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

Study Overview
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Brief Summary
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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
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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.

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

Conditions
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Pantothenate Kinase-Associated Neurodegeneration

Intervention / Treatment
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* Drug: Deferiprone oral solution


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 5 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: All participants in this study received the same intervention.
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Deferiprone<br>All patients will receive deferiprone oral solution. | Drug: Deferiprone oral solution<br>* Deferiprone oral solution at a dosage of up to 15 mg per kilogram of body weight, twice a day<br>* Other names: DFP;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Adverse Events | Safety and tolerability were assessed based on changes in: frequency of adverse events (AEs), frequency of serious adverse events (SAEs), and discontinuation due to AEs. No statistical comparison between the groups was conducted as all participants received the same study product. | 18 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Score on the BAD Scale -- Comparison of Treatment Groups Over Each Study | The Barry-Albright Dystonia (BAD) scale is an instrument for rating the severity of dystonia in eight body regions. The individual scores are summed to provide a total score that ranges from 0 to 32; the higher the score, the more severe the dystonia. Patients were assessed for the change in total BAD score over the course of both the initial study (during which one group received placebo and the other received deferiprone) and the extension study (during which both groups received deferiprone). | Baseline and Month 18 of each study | 
| Change in Score on the BAD Scale -- Comparison of Placebo-DFP Patients Across Studies | The Barry-Albright Dystonia (BAD) scale is an instrument for rating the severity of dystonia in eight body regions. The individual scores are summed to provide a total score that ranges from 0 to 32; the higher the score, the more severe the dystonia. Patients were assessed for the change in total BAD score over the course of each study. | Baseline and Month 18 of each study | 
| Change in Score on the BAD Scale -- Comparison of DFP-DFP Patients Across Studies | The Barry-Albright Dystonia (BAD) scale is an instrument for rating the severity of dystonia in eight body regions. The individual scores are summed to provide a total score that ranges from 0 to 32; the higher the score, the more severe the dystonia. Patients were assessed for the change in total BAD score over the course of the study. | Baseline and Month 18 of each study | 
| Proportion of Patients With Improved or Unchanged BAD Score | Patients were deemed to be responders if their BAD total score either improved or remained unchanged from baseline, with baseline being the start of each study for the placebo-DFP group and the start of the initial study for the DFP-DFP group | Month 18 of each study | 
| Patient Global Impression of Improvement (PGI-I) Comparison of Placebo-DFP Patients Across Studies | The Patient Global Impression of Improvement (PGI-I) is a global index used to rate the response of a condition to a therapy. Patients were asked at each post-baseline visit to rate their overall condition since the start of the extension study on a 7-point rating scale: 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, and 7 = very much worse. | Month 18 of each study | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Pantothenate Kinase-Associated Neurodegeneration, PKAN, Neurodegeneration with Brain Iron Accumulation, NBIA, Deferiprone, Ferriprox