E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Localized myxoid / round cell liposarcoma (MRCL). |
|
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 6.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10024630 |
|
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To determine the pathological complete response (pCR) rate with trabectedin in patients with localized myxoid / round cell liposarcoma (MRCL). |
|
E.2.2 | Secondary objectives of the trial |
Evaluate the objective response rate by RECIST and contrast such response with changes in radiological density and tumor pathology. Describe the incidence and severity of adverse events in this patient population. Exploratory, hypothesis-generating pharmacogenomic analyses to correlate molecular parameters in patient samples with clinical outcomes (pCR). |
|
E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
PatientŽs written informed consent before any study-specific procedure Adult patients (more than or equal to 18 years)
Pathological diagnosis of myxoid / round cell liposarcoma (MRCL) and availability of pathology specimens for central review and pharmacogenomic studies
Clinical evidence of localized, non-metastatic tumor, including locally recurring disease after initial surgery
Measurable disease (by RECIST)
No prior chemotherapy or radiation (except for adjuvant post-operative radiotherapy)
Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) 0-2
Hematologic variables:
oHemoglobin more than or equal to 9 g/dL
oAbsolute neutrophil count (ANC)more than or equal to 1,500/µL, and oPlatelet count more than or equal to 100,000/µL
Serum creatinine less than or equal to 1.5 mg/dL or creatinine clearance more than or equal to 30 mL/min
Creatinine phosphokinase (CPK) less than or equal to 2.5 ULN
Hepatic function variables:
oTotal bilirubin less than or equal to ULN
oTotal alkaline phosphatase less than or equal to 2.5 ULN, or if > 2.5 ULN consider alkaline phosphatase liver fraction or GGT or 5 nucleotidase must be less than or equal to ULN
o AST (serum aspartate transaminase [SGOT]) and ALT (serum alanine transaminase [SGPT]) must be less than or equal to 2.5 x ULN
Albumin more than or equal to 25 g/L |
|
E.4 | Principal exclusion criteria |
Known hypersensitivity to any of the components of the trabectedin i.v. formulation or dexamethasone
Pregnant or lactating women or any patient of childbearing potential who are not employing adequate contraception
History of another neoplastic disease (except basal cell carcinoma or cervical carcinoma in situ adequately treated) unless in remission for 5 years or longer
Known distant metastases
Other serious illnesses such as congestive heart failure or angina pectoris, myocardial infarction within 1 year before enrollment, uncontrolled arterial hypertension or arrhythmias
Psychiatric disorder that prevents compliance with protocol
Active viral hepatitis or chronic liver disease
Active infection
Any other unstable medical condition |
|
E.5 End points |
E.5.1 | Primary end point(s) |
The primary efficacy evaluation will be the central assessment of pathological response in the surgical specimen. |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | Yes |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Information not present in EudraCT |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
|
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
|
E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
| |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 0 |