PKAN MedDRA version: 9.1 Level: HLT Classification code 10042259 Term: Structural brain disorders NEC
Conditions
Interventions
Trade Name: FERRIPROX*100CPR RIV 500MG
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Deferiprone
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration
Sponsors
ISTITUTO NEUROLOGICO "CARLO BESTA"
Eligibility
Sex/Gender
All
Inclusion criteria
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). Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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 patient?s 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 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.;Secondary Objective: 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.;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). | — |
Countries
Italy
Outcome results
None listed