Clinical Trial: Phase 2 Study in Patients With MiT Tumors

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Phase 2 Study of ARQ 197 in Patients With Microphthalmia Transcription Factor Associated Tumors

Brief Summary: This is a multi-center, single arm intended to evaluate the anti-tumor effect of ARQ 197 in patients with microphthalmia transcription factor associated (MiT) tumors. MiT tumors include clear cell sarcoma, alveolar soft parts sarcoma, and translocation associated renal cell carcinoma.

Detailed Summary:

This is a multi-center, single arm, two-stage phase 2 study of ARQ 197 in patients with microphthalmia transcription factor associated (MiT) tumors. ARQ 197 is a novel small molecule drug designed to block the activity of c-Met, which is thought to play multiple key roles in human cancer, including cancer cell growth, survival, angiogenesis, invasion and metastasis.

The microphthalmia transcription factor tumors (MiT tumors) are clear cell sarcoma (CCS), alveolar soft part sarcoma (ASPS), and translocation associated renal cell carcinoma (RCC). These soft tissue cancers are characterized by a common transcriptional mechanism that leads to inexorable spread and resistance to all known therapies. They tend to strike adolescents and young adults, and are invariably fatal if not resectable at diagnosis. Several academic laboratories have shown that genetic translocations in these tumors upregulate c-Met, and that such tumors are dependent upon this activity.

The study will enroll adolescent (age 13 or older) and adult patients with a histologically or cytologically confirmed MiT malignant disease. Eligible patients will receive ARQ 197 twice daily. Treatment will be continued until progression of disease, unacceptable toxicity, or another discontinuation criterion is met.

During the study, tumor evaluations will be performed at baseline, then in 8-week intervals.

To evaluate each patient's safety and the drug's toxicity, physical examinations, laboratory evaluations, vitals signs, and adverse event assessments will be performed throughout the study. Blood samples for PK analysis will be collected during first cycle of treatment from up to 10 patients aged 20 or younger. Archival tissue specimens and relevant laboratory results o
Sponsor: ArQule

Current Primary Outcome: Determine the overall response rate (ORR) in patients treated with ARQ 197

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Evaluate progression-free survival (PFS) time in patients treated with ARQ 197
  • Evaluate 6-month and 1-year overall survival (OS) rates in patients treated with ARQ 197
  • Further characterize the safety of ARQ 197 in adolescent and young adult patients with MiT tumors


Original Secondary Outcome: Same as current

Information By: ArQule

Dates:
Date Received: November 12, 2007
Date Started: October 2007
Date Completion:
Last Updated: February 6, 2013
Last Verified: February 2013