Phase II trial to assess safety and efficacy of Iron chelating agent Deferiprone in patients with Pantothenate Kinase-Associated Neurodegeneration ? ICAND ? Trial - ND

• Conditions: PKAN; MedDRA version: 9.1Level: HLTClassification code 10042259Term: Structural brain disorders NEC

• Registration Number: EUCTR2008-003059-56-IT
• Lead Sponsor: ISTITUTO NEUROLOGICO CARLO BESTA

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2008-07-18
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: Not specified

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

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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified