Recurrent Childhood Soft Tissue Sarcoma, Recurrent Ewing Sarcoma, Recurrent Rhabdomyosarcoma
Conditions
Keywords
pediatric sarcomas, Ewing sarcoma, rhabdomyosarcoma, soft tissue sarcoma
Brief summary
The purpose of the study is to evaluate patients with refractory childhood sarcomas, who have been treated with a combination therapy of trabectedin and irinotecan (within compassionate use), to determine, if this is a promising treatment option with acceptable toxicity and if the results warrant a prospective study.
Interventions
DRUGCombined chemotherapy with trabectedin followed by irinotecan
Day 1: Trabectedin 1,1 - 1,5 mg/m²/day i.v. Day 3-5 and 10-12: Irinotecan 30- 90 mg/m²/day p.o. or i.v.
Sponsors
Technical University of Munich
Study design
Observational model
CASE_ONLY
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
10 Years to 40 Years
Healthy volunteers
No
Inclusion criteria
* Adolescents and young adults (age10 - 40 yoa) with refractory sarcoma, for which there are presently no further treatment options in EURO-Ewing/ EURAMOS/CWS (i.e. ≥ second relapse in ES, resistance to surgery in osteosarcoma, relapsed rhabdomyosarcoma) and have been treated with a combined chemotherapy of trabectedin and irinotecan (described in intervention) * existing matched control in existing study databases * have started of treatment within three months of last progress Key
Exclusion criteria
* Patients newly diagnosed or in first relapse.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Toxicity during therapy | As long as patient is undergoing therapy/ 6 months | WHO toxicity grading |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Quality of life | 12 months | Assessment of quality of life at the day before starting the next cycle and at months 3, 6, 9 and 12 after start of treatment, using adapted Karnofsky Score. |
| Assessment of therapeutic effect | 12 months | Read out after every two cycles (functional imaging, PET-MRT/PET-CT), evaluation according to RECIST. |
| Assessment of time to treatment failure | 12 months | Read out after every two cycles (functional imaging, PET-MRT/PET-CT), evaluation according to RECIST. |
Other
| Measure | Time frame | Description |
|---|---|---|
| Event free survival in comparison to matched controls. | 12 months | Comparison of event free survival in a matched pair analysis. |
| Overall survival in comparison to matched controls. | 12 months | Comparison of overall survival in a matched pair analysis. |
| Differences in response rate comparing response rate in Ewing Sarcoma to response rate in Soft Tissue Sarcoma | 12 months | Statistical comparison of response rate according to RECIST in Ewing Sarcoma vs. Soft Tissue Sarcoma |
Outcome results
None listed