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Completed

NCT Number: NCT04182763

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

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.

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Key information

Conditions

Pantothenate Kinase-Associated Neurodegeneration Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Movement Disorders Nervous System Diseases Neuroaxonal Dystrophies Neurodegenerative Diseases

Age range

3 month–89 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Has a diagnosis of PKAN confirmed by: a) genetic testing confirming 2 pathogenic or likely pathogenic mutations, or (b) typical findings on exam and brain MR imaging with only one pathogenic mutation +/- a second likely pathogenic or VOUS in PANK2, or (c) typical findings on exam and brain MR imaging with a single likely pathogenic or VOUS in PANK2, or (d) be a symptomatic sibling of a proband subject meeting a, b or c.
  • Be between 3 months old and 89 years old.
  • Be able to take study product by mouth or feeding tube.
  • Be willing and able to complete study procedures / telephone visits / blood draws independently, OR have a caregiver / parent willing and able to assist with these tasks.
  • Be enrolled or willing to enroll in the PKANready natural history study (eIRB 10832).
  • Be resident in North America (US or Canada) for the duration of the trial.

Exclusion criteria

  • Have had exposure to a putative PANK2 bypass therapeutic agent in the 30 days prior to screening.
  • Be concurrently enrolled in another interventional clinical trial.
  • Have concurrent medical or other condition expected to preclude completion of study procedures of confound the assessment of clinical and laboratory measures of safety.

Treatment and study plan

CoA-Z

Other

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.

Placebo

Other

The placebo is a strawberry-flavored syrup that looks and tastes like CoA-Z but has no active CoA-Z in it.

Primary outcomes

  1. Number of Treatment-emergent Adverse Events Assessed Using CTCAE v4.0

    Time frame: 6 months following first dose in double-blind phase

    Safety will be measured using the number of treatment-emergent adverse events (both AE and SAE) by treatment arm. Counts are adjusted for number of prescription medications at baseline as a measure of baseline disease severity.

  2. Number of Treatment-emergent Clinically Significant Laboratory Abnormalities on Complete Blood Count.

    Time frame: 6-month randomized, double-blind, placebo-controlled phase

    Safety will be assessed by measuring the number of treatment-emergent clinically significant laboratory abnormalities on Complete Blood Count. Clinical significance will be determined by Medical Safety Monitor and Clinical Investigators.

  3. Number of Treatment-emergent Clinically Significant Laboratory Abnormalities on Comprehensive Metabolic Profile.

    Time frame: 6-month randomized, double-blind, placebo-controlled phase

    Safety will be assessed by measuring the number of treatment-emergent clinically significant laboratory abnormalities on Comprehensive Metabolic Profile by collection month. Clinical significance will be determined by Medical Safety Monitor and Clinical Investigators.

  4. Number of Participants Retained in Each Arm.

    Time frame: 6 month randomized, double-blind, placebo-controlled period

    Tolerability will be assessed by measuring the number of participants retained in each arm over the course of the study.

  5. Mean Percent of Study Product Consumed.

    Time frame: 6-month randomized, double-blind, placebo-controlled phase

    Tolerability will be assessed by adherence to the study product regimen arm at each follow-up time point.

Secondary outcomes

  1. CoASY mRNA Expression

    Time frame: Up to 6 months after first dose

    Average relative CoASY gene expression, measured as the ratio to baseline of 1 / (2^[COASY Ct - 18s Ct]), where Ct = cycle time and 18s is a housekeeping gene. Values >1 reflect higher expression.

Sponsors and collaborators

Lead sponsor

Oregon Health and Science University

Other

Collaborators

  • Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
  • Oregon State University
  • Spoonbill
  • Spoonbill Foundation
  • Washington State University

Registry information

Official study title

A Phase 2 Study of a Vitamin Metabolite for PKAN

Important dates

Study start
2019
Primary completion
2024
Study completion
2025
First posted
Dec 2, 2019
Registry last updated
Oct 29, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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