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] - Respiratory Tract Diseases [C08] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 16.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10011762 |
E.1.2 | Term | Cystic fibrosis |
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 |
The primary objective of this study is to characterise the blood pharmacokinetic profile of oral immediate-release cysteamine (Cystagon) when administered to patients with Cystic Fibrosis at the dose licensed for use in cystinosis. |
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E.2.2 | Secondary objectives of the trial |
The secondary objectives are to demonstrate that when oral immediate-release cysteamine (Cystagon) is administered to patients with Cystic Fibrosis at the dose licensed for use in cystinosis: 1. Cysteamine enters the bronchial secretions 2. Cysteamine is tolerated
A further secondary objective is for the Contract Research Organisation (CRO) to establish a method to quantify cysteamine in the sputum of patients with CF. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. CF related suppurative lung disease who expectorate sputum 2. Clinically stable for >4 weeks, 3. Aged ≥18 years, 4. Weight >50kg. 5. Female participants of child bearing potential should be using a reliable form of contraception. |
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E.4 | Principal exclusion criteria |
1. Hypersensitivity to the active substance, any form of cysteamine, or to any of the excipients. 2. Hypersensitivity to penicillamine. 3. Lung, liver transplant, on active transplant list. 4. For women, current pregnancy or breast-feeding, or planned pregnancy during the study. 5. Any other significant disease/disorder which, in the investigator’s opinion, either puts the patient at risk because of study participation or may influence the results of the study or the patient's ability to participate in the study.
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary outcome will be blood cysteamine measured at baseline, 30mins, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 10, and 24 hours after oral administration of immediate-release cysteamine (Cystagon) to patients with Cystic Fibrosis at the dose licensed for use in cystinosis. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Baseline, 30mins, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 10, and 24 hours after oral administration of immediate-release cysteamine (Cystagon) |
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E.5.2 | Secondary end point(s) |
1. Sputum concentration of cysteamine 3 hours after final dosing. 2. Adverse events (tolerability) 3. Lung function (FEV1, FVC) 4. Weight 5. Disease specific health status using the CFQ-R 6. Quantitative sputum microbiology, sputum rheology, bacterial sensitivities
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
CFQ-R will be assessed at recruitment and after 5 weeks sputum, adverse events, lung function, weight and sputum microbiology will be assessed at recruitment, 1,2,3 and 5 weeks |
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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 |
I/IIa: CYSTAGON has been available for some time but being used in CF patients for the first time. |
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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 | 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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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 | 11 |
E.8.9.1 | In the Member State concerned days | 31 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 11 |
E.8.9.2 | In all countries concerned by the trial days | 31 |