NO_LONGER_AVAILABLENot Applicable

Compassionate Use of Deferiprone in Patients With PKAN

Plain-language 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.

Who can participate

Age range

7 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Completed study TIRCON2012V1-EXT
. Able to adhere to program appointments and evaluation schedules
. 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.
. 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
. 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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

Trial details

NCT IDNCT02635841

SponsorChiesi Canada Corp

Sponsor typeINDUSTRY

Study typeEXPANDED_ACCESS

View on ClinicalTrials.gov More Pantothenate Kinase-Associated Neurodegeneration trials