E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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)
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10065865 |
E.1.2 | Term | Clear cell sarcoma |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10001882 |
E.1.2 | Term | Alveolar soft part sarcoma |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10067946 |
E.1.2 | Term | <Manually entered code. Term in E.1.1> |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Determine the overall response rate (ORR) in patients treated with ARQ 197 |
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E.2.2 | Secondary objectives of the trial |
•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
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal 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
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E.4 | Principal 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
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E.5 End points |
E.5.1 | 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
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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Last visit of the last patient undergoing the trial. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 0 |
E.8.9.1 | In the Member State concerned months | 8 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |