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 |
This is an inherited condition where the mechanisms to re-absorb amino acids in the kidneys has failed, leading to increased excretion of amino acids in the urine. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nutritional and Metabolic Diseases [C18] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10011778 |
E.1.2 | Term | Cystinuria |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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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 |
Does paracetamol reduce the amount urinary cystine excreted in patients with cystinuria? |
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E.2.2 | Secondary objectives of the trial |
1.Do different doses of paracetamol affect the amount of urinary cystine excreted in patients with cystinuria differently? 2.To observe any serious adverse events related to paracetamol during the study |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Patients diagnosed with cystinuria |
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E.4 | Principal exclusion criteria |
1. Under 18 years old 2. Unable to give consent 3. Drinks alcohol regularly over the recommended limit 4. History of liver disease 5. History of paracetamol hypersensitivities 6. History of psychiatric disorder or self harm 7. Patiets who are likely to be glutathione deplete (e.g. patients with history of cystic fibrosis, HIV infection, starvation, cachexia) 8. Patient taking other drugs containing paracetamol regularly 9. Patients taking long term liver enzyme inducer drugs (e.g. carbamazepine, phenobarbital, phenytoin, primidone, rifampicin and St John’s Wort) 10. Pregnancy 11. Patients on another study in the last 30 days
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E.5 End points |
E.5.1 | Primary end point(s) |
The mean amount of 24-hour urinary cystine excretion. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
24-hour urinary samples taken during the week (two samples taken anythime between Day 3 to Day 7) when the patient is in either Paracetamol 2g/day, Paracetamol 4g/day or Control groups. |
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E.5.2 | Secondary end point(s) |
The mean amount of 24-hour urinary paracetamol metabolite excretion (paracetamol-cysteine, paracetamol mercapturate, paracetamol-sulphate and paracetamol-gluconate conjugates) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
24-hour urinary samples taken during the week (two samples taken anythime between Day 3 to Day 7) when the patient is in either Paracetamol 2g/day, Paracetamol 4g/day or Control groups. |
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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 | 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 | 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 | Yes |
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 |
Patients on usual baseline therapy (e.g. oral hydration, alkalinising agent) |
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E.8.2.4 | Number of treatment arms in the trial | 3 |
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.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
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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When the last recruited patient provides the necessary 24-hour urine samples |
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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 | 0 |
E.8.9.1 | In the Member State concerned months | 5 |
E.8.9.1 | In the Member State concerned days | 0 |