E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Primary soft tissue sarcoma in the upper and lower limbs of adults. |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 13 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10061271 |
E.1.2 | Term | Malignant soft tissue neoplasm |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 13 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10039491 |
E.1.2 | Term | Sarcoma |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The trial aims to investigate the safety and efficacy of verteporfin photodynamic therapy in patients with soft tissue sarcomas in the arms or legs. The primary endpoint is the diameter of necrosis of tumour achieved around a single fibre for a given light energy.
We aim to establish the minimum light dose, in the range 20J to 50J, required to induce an area of necrosis with a diameter of at least 12mm. Once we have established the light dose that achieves this in 3 consecutive patients, we will study a further 7 patients with the same light dose. |
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E.2.2 | Secondary objectives of the trial |
What effect does photodynamic therapy have on the histopathology of the tumour and normal surrounding tissues? To look at the effect of Photodynamic Therapy on the tumour and normal soft tissues and study if necrosis extends to normal tissue.
What effect does photodynamic therapy have on the MRI appearance of the sarcoma? To radiologically assess the response of the Photodynamic Therapy on sarcomas we will use pre and post PDT MRI and compare with final histology. We will measure the diameter of necrosis on MRI scan which will be compared with the histological measurement. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Primary malignant, biopsy proven soft tissue sarcoma of the upper or lower limbs. Adults (over 18 years of age). Patients able to give informed written consent. Patients undergoing limb-sparing surgery. Unifocal tumour. Women of child-bearing potential with negative pregnancy test prior to study entry AND be using an adequate contraceptive method, which must be continued for 1 week following PDT. |
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E.4 | Principal exclusion criteria |
Previous treatment for the tumour (either surgery or radiotherapy) or tumour recurrence. Skin photosensitivity (including porphyrea). Patients with known sensitivity to photosensitizers. Metastatic disease. Patients with severe cardiovascular disease. Pregnancy and lactation. Evidence of severe or uncontrolled systemic disease such as hepatic impairment or laboratory findings that makes it undesirable for the patient to participate in the trial. Any psychiatric disorder making reliable informed consent impossible. Taking part in any other trial of an experimental medicine. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary outcome is the maximum diameter of the area of necrosis (mm) achieved around a single fibre for a given light energy. We aim to achieve a 12mm zone of necrosis in the tumour. |
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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 | Yes |
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) | 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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | Information not present in EudraCT |
E.7.4 | Therapeutic use (Phase IV) | Information not present in EudraCT |
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
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
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 | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
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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The end of the trial is: • When 10 patients completed trial at minimum effective light dose • Verteporfin declared not appropriate/dangerous • Last patient last visit
Patients will all be followed up after end of trial as per protocol set up for follow-up of soft tissue sarcoma patients.
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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 | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 1 |