RIST-rNB 2011

Author: Julia Dobke, erstellt am: 2013/03/01, Last modification: 2013/05/22

RIST-rNB 2011 Prospective, open label, randomized phase II trial to assess a multimodal molecular targeted therapy in children, adolescent and young adults with relapsed or refractory high-risk neuroblastoma
Disease Children, Adolescent and Young Adults with Relapsed or Refractory High-risk Neuroblastoma
Type Prospective, Open Label, Randomized Phase II Trial
Problem / Objectives

Children, adolescents and young adults with high risk relapsed or treatment refractory neuroblastoma (rNB) represent a group of patients with dismal prognosis for whom a recommended standard salvage therapy is currently not available.
The multimodal metronomic approach combining molecular targeted drugs (rapamycin and dasatinib) with conventional chemotherapy (irinotecan and temozolomide) will be investigated in a randomized fashion as new treatment strategy for patients with rNB. The intention is to assess the therapeutic benefit of molecular targeted drugs for the treatment of rNB.
The primary objective of this trial is the evaluation of progression-free survival of rNB in children, adolescents and young adults, comparing a multimodal treatment regimen consisting of temozolomide (T), irinotecan (I), sirolimus (R) and dasatinib (S) against irinotecan and temozolomide (I/T) alone.

Inclusion Criteria
  • all relapsed neuroblastomas grade IV
  • all relapsed neuroblastomas grade I – III & IVS N-MYC pos.
  • Age12 month - <25 years
  • Signed written informed consent
  • birth control with a pearl index <1%
  • Non-smoker for at least the previous 3 months
  • Abstain from alcohol within the last 24 hours before screening and before admission to the clinical trial center as well as during the entire clinical trial
  • ANC ≥500/µL
  • hemoglobin ≥n 8g/dL (transfusion permitted)
  • platelet count ≥30, 000/µL u
  • weight ≥10 kg
Exclusion Criteria
  • Pregnancy, nursing
  • Patients who suffered from a thrombotic event and need anticoagulation (i.e. coumadine derivatives or low molecular weight heparin derivatives, LMWH)
  • Patients with cardiac arrhythmias especially prolonged QT
  • ECG with QTc>500 msec / QTc>60 msec baseline
  • Patients with chronic inflammatory bowel diseases and/or bowel obstruction
  • Patients with bilirubin serum levels 3 fold above the upper normal limit
  • Vaccination with a live virus vaccine during the clinical trial
  • Impaired liver function and/or impaired renal function (hepatic and renal index parameter two times above normal range; see below)
  • Potentially unreliable subjects, probably non compliant subjects and those judged by the investigator to be unsuitable for the study
  • Known allergic reactions to the treatment medication
  • Patients who were treated with radiation and/or chemotherapy for any other oncological condition
  • Participation in any other phase I to III trial
  • Patients with extremely poor general condition (Karnofksy or Lansky score <50%
Recruitment Prospective, Open Label, Randomized Phase II Trial
Status 07/2013-12/2018
EudraCT 2011-004062-15
Entry Study Register ClinicalTrials.gov: NCTNCTNCT01467986
Principal Investigator Prof. Dr. med. Selim Corbacioglu
Contact

Investigator

Prof. Dr. med. Selim Corbacioglu Universitätsklinikum Regensburg, Klinik und Poliklinik für Kinder- und Jugendmedizin Abt. Pädiatrische Hämatologie, Onkologie und SZT Franz-Josef-Strauss-Allee 11 93053 Regensburg Telefon +49 (941)-944 2101 Fax +49 (941)-944-2102 selim.corbacioglu@ukr.de

Coordinator

Susanne Ellinger Universitätsklinikum Regensburg, Klinik und Poliklinik für Kinder- und Jugendmedizin Abt. Päd. Hämatologie, Onkologie und SZT Franz-Josef-Strauss-Allee 11 93053 Regensburg Telefon +49 (941) 944 2063 Fax +49 (941) 944 2068 Susanne.Ellinger@ukr.de