NCT02635841

Compassionate Use of Deferiprone in Patients With PKAN

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

• Status: No Longer Available
• Phase: —
• Study type: Expanded Access
• Enrollment: —
• Sponsor: Chiesi Canada Corp · Industry
• Sex: All
• Age: 7 Years
• Healthy volunteers: —

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 description

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.

Conditions

• Pantothenate Kinase-Associated Neurodegeneration

Interventions

Type	Name	Description
DRUG	Deferiprone	Ferriprox (deferiprone) 100 mg/mL oral solution

Timeline

• First posted: 2015-12-21
• Last updated: 2022-11-14