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

Pantothenate Kinase-Associated Neurodegeneration Clinical Trial

— TIRCON-EXT  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02174848
• Other study ID #: TIRCON2012V1-EXT
• Secondary ID: 2012-000845-11
• Status: Completed
• Phase: Phase 3
• Start date: June 2014
• Est. completion date: March 16, 2018

Study information

• Verified date: August 2020
• Source: Chiesi Canada Corp
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Completed
• Enrollment: 68
• Est. completion date: March 16, 2018
• Est. primary completion date: March 16, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Nerve Degeneration
• Pantothenate Kinase-Associated Neurodegeneration

Intervention

Drug:
• Deferiprone oral solution
Deferiprone oral solution at a dosage of up to 15 mg per kilogram of body weight, twice a day

Locations

Country	Name	City	State
Germany	Klinikum der Universität München	Munich
Italy	Foundation Neurological Institute C. Besta	Milan
United Kingdom	Newcastle University Institute of Human Genetics	Newcastle Upon Tyne
United States	UCSF Benioff Children's Hospital Oakland	Oakland	California

Sponsors (1)

Lead Sponsor	Collaborator
ApoPharma

Countries where clinical trial is conducted

United States,  Germany,  Italy,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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