Liposarcoma,Myxoid
Conditions
Keywords
Myxoid, Liposarcoma, Trabectedin, Dexamethasone
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.
Interventions
DRUGTrabectedin
Trabectedin 1.5 mg/m\^2 over a 24-hour iv infusion every 3 weeks for a minimum of 3 and a maximum of 6 cycles of trabectedin.
DRUGDexamethasone
Dexamethasone 20 mg iv will be administered within 30 minutes before start of each trabectedin iv infusion
Sponsors
PharmaMar
Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With Pathological Complete Response (pCR) | Every 6 weeks until disease progression (up to Week 33) or 6 months post surgery. | Complete pathological response is complete disappearance of the tumor tissue up to the molecular level. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With Objective Tumor Response Based on Response Evaluation Criteria In Solid Tumors (RECIST) | Every 6 weeks until disease progression (up to Week 33) or 6 months post surgery. | The objective tumor response is defined as the percentage of participants achieving partial response (PR) on tumor response assessed by RECIST. The PR is at least 30 percent decrease in sum of the longest diameter of target lesions or persistence of one or more non-target lesion(s) or/and maintenance of tumor marker level above the normal limits. |
Countries
France, Germany, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Trabectedin Trabectedin at a dose of 1.5 milligram per meter square (mg/m\^2) was given as an intravenous (iv) infusion (a fluid or a medicine delivered into a vein by way of a needle) over 24-hour every 3 weeks for a minimum of 3 and a maximum of 6 cycles prior to definitive surgery. Dexamethasone 20 mg iv was also administered within 30 minutes before start of each trabectedin infusion. | 29 |
| Total | 29 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Death | 1 |
| Overall Study | End of neoadjuvant therapy | 25 |
| Overall Study | Radiation therapy before surgery | 1 |
| Overall Study | Secondary lesion | 1 |
| Overall Study | toxicity | 1 |
Baseline characteristics
| Characteristic | Trabectedin |
|---|---|
| Age, Customized 18-49 years | 15 participants |
| Age, Customized 50-69 years | 10 participants |
| Age, Customized greater than or equal to 70 years | 4 participants |
| Sex: Female, Male Female | 13 Participants |
| Sex: Female, Male Male | 16 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 28 / 29 |
| serious Total, serious adverse events | 6 / 29 |
Outcome results
Primary
Percentage of Participants With Pathological Complete Response (pCR)
Complete pathological response is complete disappearance of the tumor tissue up to the molecular level.
Time frame: Every 6 weeks until disease progression (up to Week 33) or 6 months post surgery.
Population: Participants who received at least one cycle of trabectedin and have adequate pre and post trabectedin pathologic specimens available. Six participants were non evaluable for pCR assessment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Trabectedin | Percentage of Participants With Pathological Complete Response (pCR) | 13 percentage of participants |
Secondary
Percentage of Participants With Objective Tumor Response Based on Response Evaluation Criteria In Solid Tumors (RECIST)
The objective tumor response is defined as the percentage of participants achieving partial response (PR) on tumor response assessed by RECIST. The PR is at least 30 percent decrease in sum of the longest diameter of target lesions or persistence of one or more non-target lesion(s) or/and maintenance of tumor marker level above the normal limits.
Time frame: Every 6 weeks until disease progression (up to Week 33) or 6 months post surgery.
Population: Participants who received at least one cycle of trabectedin and have at least one post baseline disease assessment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Trabectedin | Percentage of Participants With Objective Tumor Response Based on Response Evaluation Criteria In Solid Tumors (RECIST) | 24.1 percentage of participants |