NCT01704716 High Risk Neuroblastoma Study 1.8 of SIOP-Europe (SIOPEN)
| NCT ID | NCT01704716 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | St. Anna Kinderkrebsforschung |
| Condition | Neuroblastoma |
| Study Type | INTERVENTIONAL |
| Enrollment | 3,300 participants |
| Start Date | 2002-02 |
| Primary Completion | 2021-09-30 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 3 trials are large pivotal studies comparing the treatment to current standard of care or placebo. Your participation directly contributes to the evidence needed for regulatory approval.
This trial targets 3,300 participants in total. It began in 2002-02 with a primary completion date of 2021-09-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
This is a randomized study of the European SIOP Neuroblastoma Group (SIOPEN) in high-risk neuroblastoma (stages 2, 3, 4 and 4s MYCN-amplified neuroblastoma, stage 4 MYCN non amplified \> 12 months at diagnosis). The protocol consists of a rapid, dose intensive induction chemotherapy, peripheral blood stem cell harvest, attempted complete excision of the primary tumour, myeloablative therapy followed by peripheral blood stem cell rescue, radiotherapy to the site of the primary tumour and immunotherapy (R4 randomization - isotretinoin and ch14.18/CHO (Dinutuximab beta, Qarziba ®).), with or without s.c. aldesleukin (IL-2)). Patients diagnosed after the closure of R3 randomization will not be R4 randomized. For these patients the use of ch14.18/CHO antibody is recommended without scIL-2 as continuous infusion as standard of care outside of controlled trials. ch14.18/CHO received marketing authorization by EMA in May 2017 (Qarziba ®). In the induction phase, all patients receive Rapid COJEC following the result of the R3 randomization which was closed on June 8th, 2017 after inclusion of 630 patients as planned. Following induction treatment peripheral blood stem cell harvest (PBSCH) is performed and complete excision of the primary tumour will be attempted. Patients with an inadequate metastatic response to allow BuMel MAT followed by PBSCR at the end of induction should receive 2 TVD (Topotecan, Vincristine, Doxorubicin) cycles. After Rapid COJEC induction, localized patients will proceed to consolidation. Patients aged 12-18 months at diagnosis, with stage 4 neuroblastoma, no MYCN amplification and without segmental chromosomal alterations (SCAs) are thought to have a good prognosis and will stop treatment after induction therapy and surgery to the primary tumour. Consolidation consists of BuMel MAT based on the results of the R1 randomization followed by peripheral blood stem cell rescue (PBSCR) and radiotherapy to the site of the primary tumour. The R2 immunotherapy randomization using ch14.18/CHO as 8 hour infusion on 5 consecutive days ( total dose (100mg/m²) with or without aldesleukin (IL-2) alternated with isotretinoin (13-cis-RA) is closed. The amended R4 immunotherapy randomization using ch14.18/CHO as continuous infusion (total dose 100mg/m² over 10 days) with or without aldesleukin (IL-2) alternated with isotretinoin (13-cis-RA) has accrued according to plan with results pending awaiting data maturity and DMC approval.
Eligibility 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 \-
Contact & Investigator
Ruth L Ladenstein, MD, MBA, cPM
PRINCIPAL INVESTIGATOR
St. Anna Kinderkrebsforschung
Frequently Asked Questions
Who can join the NCT01704716 clinical trial?
This trial is open to participants of all sexes, aged 1 Month or older, up to 21 Years, studying Neuroblastoma. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT01704716 trial and what does that mean for participants?
Phase 3 trials are large-scale studies comparing the new treatment to existing standards of care or a placebo. They provide the evidence needed for regulatory approval. This trial targets 3,300 participants.
Is NCT01704716 currently recruiting?
Yes, NCT01704716 is actively recruiting participants. Contact the research team at ruth.ladenstein@ccri.at for enrollment information.
Where is the NCT01704716 trial being conducted?
This trial is being conducted at Adelaide, Australia, Brisbane, Australia, Newcastle, Australia, Parkville, Australia and 11 additional locations.
Who is sponsoring the NCT01704716 clinical trial?
NCT01704716 is sponsored by St. Anna Kinderkrebsforschung. The principal investigator is Ruth L Ladenstein, MD, MBA, cPM at St. Anna Kinderkrebsforschung. The trial plans to enroll 3,300 participants.