Sargramostim for Blood Cancer

Overseen By Stacey Brown, BA

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Northside Hospital, Inc.

Disqualifiers: Poor organ function, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: Approximately 3 weeks

38 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called Sargramostim for individuals with blood cancers who have undergone a type of stem cell transplant. Researchers aim to determine if Sargramostim can aid blood cell recovery and reduce infection risk post-transplant. Eligible participants have blood cancers like AML or CLL and a partially matched donor for their transplant. The trial seeks participants willing to explore new recovery methods after their transplant. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to significant advancements in recovery methods.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that Sargramostim is likely to be safe for humans?

Research has shown that Sargramostim, also known as GM-CSF, is generally safe for people. Studies have found that it helps the body recover blood cells more quickly in various medical situations. This faster recovery can lower the risk of infections and other complications.

For over twenty years, Sargramostim has been used in older patients with low white blood cell counts, and the safety information collected during this time is reassuring. A review of studies also suggests it might help boost the immune system in patients with certain types of cancer.

Overall, evidence suggests Sargramostim is safe for most people, with few serious side effects. However, like any treatment, some risks may exist, so it is important for each person to consult their healthcare provider before joining a clinical trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard care for blood cancers, which often includes chemotherapy and stem cell transplants, sargramostim (GM-CSF) stands out due to its role in boosting white blood cell production after a transplant. While most treatments focus on directly attacking cancer cells, sargramostim enhances the body's immune recovery by stimulating the production of neutrophils, a type of white blood cell crucial for fighting infections. Researchers are excited because this approach could lead to faster immune recovery post-transplant, potentially reducing infection risks and improving overall patient outcomes.

What evidence suggests that Sargramostim might be an effective treatment for blood cancer?

Research has shown that sargramostim, also known as GM-CSF, helps the body produce more blood cells. Studies have found it aids blood cell count recovery after stem cell transplants, which is crucial for reducing the risk of infections that often occur post-transplant. In this trial, participants will receive sargramostim after their transplant to support blood cell recovery. Although limited data exists specifically for blood cancers, sargramostim has proven safe and effective in other treatments. Its ability to enhance blood cell recovery makes it a promising option for reducing complications in blood cancer patients undergoing transplants. ^{1 2 3 6 7}

Who Is on the Research Team?

Melhem Solh, MD

Principal Investigator

Northside Hospital

Are You a Good Fit for This Trial?

This trial is for patients with various blood cancers who need a stem cell transplant and have a partially matched related donor. They must be in good physical shape (KPS >/= 70%) and able to consent. It's not for those with poor heart, lung, liver, or kidney function, HIV-positive individuals, or anyone allergic to GM-CSF or yeast products.

Inclusion Criteria

I have a blood disorder that qualifies for a bone marrow transplant.

You need to have a family member who is a good match for a transplant (with a matching score of 5/10 to 8/10).

I am mostly able to care for myself.

Exclusion Criteria

History of severe or serious allergic reaction to human GM-CSF or yeast-derived products

HIV-positive

You have a serious medical or mental condition that would prevent you from understanding and agreeing to participate in the study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Sargramostim (GM-CSF) starting on Day +5 post-transplant until ANC >1000 x3 days or >1500 x1 day, administered not less than 24 hours after the last dose of cyclophosphamide

Approximately 3 weeks

Daily visits for infusion administration

Follow-up

Participants are monitored for safety, engraftment, and infection rates post-treatment

3 months

Regular visits for monitoring and assessments

Long-term follow-up

Participants are monitored for relapse rates, GVHD, and overall survival

12 months

What Are the Treatments Tested in This Trial?

Interventions

• Sargramostim

Trial Overview

The study tests if GM-CSF can help recover blood counts after a haploidentical stem cell transplant with post-transplant cyclophosphamide treatment while reducing infection risks compared to the standard G-CSF approach.

How Is the Trial Designed?

Group I: GM-CSF post-transplant Experimental Treatment 2 Interventions

Sargramostim (GM-CSF) will start on Day +5 and continue until ANC \>1000 x3 days or \>1500 x1 day. GM-CSF will be administered not less than 24 hours after the last dose of cyclophosphamide and will be given at a dose of 250mcg/m2/day as an infusion over 2 hours.

Who Is Running the Clinical Trial?

Northside Hospital, Inc.

Lead Sponsor

Trials

26

Recruited

1,100+

Published Research Related to This Trial

In a study involving 35 patients (18 receiving GM-CSF and 17 controls), GM-CSF did not enhance T cell or natural killer cell recovery after allogeneic stem cell transplantation, contrary to expectations.

However, GM-CSF administration improved dendritic cell reconstitution in patients undergoing autologous stem cell transplantation, suggesting its benefits may vary based on the type of transplant.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Granulocyte-macrophage colony-stimulating factor increases the proportion of circulating dendritic cells after autologous but not after allogeneic hematopoietic stem cell transplantation. Eksioglu, EA., Kielbasa, J., Eisen, S., et al.[2018]

A case report described an adverse reaction to sargramostim (rhu GM-CSF) involving symptoms like itching, hives, and throat tightness, highlighting the potential for allergic reactions to this treatment.

Prick skin testing showed that the patient was sensitized to sargramostim but not to filgrastim (rhu G-CSF), suggesting that skin testing could help identify patients at risk for allergic reactions to GM-CSF therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Immediate hypersensitivity to human recombinant granulocyte-macrophage colony-stimulating factor associated with a positive prick skin test reaction. Engler, RJ., Weiss, RB.[2017]

In a study involving 137 cancer patients undergoing chemotherapy, both sargramostim and filgrastim were found to be well tolerated for preventing chemotherapy-induced neutropenia, with only a slightly higher incidence of mild fever associated with sargramostim.

There were no significant differences in adverse events, hospitalization days, or the need for intravenous antibiotics between the two treatments, indicating that both growth factors are comparably effective in this context.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Randomized trial comparing the tolerability of sargramostim (yeast-derived RhuGM-CSF) and filgrastim (bacteria-derived RhuG-CSF) in cancer patients receiving myelosuppressive chemotherapy. Beveridge, RA., Miller, JA., Kales, AN., et al.[2019]

In a study analyzing 990 patients treated with sargramostim and matched cohorts receiving filgrastim or pegfilgrastim, sargramostim was associated with a 56% lower risk of infection-related hospitalizations for those undergoing chemotherapy-induced neutropenia.

Patients using sargramostim also experienced significantly lower hospitalization costs, with reductions of 84% compared to filgrastim and 62% compared to pegfilgrastim, highlighting its potential economic benefits in clinical settings.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Comparison of hospitalization risk and associated costs among patients receiving sargramostim, filgrastim, and pegfilgrastim for chemotherapy-induced neutropenia. Heaney, ML., Toy, EL., Vekeman, F., et al.[2022]

In a study of 15 pediatric patients with malignancies and invasive fungal diseases (IFDs), sargramostim showed a high overall response rate of 92%, indicating its potential effectiveness as an adjunctive treatment for patients who are neutropenic and refractory to antifungal therapy.

A systematic review of 65 cases, including the 15 new patients, demonstrated an overall response rate of 82% for sargramostim in treating IFDs, suggesting it may serve as a valuable immunomodulator for patients with hematological malignancies facing difficult-to-treat fungal infections.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Recombinant Human Granulocyte-Macrophage Colony-Stimulating Factor (rhu GM-CSF) as Adjuvant Therapy for Invasive Fungal Diseases. Chen, TK., Batra, JS., Michalik, DE., et al.[2022]

Sargramostim is effective in accelerating hematologic recovery in patients exposed to bone marrow-suppressing agents, leading to fewer infections and improved survival outcomes in various clinical settings.

As an immune modulator, sargramostim enhances anti-cancer responses when used alongside conventional therapies, showing a favorable safety profile and potential for expanded use in treating immune-related conditions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Sargramostim (rhu GM-CSF) as Cancer Therapy (Systematic Review) and An Immunomodulator. A Drug Before Its Time? Lazarus, HM., Ragsdale, CE., Gale, RP., et al.[2022]

Citations

1.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC8416151/

Sargramostim (rhu GM-CSF) as Cancer Therapy (Systematic ...

Sargramostim may be less efficacious in hematologic malignancies, although data are limited. The use of sargramostim in combination with immune checkpoint ...

2.

accessdata.fda.gov

accessdata.fda.gov/drugsatfda_docs/label/2017/103362s5237lbl.pdf

Leukine (sargramostim) Label - accessdata.fda.gov

GM-CSF belongs to a group of growth factors termed colony stimulating factors which support survival, clonal expansion, and differentiation of hematopoietic ...

3.

clinicaltrials.gov

clinicaltrials.gov/study/NCT01409915

NCT01409915 | Study of the Safety & Efficacy of Leukine® ...

Preliminary preclinical results demonstrated that GM-CSF (Granulocyte macrophage colony-stimulating factor, e.g. Leukine®/Sargramostim) rapidly reduced ...

4.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC7988877/

Safety and efficacy of sargramostim (GM‐CSF) in the ...

The data presented herein show that GM‐CSF/sargramostim treatment was safe and well‐tolerated and provided measurable disease‐modifying and ...

5.

hemonc.org

hemonc.org/w/images/archive/e/ec/20201126004019%21Sargramostim.pdf

Leukine® (sargramostim)

GM-CSF was last detected in blood samples obtained at three or six hours. When LEUKINE (either liquid or lyophilized) was administered SC to ...

6.

partnertx.com

partnertx.com/a-drug-before-its-time-leukine-systematic-review-highlights-innate-and-adaptive-immune-activity-that-may-improve-cancer-outcomes-and-reduce-toxicity/

“A Drug Before Its Time?” LEUKINE® systematic review ...

This review article highlights Leukine's potential to generate benefit for patients in numerous diseases that feature GM-CSF deficiency.

7.

alz-journals.onlinelibrary.wiley.com

alz-journals.onlinelibrary.wiley.com/doi/10.1002/trc2.12158

Safety and efficacy of sargramostim (GM‐CSF) in the ...

The data presented herein show that GM-CSF/sargramostim treatment was safe and well-tolerated and provided measurable disease-modifying and ...