E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Patients with locally recurrent or second primary SCC of the anterior oral cavity, soft palate, or tonsil that have failed primary curative therapy in whom surgical resection is seen as an option for disease control
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MedDRA Classification |
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 |
To compare function status at 4 months after treatment with bleomycin-EPT or surgery in patients with locally recurrent or second primary SCC of the anterior oral cavity, soft palate, or tonsil that have failed primary curative therapy and in whom surgical resection is seen as an option for disease control. |
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E.2.2 | Secondary objectives of the trial |
- To evaluate local tumor recurrence, disease free survival and overall survival rates through 2 years after bleomycin-EPT or surgery treatment. - To evaluate safety through 6 months after the study treatment. - To evaluate the durability of function status outcomes at 8 months after bleomycin-EPT or surgery treatment. - To evaluate pharmacoeconomic parameters through 8 months after bleomycin-EPT or surgery treatment. - To characterize bleomycin systemic absorption and plasma pharmacokinetics following IT bleomycin-EPT administration.
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
1. The presence of SCC of the anterior oral cavity, soft palate or tonsil must be confirmed by histological examination of a tissue sample (e.g., biopsy) obtained within 1 month of the patient receiving the study treatment. 2. Recurrent or second primary disease in patients where surgical resection is seen as an option for disease control. 3. The length of the longest diameter of the study lesion must be < 5 cm and the calculated treatment volume must be < 60.0 cm3 (tumor volume plus a 0.5 cm margin around the tumor) for the study lesion [where treatment volume = 0.5 (A+1)(B+1)^2 and where A = length of the longest diameter (cm), B = the next longest diameter perpendicular to “A” (cm)]. 4. Tumor burden must be completely encompassed by surgery or bleomycin-EPT. 5. Age: 18 years or older. 6. Men and women of childbearing potential must use physician approved contraceptive methods for 7 days following bleomycin-EPT. 7. Hematopoietic status: ANC > 1000/uL Platelets > 75,000/mm3 PT: INR ≤ 1.5 (correctable with Vitamin K injection) 8. Blood Chemistry status: Transaminases < 3 times upper limit of normal Total Bilirubin < 2.5 mg/dL Creatinine < 2.5mg/dL 9. A written Informed Consent Form must be signed prior to the patient receiving any study procedures or treatments.
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E.4 | Principal exclusion criteria |
1. Patients with tumors suspected of involving a 50% or greater encasement of a blood vessel as measured by MRI or CT scan. 2. Patients with tumors having bone invasion. 3. Patients with any metallic implants in the treatment field. 4. Patients with hypersensitivity to bleomycin. 5. Patients who have received or will exceed a total lifetime dose of bleomycin greater than 400 Units. 6. Patients deemed unsuitable for general anesthesia. 7. Patients with a significant history of emphysema or pulmonary fibrosis. 8. Patients with indwelling cardiac pacemakers who cannot tolerate a period with pacemaker turned off. 9. Patients with a history of uncontrolled cardiac arrhythmia. 10. Women who are pregnant, or are nursing. Women of childbearing potential must have a negative βHCG test within 7 days of study treatment. 11. Radiation therapy to the treatment area within 8 weeks of study treatment. 12. Chemotherapy or other cancer therapy (e.g., surgery, cryotherapy, etc.) to the treatment area within 4 weeks of study treatment. 13. Patients participating in a clinical study for an investigational drug or device within 4 weeks prior to the study treatment. 14. Patients previously randomized to this study.
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E.5 End points |
E.5.1 | Primary end point(s) |
Function status, evaluated at 4 months after initiation of treatment by the validated, clinicianrated disease specific, head and neck Performance Status Scale (PSSHN), will be the primary efficacy endpoint to determine clinical benefit. The PSSHN will be administered at each investigational site by a clinician or health professional trained according to the standard recommendations for administering the PSSHN. Clinical benefit will be defined as at least a 20% difference between treatments for the proportion of patients that improved or had no change from baseline in at least one of the discrete PSSHN function status scales (i.e., functional performance related to normalcy of diet, eating in public and understandability of speech). No change is defined as patients who did not change from baseline in understandability of speech or eating in public by one level (25 points) or no more than two levels (20 points) for normalcy of diet.
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Information not present in EudraCT |
E.6.2 | Prophylaxis | Information not present in EudraCT |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Information not present in EudraCT |
E.6.8 | Bioequivalence | Information not present in EudraCT |
E.6.9 | Dose response | Information not present in EudraCT |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | Information not present in EudraCT |
E.6.12 | Pharmacoeconomic | Yes |
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) | Information not present in EudraCT |
E.7.1.1 | First administration to humans | Information not present in EudraCT |
E.7.1.2 | Bioequivalence study | Information not present in EudraCT |
E.7.1.3 | Other | Information not present in EudraCT |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Information not present in EudraCT |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | Information not present in EudraCT |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | Yes |
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 | Yes |
E.8.2.3.1 | Comparator description |
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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.8.5 | The trial involves multiple Member States | Yes |
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 | Information not present in EudraCT |
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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- in the MS concerned: clinical evaluation after treatment: week one (1) and every one (1) month for the first (1st) year, every two (2) months for the second (2nd) year. - in all countries involved in the trial: clinical evaluation after treatment: week one (1) and every one (1) month for the first (1st) year, every two (2) months for the second (2nd) year.
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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 | 3 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 4 |