E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Locally Advanced, Recurrent or Metastatic Squamous Cell Carcinoma of the Head and Neck
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 8.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10063569 |
E.1.2 | Term | Metastatic squamous cell carcinoma |
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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 |
Assess the overall response rate (ORR) of patients treated with ARQ501 |
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E.2.2 | Secondary objectives of the trial |
Determine the progression-free survival (PFS) rate at six months for patents treated with ARQ501. Further characterize the safety of ARQ501 |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Able to provide signed and dated informed consent document prior to study-specific screening procedures 2. Histologically or cytologically confirmed locally advanced, recurrent or metastatic SCCHN 3. Measurable disease as defined by RECIST (see section 9.0) 4. Age greater or equal 18years 5. Karnofsky performance status greater or equal 70 % (see Appendix B) 6. Male or female patients of child-producing potential must agree to use contraception or avoidance of pregnancy measures during the study and for 30 days after the last infusion ARQ501. 7. Hemoglobin (Hgb) greater or equal 10 g/dl 8. Absolute neutrophil count (ANC) greater or equal 1500/mm³ 9. Platelet count greater or equal 100.000/mm³ 10. Total bilirubin lower or equal 1.5 * upper limit of normal (ULN) or lower or equal 3.0 * ULN with metastatic liver disease 11. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) lower or equal 2.5 * ULN or lower or equal 5.0 ULN with metastatic liver disease 12. Creatinine lower or equal 1.5 ULN |
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E.4 | Principal exclusion criteria |
1. Primary tumor of nasopharyngeal origin 2. Eligible for curative surgery or radiotherapy 3. Received three or more anticancer regimens 4. Have active, uncontrolled systemic infection considered opportunistic, life threatening or clinically significant at the time of treatment 5. Have received anticancer chemotherapy, immunotherapy, radiotherapy, or investigational agents within three weeks of first infusion 6. Surgery within two weeks of first infusion 7. Have symptomatic or untreated central nervous system (CNS) involvement 8. Are pregnant or lactating 9. Previous exposure to ARQ501 |
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary Efficacy Analyses: Overall Response Rate Tumor response will be characterized as the best overall response recorded from the time of first treatment. Best overall response will be determined using the RECIST response criteria described in Section 9.0 of this protocol. Assessment of clinical activity of ARQ 501 will be determined by the percentage of evaluable patients who exhibit a complete or partial response (i.e., CR + PR). Ninety-five percent confidence intervals (CIs) will be calculated for the response rate. The number and percent of patients in each RECIST response category (CR, PR, SD, and PD) will also be summarized for the ITT patients. |
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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 | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
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 | Information not present in EudraCT |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 9 |
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 subject undergoing the trial including protocol defined follow up period. |
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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 | 1 |
E.8.9.1 | In the Member State concerned months | 2 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 2 |