manufacturing process claims (cell lines, purification steps, analytics).
• Method-of-use claims tied to dosing regimens and patient subsets.

What is the Orange Book status of LEUKINE?

Featured snippet: Orange Book listing status is required to answer this, including patents and exclusivity codes. No Orange Book record details were provided, so a complete status report cannot be generated.

What formulations are protected by LEUKINE patents?

Featured snippet: Formulation protection depends on claim coverage for the specific marketed presentation(s). Those claim details are not provided.

Typical formulation claim categories

• Lyophilized versus liquid presentations.
• Excipients and buffers.
• Particle control, aggregation limits, and stability targets.
• Delivery system or reconstitution guidance.

What patent litigation affects LEUKINE, including Paragraph IV challenges?

Featured snippet: Paragraph IV challenges apply to small-molecule generics; sargramostim is a biologic and would face biosimilar pathways (BPCIA) and related litigation. No litigation dockets, parties, or events were supplied, so a legal status summary cannot be assembled.

What would a litigation section include for decision makers?

• Court, case number, and filing dates.
• Asserted patents, infringement theories, and claim construction outcomes.
• Settlement terms and any licensed exclusivity or “at-risk” launch dates.

How does LEUKINE compare with other GM-CSF therapies on clinical and commercial outcomes?

Featured snippet: A defensible comparison needs specific endpoints from trials and head-to-head or real-world comparative utilization data, neither of which is included.

Where real-world differentiation usually shows up

• Time-to-neutrophil recovery.
• Hospital length of stay associated with supportive care protocols.
• Utilization patterns across transplant vs non-transplant indications.

What generic or biosimilar entry risks exist for LEUKINE?

Featured snippet: Biosimilar entry risk depends on regulatory approval status and whether patent estates and exclusivity withstand biosimilar application challenges. No current biosimilar pipeline or approval status details were provided.

Entry risk map typically used in investment models

• Near-term: filing or scientific advice signals.
• Mid-term: biosimilar approval timeline and labeling scope.
• Long-term: manufacturing scalability and interchangeability-like adoption patterns.

What is the FDA regulatory status of LEUKINE right now?

Featured snippet: Up-to-date FDA status requires access to the current label, supplement history, and any safety communications or REMS-like elements. None of these details were provided.

What matters for commercial forecasting

• Label expansions or restrictions.
• Safety label updates affecting contraindication language or monitoring.
• Manufacturing changes that trigger stability/packaging adjustments and channel disruption.

LEUKINE revenue projection: what is the base-case, bull-case, and bear-case trajectory?

Featured snippet: A revenue projection cannot be produced with the necessary foundational inputs (current sales baseline, payer coverage, utilization trends, and exclusivity-to-competition timeline). The request did not include those data.

How to structure a decision-grade projection model (inputs required)

A complete projection would tie together:

• Current U.S. sales and unit volumes by indication.
• Channel mix: hospital vs specialty.
• Price net of rebates and contract erosion assumptions.
• Competitive substitution rates among GM-CSFs and supportive regimens.
• Any biosimilar or competitive product entry dates.
• Forecast utilization growth from guideline changes or care pathway shifts.

Key Takeaways

• LEUKINE is a niche-but-established GM-CSF with demand tied to oncology supportive care protocols and labeled clinical workflows.
• A true “clinical trials update” requires trial identifiers, phase details, enrollment and readout schedules, and sponsor/regulatory plans that are not present in the provided inputs.
• Exclusivity-to-revenue projection requires current U.S. regulatory status, patent estate mapping, and competition entry timing; none of the necessary status records were included.

FAQs

1. What are the most common uses of sargramostim in hospital supportive care pathways?
2. Which GM-CSF competitors most often substitute for sargramostim in oncology supportive care protocols?
3. What endpoints are typically used to support label expansions for GM-CSF biologics?
4. How do biosimilar label scopes affect uptake for GM-CSF products?
5. What contracting and rebate mechanisms drive GM-CSF market share shifts in U.S. hospitals?

References

No sources were cited because no specific regulatory, clinical, litigation, or market dataset was provided in the prompt.