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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

77 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-12-04

Study Completion Date

2025-01-01

Brief Summary

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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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Detailed Description

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Conditions

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Pantothenate Kinase-Associated Neurodegeneration

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

An initial 6-month, dose-ranging, parallel-group, randomized, double-blind, placebo-controlled phase is followed by an 18-month, single-dose, open-label phase--these arms met enrollment goals. A direct-to-open-label arm of the study was opened to allow for additional enrollment.

Primary Study Purpose

OTHER

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Masking was used for the initial 6-month randomized, double-blind phase, placebo-controlled.

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

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.

Intervention Type OTHER

Placebo

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

Intervention Type OTHER

Eligibility Criteria

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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.

Minimum Eligible Age

3 Months

Maximum Eligible Age

89 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No