An international multicenter phase II randomised trial evaluating and comparing two intensification treatment strategies for metastatic neuroblastoma patients with a poor response to induction chemotherapy

Phase 2

Completed

Conditions: Neuroblastoma
tumour of the peripheral autonomic symphatetic nervous system
10029211

Registration Number: NL-OMON49491

Lead Sponsor: Gustave Roussy

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 16

Inclusion Criteria

1. Metastatic neuroblastoma (NBL).
2. Previously treated within the ongoing High Risk Neuroblastoma SIOPEN study.
3. 131I-mIBG scintigraphy positive at diagnosis and after induction
chemotherapy (before high-dose BuMel + ASCT).
4. Metastatic response after induction chemotherapy lower to the ongoing High
Risk Neuroblastoma
SIOPEN trial criteria to be eligible for High Dose Chemotherapy; metastatic
response worse than
partial response (< PR) or SIOPEN score >= 3.
5. Females of childbearing potential must have a negative serum pregnancy test
within 7 days prior
to initiation of treatment. Sexually active patients must agree to use
acceptable and appropriate
contraception while on study drug and for one year after stopping the study
drug. Acceptable
contraception are defined in CTFG Guidelines *Recommendations related to
contraception and
pregnancy testing in clinical trials*. Female patients who are lacting must
agree to stop breastfeeding.
6. Written informed consent from parents/legal representative, patient, and
age-appropriate assent
before any study-specific screening procedures are conducted according to
local, regional or
national guidelines.
7. Patient affiliated to a social security regimen or beneficiary of the same
according to local
requirements.

Exclusion Criteria

1. Parenchymal brain metastasis(es) (even one)
2. Progressive disease at study entry
3. Previous high-dose therapy and PBSCT
4. Performance status (Karnofsky or Lansky) <70%
5. Patient having received other therapy for cancer treatment than those
allowed as per the ongoing
High Risk Neuroblastoma SIOPEN trial or as defined in the future frontlines
protocol (for HRNBL1
trial : after induction + 2 TVD)
6. Impaired organ function (liver, kidney, heart, lungs) according to the
following definitions
o Shortening fraction <28%, or ejection fraction <55%, or clinical evidence of
congestive heart
failure or uncontrolled cardiac rythm disturbance
o Dyspnea at rest and/or pulse oxymetry <95% in air.
o ALT, Bilirubin >= 2 ULN
o Creatinine clearance and/or GFR <= 60 ml/min/1.73m2 and serum creatinine < 1.5
mg/dl
7. Any uncontrolled intercurrent illness or infection that in the
investigator*s opinion would impair
study participation
8. Concomitant use with yellow fever vaccine and with live virus and bacterial
vaccines
9. Patient allergic to peanut or soya
10. Chronic inflammatory bowel disease and/or bowel obstruction
11. Pregnant or breastfeeding women
12. Known hypersensitivity to the active substance or to any of the excipients
of study drugs
13. Known hypersensitivity to dacarbazine
14. Concomitant use with St John's Wort

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The primary endpoint is the 3 years Event-Free Survival from the date of<br /><br>randomisation into the<br /><br>VERITAS trial, considering as events: disease progression or relapse, death<br /><br>from any cause, and secondary malignancy. Patients without event are censored<br /><br>at the date of their last follow up evaluation.</p><br>

Secondary Outcome Measures