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)
Lead Sponsor: ApoPharma
|Source||Chiesi Canada Corp|
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.
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.
|Start Date||June 2014|
|Completion Date||March 16, 2018|
|Primary Completion Date||March 16, 2018|
Intervention Type: Drug
Intervention Name: Deferiprone oral solution
Description: Deferiprone oral solution at a dosage of up to 15 mg per kilogram of body weight, twice a day
Arm Group Label: Deferiprone
Other Name: DFP
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
- Completed study TIRCON2012V1
- 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
Minimum Age: 5 Years
Maximum Age: N/A
Healthy Volunteers: No
|Has Expanded Access||No|
|Number Of Arms||1|
Description: All patients will receive deferiprone oral solution.
|Study Design Info||
Intervention Model: Single Group Assignment
Intervention Model Description: All participants in this study received the same intervention.
Primary Purpose: Treatment
Masking: None (Open Label)