Clinical trial NCT02174848: Long-term Deferiprone Treatment in Patients With Pantothenate Kinase-Associated Neurodegeneration
Official title: Long-term Safety and Efficacy Study of Deferiprone in Patients With Pantothenate Kinase-Associated Neurodegeneration (PKAN)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Pantothenate Kinase-Associated Neurodegeneration
Keywords: Pantothenate Kinase-Associated Neurodegeneration
PKAN
Neurodegeneration with Brain Iron Accumulation
NBIA
Deferiprone
Ferriprox
Lead sponsor: Chiesi Canada Corp
Sponsor class: INDUSTRY
First posted: 2014-06-26
Enrollment: 68
Sex: ALL
Minimum age (years): 5
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: All participants in this study received the same intervention.
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Number of Participants With Adverse Events
timeFrame: 18 months
Secondary outcomes:
measure: Change in Score on the BAD Scale -- Comparison of Treatment Groups Over Each Study
timeFrame: Baseline and Month 18 of each study
measure: Change in Score on the BAD Scale -- Comparison of Placebo-DFP Patients Across Studies
timeFrame: Baseline and Month 18 of each study
measure: Change in Score on the BAD Scale -- Comparison of DFP-DFP Patients Across Studies
timeFrame: Baseline and Month 18 of each study
measure: Proportion of Patients With Improved or Unchanged BAD Score
timeFrame: Month 18 of each study
measure: Patient Global Impression of Improvement (PGI-I) Comparison of Placebo-DFP Patients Across Studies
timeFrame: Month 18 of each study
Other outcomes:
Locations:
city: Oakland
country: United States
facility: UCSF Benioff Children's Hospital Oakland
state: California
city: Munich
country: Germany
facility: Klinikum der Universität München
city: Milan
country: Italy
facility: Foundation Neurological Institute C. Besta
city: Newcastle Upon Tyne
country: United Kingdom
facility: Newcastle University Institute of Human Genetics
Study officials:
affiliation: UCSF Benioff Children's Hospital Oakland
name: Elliott Vichinsky, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Klinikum der Universität München
name: Thomas Klopstock, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Foundation Neurological Institute C. Besta
name: Nardo Nardocci, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Newcastle University Institute of Human Genetics
name: Patrick Chinnery, MD
role: PRINCIPAL_INVESTIGATOR
References: