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

• Conditions: Microphthalmia Transcription Factor Associated Tumors: Clear cell sarcoma Alveolar soft part sarcoma Translocation associated renal cell carcinoma (Renal cell carcinoma; MedDRA code manually entered in E.1.2); MedDRA version: 9.1Level: LLTClassification code 10065865Term: Clear cell sarcoma; MedDRA version: 9.1Level: LLTClassification code 10001882Term: Alveolar soft part sarcoma; MedDRA version: 12.0Level: LLTClassification code 10067946Term: <Manually entered code. Term in E.1.1>

• Registration Number: EUCTR2009-011669-10-GB
• Lead Sponsor: ArQule, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2009-05-22
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 39

Inclusion Criteria

Each patient must meet the following criteria to be enrolled in this study:
1.Provide signed and dated informed consent/assent prior to study-specific screening procedures
2.Histologically or cytologically confirmed unresectable locally advanced or metastatic alveolar soft part sarcoma, clear cell sarcoma, or translocation associated renal cell carcinoma
3.=13 years old
4.Measurable disease as defined by Response Evaluation Criteria in Solid Tumors (RECIST)
5.Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1 (see Appendix 2)
6.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
7.Females of childbearing potential must have a negative serum pregnancy test
8.Aspartate transaminase (AST) and alanine transaminase (ALT) = 2.5 × upper limit of normal (ULN) or = 5 × ULN with metastatic liver disease
9.Hemoglobin = 8.5 g/dl without transfusion
10.Total bilirubin = 1.5 × ULN
11.Serum creatinine = 1.5 x ULN
12.Absolute neutrophil count (ANC) = 1.5 x 109/L
13.Platelets = 100 x 109/L without transfusion

Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Patients who meet any of the following criteria will be excluded from the study:
1.Received anti-cancer therapy including surgery, chemotherapy, radiotherapy and investigational drug within four weeks prior to first dose of ARQ 197
2.Central nervous system metastasis unless it has been stable for = 3 months after treatment and patient has no neural symptoms
3.Pregnant or lactating
4.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)
5.Unable or unwilling to swallow ARQ 197 capsules twice daily
6.Significant co-morbid conditions that in the opinion of the Investigator would impair study participation
7.Known human immunodeficiency virus (HIV), Hepatitis B virus (HBV) or hepatitis C virus (HCV) infection
8.Bradycardia at baseline or known history of arrhythmia
9.Received ARQ 197 previously

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Determine the overall response rate (ORR) in patients treated with ARQ 197;Secondary Objective: •Evaluate progression-free survival (PFS) time in patients treated with ARQ 197<br>•Evaluate 6-month and 1-year overall survival (OS) rates in patients treated with ARQ 197<br>•Further characterize the safety of ARQ 197 in adolescent and young adult patients with MiT tumors<br>;Primary end point(s): For patients treated with ARQ 197 in this study, the primary end points are:<br>To determine the overall response rate (ORR):<br>To evaluate progression-free survival (PFS) time; <br>To evaluate 6-month and 1-year overall survival (OS) rates: <br>and further characterize the safety of ARQ 197 in adolescent and young adult patients with MiT tumors<br>