Compassionate Use of Deferiprone in Patients With PKAN

• Status: Available
• Trial end date: 1969-12-31
• Target enrollment: 0
• Participant gender: All

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.

Details

• Lead Sponsor: ApoPharma; Chiesi Canada Corp
• Treatments: Deferiprone

Criteria

Inclusion Criteria:

1. Completed study TIRCON2012V1-EXT

2. Able to adhere to program appointments and evaluation schedules

3. Females of childbearing potential must have a negative pregnancy test result at the start of the program, unless the treating physician determines there is no reasonable risk of pregnancy because of significant incapacity. If applicable, they must meet at least one of the following criteria: Use an effective method of contraception during the program and for 30 days following the last dose of deferiprone, OR participate in a non-heterosexual lifestyle, OR have a male sexual partner who has been sterilized, OR be abstinent during the program and for at least 30 days after its completion.

4. Fertile sexually active heterosexual males must agree to use an effective method of contraception during the program and for at least 30 days after its completion

5. Patients and/or their authorized legal representatives must provide signed and dated written informed consent, and minors must additionally sign an assent form as per local regulatory requirements.

Exclusion Criteria:

Patients will be excluded from taking part in the compassionate use program if the treating physician determines the presence of any medical, psychological, or psychiatric condition which in his/her opinion would cause participation in the program to be unwise