High-Risk Neuroblastoma Study 2 of SIOP-Europa-Neuroblastoma (SIOPEN)

Recruiting now · Phase 3

Conditions studied: High-Risk Neuroblastoma, Patient With Insufficient Response Chemoimmunotherapy

In brief

This is an international multicenter, open-label, randomized phase III trial including three sequential randomizations to assess efficacy of induction and consolidation chemotherapies and radiotherapy for patients with high-risk neuroblastoma.

Key facts

Study ID

NCT04221035

Run by

Gustave Roussy, Cancer Campus, Grand Paris

People needed

800

Starts

2019-11-05

Expected to finish

2032-11-01

Last updated by the study team

2025-06-26

Who can join

Age: any, up to 21. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Enrollment in HR-NBL2 will be performed:
• at diagnosis before the beginning of chemotherapy or
• up to 21 days after one course of Carboplatin-Etoposide for patients with localized neuroblastoma or infants with metastatic neuroblastoma with MYCN amplification or patients with metastatic neuroblastoma treated in emergency or
• up to 21 days after one course of the current protocol for R-I randomisation (RAPID COJEC/GPOH) low/intermediate risk neuroblastoma in Germany/Netherlands for patients with localized neuroblastoma or infants with metastatic neuroblastoma with MYCN amplification
• HR-NBL2 eligibility criteria:
• Established diagnosis of neuroblastoma according to the SIOPEN- modified International Neuroblastoma Risk Group (INRG) criteria, High-risk neuroblastoma defined as:
• Stage M neuroblastoma above 365 days of age at diagnosis (no upper age limit) and Ms neuroblastoma 12-18 months old, any MYCN status or
• L2, M or Ms neuroblastoma any age with MYCN amplification, or focal high level MYC or MYCL amplification.
• In Germany, patients aged less than 18 months with stage M and without MYCN amplification will not be enrolled in HR-NBL2 trial.
• No previous chemotherapy or up to 21 days after one cycle of Carboplatin-Etoposide chemotherapy for patients with localized neuroblastoma or infants with metastatic neuroblastoma with MYCN amplification or patients with metastatic neuroblastoma treated in emergency or up to 21 days after one course of the current protocol for low/intermediate risk neuroblastoma in Germany/Netherlands for patients with localized neuroblastoma or infants with metastatic neuroblastoma with MYCN amplification
• Females of childbearing potential must have a negative serum or urine pregnancy test within 7 days prior to initiation of treatment. Sexually active patients must agree to use acceptable and appropriate contraception while on HR-NBL2 study and for one year after stopping the study. Acceptable contraception is defined in CTFG Guidelines "Recommendations related to contraception and pregnancy testing in clinical trials" (Appendix 11). Female patients who are lactating must agree to stop breast-feeding.
• Written informed consent to enter the HR-NBL2 protocol from patient or parents/legal representative, patient, and age-appropriate assent.
• Patient affiliated to a social security regimen or beneficiary of the same according to local requirements.
• Patients should be able and willing to comply with study visits and procedures as per protocol
• R-I eligibility criteria:
• Written informed consent to enter the R-I randomisation from patient or parents/legal representative, patient, and age- appropriate assent.
• In case of parents'/patient's refusal to R-I, or Organ toxicity exclusion criteria at diagnosis, patients can still be enrolled in HR- NBL2 trial with parents'/patient's consent before or within 3 weeks from the beginning of chemotherapy
• R-HDC randomisation (Single HDC Bu-Mel/ Tandem HDC Thiotepa+Bu-Mel) Etoposide or one course of the current protocol for low/intermediate risk neuroblastoma in Germany/Netherlands). Patients will be treated with the standard induction regimen per country (Rapid COJEC or GPOH) and will be potentially eligible for subsequent randomisations.
• Randomisation for HDC strategy will be performed at the end of induction after the disease evaluation and after surgery of the primary tumour for those patients who will receive surgery before HDC.
• R-HDC eligibility criteria:
• - Stage M neuroblastoma above 365 days of age at diagnosis, any MYCN status, EXCEPT patients with stage M or Ms 12-18 months old with numerical chromosomal alterations only, and in complete metastatic response at the end of induction: in this case, patients will have surgery and no further treatment.
• OR
• L2, M or Ms neuroblastoma, any age, with MYCN amplification, or focal high level MYC or MYCL amplification
• Age < 21 years at the time of randomization
• Complete response (CR) or partial response (PR) at metastatic sites:

Where it is running

• CHU angers — Angers, France (enrolling)
• Groupe Hospitalier Pellegrin - Chu - Bordeaux — Bordeaux, France (enrolling)
• Gustave Roussy — Villejuif, Val De Marne, France (enrolling)
• CHU d'AMIENS — Amiens, France (enrolling)
• CHU Bordeaux — Bordeaux, France (enrolling)
• Sydney children Hospital — Sydney, Randwick, Australia (enrolling)
• Medical University Graz — Graz, Austria (enrolling)
• Children's Cancer & Haematology Services, John Hunter Children's Hospital — New Lambton Heights, Australia (enrolling)
• Kepler Universitatsklinikum Linz — Linz, Austria (enrolling)
• Children's Cancer Centre, Monash Children's Hospital — Clayton, Australia (enrolling)
• CHU-Pôle Médico-Chirurgical de l'Enfant et l'Adolescant — Besançon, France (enrolling)
• Hôpital Universitaire des Enfants Reine Fabiola (ULB) — Brussels, Belgium (enrolling)
• Cliniques Universitaires Saint-Luc (UCL) — Brussels, Belgium (enrolling)
• University Hospital Gent — Ghent, Belgium (enrolling)
• University Hospitals Leuven — Leuven, Belgium (enrolling)
• CHR Citadelle — Liège, Belgium (enrolling)
• University Hospital Motol — Prague, Prague, Czechia (enrolling)
• Landeskrankenhaus-Universitätsklinikum Innsbruck — Innsbruck, Austria (enrolling)
• Aarhus University Hospital — Aarhus, Denmark (enrolling)
• Department of Paediatrics and Adolescent Medicine, Rigshospitalet — Copenhagen, Denmark (enrolling)
• The Hans Christian Andersen Children's Hospital, University of Southern Denmark — Odense, Denmark (enrolling)
• Universitastsklinikum Salzburg — Salzburg, Austria (enrolling)
• St Anna'S Children Hospital — Vienna, Austria (enrolling)
• sydney children Hospital — Sydney, Australia (enrolling)
• CHU Brest — Brest, France (enrolling)

Full record on ClinicalTrials.gov

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