Unituxin (dinutuximab) / United Therapeutics Corporation, Ohara Pharma - LARVOL DELTA

Rapid Administration Pilot for Infusing Dinutuximab (clinicaltrials.gov) - P2 | N=30 | Active, not recruiting | Sponsor: Children's Hospital Los Angeles | Phase classification: P1 ➔ P2 | N=11 ➔ 30 | Trial completion date: Jan 2026 ➔ Nov 2029 | Trial primary completion date: Jan 2026 ➔ Nov 2028

Enrollment change • Phase classification • Trial completion date • Trial primary completion date • Neuroblastoma • Oncology • Solid Tumor

A pilot induction regimen incorporating dinutuximab and sargramostim for the treatment of newly diagnosed high-risk neuroblastoma: A report from the Children's Oncology Group. (ASCO 2022) - P2 | "Chemoimmunotherapy including irinotecan, temozolomide, DIN and sargramostim (GM-CSF) in patients with recurrent or refractory neuroblastoma results in robust objective clinical responses. The administration of DIN and GM-CSF to COG Induction Cycles 3-5 for patients with newly-diagnosed high-risk neuroblastoma was tolerable and feasible. The objective response rate at EOI appears encouraging. This therapeutic regimen will be studied in a randomized phase 3 trial to further evaluate the efficacy of Induction phase chemoimmunotherapy for high-risk neuroblastoma."

Clinical • Hypotension • Neuroblastoma • Oncology • Pain • Solid Tumor • CSF2

STING: NK Cells Infusions With Irinotecan, Temozolomide, and Dinutuximab (clinicaltrials.gov) - P1/2 | N=31 | Active, not recruiting | Sponsor: Nationwide Children's Hospital | Recruiting ➔ Active, not recruiting

Enrollment closed • Neuroblastoma • Oncology • Solid Tumor

Preliminary experience of ex-vivo expanded allogeneic universal donor TGFβi NK cell infusions in combination with irinotecan, temozolomide, and dinutuximab in patients with relapsed or refractory neuroblastoma: The Allo - STING trial. (ASCO 2024) - P1/2 | "UD allo-TGFβi NK cells can be safely and feasibly administered to children with RR NBL after treatment with CIT, with early objective responses observed in the preliminary cohort of patients. Ongoing studies will assess markers of response, NK cell persistence, pharmacokinetics and pharmacodynamics."

Combination therapy • IO biomarker • Preclinical • CNS Tumor • Hematological Disorders • Neuroblastoma • Oncology • Solid Tumor • STING • TGFB1 • TGFBI

Phase I Study of 131I-Metaiodobenzylguanidine With Dinutuximab ± Vorinostat for Patients With Relapsed or Refractory Neuroblastoma: A New Approaches to Neuroblastoma Therapy Trial. (PubMed, J Clin Oncol) - "MIBG combined with dinutuximab was well tolerated with encouraging antitumor activity. Vorinostat added to this combination may augment responses in this heavily pretreated patient population."

Journal • P1 data • Fatigue • Neuroblastoma • Oncology • Pneumonia • Solid Tumor

NANT 2021-02: Randomized MIBG With Vorinostat/Dinutuximab/Vorinostat + Dinutuximab (clinicaltrials.gov) - P2 | N=118 | Not yet recruiting | Sponsor: New Approaches to Neuroblastoma Therapy Consortium | Trial completion date: Jul 2031 ➔ Dec 2031 | Initiation date: Jul 2026 ➔ Dec 2026 | Trial primary completion date: Jul 2030 ➔ Dec 2030

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

Combination Therapy with Pregabalin and Morphine Relieves Dinutuximab-Induced Pain in a 3-Year-Old Girl: A Case Report. (PubMed, Palliat Med Rep) - "This approach significantly reduced abdominal pain, with the maximum FPS score decreasing to 2. This case highlights that adding pregabalin to standard opioid therapy can contribute to improved management of DIN-induced pain."

Journal • Neuralgia • Neuroblastoma • Oncology • Pain • Pediatrics • Solid Tumor • Transplantation

Testing the Addition of Iberdomide to Therapy in People With Neuroblastoma That Has Come Back, Not Responded to Treatment, or Gotten Worse (clinicaltrials.gov) - P1/2 | N=76 | Not yet recruiting | Sponsor: National Cancer Institute (NCI) | Initiation date: Jul 2026 ➔ Oct 2026

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

Effect of Endocytosis Inhibitors on the Cytotoxicity and Antitumor Activity of Anti-GD2 ADCs. (PubMed, Acta Naturae) - "This study focuses on the mechanisms of receptor-mediated endocytosis for the conjugate formed between the anti-GD2 antibody ch14.18 and monomethyl auristatin E (MMAE), as well as the approaches for modulating this process using endocytosis inhibitors. Nystatin, a specific inhibitor of caveolin-mediated endocytosis, increased ADC accumulation in cells and reduced the IC50 1.5- to 2.5-fold depending on the cell line. Although administration of nystatin together with anti-GD2 ADCs did not enhance tumor growth inhibition compared to ADC monotherapy in the GD2- positive mouse tumor model, further optimization of combination therapy conditions aimed at enhancing receptor-mediated endocytosis is a promising strategy for potentiating the antitumor effects of anti-GD2 ADCs."

Journal • Oncology

Efficacy of anti-GD2 antibody immunotherapy with filgrastim and teceleukin versus standard treatment with sargramostim, aldesleukin and isotretinoin in children with high-risk neuroblastoma. (PubMed, Int J Clin Oncol) - "Our results suggest that the efficacy of the alternative regimen containing G-CSF is comparable to that of the ANBL0032 regimen, providing a rationale for further evaluation in a phase III trial."

Journal • Hematological Disorders • Infectious Disease • Neuroblastoma • Oncology • Solid Tumor • Transplantation • CSF2

Long-Term Outcomes of Patients With Relapsed/Refractory Neuroblastoma Treated on ANBL1221: A Report From the Children's Oncology Group. (PubMed, Pediatr Blood Cancer) - "Chemoimmunotherapy with irinotecan/temozolomide/dinutuximab is now used as both bridge therapy following suboptimal response to induction and as treatment in first relapse. Patients with early disease progression following treatment on ANBL1221 tended to have been diagnosed with high-risk neuroblastoma at a younger age than those without early disease progression. A subset of chemoimmunotherapy-treated patients survived ≥2 years, but most all received additional therapy."

Journal • Neuroblastoma • Oncology • Solid Tumor

Dinutuximab-beta and Chemotherapy in Newly Diagnosed High-Risk Neuroblastoma (clinicaltrials.gov) - P1 | N=30 | Not yet recruiting | Sponsor: Chang Gung Memorial Hospital

New P1 trial • Neuroblastoma • Oncology • Solid Tumor • MYCN

Circumventing Ewing sarcoma tumor microenvironment resistance by IL1RAP CAR-modified TGFβ1-imprinted natural killer cells in combination with IL-15 agonist and anti-GD2 antibody. (PubMed, J Immunother Cancer) - "Our preclinical data demonstrate that combinatorial innate immunotherapy leveraging tumor-targeting TGFβ1-imprinted IL1RAP-CAR-NK cells combined with an IL-15 agonist and an anti-GD2 antibody is a promising novel therapeutic strategy for targeting metastatic/relapsed/refractory ES."

Biomarker • IO biomarker • Journal • Ewing Sarcoma • Oncology • Osteosarcoma • Sarcoma • Solid Tumor • IL15 • IL1RAP • IL21 • TGFB1

Testing the Combination of Two Immunotherapy Drugs (Magrolimab and Dinutuximab) in Patients With Relapsed or Refractory Neuroblastoma or Relapsed Osteosarcoma (clinicaltrials.gov) - P1 | N=12 | Terminated | Sponsor: National Cancer Institute (NCI) | Completed ➔ Terminated; Drug supply issues

Trial termination • Neuroblastoma • Oncology • Osteosarcoma • Sarcoma • Solid Tumor

Comparative efficacy of tarlatamab and second-line therapies in extensive-stage small cell lung cancer: A network meta-analysis. (PubMed, Lung Cancer) - "Tarlatamab demonstrated statistically significant and clinically meaningful improvement in OS compared with all currently available 2L treatments for ES-SCLC in the overall 2L population and platinum-sensitive subgroup, supporting its potential as a new SOC."

Journal • Retrospective data • Lung Cancer • Oncology • Small Cell Lung Cancer • Solid Tumor

RAPID: A pilot feasibility study of rapid dinutuximab infusion in patients with relapsed/refractory (RR) high-risk neuroblastoma (HRNBL). (ASCO 2026) - P1 | "Patients received chemoimmunotherapy consisting of dinutuximab 17.5 mg/m²/day for 4 days (over 2-4 hours) combined with irinotecan/temozolomide (ITD) or cyclophosphamide/topotecan (CTD) for up to 6 cycles...Secondary and exploratory endpoints included infusion duration, serial pain scores assessed during and post-infusion, opioid use in morphine milligram equivalents (MME), toxicity, feasibility of outpatient administration, pharmacokinetics (PK) and human anti-chimeric antibody (HACA)... RID was feasible and well tolerated, including in the outpatient setting. Pain control was achieved with standard premedication and significantly reduced opioid requirements compared with standard infusion. These data support planned multi-institutional evaluation of RID."

Clinical • Cardiovascular • Endocrine Disorders • Heart Failure • Neuroblastoma • Pulmonary Disease • Solid Tumor

A study of the safety, efficacy, and pharmacokinetics of SPJ-101CA as maintenance therapy for newly diagnosed high-risk neuroblastoma. (ASCO 2026) - P | "Clinical Trial Registration Number: jRCT2031220687 Background: Isotretinoin (ISO), an oral vitamin A derivative, induces differentiation of neuroblastoma (NB) cells and is used as maintenance therapy...Dinutuximab was permitted only before or after, but not during, SPJ‑101CA administration... SPJ-101CA was well-tolerated and effective as maintenance therapy following multidisciplinary treatment for high-risk NB. The PK findings were consistent with the known profile of ISO and indicated good absorption of the formulation."

Clinical • PK/PD data • Atopic Dermatitis • CNS Disorders • Dyslipidemia • Endocrine Disorders • Hypertriglyceridemia • Immunology • Mental Retardation • Metabolic Disorders • Neuroblastoma • Neutropenia • Pruritus • Solid Tumor

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

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

PET Imaging of GD2 expression with 89Zr-DFO-Dinutuximab as a biomarker for malignant peripheral nerve sheath tumors (SNMMI 2026) - "Thus, GD2 expression could serve as a biomarker to differentiate between benign PNF and the transition to MPNST. In addition, uniformly high GD2 expression in MPNST supports the potential utility of GD2-targeted therapies for patients with metastatic or unresectable disease."

Biomarker • ADHD (Impulsive Aggression) • Astrocytoma • Attention Deficit Hyperactivity Disorder • Brain Cancer • Breast Cancer • CNS Disorders • Embryonal Tumor • Gastrointestinal Stromal Tumor • Genetic Disorders • Glioma • Hematological Malignancies • Leukemia • Melanoma • Neuroblastoma • Neurofibromatosis • Neurofibrosarcoma • Oncology • Osteosarcoma • Pilocytic Astrocytoma • Rhabdomyosarcoma • Sarcoma • Soft Tissue Sarcoma • Solid Tumor • B4GALNT1 • CDKN2A • NF1

PET Imaging of GD2 expression with 89Zr-DFO-Dinutuximab as a biomarker for malignant peripheral nerve sheath tumors (SNMMI 2026) - "Thus, GD2 expression could serve as a biomarker to differentiate between benign PNF and the transition to MPNST. In addition, uniformly high GD2 expression in MPNST supports the potential utility of GD2-targeted therapies for patients with metastatic or unresectable disease."

Biomarker • ADHD (Impulsive Aggression) • Astrocytoma • Attention Deficit Hyperactivity Disorder • Brain Cancer • Breast Cancer • CNS Disorders • Embryonal Tumor • Gastrointestinal Stromal Tumor • Genetic Disorders • Glioma • Hematological Malignancies • Leukemia • Melanoma • Neuroblastoma • Neurofibromatosis • Neurofibrosarcoma • Oncology • Osteosarcoma • Pilocytic Astrocytoma • Rhabdomyosarcoma • Sarcoma • Soft Tissue Sarcoma • Solid Tumor • B4GALNT1 • CDKN2A • NF1

PET Imaging of GD2 expression with 89Zr-DFO-Dinutuximab as a biomarker for malignant peripheral nerve sheath tumors (SNMMI 2026) - "Thus, GD2 expression could serve as a biomarker to differentiate between benign PNF and the transition to MPNST. In addition, uniformly high GD2 expression in MPNST supports the potential utility of GD2-targeted therapies for patients with metastatic or unresectable disease."

Biomarker • ADHD (Impulsive Aggression) • Astrocytoma • Attention Deficit Hyperactivity Disorder • Brain Cancer • Breast Cancer • CNS Disorders • Embryonal Tumor • Gastrointestinal Stromal Tumor • Genetic Disorders • Glioma • Hematological Malignancies • Leukemia • Melanoma • Neuroblastoma • Neurofibromatosis • Neurofibrosarcoma • Oncology • Osteosarcoma • Pilocytic Astrocytoma • Rhabdomyosarcoma • Sarcoma • Soft Tissue Sarcoma • Solid Tumor • B4GALNT1 • CDKN2A • NF1

KAT6A/B are Therapeutic Targets to Enhance the Response to GD2 Immunotherapy in Neuroblastoma (ASPHO 2026) - "Together, these studies identify KAT6A and KAT6B as previously unrecognized epigenetic regulators of GD2 expression and nominate KAT6A/B inhibition as a novel strategy to enhance differentiation therapy and GD2-directed immunotherapy for patients with high-risk neuroblastoma."

IO biomarker • Neuroblastoma • Solid Tumor • GATA3 • HOXB6 • KAT6A • KAT6B • MYCN • PHOX2B

Defining And Targeting Gamma Delta (γδ) T-Cell Induced Apoptosis in Neuroblastoma (ASPHO 2026) - "Design/ γδ T cells and NB cells were cocultured with and without venetoclax and dinutuximab for 48 hrs and cleaved caspase 3 was evaluated by IncuCyte. γδ T cells induce apoptosis in sensitive NBs via the mitochondrial pathway. Bcl-2 dependence in NBs induce γδ T cell resistance that can be overcome with the addition of venetoclax, supporting a novel therapy approach."

IO biomarker • Neuroblastoma • Solid Tumor • CASP3 • CASP8 • CASP9 • HAVCR2 • LAMP1 • PD-L1 • TIGIT

Ganglioside targeting: Exerting sweet revenge on neuroblastoma (AACR 2026) - "Dinutuximab is an FDA approved monoclonal antibody (mAb) against GD2, used in the treatment of high-risk NBL...These samples showed heterogeneous GD2, yet high GM2.Together these data shows, GD2 loss driven by immunotherapy and a concomitant increase in GM2. More importantly, it validates GM2 as an alternative target for GD2 low NBL and because GM2 has limited normal-tissue expression, it can be safely targeted alongside GD2 to prevent antigen escape"

IO biomarker • Neuroblastoma • Oncology • Solid Tumor

Efficacy and Safety of Anti-GD2 Monoclonal Antibodies Combined with GM-CSF in Relapsed and Refractory Neuroblastoma: A Systematic Review and Meta-analysis (AAN 2026) - "Results Thirteen studies (n = 2008) were included, evaluating hu14.18K322A (n = 172) and dinutuximab (n = 1836). Common adverse events included fever (31%), pain (42%), and hypotension (21%), with variable heterogeneity across studies. Conclusions Anti-GD2 combined with GM-CSF shows promising efficacy and tolerable toxicity in relapsed and refractory neuroblastoma, though further studies are needed to confirm consistency across treatment settings."

Retrospective data • Review • Hypotension • Neuroblastoma • Solid Tumor • CSF2