Ewing's sarcoma, neuroblastoma
Conditions
Interventions
Product Name: melphalan
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: MELPHALAN
CAS Number: 148-82-3
Concentration unit: mg milligram(s)
Concentration type: e
Sponsors
Masarykova univerzita
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 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 therapyb. 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
Exclusion criteria
Exclusion criteria: 1. Performance status (Karnofsky/Lansky < 40). 2. Toxicity levels related to prior therapy preventing the HDCt application 3. Pregnancy or breastfeeding. 4. Patients taking part in a clinical trial testing another anticancer drug or a drug with a potential anticancer effect 14 days or less before the screening visit. 5. Confirmed allergy reaction to any of the study drug. 6. Patient with uncontrolled psychiatric disorder.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 6 months. ;Secondary Objective: • To evaluate occurrence of veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) 4 weeks after high dose chemotherapy. • To evaluate safety and tolerability of high dose chemotherapy conditioning Treo/Mel regimen and aPBSCT in children, adolescents and young adults with rES or rNB 6, 12, 18 and 24 months after hight dose chemotherapy with TREO / MEL. • To evaluate time to progression in the study population. • To evauate overall survival (OS) of the study population 6, 12, 18 and 24 months after hight dose chemotherapy with TREO / MEL. • To compare the study population survival data trends with available historical data. ;Primary end point(s): Primary endpoint of efficacy • Absence of the primary disease progression 6 months after aPBSCT. ;Timepoint(s) of evaluation of this end point: 6 months after aPBSCT | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary endpoints • Occurrence of VOD/SOS in each patient. • Occurrence of organ toxicity grade III or IV according to CTCAE (v4.0)* (with special interest to cardiac, renal, hepatic toxicity) *Common Terminology Criteria for Adverse Events (CTCAE), v4.0 • Time to progression (TTP). • Overall survival (OS) in 6, 12, 18, 24 months. ;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. In 6, 12, 18 and 24 months for OS | — |
Countries
Czech Republic, Hungary, Slovenia
Contacts
Public ContactCentrum pro klinická hodnocení
Masarykova univerzita- Lékarská fakulta
Outcome results
None listed