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.
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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