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

Brief Title

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)

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.
    

Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Number of Participants With Adverse Events

Secondary Outcome

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

Condition

Pantothenate Kinase-Associated Neurodegeneration

Intervention

Deferiprone oral solution

Study Arms / Comparison Groups

 Deferiprone
Description:  All patients will receive deferiprone oral solution.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

68

Start Date

June 2014

Completion Date

March 16, 2018

Primary Completion Date

March 16, 2018

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

Ages

5 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Elliott Vichinsky, MD, , 

Location Countries

Germany

Location Countries

Germany

Administrative Informations


NCT ID

NCT02174848

Organization ID

TIRCON2012V1-EXT

Secondary IDs

2012-000845-11

Responsible Party

Sponsor

Study Sponsor

ApoPharma


Study Sponsor

Elliott Vichinsky, MD, Principal Investigator, UCSF Benioff Children's Hospital Oakland


Verification Date

August 2020