Clinical trial NCT00579501: Safety and Efficacy Study of Trabectedin for the Treatment of Localized Myxoid / Round Cell Liposarcoma
Official title: A Multicenter Phase II Clinical Trial of Neoadjuvant Trabectedin (Yondelis) in Patients With Localized Myxoid / Round Cell Liposarcoma
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Liposarcoma,Myxoid
Keywords: Myxoid
Liposarcoma
Trabectedin
Dexamethasone
Lead sponsor: Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Sponsor class: INDUSTRY
Collaborators: name: PharmaMar
First posted: 2007-12-24
Enrollment: 29
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the safety and efficacy of trabectedin for the treatment of localized (non-metastatic) myxoid / round cell liposarcoma (malignant tumor derived from primitive or embryonal lipoblastic cells).

Detailed description: This is an open-label (all people know the identity of the intervention), prospective (study following participants forward in time), multicenter (when more than one hospital or medical school team work on a medical research study) study of trabectedin for the treatment of localized myxoid / round cell liposarcoma (MRCL). Trabectedin will be given at 1.5 milligram per meter square (mg/m\^2) over a 24-hour intravenous (iv) infusion every 3 weeks for a minimum of 3 and a maximum of 6 cycles along with dexamethasone 20 mg iv which will be given within 30 minutes before start of each trabectedin iv infusion. Participants whose myxoid/round cell liposarcoma (MRCL) do not progress at the end of the neoadjuvant treatment will be followed every 6 weeks for disease progression or until 6 months post definitive surgery, in the absence of unacceptable toxicity and/or disease progression. Efficacy will be assessed by determining the pathologic Complete Response (pCR) rate assessed in the tumor surgical specimen by a central pathology review. Participants' safety will be monitored throughout the trial.

Eligibility criteria: Inclusion Criteria:

* Pathological diagnosis of myxoid / round cell liposarcoma (MRCL) and availability of pathology specimens for central review and pharmacogenomic studies
* Clinical evidence of locally advanced (Stage III), non-metastatic tumor, including locally recurring disease after initial surgery
* Measurable disease (by Response Evaluation Criteria In Solid Tumors \[RECIST\])
* No prior chemotherapy or radiation (except for adjuvant post-operative radiotherapy)
* Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) 0-2

Exclusion Criteria:

* Known hypersensitivity to any of the components of the trabectedin intravenous (iv) formulation or dexamethasone
* Pregnant or lactating women and women of reproductive potential who are not using effective contraceptive methods
* 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

Primary outcomes:
measure: Percentage of Participants With Pathological Complete Response (pCR)
timeFrame: Every 6 weeks until disease progression (up to Week 33) or 6 months post surgery.
Secondary outcomes:
measure: Percentage of Participants With Objective Tumor Response Based on Response Evaluation Criteria In Solid Tumors (RECIST)
timeFrame: Every 6 weeks until disease progression (up to Week 33) or 6 months post surgery.
Other outcomes:
Locations:
city: Coeur D Alene
country: United States
state: Idaho
city: Park Ridge
country: United States
state: Illinois
city: Iowa City
country: United States
state: Iowa
city: Boston
country: United States
state: Massachusetts
city: Bourdeaux
country: France
city: Lyon
country: France
city: Villejuif
country: France
city: Mannheim
country: Germany
Study officials:
affiliation: Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
name: Johnson & Johnson Pharmaceutical Research & Development, L.L. C. Clinical Trial
role: STUDY_DIRECTOR
References:
citation: Gronchi A, Bui BN, Bonvalot S, Pilotti S, Ferrari S, Hohenberger P, Hohl RJ, Demetri GD, Le Cesne A, Lardelli P, Perez I, Nieto A, Tercero JC, Alfaro V, Tamborini E, Blay JY. Phase II clinical trial of neoadjuvant trabectedin in patients with advanced localized myxoid liposarcoma. Ann Oncol. 2012 Mar;23(3):771-776. doi: 10.1093/annonc/mdr265. Epub 2011 Jun 3.