E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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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 | HLT |
E.1.2 | Classification code | 10042259 |
E.1.2 | Term | Structural brain disorders NEC |
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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 efficacy outcome will be a reduction of iron concentration in the exposed patients at six months, assessed by measuring the iron concentration in both globus pallidus on MRI. |
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E.2.2 | Secondary objectives of the trial |
Secondary efficacy endpoints are changes in the following clinical scales: Burke-Fahn -Marsden Dystonia Rating Scales (BFMDRS) (Burke et al, 1985) and health-related quality of life (assessed with the 36 item Short Form questionnaire [SF-36] in patients aged 14 year or older (Ware 1992; Apolone et al, 1998), and with the Child Health Questionnaire Parent Form (CHQ-PF50) in 6 -13 year old children (Landgraf et al, 1996) and will be assessed every two months. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Written IC; Clinical diagnosis of NBIA (neurodegeneration with brain iron accumulation); Presence of Eye of the tiger sign on brain MRI; Genetic confirmation of PKAN diagnosis, with presence of mutation in the PANK2 gene; Age 6 to 40 years; Effective contraception (i.e. hormonal or IUD) during the study (women in childbearing age); No conditions known to be contraindications to the use of Deferiprone (history of recurrent episodes of neutropenia; history of agranulocytosis). |
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E.4 | Principal exclusion criteria |
Neurological disorders other than PKAN, or major co-morbidities (e.g. psychiatric disease other than OCD, overt heart, kidney or lung failure, overt malignancy, HIV infection). Known haemathological disorders (presence of any disorders associated with neutropenia [ANC < 1.5 x 109/L] or thrombocytopenia [platelet count <50 x 109/L) during the 12 months prior to screening. Treatment with medicinal products known to be associated with neutropenia or agranulocytosis during the three months prior to screening. Any condition preventing from brain MRI assessment (i.e pace-maker, metal prostheses or other metallic devices). Patients with intrathecal infusion pump can be included in the study but will undergo the following procedures before the radiological examination: the pump is turned off, then it is empted, secured to the patients abdomen with a cotton belt and drug infusion suspended for the entire duration of MRI exposure (Medtronic Inc.); Female subjects who are pregnant or lactating; Participation in a clinical trial with experimental drugs during the three months prior to screening. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The predefined primary outcome criterion is the effect of Deferiprone treatment on MRI-determined iron concentration in both globus pallidus (mean R 2* changes at six months). |
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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 | Yes |
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) | 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
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
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 | 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
| |
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 | 12 |
E.8.9.1 | In the Member State concerned days | |
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 | 12 |