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

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

Sponsors

Lead Sponsor: ApoPharma

Source Chiesi Canada Corp
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.

Overall Status Completed
Start Date June 2014
Completion Date March 16, 2018
Primary Completion Date March 16, 2018
Phase Phase 3
Study Type Interventional
Primary Outcome
Measure Time Frame
Number of Participants With Adverse Events 18 months
Secondary Outcome
Measure Time Frame
Change in Score on the BAD Scale -- Comparison of Treatment Groups Over Each Study Baseline and Month 18 of each study
Change in Score on the BAD Scale -- Comparison of Placebo-DFP Patients Across Studies Baseline and Month 18 of each study
Change in Score on the BAD Scale -- Comparison of DFP-DFP Patients Across Studies Baseline and Month 18 of each study
Proportion of Patients With Improved or Unchanged BAD Score Month 18 of each study
Patient Global Impression of Improvement (PGI-I) Comparison of Placebo-DFP Patients Across Studies Month 18 of each study
Enrollment 68
Condition
Intervention

Intervention Type: Drug

Intervention Name: Deferiprone oral solution

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

Arm Group Label: Deferiprone

Other Name: DFP

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

Gender: All

Minimum Age: 5 Years

Maximum Age: N/A

Healthy Volunteers: No

Overall Official
Location
Facility:
UCSF Benioff Children's Hospital Oakland | Oakland, California, 94609, United States
Klinikum der Universität München | Munich, 80336, Germany
Foundation Neurological Institute C. Besta | Milan, 20133, Italy
Newcastle University Institute of Human Genetics | Newcastle Upon Tyne, NE1 3BZ, United Kingdom
Location Countries

Germany

Italy

United Kingdom

United States

Verification Date

August 2020

Responsible Party

Type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 1
Arm Group

Label: Deferiprone

Type: Experimental

Description: All patients will receive deferiprone oral solution.

Acronym TIRCON-EXT
Study Design Info

Allocation: N/A

Intervention Model: Single Group Assignment

Intervention Model Description: All participants in this study received the same intervention.

Primary Purpose: Treatment

Masking: None (Open Label)

Source: ClinicalTrials.gov