NCT04182763

CoA-Z in Pantothenate Kinase-associated Neurodegeneration (PKAN)

A Phase 2 Study of a Vitamin Metabolite for PKAN

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 77 (actual)
• Sponsor: Oregon Health and Science University · Academic / Other
• Sex: All
• Age: 3 Months – 89 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Pantothenate Kinase-Associated Neurodegeneration

Interventions

Type	Name	Description
OTHER	CoA-Z	People with PKAN lack a chemical to process or metabolize a certain vitamin in the brain. CoA-Z is designed to bypass this metabolic defect that causes PKAN.
OTHER	Placebo	The placebo is a strawberry-flavored syrup that looks and tastes like CoA-Z but has no active CoA-Z in it.

Timeline

• Start date: 2019-12-04
• Primary completion: 2024-09-01
• Completion: 2025-01-01
• First posted: 2019-12-02
• Last updated: 2025-10-29
• Results posted: 2025-10-29

Locations

1 site across 1 country: United States