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

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

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 (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 Summary: TIRCON2012V1-EXT is a multi-center, single-arm, open-label study. All patients who completed the earlier study TIRCON2012V1 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.
Sponsor: ApoPharma

Current Primary Outcome: Safety and tolerability of deferiprone in patients with PKAN [ Time Frame: 18 months ]

Safety and tolerability will be assessed based on changes in: frequency of adverse events (AEs), frequency of serious adverse events (SAEs), discontinuation due to AEs, clinical laboratory tests (including hematology and biochemistry)and ECG from baseline to month 18.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Change in severity of dystonia as assessed by the total score on the Barry-Albright Dystonia (BAD) scale. [ Time Frame: Baseline to 18 months, where baseline is defined as prior to the start of deferiprone therapy (i.e., this will be baseline of the initial TIRCON2012V1 study for patients who received deferiprone in that study) ]
  • Proportion of patients with improved or unchanged total score on the BAD scale. [ Time Frame: Baseline to 18 months ]
  • Change in score on the Patient Global Impression of Improvement (PGI) [ Time Frame: Visit 1 to 18 months ]
  • Proportion of patients showing an improvement on the Patient Global Impression of Improvement at the last visit [ Time Frame: Visit 1 to 18 months ]
  • For patients who earlier received placebo, comparison of change in BAD total score from baseline to end of initial study vs. change in BAD total score from baseline to end of extension study [ Time Frame: Baseline to 18 months ]
  • For patients who earlier received placebo, comparison of proportion with improved or unchanged BAD total score between baseline and end of initial study vs. from baseline to end of extension study [ Time Frame: Baseline of initial study to end of extension study (up to 36 months) ]
  • For only those patients who received placebo in the initial study, comparison of change in PGI-I score from baseline to completion of the initial study vs. change in PGI-I score from baseline to completion of the extension study [ Time Frame: Baseline of initial study to end of extension study (up to 36 months) ]
  • For patients who earlier received placebo, comparison of proportion of patients showing an improvement on PGI-I at end of initial study vs. at end of extension study [ Time Frame: Baseline of initial study to end of extension study (up to 36 months) ]


Original Secondary Outcome: Same as current

Information By: ApoPharma

Dates:
Date Received: June 5, 2014
Date Started: June 2014
Date Completion: February 2018
Last Updated: March 22, 2017
Last Verified: April 2016