E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Erythropoietic protoporphyria (EPP) |
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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 | 10015289 |
E.1.2 | Term | Erythropoietic protoporphyria |
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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 |
Erythropoietic protoporphyria (EPP) is an inherited disease in which protoporphyrin accumulates in red blood cells and causes 2-3 day long bouts of severe pain following exposure of the skin to sunlight. It is a lifelong, severe and disabling condition for which there is no effective treatment.
Colestyramine is an anion-exchange resin. It has been shown to bind to protoporphyrin (Stathers, 1966) in the gut and to increase its faecal excretion (McCullough et al., 1988; Bloomer, 1979) (by interrupting the porphyrin's enterohepatic recirculation).
The main objective of this trial is to assess whether oral colestyramine is likely to be a useful therapy in EPP, by seeing whether it reduces the plasma and red cell protoporphyrin concentration in EPP patients when given over a three month period.
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients who regularly attend the Photodermatoses clinic at St Thomas’ Hospital and meet the inclusion criteria below will be invited to participate in this trial: -
•Patients with a diagnosis of EPP. •Patients aged 18 years or over. •Patients who are willing and able to give written informed consent to participate in the trial.
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E.4 | Principal exclusion criteria |
•Patients under 18 years of age •Patients who do not adequately understand verbal explanations or written information given in English, or who have special communication needs and unable to give informed consent. •Patients who are pregnant or breast feeding. •Patients who are unwilling or unable to comply with the study protocol •Patients with complete biliary obstruction •Patients who have diabetes mellitus •Patients with epilepsy •Patients prescribed thyroxine sodium for hypothyroidism
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E.5 End points |
E.5.1 | Primary end point(s) |
Does colestyramine decrease the plasma and red cell protoporphyrin concentration by at least 15% in EPP (15% chosen because it is the level below which a fall in plasma and red cell protoporphyrin is not likely to be clinically significant in terms of symptom control).
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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 | No |
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 | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Information not present in EudraCT |
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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | No |
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 | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
treatment versus no-treatment within each subject |
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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
| 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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Last visit of the last subject |
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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 | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
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 |