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

Phase 2
13 Years
Not Enrolling
Renal Cell Carcinoma (RCC), Alveolar Soft Part Sarcoma (ASPS), Clear Cell Sarcoma (CCS)

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Trial Information

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

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

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 on patients' gene translocation/fusion status will be collected.

Tumor biopsies may also be collected (optional) with patient's consent. If patients agree
tumor samples may be collected using core needle biopsy.

In addition, to explore biological responses of tumors to ARQ 197 treatment, FDG-PET
scanning will be performed at three time points: within 14 days prior to the treatment, on
Day 8 (± 2 days) of Cycle 1 and after two cycles of treatment coinciding with tumor

Inclusion Criteria:

1. Histologically or cytologically confirmed unresectable locally advanced or metastatic
alveolar soft part sarcoma, clear cell sarcoma, or translocation associated renal
cell carcinoma

2. ≥13 years old

3. Male or female patients of child-producing potential must agree to use double barrier
contraception, oral contraceptives or avoidance of pregnancy measures during the
study and for 30 days after the last ARQ 197 dose

4. Females of childbearing potential must have a negative serum pregnancy test

Exclusion Criteria:

1. Central nervous system metastasis unless it has been stable for ≥ 3 months after
treatment and patient has no neural symptoms

2. Pregnant or lactating

3. Significant gastrointestinal disorder(s), in the opinion of a Investigator, could
interfere with the absorption of ARQ 197 (e.g., Crohn's disease, ulcerative colitis,
extensive gastric or small bowel resection)

4. Unable or unwilling to swallow ARQ 197 capsules twice daily

5. Known human immunodeficiency virus (HIV), Hepatitis B virus (HBV) or hepatitis C
virus (HCV) infection

6. Bradycardia at baseline or known history of arrhythmia

7. Received ARQ 197 previously

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Determine the overall response rate (ORR) in patients treated with ARQ 197


United States: Food and Drug Administration

Study ID:

ARQ 197-204



Start Date:

October 2007

Completion Date:

February 2011

Related Keywords:

  • Renal Cell Carcinoma (RCC)
  • Alveolar Soft Part Sarcoma (ASPS)
  • Clear Cell Sarcoma (CCS)
  • Microphthalmia transcription (MiT) factor associated tumors
  • Alveolar soft part sarcoma (ASPS)
  • Clear cell sarcoma (CCS)
  • Renal cell carcinoma (RCC)
  • Carcinoma
  • Carcinoma, Renal Cell
  • Microphthalmos
  • Sarcoma, Clear Cell
  • Sarcoma, Alveolar Soft Part
  • Sarcoma



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