E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Wilson disease, also known as hepatolenticular degeneration, a rare autosomal, recessively inherited disorder which results in chronic copper intoxication. |
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E.1.1.1 | Medical condition in easily understood language |
Wilson disease, an inherited disease in which the body cannot metabolise copper. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nutritional and Metabolic Diseases [C18] |
MedDRA Classification |
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 |
To evaluate the pharmacokinetics of a single dose of trientine in children ≥6 years and adult patients with Wilson’s disease by pharmacokinetic (PK) analysis. |
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E.2.2 | Secondary objectives of the trial |
To evaluate the tolerability and safety of a single dose of trientine dihydrochloride |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Children ≥ 6 years and adult patients
2. Confirmed diagnosis of Wilson’s disease by Leipzig-Score >3 (Ferenci et al 2003)
3. Current treatment with trientine dihydrochloride
4. Signed informed consent including parental consent in patients ≤ 18 years
5. Agree to remain in the study site for the PK measurements period.
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E.4 | Principal exclusion criteria |
1. Known clinically significant allergy or hypersensitivity to drugs that, in the opinion of the investigator, may affect the patient's safety
2. Have any clinically significant conditions that would interfere with the collection or interpretation of the study results or would compromise the patient’s health
3. Women of child bearing potential who do not use contraceptives, breastfeeding, or pregnant women
4. Severe anaemia (haemoglobin <9 mg/dL)
5. In the judgment of the Investigator, is likely to be noncompliant or uncooperative during the study, or unable to cooperate because of a language problem or poor mental development
6. Participation in any interventional clinical study at the same time or within the 4 weeks prior his study.
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E.5 End points |
E.5.1 | Primary end point(s) |
This clinical trial is a phase 1 pharmacokinetic profiling study and therefore has no clinical endpoint. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | Yes |
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) | Yes |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | Yes |
E.7.1.3.1 | Other trial type description |
Pharmacokinetic Profiling study |
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E.7.2 | Therapeutic exploratory (Phase II) | No |
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 | 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 | No |
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 | No |
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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End of study is defined as the last study visit of the last patient. |
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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 | |