Clinical Trial: Compassionate Use of Deferiprone in Patients With PKAN

Study Status: Available
Recruit Status: Available
Study Type: Expanded Access

Official Title: The Compassionate Use of Deferiprone in Patients With Pantothenate Kinase-Associated Neurodegeneration

Brief Summary: Patients with pantothenate kinase-associated neurodegeneration (PKAN) who have completed the ApoPharma-sponsored study TIRCON2012V1-EXT and who wish to continue to take deferiprone will be offered the opportunity to receive it on a compassionate basis. Patients will be followed locally by their own neurologist or other appropriate specialist.

Detailed Summary: Patients with PKAN who have completed both the randomized, blinded, placebo-controlled trial TIRCON2012V1 and its follow-up, the single-arm open-label extension study TIRCON2012V1-EXT, may enroll in this compassionate-use/expanded access program in order to continue receiving the iron chelator deferiprone. Participants in this program will be followed locally by their own neurologist or other appropriate specialist. Patients will take deferiprone oral solution 80 mg/mL at the same dose they were prescribed in the TIRCON2012V1-EXT clinical trial. Safety monitoring of the patients will be performed. At a minimum, the program will continue until the analysis of the currently blinded TIRCON2012V1 trial is completed (approximately Q4 2016), at which time it will be re-evaluated.
Sponsor: ApoPharma

Current Primary Outcome:

Original Primary Outcome:

Current Secondary Outcome:

Original Secondary Outcome:

Information By: ApoPharma

Dates:
Date Received: December 17, 2015
Date Started:
Date Completion:
Last Updated: April 1, 2016
Last Verified: November 2015