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Treatment for high-risk neuroblastoma in children

High Risk Neuroblastoma Study 1 of SIOP-Europe (SIOPEN)

Phase 3 Interventional St. Anna Kinderkrebsforschung · NCT01704716

This study is testing a new treatment plan for children with high-risk neuroblastoma to see if a mix of chemotherapy, surgery, radiotherapy, and immunotherapy can help them survive better.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment3300 (estimated)
Ages1 Month to 21 Years
SexAll
SponsorSt. Anna Kinderkrebsforschung Academic / other
Drugs / interventionschemotherapy, Dinutuximab, immunotherapy, Doxorubicin
Locations126 sites (Adelaide and 125 other locations)
Trial IDNCT01704716 on ClinicalTrials.gov

What this trial studies

This study investigates a comprehensive treatment approach for children with high-risk neuroblastoma, which includes a combination of intensive chemotherapy, surgery, radiotherapy, and immunotherapy. The protocol specifically targets children with advanced stages of neuroblastoma, particularly those with MYCN amplification, to improve their chances of survival. Participants will undergo a series of treatments including peripheral blood stem cell harvest and myeloablative therapy, followed by either standard immunotherapy or an additional treatment with aldesleukin. The study aims to evaluate the effectiveness of these interventions in improving patient outcomes.

Who should consider this trial

Good fit: Ideal candidates for this study are children under 21 years old diagnosed with high-risk neuroblastoma, specifically those with MYCN amplification or advanced stages of the disease.

Not a fit: Patients who have previously received extensive chemotherapy or those with low-risk neuroblastoma may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve survival rates and reduce the risk of relapse in children with high-risk neuroblastoma.

How similar studies have performed: Previous studies have shown promising results with similar aggressive treatment approaches for high-risk neuroblastoma, indicating potential for success in this trial.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* • Established diagnosis of neuroblastoma according to the International Neuroblastoma Staging System (INSS).

  * Age below 21 years.
  * High risk neuroblastoma defined as either:

    1. INSS stage 2, 3, 4, and 4s with MYCN amplification, or
    2. INSS stage 4 without MYCN amplification aged \> 12 months at diagnosis
  * Patients who have received no previous chemotherapy except for one cycle of etoposide and carboplatin (VP16/Carbo). In this situation patients will receive Rapid COJEC induction and the first Rapid COJEC cycle may be replaced by the first cycle VP16/Carbo (etoposide / carboplatin).
  * Written informed consent, including agreement of parents or legal guardian for minors, to enter a randomised study if the criteria for randomisation are met.
  * Tumour cell material available for determination of biological prognostic factors.
  * Females of childbearing potential must have a negative pregnancy test. Patients of childbearing potential must agree to use an effective birth control method. Female patients who are lactating must agree to stop breast-feeding.
  * Registration of all eligibility criteria with the data centre within 6 weeks from diagnosis.
  * Provisional follow up of 5 years.
  * National and local ethical committee approval.

Exclusion Criteria:

Any negative answer concerning the inclusion criteria of the study

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Where this trial is running

Adelaide and 125 other locations

+76 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions NeuroblastomaneuroblastomaimmunotherapyMATantibody treatment
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.