TREATMENT OF TUMORS THAT OCCURRED AGAIN (NEUROBLASTOMA AND EWING´S SARCOMA) BASED ON HIGH DOSE CHEMOTHERAPY WITH TRANSPLANTATION OF PATIENT'S PROPER CELLS
- Conditions
- Ewing's sarcoma, neuroblastomaTherapeutic area: Diseases [C] - Cancer [C04]
- Registration Number
- EUCTR2015-002584-41-HU
- Lead Sponsor
- CEPOETA (Central European Pediatric Oncology Early Trials Alliance, z.s.)
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 35
1.Signed written informed consent (participant and parents when required).
2.Age: less than 25 years at the time of enrolement.
3.Diagnosis: relapsed high-risk neuroblastoma (rNB) or relapsed Ewing´s sarcoma (rES).
a.Confirmation of rES or rNB by biopsy or cytology.
b.At least partial remission to reinduction chemotherapy (at least 2 cycles of conventional chemotherapy or 3 months of metronomic therapy) and/or local therapy.
4.Performance status (Karnovsky/Lansky) = 40.
5.Renal, liver and cardiac functions not worse than grade III (CTC v4.0).
6.Negative pregnancy test in fertile women.
7.Stem cell product with a minimum of 2,0 x 106 CD34+/kg.
8.At least 28 days from the prior antitumour therapy.
Are the trial subjects under 18? yes
Number of subjects for this age range: 30
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 5
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1. Performance status (Karnofsky/Lansky < 40).
2. Toxicity levels related to prior therapy preventing the high-dose chemotherapy administration.
3. Pregnancy or breastfeeding.
4. Patient taking experimental study treatment with anticancer drug 14 days or less before the RENETA screening visit and concomitantly until the assessment of the primary objective at 3 months.
5. Confirmed allergic reaction to any of the study drugs.
6. Confirmed contraindication according to SmPC of any study drug.
7. Patient with uncontrolled psychiatric disorder and/or other life threatening conditions.
8. Refusal to use adequate contraception in fertile women.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate efficacy of TREO/MEL high dose chemotherapy conditioning + aPBSCT in patients with rNB and rES in terms of response rate defined as absence of progression* in 3 months.;Secondary Objective: •To evaluate occurrence of veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) 4 weeks after high dose chemotherapy. <br>•To evaluate safety and tolerability of high-dose chemotherapy TREO/MEL regimen and aPBSCT in children, adolescents and young adults with rES or rNB after FU 1 Visit (4 weeks after high-dose chemotherapy with TREO/MEL). <br>•To evaluate time to progression in the study population. <br>•To evaluate overall survival (OS) of the study population.<br>•To compare the study population survival data trends with available historical data.;Primary end point(s): Primary endpoint of efficacy<br>•Absence of the primary disease progression 3 months after aPBSCT.;Timepoint(s) of evaluation of this end point: <br>3 months after aPBSCT
- Secondary Outcome Measures
Name Time Method Secondary end point(s): Secondary endpoints<br>•Occurrence of VOD/SOS in each patient. <br>•Occurrence of organ toxicity grade III or IV according to CTCAE (v4.0)* (with special interest to cardiac, renal, hepatic toxicity) <br>*Common Terminology Criteria for Adverse Events (CTCAE), v4.0<br>•Time to progression (TTP). <br>•Overall survival (OS). ;Timepoint(s) of evaluation of this end point: During the whole course of the clinical trial for occurence of VOD/SOS, occurence of organ toxicity grade III or IV and TTP.<br>In 3, 6, 12, 18 and 24 months for OS