Trabectedin and Irinotecan for Refractory Pediatric Sarcomas

Technical University of Munich30 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Recurrent Childhood Soft Tissue Sarcoma Recurrent Ewing Sarcoma Recurrent Rhabdomyosarcoma

Interventions

Combined chemotherapy with trabectedin followed by irinotecanDRUG

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.

Eligibility

Sex: ALLMin age: 10 YearsMax age: 40 Years

Medical Language ↔ Plain English

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.

Outcomes

Primary Outcomes

Toxicity during therapy

WHO toxicity grading

Time frame: As long as patient is undergoing therapy/ 6 months

Secondary Outcomes

Quality of life

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.

Time frame: 12 months

Assessment of therapeutic effect

Read out after every two cycles (functional imaging, PET-MRT/PET-CT), evaluation according to RECIST.

Time frame: 12 months

Assessment of time to treatment failure

Read out after every two cycles (functional imaging, PET-MRT/PET-CT), evaluation according to RECIST.

Time frame: 12 months

Data from ClinicalTrials.gov

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