Clinical trial NCT00210665: A Study to Provide Access to Trabectedin in Participants With Locally Advanced or Metastatic Soft Tissue Sarcoma Who Have Persistent or Recurrent Disease and Who Are Not Expected to Benefit From Currently Available Standard of Care Treatment
Official title: A Multicenter, Open-Label, Single-Arm Study of YONDELIS (Trabectedin) for Subjects With Locally Advanced or Metastatic Soft Tissue Sarcoma Who Have Relapsed or Are Refractory to Standard of Care Treatment
Study type: EXPANDED_ACCESS
Overall status: NO_LONGER_AVAILABLE
Conditions: Sarcoma
Keywords: Sarcoma
Soft tissue sarcoma
Standard care
Trabectedin
Antitumor agent
Safety
Lead sponsor: Janssen Research & Development, LLC
Sponsor class: INDUSTRY
First posted: 2005-09-21
Sex: ALL
Minimum age (years): 15
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no

Brief summary: The purpose of this study is to facilitate access to trabectedin for eligible previously treated patients with soft tissue sarcoma (STS), who cannot be expected to benefit from currently available therapeutic options but who may benefit from treatment with trabectedin. The safety profile of trabectedin will be evaluated to further assess the potential risks of trabectedin treatment.

Detailed description: This is a multicenter, open-label (all people know the identity of the intervention), single-arm study. It will consist of 2 Phases: a Screening Phase (up to 21 days before the first dose administration), and Treatment Phase (for patients meeting the continuation criteria). During the Treatment Phase, patients will receive a dose of 1.5 mg/m2 trabectedin intravenous formulation administered as a 24-hour infusion on Day 1 of each suggested 21-day treatment cycle. All patients will receive 20 mg dexamethasone (or corticosteroid equivalent to dexamethasone). Number of cycles is not specified for this study. Patients may continue to receive treatment as long as they obtain an overall clinical benefit, ie, until there is clear evidence of disease progression or unacceptable toxicity, as judged by the investigator. Trabectedin is the first of a new class of antitumor agents. Previous studies with trabectedin in patients who had been previously treated for soft tissue sarcoma have suggested that treatment with trabectedin resulted in tumor shrinkage, disease stabilization, and improved survival rates. However, hematologic toxicity, hepatic toxicity, and renal impairment were also observed in these patients. The safety profile of trabectedin will be evaluated to further assess the potential risks of trabectedin treatment in patients previously treated for soft tissue sarcoma who are not expected to benefit from currently available therapeutic options for treatment of soft tissue sarcoma. Safety will be monitored throughout the study.

Eligibility criteria: Inclusion Criteria:

* Unresectable advanced or metastatic histologically proven soft tissue sarcoma (STS). Eligibility will include adult participants with desmoplastic small round cell tumor
* Must have relapsed or had progressive disease following standard of care treatment with chemotherapy prior to enrollment or intolerant to prior standard of care treatment with chemotherapy due to safety issues
* Recovery from toxic effects of prior therapies to Grade 1 or better according to National Cancer Institute-Common Terminology Criteria of Adverse Events (criteria used to grade the severity of toxic effects on a scale from 0 to 5 - Grade 1 = mild in severity, Grade 0 = no severity)
* Clinical test results within acceptable limits (ie, hematologic, clinical chemistry and hepatic function test results)
* Female participants must be postmenopausal, surgically sterile, abstinent, or if sexually active, be practicing 2 effective methods of birth control (eg, prescription hormonal contraceptive, intrauterine device, double-barrier method \[eg, condoms, occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam, cream, gel, film, or suppository\]), before entry, and must agree to continue to use these same methods of contraception throughout the study and for 3 months thereafter. Male participants must agree to use an adequate contraception method as deemed appropriate by the investigator (eg, vasectomy, double-barrier, partner using effective contraception) and to not donate sperm for a minimum of 5 months after treatment discontinuation

Exclusion Criteria:

* Diagnosis of Ewing's sarcoma or osteosarcoma, less than 3 weeks from last dose of radiation, systemic cytotoxic therapy (or 4 half lives, whichever is longer)
* Active symptomatic viral hepatitis or chronic liver disease
* Significant uncontrolled cardiac condition, including New York Heart Association Class II or greater heart failure, uncontrolled angina pectoris, myocardial infarction within 6 months before enrollment, significant pericardial disease, or uncontrolled or arrhythmias
* Active infection
* Female participant who is pregnant or breastfeeding

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Anchorage
country: United States
state: Alaska
city: San Diego
country: United States
state: California
city: Santa Monica
country: United States
state: California
city: Aurora
country: United States
state: Colorado
city: Daytona Beach
country: United States
state: Florida
city: Hollywood
country: United States
state: Florida
city: Atlanta
country: United States
state: Georgia
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: Overland Park
country: United States
state: Kansas
city: Louisville
country: United States
state: Kentucky
city: Metairie
country: United States
state: Louisiana
city: Baltimore
country: United States
state: Maryland
city: Boston
country: United States
state: Massachusetts
city: Ann Arbor
country: United States
state: Michigan
city: Detroit
country: United States
state: Michigan
city: Rochester
country: United States
state: Minnesota
city: Saint Joseph
country: United States
state: Missouri
city: Omaha
country: United States
state: Nebraska
city: Newark
country: United States
state: New Jersey
city: New York
country: United States
state: New York
city: Cleveland
country: United States
state: Ohio
city: Tulsa
country: United States
state: Oklahoma
city: Portland
country: United States
state: Oregon
city: Philadelphia
country: United States
state: Pennsylvania
city: Charleston
country: United States
state: South Carolina
city: Houston
country: United States
state: Texas
city: San Antonio
country: United States
state: Texas
city: Seattle
country: United States
state: Washington
city: Milwaukee
country: United States
state: Wisconsin
city: Calgary
country: Canada
state: Alberta
city: Edmonton
country: Canada
state: Alberta
city: Toronto
country: Canada
state: Ontario
city: Edmonton N/A
country: Canada
city: Tel Aviv
country: Israel
Study officials:
affiliation: Janssen Research & Development, LLC
name: Janssen Research & Development, LLC & Development, L.L.C. Clinical Trial
role: STUDY_DIRECTOR
References:
citation: Samuels BL, Chawla S, Patel S, von Mehren M, Hamm J, Kaiser PE, Schuetze S, Li J, Aymes A, Demetri GD. Clinical outcomes and safety with trabectedin therapy in patients with advanced soft tissue sarcomas following failure of prior chemotherapy: results of a worldwide expanded access program study. Ann Oncol. 2013 Jun;24(6):1703-9. doi: 10.1093/annonc/mds659. Epub 2013 Feb 5.
citation: Charytonowicz E, Terry M, Coakley K, Telis L, Remotti F, Cordon-Cardo C, Taub RN, Matushansky I. PPARgamma agonists enhance ET-743-induced adipogenic differentiation in a transgenic mouse model of myxoid round cell liposarcoma. J Clin Invest. 2012 Mar;122(3):886-98. doi: 10.1172/JCI60015. Epub 2012 Feb 1.