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

Phase 3

Completed

Conditions: Pantothenate Kinase-Associated Neurodegeneration

Interventions: Drug: Deferiprone oral solution

Registration Number: NCT02174848

Lead Sponsor: ApoPharma

Brief 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.

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 68

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

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Deferiprone	Deferiprone oral solution	All patients will receive deferiprone oral solution.

Primary Outcome Measures

Name	Time	Method
Number of Participants With Adverse Events	18 months	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.

Secondary Outcome Measures

Name	Time	Method
Change in Score on the BAD Scale -- Comparison of DFP-DFP Patients Across Studies	Baseline and Month 18 of 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 the study.
Proportion of Patients With Improved or Unchanged BAD Score	Month 18 of each study	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
Change in Score on the BAD Scale -- Comparison of Placebo-DFP Patients Across Studies	Baseline and Month 18 of 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 each study.
Patient Global Impression of Improvement (PGI-I) Comparison of Placebo-DFP Patients Across Studies	Month 18 of each study	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.
Change in Score on the BAD Scale -- Comparison of Treatment Groups Over Each Study	Baseline and Month 18 of 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).

Trial Locations

Locations (4): Foundation Neurological Institute C. Besta
🇮🇹
Milan, Italy
UCSF Benioff Children's Hospital Oakland
🇺🇸
Oakland, California, United States
Klinikum der Universität München
🇩🇪
Munich, Germany
Newcastle University Institute of Human Genetics
🇬🇧
Newcastle Upon Tyne, United Kingdom