The purpose of this study is to learn more about how people with the condition pantothenate kinase-associated neurodegeneration (PKAN) respond to a specialized study product. We are hoping to find out if the study product is safe, what effects-good and bad-the study product causes, and whether the study product changes certain measures of disease in PKAN.
Age range
3 Months – 89 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.
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.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
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.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Number of Treatment-emergent Adverse Events Assessed Using CTCAE v4.0
Timeframe: 6 months following first dose in double-blind phase
Number of Treatment-emergent Clinically Significant Laboratory Abnormalities on Complete Blood Count.
Timeframe: 6-month randomized, double-blind, placebo-controlled phase
Number of Treatment-emergent Clinically Significant Laboratory Abnormalities on Comprehensive Metabolic Profile.
Timeframe: 6-month randomized, double-blind, placebo-controlled phase
Number of Participants Retained in Each Arm.
Timeframe: 6 month randomized, double-blind, placebo-controlled period
Mean Percent of Study Product Consumed.
Timeframe: 6-month randomized, double-blind, placebo-controlled phase