NCT01857934CompletedPhase 2
Therapy for Children With Advanced Stage Neuroblastoma
Neuroblastoma Protocol 2012: Therapy for Children With Advanced Stage High-Risk Neuroblastoma
Source: ClinicalTrials.gov · Last updated 22 Sept 2026
Brief summary (registry, in English)
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).
- Status (registry)
- Completed
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 153 (actual)
- First submitted
- 16 May 2013
- Start date
- 5 Jul 2013
- Primary completion date
- 21 Oct 2021
- Lead sponsor
- St. Jude Children's Research Hospital
- Diseases
- Neuroblastoma, Childhood cancers
- NCT number
- NCT01857934
What you commit to
- Study type: Interventional · Phase 2
- Participants enrolled: 153
- From 5 Jul 2013 to the primary completion date, 21 Oct 2021
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Criteria the registry did not label
- PARTICIPANT Inclusion Criteria:
- Participants \<19 years of age (eligible until 19th birthday).
- Newly diagnosed, advanced stage, high-risk neuroblastoma defined as one of the following:
- Children \< 1 year with International Neuroblastoma Staging System (INSS) stage 2a, 2b, 3, 4 or 4S disease AND MYCN amplification (\>10 copies, or greater than four-fold increase in MYCN signal as compared to reference signal).
- INSS 2a or 2b disease AND MYCN amplification, regardless of age or additional biologic features
- INSS stage 3 AND:
- MYCN amplification (\>10 copies, or greater than four-fold increase in MYCN signal as compared to reference signal, regardless of age or additional biologic features
- Age \> 18 months (\> 547 days) with unfavorable pathology, regardless of MYCN status
- INSS stage 4 and:
- MYCN amplification, regardless of age or additional biologic features
- Age \> 18 months (\> 547 days) regardless of biologic features
- Age 12 - 18 months (365 - 547 days) with any of the following three unfavorable biologic features (MYCN amplification, unfavorable pathology and/or DNA index =1) or any biologic feature that is indeterminant/unknown
- Children at least 365 days initially diagnosed with: INSS stage 1, 2, 4S who progressed to a stage 4 without interval chemotherapy.
- Histologic proof of neuroblastoma or positive bone marrow for tumor cells with increased urine catecholamines.
- Adequate renal and hepatic function (serum creatinine \<3 x upper limit of normal for age, AST\< 3 x upper limit of normal).
- No prior therapy, unless an emergency situation requires local tumor treatment (discuss with principal investigator).
- Written, informed consent according to institutional guidelines.
- PARTICIPANT Exclusion Criteria:
- Any evidence, as judged by the investigator, of severe or uncontrolled systemic disease (e.g., unstable or uncompensated respiratory, cardiac, hepatic, or renal disease).
- Pregnant or breast feeding (female of child-bearing potential).
- Children with INSS 4 disease, age \<18 months with all 3 favorable biologic features (non-amplified MYCN, favorable pathology and DNA index \>1).
- DONOR Inclusion Criteria:
- Potential donor is a biologic parent
- Potential donor is at least 18 years of age.
Age in the registry: - to 18 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| St. Jude Children's Research Hospital Coordinator of this site? Confirm its status | Memphis | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.