NCT02174848

Long-term Deferiprone Treatment in Patients With Pantothenate Kinase-Associated Neurodegeneration

Long-term Safety and Efficacy Study of Deferiprone in Patients With Pantothenate Kinase-Associated Neurodegeneration (PKAN)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 68 (actual)
• Sponsor: ApoPharma · Industry
• Sex: All
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed 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.

Conditions

• Pantothenate Kinase-Associated Neurodegeneration

Interventions

Type	Name	Description
DRUG	Deferiprone oral solution	Deferiprone oral solution at a dosage of up to 15 mg per kilogram of body weight, twice a day

Timeline

• Start date: 2014-06-01
• Primary completion: 2018-03-16
• Completion: 2018-03-16
• First posted: 2014-06-26
• Last updated: 2020-08-25
• Results posted: 2019-07-17

Locations

4 sites across 4 countries: United States, Germany, Italy, United Kingdom