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Clinical Trials/NCT04856176
NCT04856176
Terminated
Phase 2

A Phase II Trial of GM-CSF Plus Maintenance Pembrolizumab +/- Pemetrexed After Completion of First Line Chemo-Immunotherapy in Advanced Non-Small Cell Lung Cancer Patients with PDL-1 of 1%-49%

Tufts Medical Center1 site in 1 country5 target enrollmentJanuary 3, 2022

Overview

Phase
Phase 2
Intervention
Paclitaxel
Conditions
Advanced Lung Non-Small Cell Carcinoma
Sponsor
Tufts Medical Center
Enrollment
5
Locations
1
Primary Endpoint
Overall Survival (OS)
Status
Terminated
Last Updated
last year

Overview

Brief Summary

Metastatic lung cancer is the leading cause of cancer mortality worldwide with a 5-year survival of less than 5%. With the approval of programmed cell death 1 (PD-1) inhibitors in advanced lung cancer, such as pembrolizumab, there has been an improvement in overall response rates (ORR) and survival compared to chemotherapy.

However, there is still a need for improvement in response rates in first-line treatments for patients with stage 4 NSCLC without genetically targetable alterations, especially in those patients with PDL-1 <50%.

This trial is important because it seeks to discover whether the responses seen in first line treatments with PD-1 inhibitors + chemotherapy can be augmented with the addition of GM-CSF during the maintenance phase with pembrolizumab +/- pemetrexed.

Detailed Description

Lung cancer is the most commonly diagnosed cancer worldwide. Metastatic lung cancer is the leading cause of cancer mortality worldwide with a 5-year survival of less than 5%. With the approval of programmed cell death 1 (PD-1) inhibitors in advanced lung cancer, there has been an improvement in overall response rates and survival compared to chemotherapy. Checkpoint inhibition has become a primary treatment modality for vast number of cancers including lung cancer, prolonging survival in some patients. However, an important consideration is how to best select those patients who will respond to checkpoint inhibition. The biomarker that has been studied most extensively is PD-L1 expression. Studies have shown trends for increased response rates to PD-1 blockade in PD-L1 positive tumors. NSCLC patients are now approved for pembrolizumab monotherapy (PDL-1\>1%) or for pembrolizumab in combination with chemotherapy (carboplatin/pemetrexed for non-squamous or carboplatin/paclitaxel) (no minimum PDL-1). As the ORR and PFS in both these trials indicate, however, there is a need for improvement in response rates and PFS in first-line treatments for patients with stage 4 NSCLC without genetically targetable alterations especially in those patients with PDL-1 \<50%. There are both pre-clinical and clinical evidence supporting the combination of granulocyte macrophage colony stimulating factor (GM-CSF) with immunotherapy. GM-CSF is a hematopoietic growth factor that triggers proliferation and differentiation of hematopoietic progenitor cells, mainly neutrophils, monocytes/macrophages and myeloid-derived dendritic cells, and is approved by the FDA for this purpose. A phase II randomized clinical trial of unresectable stage III or IV melanoma patients comparing the effects of ipilimumab plus GM-CSF vs ipilimumab alone was shown to be both safe and had longer overall survival with lower toxicity than immunotherapy alone; 1 year survival for ipilimumab plus sargramostim was 68.9% (95% CI, 60.6%-85.5%) compared to 52.9% (95% CI, 43.6%-62.2%) for ipilimumab alone. It is hypothesized that the use of GM-CSF along with a PD-1 inhibitor +/- pemetrexed is safe and will increase the overall response rate and progression-free survival in advanced NSCLC patients with PDL-1 of 1%-49%. This will establish the basis for further evaluation of GM-CSF+PD-1 in advanced NSCLC patients.

Registry
clinicaltrials.gov
Start Date
January 3, 2022
End Date
December 6, 2024
Last Updated
last year
Study Type
Interventional
Study Design
Single Group
Sex
All

Investigators

Responsible Party
Sponsor

Eligibility Criteria

Inclusion Criteria

  • 18 years of age or older
  • Histologically confirmed stage 4 NSCLC or stage 3B/3C not able to receive chemoradiation with no sensitizing EGFR or ALK mutations.
  • PDL-1 of 1%-49%
  • No previous history of immunotherapy treatment
  • ECOG PS 0-1
  • At least one measurable lesion according to RECIST version 1.1
  • Life expectancy of at least 3 months.
  • Able to self-administer daily GM-CSF injections
  • Eligible for treatment with 4 cycles of chemoimmunotherapy followed by maintenance therapy with pembrolizumab +/- pemetrexed.

Exclusion Criteria

  • Receiving systemic glucocorticoids or other immunosuppressive treatment
  • Untreated brain metastases
  • Active autoimmune disease
  • Active interstitial lung disease, pneumonitis
  • Solid organ transplant recipients
  • Subject may not participate in another drug research study while participating in this research study
  • Pregnant patients
  • Known hypersensitivity to GM-CSF (sargramostim)

Arms & Interventions

GM-CSF Plus Maintenance Pembrolizumab +/- Pemetrexed

All patients will receive GM-CSF plus maintenance pembrolizumab with or without pemetrexed, following completion of 4 cycles of chemo-immunotherapy

Intervention: Paclitaxel

GM-CSF Plus Maintenance Pembrolizumab +/- Pemetrexed

All patients will receive GM-CSF plus maintenance pembrolizumab with or without pemetrexed, following completion of 4 cycles of chemo-immunotherapy

Intervention: Granulocyte-Macrophage Colony-Stimulating Factor

GM-CSF Plus Maintenance Pembrolizumab +/- Pemetrexed

All patients will receive GM-CSF plus maintenance pembrolizumab with or without pemetrexed, following completion of 4 cycles of chemo-immunotherapy

Intervention: Pembrolizumab

GM-CSF Plus Maintenance Pembrolizumab +/- Pemetrexed

All patients will receive GM-CSF plus maintenance pembrolizumab with or without pemetrexed, following completion of 4 cycles of chemo-immunotherapy

Intervention: pemetrexed

GM-CSF Plus Maintenance Pembrolizumab +/- Pemetrexed

All patients will receive GM-CSF plus maintenance pembrolizumab with or without pemetrexed, following completion of 4 cycles of chemo-immunotherapy

Intervention: Carboplatin

Outcomes

Primary Outcomes

Overall Survival (OS)

Time Frame: 24 months

Patient survival status throughout their participation in the study

Progression free survival (PFS)

Time Frame: 24 Months

Progression is measured according to RECIST 1.1 criteria.

Secondary Outcomes

  • To evaluate changes in myeloid derived suppressor cells at different time points during study treatment(24 Months)
  • To evaluate changes in PD-1+ CD8 at different time points during study treatment(24 Months)
  • To evaluate changes in CD4 T at different time points during study treatment(24 Months)
  • To evaluate changes in CD8 T at different time points during study treatment(24 Months)
  • To evaluate changes in monocytes at different time points during study treatment(24 Months)
  • To evaluate changes in PD-1+ CD4 at different time points during study treatment(24 Months)

Study Sites (1)

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