MedTrace Logo

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

NCT04182763 · Status: COMPLETED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 77

Last updated 2025-10-29

Study results available
· View outcomes & findings →

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

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.

Sponsors & Collaborators

  • Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

    collaborator NIH

  • Washington State University

    collaborator OTHER

  • Oregon State University

    collaborator OTHER

  • Spoonbill Foundation

    collaborator UNKNOWN

  • Spoonbill

    collaborator UNKNOWN

  • Oregon Health and Science University

    lead OTHER

Principal Investigators

  • Penelope Hogarth, M.D. · Oregon Health and Science University

  • Susan J Hayflick, MD · Oregon Health and Science University

Study Design

Allocation
RANDOMIZED
Purpose
OTHER
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
3 Months
Max Age
89 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-12-04
Primary Completion
2024-09-01
Completion
2025-01-01

Countries

  • United States

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04182763 on ClinicalTrials.gov