E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10036223 |
E.1.2 | Term | Positron emission tomography |
E.1.2 | System Organ Class | 100000004848 |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10047988 |
E.1.2 | Term | Wilson's disease |
E.1.2 | System Organ Class | 100000004850 |
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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 |
We wish to optimize the diagnostic potential of 64Cu PET in Wilson's disease by determining the optimal time point for the scan following tracer injection. |
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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 |
1. Age above 18 years 2. BMI below 30 3. For WD patients: A diagnosis based on the Leipzig criteria 4. For heterozygotes: Having a parent or child with WD diagnosis based on Leipzig criteria 5. Fertile women must accept to use contraceptives at the time of the PET scan (spiral or hormonal contraceptive) 6. On the day of the tracer injection, a negative pregnancy urine test must be available for fertile women |
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E.4 | Principal exclusion criteria |
1. For healthy and heterozygote participants: Chronic liver or kidney disease 2. Participation in a PET trial during the past year 3. Known hypersensitivity toward 64Cu, copper in general, or another tracer formula ingredient 4. Claustrophobia 5. Pregnancy, breastfeeding or a wish to become pregnant before the end of the trial
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E.5 End points |
E.5.1 | Primary end point(s) |
A comparison of liver SUV in WD patients, heterozygotes and healthy after 20, 48, 54 and 68 hours. We expect to be able to completely separate individuals in the groups at minimum one time point. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
After the last trial participant has completed the last scan. |
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E.5.2 | Secondary end point(s) |
A comparison of gallbladder SUV in WD patients, heterozygotes and healthy after 20, 48, 54 and 68 hours. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
After the last trial participant has completed the last scan. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Yes |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | No |
E.6.5 | Efficacy | No |
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 | No |
E.8.1.2 | Open | No |
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) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
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 | Yes |
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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When the last participant has completed the last scan. Criteria for total discontinuation of the study a)Serious adverse event related to the tracer in more than one participant. |
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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 | |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |