Leukine (sargramostim) / Partner Therap - LARVOL DELTA

Tebentafusp-tebn With LDT in Metastatic UM (clinicaltrials.gov) - P1/2 | N=109 | Recruiting | Sponsor: Thomas Jefferson University | Not yet recruiting ➔ Recruiting | Trial completion date: Mar 2032 ➔ Aug 2032 | Trial primary completion date: May 2030 ➔ Oct 2030

Enrollment open • Trial completion date • Trial primary completion date • Eye Cancer • Melanoma • Oncology • Solid Tumor • Uveal Melanoma

Intermittent Sargramostim Administration Expands Proliferating Naïve T Cells, Tregs, HLA-DR+ PD-L1+ Monocytes and Myeloid-Derived Suppressor Cells: Results from a Randomized Placebo-Controlled Clinical Trial of GM-CSF in Patients with Peripheral Artery Disease (ASH 2023) - "Our findings demonstrate that intermittent dosing with sargramostim is well-tolerated and induces significant but transient increases in the numbers and activation status of T cells in the bloodstream. Sargramostim treatment also increased numbers of proliferating naïve T cells, Tregs, and certain subsets of myelo-monocytic cells with immunosuppressive phenotypes. These results support the use of intermittent sargramostim dosing to avoid excessive leukocytosis and associated adverse effects."

Clinical • Myeloid-derived suppressor cells • Acute Myelogenous Leukemia • Atherosclerosis • Bone Marrow Transplantation • Cardiovascular • Hematological Malignancies • Immune Modulation • Immunology • Leukemia • Oncology • Peripheral Arterial Disease • Transplantation • CD14 • CD8 • CSF2 • FOXP3 • IL2RA • PD-1 • PD-L1

Durable Tumor Control Following Off-the-Shelf Tumor Peptide Vaccination in Metastatic Ewing Sarcoma: A Case Report (DGHO 2025) - "Each vaccination was administered intracutaneously with sargramostim and topical imiquimod. This case illustrates the safety, feasibility, and immunogenicity of fusion-specific peptide vaccination in metastatic Ewing sarcoma. The durable clinical response, paired with functional immune data, suggests a potential benefit of this off-the-shelf immunotherapeutic strategy. Further investigation in fusion-driven sarcomas is warranted, particularly regarding optimal timing, combination with chemotherapy, and prevention of T cell exhaustion."

Case report • Clinical • Metastases • Ewing Sarcoma • Oncology • Sarcoma • Solid Tumor • CD4 • EWSR1 • FLI1

NANT 2021-01 Phase II STING (Sequential Temozolomide, Irinotecan, NK Cells and GD2 mAb) Trial (clinicaltrials.gov) - P2 | N=62 | Recruiting | Sponsor: New Approaches to Neuroblastoma Therapy Consortium | Active, not recruiting ➔ Recruiting

Enrollment open • Neuroblastoma • Oncology • Solid Tumor

ANBL19P1: Treatment With Dinutuximab, Sargramostim (GM-CSF), and Isotretinoin in Combination With Irinotecan and Temozolomide After Intensive Therapy for People With High-Risk Neuroblastoma (NBL) (clinicaltrials.gov) - P2 | N=41 | Active, not recruiting | Sponsor: Children's Oncology Group | Trial completion date: Sep 2025 ➔ Sep 2027

Trial completion date • Ganglioneuroblastoma • Neuroblastoma • Oncology • Solid Tumor • MYCN

GM-CSF With Post-Transplant Cyclophosphamide (clinicaltrials.gov) - P2 | N=38 | Recruiting | Sponsor: Northside Hospital, Inc. | Trial completion date: Sep 2025 ➔ Sep 2026 | Trial primary completion date: Sep 2025 ➔ Sep 2026

Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Oncology • Transplantation

STEMVAC in Patients With Early Stage Triple Negative Breast Cancer (clinicaltrials.gov) - P2 | N=33 | Active, not recruiting | Sponsor: University of Washington | Recruiting ➔ Active, not recruiting | Trial completion date: Jun 2026 ➔ Jul 2028 | Trial primary completion date: Dec 2025 ➔ Jul 2026

Enrollment closed • Trial completion date • Trial primary completion date • Breast Cancer • Hormone Receptor Negative Breast Cancer • Oncology • Solid Tumor • Triple Negative Breast Cancer • ER • HER-2 • PGR

Tebentafusp-tebn With LDT in Metastatic UM (clinicaltrials.gov) - P1/2 | N=109 | Not yet recruiting | Sponsor: Thomas Jefferson University | Trial completion date: Feb 2027 ➔ Mar 2032 | Trial primary completion date: Jan 2027 ➔ May 2030

Trial completion date • Trial primary completion date • Eye Cancer • Melanoma • Oncology • Solid Tumor • Uveal Melanoma

NCI-2018-03732: Dinutuximab, Sargramostim, and Combination Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma (clinicaltrials.gov) - P2 | N=42 | Active, not recruiting | Sponsor: National Cancer Institute (NCI) | Trial completion date: Sep 2025 ➔ Sep 2026

Trial completion date • Ganglioneuroblastoma • Neuroblastoma • Oncology • Solid Tumor • MYCN

Recombinant GM-CSF drug evaluation review. (PubMed, Immunotherapy) - "Molgramostim and sargramostim are rGM-CSF which can be self-administered at home by patients with aPAP either subcutaneously or using a handheld nebulizer. In all clinical studies to date, rGM-CSF has shown a favorable safety profile with no dose limiting toxicity. This supports the application of inhaled rGM-CSF for the treatment of aPAP and the possibility of WLL as a rescue therapy."

Journal • Review • Immunology • Interstitial Lung Disease • Pulmonary Disease • Rare Diseases • Respiratory Diseases • CSF2

Testing the Addition of 131I-MIBG or Lorlatinib to Intensive Therapy in People With High-Risk Neuroblastoma (NBL) (clinicaltrials.gov) - P3 | N=750 | Recruiting | Sponsor: Children's Oncology Group | Trial completion date: Sep 2026 ➔ Sep 2030 | Trial primary completion date: Sep 2026 ➔ Sep 2030

Trial completion date • Trial primary completion date • Ganglioneuroblastoma • Neuroblastoma • Oncology • Solid Tumor • MYCN

Testing the Addition of 131I-MIBG or Lorlatinib to Intensive Therapy in People With High-Risk Neuroblastoma (NBL) (clinicaltrials.gov) - P3 | N=750 | Recruiting | Sponsor: Children's Oncology Group | Active, not recruiting ➔ Recruiting

Enrollment open • Ganglioneuroblastoma • Neuroblastoma • Oncology • Solid Tumor • MYCN

A Phase II Study With a Safety Run-In of the Addition of N-803 to a Chemoimmunotherapy Backbone for the Treatment of Patients With Relapsed or Refractory Neuroblastoma (clinicaltrials.gov) - P2 | N=54 | Recruiting | Sponsor: St. Jude Children's Research Hospital | Not yet recruiting ➔ Recruiting

Enrollment open • Neuroblastoma • Oncology • Solid Tumor • CSF2

REGAL: Galinpepimut-S Versus Investigator's Choice of Best Available Therapy for Maintenance in AML CR2/CRp2 (clinicaltrials.gov) - P3 | N=127 | Active, not recruiting | Sponsor: Sellas Life Sciences Group | Trial primary completion date: Sep 2025 ➔ Dec 2025

Monotherapy • Trial primary completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

A Rare Case of Pulmonary Alveolar Proteinosis. (PubMed, Cureus) - "Daily subcutaneous followed by inhaled recombinant GM-CSF (sargramostim 5 µg/kg) provided lasting symptom relief, reduced ground-glass lung infiltrates, and improved DLCO to 65% of predicted. PAP should be suspected in progressive respiratory failure with typical imaging findings, particularly when no secondary cause is identified. For patients who do not respond to WLL or recombinant GM-CSF, further studies are needed to define the best treatment approach."

Journal • Cough • Immunology • Infectious Disease • Interstitial Lung Disease • Pneumonia • Pulmonary Disease • Respiratory Diseases • CSF2

Pulmonary Alveolar Proteinosis: a 3-country cohort study Greece, Türkiye, Cyprus (ERS 2025) - "Whole lung lavage (WLL) in 51%, history of inhaled-GMCSF (sargramostim or molgramostim) in 44%. In Türkiye WLL was the mainstay of treatment, while in Greece i-GM-CSF. Secondary PAP, age and pulmonary fibrosis negatively influenced prognosis. i-GMCSF as the sole therapy was not inferior to WLL or WLL followed by i-GMCSF irrespective of the severity of patients."

Immunology • Pulmonary Disease • Rare Diseases • Respiratory Diseases • CSF2

Quadruple Immunotherapy for Neuroblastoma (clinicaltrials.gov) - P2 | N=29 | Recruiting | Sponsor: Hong Kong Children's Hospital | Trial primary completion date: Dec 2024 ➔ Dec 2025

Trial primary completion date • Neuroblastoma • Oncology • Pediatrics • Solid Tumor

Temozolomide and Survivin Long Peptide Vaccine (SurVaxM) for the Treatment of Patients With Progressing Metastatic Neuroendocrine Carcinomas (clinicaltrials.gov) - P2 | N=60 | Not yet recruiting | Sponsor: Roswell Park Cancer Institute | N=132 ➔ 60

Enrollment change • Endocrine Cancer • Neuroendocrine Carcinoma • Neuroendocrine Tumor • Oncology • Solid Tumor

Testing the SurVaxM Vaccine for Lung Cancer Prevention (clinicaltrials.gov) - P2 | N=80 | Not yet recruiting | Sponsor: National Cancer Institute (NCI)

New P2 trial • Lung Cancer • Oncology • Solid Tumor

Survivin Long Peptide Vaccine in Treating Patients With Metastatic Neuroendocrine Tumors (clinicaltrials.gov) - P1 | N=19 | Completed | Sponsor: Roswell Park Cancer Institute | Recruiting ➔ Completed | N=14 ➔ 19 | Trial completion date: Dec 2026 ➔ Mar 2025 | Trial primary completion date: Dec 2025 ➔ Feb 2025

Enrollment change • Trial completion • Trial completion date • Trial primary completion date • Carcinoid Tumor • Gastrointestinal Cancer • Neuroendocrine Tumor • Solid Tumor

Pharmacotherapy for Autoimmune Pulmonary Alveolar Proteinosis. (PubMed, Drugs) - "Inhaled granulocyte-macrophage colony-stimulating factor supplementation with molgramostim or sargramostim is now considered a first-line treatment in the international guidelines for autoimmune pulmonary alveolar proteinosis, following the positive results of recent randomized placebo-controlled studies. Rituximab and plasmapheresis can be prescribed as third- and fourth-line treatments, respectively. Lung transplantation may be considered for eligible patients experiencing terminal respiratory failure. A deeper understanding of the pathogenesis of autoimmune pulmonary alveolar proteinosis has opened up new therapeutic avenues, such as the use of PPARγ agonists or statins."

Journal • Immunology • Respiratory Diseases • Transplantation

SESAD: Phase II Trial to Evaluate Safety and Efficacy of GM-CSF/Sargramostim in Alzheimer's Disease (clinicaltrials.gov) - P2 | N=42 | Recruiting | Sponsor: University of Colorado, Denver | Trial completion date: Oct 2026 ➔ Oct 2027 | Trial primary completion date: Apr 2026 ➔ Apr 2027

Trial completion date • Trial primary completion date • Alzheimer's Disease • CNS Disorders

A Phase II Study With a Safety Run-In of the Addition of N-803 to a Chemoimmunotherapy Backbone for the Treatment of Patients With Relapsed or Refractory Neuroblastoma (clinicaltrials.gov) - P2 | N=54 | Not yet recruiting | Sponsor: St. Jude Children's Research Hospital | Initiation date: Jul 2025 ➔ Dec 2025

Trial initiation date • Neuroblastoma • Oncology • Solid Tumor • CSF2

Unravelling Neuronal Death Mechanisms: The Role of Cytokines and Chemokines in Immune Imbalance in Alzheimer's Disease Progression. (PubMed, Ageing Res Rev) - "Therapeutic drugs such as Magnolol, Necrostatin-1, Salidroside, Azeliragon, DNL788, Baricitinib, Sargramostim, etc. targeting neuroinflammation-associated signaling pathways, have shown efficacy in preclinical and clinical studies mitigating AD pathology. Enhancing our comprehension of neuronal death mechanisms could elucidate disease pathogenesis, offer insights for therapeutic approaches, and aid in developing modified animal models of AD."

Journal • Review • Alzheimer's Disease • CNS Disorders • Inflammation • AMPK • GPX4 • NLRP3 • RIPK1 • STING

A Multiple Antigen Vaccine (STEMVAC) for the Treatment of Patients With Stage IV Non-Small Cell Lung Cancer (clinicaltrials.gov) - P2 | N=5 | Active, not recruiting | Sponsor: University of Washington | N=40 ➔ 5

Enrollment change • Lung Cancer • Lung Non-Squamous Non-Small Cell Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor • ENG