Sargramostim for Blood Cancer

Recruiting in Palo Alto (17 mi)

Overseen by Melhem Solh, MD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Recruiting

Sponsor: Northside Hospital, Inc.

Disqualifiers: Poor organ function, HIV, others

No Placebo Group

Prior Safety Data

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?

Given the increased number of HLA-mismatched haploidentical transplantation with post-transplant cyclophosphamide performed each year and the high risk of infectious complications associated with this type of transplant, the investigators suggest that GM-CSF administration post-infusion of T-replete haploidentical stem cells and post-transplant cyclophosphamide can yield similar count recovery rates to G-CSF with a potential of lowering risk of infectious complications.

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.

What data supports the effectiveness of the drug Sargramostim for blood cancer?

Sargramostim, also known as rhu GM-CSF, is used to help the body recover after bone marrow transplants and chemotherapy by boosting the immune system. It has been shown to improve immune recovery and increase certain immune cells, which can be beneficial in cancer treatment. ^{1 2 3 4 5}

Is Sargramostim safe for humans?

Sargramostim, also known as rhu GM-CSF, has been used since 1991 to help recover bone marrow after chemotherapy and is generally considered safe, but some people have reported allergic reactions. A study comparing it to another similar drug found it to be well-tolerated, with only a slightly higher chance of mild fever. ^{1 2 4 5 6}

How is the drug sargramostim unique for treating blood cancer?

Sargramostim is unique because it boosts the immune system by increasing the number of certain immune cells, which can help the body recover from chemotherapy and fight infections. Unlike some other treatments, it is derived from yeast and is used to speed up recovery of bone marrow and enhance immune responses. ^{1 2 3 4 5}

Eligibility Criteria

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.

Trial Timeline

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

Treatment Details

Interventions

• Sargramostim (Cytokine)

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.

Participant Groups

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.

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Northside Hospital Atlanta, GA

Who Is Running the Clinical Trial?

Northside Hospital, Inc. Lead Sponsor

References

1. United States pubmed.ncbi.nlm.nih.gov

Immediate hypersensitivity to human recombinant granulocyte-macrophage colony-stimulating factor associated with a positive prick skin test reaction. [2017] Recombinant human granulocyte-macrophage colony-stimulating factor (rhu GM-CSF), also known as sargramostim, is used to accelerate myeloid recovery following bone marrow transplantation or cytotoxic chemotherapy. "Anaphylactic" reactions to sargramostim have been reported on a limited basis and are poorly characterized.

2. United States pubmed.ncbi.nlm.nih.gov

Recombinant Human Granulocyte-Macrophage Colony-Stimulating Factor (rhu GM-CSF) as Adjuvant Therapy for Invasive Fungal Diseases. [2022] Sargramostim (yeast-derived, glycosylated recombinant human granulocyte-macrophage colony-stimulating factor [rhu GM-CSF]) augments innate and adaptive immune responses and accelerates hematopoietic recovery of chemotherapy-induced neutropenia. However, considerably less is known about its efficacy as adjunctive immunotherapy against invasive fungal diseases (IFDs).

3. England 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. [2018] Granulocyte-macrophage (GM) colony-stimulating factor (CSF) has been used as an adjuvant in cancer immunotherapy. We tested the hypothesis that GM-CSF (Leukine(®); sargramostim) improves immune reconstitution after hematopoietic stem cell transplantation (HSCT) based on our prior in vitro work that demonstrated the pro-inflammatory effects of GM-CSF on dendritic cells (DC).

4. Germany 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. [2019] A prospective, randomized, double-blind, multicenter study in cancer patients receiving myelosuppressive chemotherapy was undertaken to evaluate and compare the tolerability of sargramostim (yeast-derived recombinant human granulocyte-macrophage colony-stimulating factor, RhuGM-CSF) and filgrastim (bacteria-derived recombinant human granulocyte colony-stimulating factor, RhuG-CSF) in the prophylaxis or treatment of chemotherapy-induced neutropenia. In all, 137 evaluable patients received sargramostim (300 micrograms; 193 mg/m2) or filgrastim (481 mg; 7 mg/kg) once daily by self-administered s.c. injection, usually beginning within 48 h after completion of chemotherapy. With the exception of a slightly higher incidence of grade 1 fever (

5. Switzerland pubmed.ncbi.nlm.nih.gov

Sargramostim (rhu GM-CSF) as Cancer Therapy (Systematic Review) and An Immunomodulator. A Drug Before Its Time? [2022] Label="BACKGROUND" NlmCategory="BACKGROUND">Sargramostim [recombinant human granulocyte-macrophage colony-stimulating factor (rhu GM-CSF)] was approved by US FDA in 1991 to accelerate bone marrow recovery in diverse settings of bone marrow failure and is designated on the list of FDA Essential Medicines, Medical Countermeasures, and Critical Inputs. Other important biological activities including accelerating tissue repair and modulating host immunity to infection and cancer via the innate and adaptive immune systems are reported in pre-clinical models but incompletely studied in humans.

6. United States pubmed.ncbi.nlm.nih.gov

Comparison of hospitalization risk and associated costs among patients receiving sargramostim, filgrastim, and pegfilgrastim for chemotherapy-induced neutropenia. [2022] Sargramostim is a granulocyte-macrophage-colony-stimulating factor (GM-CSF). Unlike filgrastim and pegfilgrastim, which are granulocyte-colony-stimulating factors (G-CSFs), sargramostim activates a broader range of myeloid lineage-derived cells. Therefore, GM-CSF might reduce infection risk more than the G-CSFs. This study compared real-world infection-related hospitalization rates and costs in patients using G/GM-CSF for chemotherapy-induced neutropenia.