G-CSF for Glioblastoma

Overseen By Jorg Dietrich, MD, PhD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Massachusetts General Hospital

Must be taking: Temozolomide

Must not be taking: Investigational agents

Disqualifiers: Pregnancy, HIV, Sickle cell, 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 4 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 2

60 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether the drug G-CSF (granulocyte-colony stimulating factor) can improve brain health and cognitive function in individuals with glioblastoma multiforme (GBM). Participants will receive standard treatments like chemotherapy and radiation, with some also receiving G-CSF to assess any additional benefits. The trial seeks individuals newly diagnosed with MGMT-methylated GBM who can undergo standard radiation and chemotherapy. As a Phase 2 trial, the research focuses on evaluating the treatment's effectiveness in an initial, smaller group, providing an opportunity to contribute to significant findings.

Do I have to stop taking my current medications for the trial?

The trial protocol does not specify whether you need to stop taking your current medications. However, if you are on high doses of corticosteroids, you may need to taper down to a lower dose before starting the trial.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Studies have shown that G-CSF, also known as Filgrastim, is generally safe and well-tolerated by patients. Research suggests that using G-CSF after chemotherapy and radiation may help protect brain function without causing significant harm. In earlier studies, most patients experienced only mild side effects, such as bone pain or slight reactions at the injection site. G-CSF is also used in other treatments, which supports its safety.

Temozolomide (TMZ), the chemotherapy drug used with G-CSF, is a standard treatment for glioblastoma and is known to be safe. Common side effects of TMZ include nausea and tiredness, but these are usually manageable.

Overall, both G-CSF and TMZ have demonstrated safety in similar treatments, with side effects that are generally mild and manageable. ^{1 2 3 4 5}

Why are researchers excited about this study treatment for glioblastoma?

Researchers are excited about using G-CSF for glioblastoma because it introduces a novel way to potentially enhance recovery and counteract the side effects of standard treatments. Unlike traditional chemoradiation therapies, G-CSF stimulates the production of white blood cells, which helps bolster the immune system during the challenging treatment cycles. This boost could mean improved resilience to infections and better overall tolerance to chemotherapy. G-CSF is also easy to administer, as it can be injected under the skin by the patient or a caregiver, adding convenience and flexibility to the treatment regimen.

What evidence suggests that G-CSF might be an effective treatment for glioblastoma?

This trial will compare standard chemoradiation with and without the addition of G-CSF for patients with glioblastoma. Studies have shown that adding G-CSF to standard treatment can benefit patients with glioblastoma, a type of brain cancer. Research suggests that combining G-CSF with chemotherapy is safe and effective. Specifically, one study found that this combination helped patients live longer without their cancer worsening. G-CSF increases the production of white blood cells, supporting the body during cancer treatment. This may also help preserve brain health and maintain cognitive function. Early results are promising, but further research is needed to confirm these benefits. ^{1 4 6 7 8}

Who Is on the Research Team?

Jorg Dietrich

Principal Investigator

Massachusetts General Hospital

Are You a Good Fit for This Trial?

This trial is for adults over 18 with newly diagnosed glioblastoma multiforme (GBM), WHO grade 4, IDH wildtype. They must have a positive MGMT promoter methylation status, good performance status (KPS > 60), and life expectancy over 6 months. Patients need to be able to undergo standard chemoradiation therapy and brain MRIs. Exclusions include pregnancy, other investigational drugs use, severe infections or HIV/AIDS, prior cranial irradiation, certain allergies, non-English/Spanish speakers due to cognitive testing requirements.

Inclusion Criteria

I am set to undergo a 6-week radiation therapy and 6 months of chemotherapy with temozolomide.

I have another cancer, but it won't affect this trial's treatment.

Ability to understand and the willingness to sign a written informed consent document

Exclusion Criteria

I am not pregnant, as the treatment in this study could harm a pregnancy.

Participants who are receiving any other investigational agents

I do not have any severe infections or HIV that could affect my white blood cell count.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

Chemoradiation

Participants undergo a 6-week chemoradiation (chemo-RT) cycle followed by a 4-week break

10 weeks

Weekly visits during chemoradiation

G-CSF Treatment

Participants receive G-CSF during the 4-week break and during up to 6 additional 4-week cycles of chemotherapy

7 months

Monthly visits for G-CSF administration

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

Regular follow-up visits every 2 months

What Are the Treatments Tested in This Trial?

Interventions

• G-CSF

Trial Overview The study tests the effects of granulocyte colony stimulating factor (G-CSF) on brain health and cognitive function in GBM patients undergoing standard chemoradiation therapy. G-CSF is given alongside Temozolomide chemotherapy during a planned course of radiation treatment.

How Is the Trial Designed?

Group I: Standard Chemoradiation combined with G-CSF Experimental Treatment 2 Interventions

Per standard of care, participants undergo a 6-week chemoradiation (chemo-RT) cycle followed by a 4-week break. During this 4-week break after chemo-RT, participants receive granulocyte colony stimulating factor (G-CSF) once per day for 5 days (Days 7-11). After the 4-week break, as a part of standard care, participants may then receive up to 6 additional 4-week cycles of chemotherapy (temozolomide (TMZ)). During these additional TMZ cycles, participants will receive G-CSF once per day for 3 consecutive days (Days 14-16). G-CSF is injected under the skin (subcutaneously injected) by a study staff member, or self-administered (or a caregiver) can be trained by a study staff member to administer it at home. The chemo-RT (radiation therapy and chemotherapy (TMZ)) are standard of care in this study.

Group II: Standard Chemoradiation Experimental Treatment 1 Intervention

Participants receive standard of care chemoradiation (chemo-RT) which includes an initial 6-week chemo-RT cycle followed by a 4-week break, and up to 6 additional 4-week chemotherapy (temozolomide (TMZ)) cycles.

Who Is Running the Clinical Trial?

Massachusetts General Hospital

Lead Sponsor

Trials

3,066

Recruited

13,430,000+

Published Research Related to This Trial

In a study involving 31 patients with breast cancer and ovarian carcinoma, the recombinant human G-CSF-Leukostim (filgrastim) effectively treated or prevented chemotherapy-induced neutropenia after just 2-6 injections.

Leukostim allowed 5 breast cancer patients to undergo an additional 10 cycles of chemotherapy without inhibiting blood cell production, and no adverse side effects were reported, indicating its safety and efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Effectiveness of Leukostim, a Russian preparation of granulocyte colony-stimulating factor, in the treatment of chemotherapy-induced neutropenia in patients with malignant tumors]. Korman, DB., Boronovskaia, LE., Maslova, IA., et al.[2015]

In a study involving 348 breast cancer patients undergoing chemotherapy, the new G-CSF XM02 was found to significantly reduce the duration of severe neutropenia compared to a placebo, with a mean duration of 1.1 days for both XM02 and Neupogen, versus 3.9 days for placebo.

XM02 demonstrated similar safety profiles to Neupogen, indicating that it is an effective alternative for managing chemotherapy-induced neutropenia without increased toxicity.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

XM02 is superior to placebo and equivalent to Neupogen in reducing the duration of severe neutropenia and the incidence of febrile neutropenia in cycle 1 in breast cancer patients receiving docetaxel/doxorubicin chemotherapy. del Giglio, A., Eniu, A., Ganea-Motan, D., et al.[2022]

Tbo-filgrastim, a recombinant human G-CSF, has been shown to be as effective as Neupogen in reducing the duration of severe neutropenia in patients undergoing myelosuppressive chemotherapy, based on data from three phase III studies involving 677 patients.

The safety profile of tbo-filgrastim is comparable to Neupogen, with most adverse events related to chemotherapy rather than the drug itself, and only a small percentage of patients experiencing severe drug-related adverse events.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical safety of tbo-filgrastim, a short-acting human granulocyte colony-stimulating factor. Pettengell, R., Bias, P., Mueller, U., et al.[2018]

Pegfilgrastim is as effective or even more effective than filgrastim in reducing chemotherapy-induced neutropenia, making it a strong option for patients undergoing cancer treatment.

One of the key benefits of pegfilgrastim is that it can be administered just once per chemotherapy cycle, which may improve patient compliance and enhance their quality of life.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pegfilgrastim; a neutrophil mediated granulocyte colony stimulating factor-expanding uses in cancer chemotherapy. Morishita, M., Leonard, RC.[2022]

The biosimilar filgrastim (BK0023) demonstrated comparable biological activity, efficacy, and safety to the reference drug Neupogen® in both in vitro and in vivo studies, including a phase I trial with healthy volunteers.

BK0023 showed similar pharmacokinetic profiles to Neupogen®, indicating it could be a viable alternative for treating neutropenia in patients undergoing cancer treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Preclinical and clinical phase I studies of a new recombinant Filgrastim (BK0023) in comparison with Neupogen®. Crobu, D., Spinetti, G., Schrepfer, R., et al.[2021]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06649851

G-CSF After Chemo-radiation in Patients With Glioblastoma This is an open-label, randomized, phase II clinical study of using G-CSF in patients with newly diagnosed, MGMT-methylated, GBM treated with standard of care ...

2. sciencedirect.com sciencedirect.com/science/article/pii/S1476558625000351

Granulocyte-macrophage colony-stimulating factor for ... The combination of hypofractionated IMRT, TMZ and GM-CSF was efficacious and safe in patients with newly diagnosed GBM. Consent for publication. All patients ...

3. careacross.com careacross.com/clinical-trials/trial/NCT06649851

G-CSF After Chemo-radiation in Patients with Glioblastoma Treatment with G-CSF will be initiated after chemo-RT and be completed after 6 cycles of adjuvant chemotherapy with Temozolomide. This study involves screening ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC4234180/

Antiangiogenic Therapy for Glioblastoma: Current Status and ... Outcome was notably improved for the combination arm including PFS-6 of 41%, compared to 11% and 18% for lomustine and bevacizumab alone, respectively. The ...

5. journals.sagepub.com journals.sagepub.com/doi/10.1177/1010428317699797

Glioblastoma-synthesized G-CSF and GM-CSF contribute ... Data reviewed establish that any damage to brain tissue tends to cause an increase in G-CSF and/or GM-CSF (G(M)-CSF) synthesized by the brain.

6. massgeneral.trialstoday.org massgeneral.trialstoday.org/trial/NCT06649851

G-CSF After Chemo-radiation in Patients With Glioblastoma The study aims to evaluate G-CSF's effects on brain health and cognitive function. ... data exists ... safety and tolerability of G-CSF. Participants ...

7. cancer.gov cancer.gov/research/participate/clinical-trials-search/v?id=NCI-2025-02420

G-CSF Following Standard Chemoradiation in Patients ... Giving G-CSF after treatment with chemotherapy radiation therapy and temozolomide may be safe, tolerable and/or effective at preserving cognitive brain function ...

8. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7217575/

Chemoradiation and granulocyte-colony or ... Use of G-CSF during concurrent chemotherapy and thoracic radiotherapy in patients with limited-stage small-cell lung cancer safety data from a phase II trial.