Long-term Deferiprone Treatment in Patients With Pantothenate Kinase-Associated Neurodegeneration (TIRCON-EXT)

ApoPharma

Status and phase

Completed

Phase 3

Conditions

Pantothenate Kinase-Associated Neurodegeneration

Treatments

Drug: Deferiprone oral solution

Study type

Interventional

Funder types

Industry

Identifiers

NCT02174848

TIRCON2012V1-EXT

2012-000845-11 (EudraCT Number)

Details and patient eligibility

About

Patients with PKAN will be treated with the iron chelator deferiprone for 18 months. Only patients who have completed the earlier study TIRCON2012V1 (NCT01741532), a double-blind placebo-controlled trial in which participants were randomized to receive either deferiprone or placebo for 18 months, are eligible to enroll.

Full description

TIRCON2012V1-EXT is a multi-center, single-arm, open-label study. All patients who completed the earlier study TIRCON2012V1 (NCT01741532) are eligible to take part. In the initial study, patients were randomized in a 2:1 ratio to receive 18 months of treatment with either the iron chelator deferiprone or placebo, respectively. In this extension study, all participants will receive deferiprone for 18 months. Thus, depending on which product was received earlier, patients will be on deferiprone for a total of either 1.5 years or 3 years. As in the earlier study, assessments will be carried out every six months to look at the safety of the drug and to see if patients are showing any improvement in dystonia and other symptoms of PKAN.

Enrollment

68 patients

Sex

All

Ages

5+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Completed study TIRCON2012V1

Exclusion criteria

Withdrew from the study TIRCON2012V1 for reasons of safety
Plan to participate in another clinical trial at any time from the day of enrolment until 30 days post-treatment in the current study