Phase II pilot study of hu14.18K322A plus chemotherapy in 153 children with high-risk neuroblastoma

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ClinicalTrials.gov Published June 4, 2026 Medically reviewed June 4, 2026 Study authors: St. Jude Children's Research Hospital NCT01857934 ↗ By Dr. Sofia Müller, MD · Lifespan & Whole-Person Care

Key Takeaway

Note that specific efficacy and safety numbers were not reported for this Phase II pilot study of hu14.18K322A in neuroblastoma.

This Phase II pilot study evaluated a regimen involving hu14.18K322A combined with induction chemotherapy consisting of cyclophosphamide and topotecan. The population comprised 153 previously untreated children with high-risk neuroblastoma. The intervention also included peripheral blood stem cell harvest, surgical resection, GM-CSF, interleukin-2, and allogeneic natural killer (NK) cells. The study setting was not reported, and the follow-up duration was 99.5 months.

Primary outcomes assessed were overall response rate, defined as the sum of complete response, very good partial response, and partial response, and event-free survival. Secondary outcomes included the feasibility of delivering hu14.18K322A to six cycles of induction chemotherapy, antitumor activity, local control patterns, and tolerability of various dosing schedules with allogeneic NK cells, interleukin-2, and GM-CSF.

Specific numerical results for the primary and secondary outcomes were not reported in the available data. Furthermore, details regarding adverse events, serious adverse events, discontinuations, and overall tolerability were not reported. Consequently, the safety profile and specific efficacy rates cannot be quantified from this summary.

As a Phase II pilot study with unreported safety data and missing specific outcome numbers, the practice relevance remains uncertain. The evidence is observational in nature regarding the pilot phase, and causality cannot be overstated. Clinicians should interpret these findings as preliminary and await further data before integrating these complex regimens into standard care.

Study Details

Study typePhase2

Sample sizen = 153

EvidenceLevel 3

Follow-up99.5 mo

PublishedApr 2026

TrialNCT01857934

View Original Abstract ↓

Status: COMPLETED | Phase: PHASE2 Condition(s): Neuroblastoma Intervention(s): cyclophosphamide (DRUG), topotecan (DRUG), hu14.18K322A (BIOLOGICAL), peripheral blood stem cell harvest (PROCEDURE), surgical resection (PROCEDURE) Neuroblastoma is the most common extracranial solid tumor in childhood, with nearly 50% of patients presenting with widespread metastatic disease. The current treatment for this group of high-risk patients includes intensive multi-agent chemotherapy (induction) followed by myeloablative therapy with stem-cell rescue (consolidation) and then treatment of minimal residual disease (MRD) with isotretinoin. Recently a new standard of care was established by enhancing the treatment of MRD with the addition of a monoclonal antibody (ch14.18) which targets a tumor-associated antigen, the disialoganglioside GD2, which is uniformly expressed by neuroblasts. Despite improvement in 2-year event-free survival (EFS) of 20%, more than one-third of children with high-risk neuroblastoma (HR defined in) still cannot be cured by this approach. Therefore, novel therapeutic approaches are needed for this subset of patients. This study will be a pilot Phase II study of a unique anti-disialoganglioside (anti-GD2) monoclonal antibody (mAb) called hu14.18K322A, given with induction chemotherapy. PRIMARY OBJECTIVE: * To study the efficacy \[response: complete remission + partial remission (CR+PR)\] to two initial courses of cyclophosphamide and topotecan combined with hu14.18K322A (4 doses/course followed by GM-CSF) in previously untreated children with high-risk neuroblastoma. * To estimate the event-free survival of patients with newly diagnosed high-risk neuroblastoma treated with the addition of hu14.18K322A to treatment. SECONDARY OBJECTIVES: * To study the feasibility of delivering hu14.18K322A to 6 cycles induction chemotherapy and describe the antitumor activity (CR+PR) of this 6 course induction therapy. * To estimate local control and pattern of failure associated with focal intensity modulated or proton beam radiation therapy dose delivery in high-risk abdominal neuroblastoma. * To describe the tolerability of four doses of hu14.18K322A with allogeneic natural killer (NK) cells from an acceptable parent, in the immediate post-transplant period \[day +2 - +5 after peripheral blood stem cell (PBSC) infusion\] in consenting participants. * To describe the tolerability of hu14.18K322A with interleukin-2 and GM-CSF as treatment for minimal residual disease (MRD). Detailed: The phases of the study are: 1. Screening phase: Tests and evaluations will be done before treatment starts. 2. Induction phase: Includes chemotherapy plus hu14.18K322A mAb. Participants will also have surgery during this part of the study to remove as much tumor as possible. 3. Consolidation/Intensification phase: Includes high doses of chemotherapy and blood stem cell transplantation with additional, experimental "minimal residual disease" (MRD) treatment. Participants will also get radiation treatment to all sites of the tumor(s) after recovery from the stem cell transplant. 4. Maintenance/MRD treatment phase: With immune therapy in addition to the standard treatment with the drug isotretinoin. Primary Outcome(s): Overall Response Rate [Complete Response + Very Good Partial Response + Partial Response (CR + VGPR + PR)]; Event-free Survival (EFS) Enrollment: 153 (ACTUAL) Lead Sponsor: St. Jude Children's Research Hospital Start: 2013-07-05 | Primary Completion: 2021-10-21 Results posted: 2023-02-24