Compassionate Use of Deferiprone in Patients With PKAN

NCT02635841 | Unknown

• 1 other identifier: TIRCON2012V1-COMP
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Pantothenate Kinase-Associated Neurodegeneration

Keywords

Pantothenate Kinase-Associated Neurodegeneration; PKAN; Neurodegeneration with Brain Iron Accumulation; NBIA; Deferiprone; Ferriprox

Interventions

Deferiprone DRUG

Ferriprox (deferiprone) 100 mg/mL oral solution

Also known as: Ferriprox

Eligibility Criteria

• Age: 7 Years+
• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Chiesi Canada Corp: lead

MeSH Terms

Conditions

Pantothenate Kinase-Associated Neurodegeneration

Interventions

Deferiprone

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuroaxonal Dystrophies; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Pyridones; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 17, 2015
• First Posted: December 21, 2015
• Last Updated: November 14, 2022

Record last verified: 2022-11