Microphthalmia Transcription Factor Associated Tumors: Clear cell sarcoma Alveolar soft part sarcoma Translocation associated renal cell carcinoma (Renal cell carcinoma
Conditions
Interventions
Trade Name: NA
Product Name: ARQ 197
Product Code: ARQ 197
Pharmaceutical Form: Capsule*
INN or Proposed INN: (-)-trans-3-(5,6-dihydro-4H-pyrrolo [3,2,1-ij] quinolin-1-yl)-4(1H-indol-3-yl)pyrrolidine-
Sponsors
ArQule, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
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
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 •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 ;Primary end point(s): For patients treated with ARQ 197 in this study, the primary end points are: To determine the overall response rate (ORR): To evaluate progression-free survival (PFS) time; To evaluate 6-month and 1-year overall survival (OS) rates: and further characterize the safety of ARQ 197 in adolescent and young adult patients with MiT tumors | — |
Countries
United Kingdom
Outcome results
None listed