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NCT Number: NCT02174848

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

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.

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Key information

Conditions

Pantothenate Kinase-Associated Neurodegeneration Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Movement Disorders Nervous System Diseases Neuroaxonal Dystrophies Neurodegenerative Diseases

Age range

5 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Klinikum der Universität München, Munich, Germany

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Deferiprone oral solution

Drug

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

Other names: DFP

Primary outcomes

  1. Number of Participants With Adverse Events

    Time frame: 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 outcomes

  1. Change in Score on the BAD Scale -- Comparison of Treatment Groups Over Each Study

    Time frame: 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).

  2. Change in Score on the BAD Scale -- Comparison of Placebo-DFP Patients Across Studies

    Time frame: 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.

  3. Change in Score on the BAD Scale -- Comparison of DFP-DFP Patients Across Studies

    Time frame: 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.

  4. Proportion of Patients With Improved or Unchanged BAD Score

    Time frame: 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

  5. Patient Global Impression of Improvement (PGI-I) Comparison of Placebo-DFP Patients Across Studies

    Time frame: 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.

Sponsors and collaborators

Lead sponsor

ApoPharma

Industry

Registry information

Official study title

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

Acronym: TIRCON-EXT

Important dates

Study start
2014
Primary completion
2018
Study completion
2018
First posted
Jun 26, 2014
Registry last updated
Aug 25, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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