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

Overview

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 PKAN disease.

Full Title of Study: “A Phase 2 Study of a Vitamin Metabolite for PKAN”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Crossover Assignment
    • Primary Purpose: Other
    • Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
  • Study Primary Completion Date: January 2024

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.

Arms, Groups and Cohorts

  • Experimental: CoA-Z dose 1
    • 6 months of CoA-Z at the highest assigned dose followed by 18 months of CoA-Z at dose 2
  • Experimental: CoA-Z dose 2
    • 6 months of CoA-Z at the medium assigned dose followed by 18 months of CoA-Z at dose 2
  • Experimental: CoA-Z dose 3
    • 6 months of CoA-Z at the lowest assigned dose followed by 18 months of CoA-Z at dose 2
  • Placebo Comparator: Placebo
    • 6 months of placebo, followed by 18 months of CoA-Z at dose 2

Clinical Trial Outcome Measures

Primary Measures

  • Number of participants experiencing adverse events assessed by CTCAE v4.0
    • Time Frame: 24 months
    • Safety will be measured by measuring the number of participants experiencing adverse events by study period and treatment arm
  • Percentage of participants experiencing adverse events assessed by CTCAE v4.0
    • Time Frame: 24 months
    • Safety will be measured by measuring the percentage of participants experiencing adverse events by study period and treatment arm
  • Percentage of participants experiencing clinically significant laboratory abnormalities on Complete Blood Count.
    • Time Frame: 24 months
    • Safety will be assessed by measuring the percentage of participants experiencing clinically significant laboratory abnormalities on Complete Blood Count by collection month. Clinical significance will be determined by Medical Safety Monitor.
  • Number of participants experiencing clinically significant laboratory abnormalities on Complete Blood Count.
    • Time Frame: 24 months
    • Safety will be assessed by measuring the number of participants experiencing clinically significant laboratory abnormalities on Complete Blood Count by collection month. Clinical significance will be determined by Medical Safety Monitor.
  • Percentage of participants experiencing clinically significant laboratory abnormalities on Comprehensive Metabolic Profile.
    • Time Frame: 24 months
    • Safety will be assessed by measuring the percentage of participants experiencing clinically significant laboratory abnormalities on Comprehensive Metabolic Profile by collection month. Clinical significance will be determined by Medical Safety Monitor.
  • Number of participants experiencing clinically significant laboratory abnormalities on Comprehensive Metabolic Profile.
    • Time Frame: 24 months
    • Safety will be assessed by measuring the number of participants experiencing clinically significant laboratory abnormalities on Comprehensive Metabolic Profile by collection month. Clinical significance will be determined by Medical Safety Monitor.
  • Number of participants retained in each arm.
    • Time Frame: 24 months
    • Tolerability will be assessed by measuring the number of participants retained in each arm at each follow up time point.
  • Mean percent of study product consumed.
    • Time Frame: 24 months
    • Tolerability will be assessed by adherence to the study product regimen arm at each follow-up time point.

Secondary Measures

  • Ratio of CoASY mRNA expression level to that of 18s, an internal control
    • Time Frame: 24 months
    • The pharmacodynamic profile of a putative disease biomarker will be assessed by measuring the mean level of CoASY gene expression by treatment arm across study timepoints.

Participating in This Clinical Trial

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, likely pathogenic or VOUS mutation +/- a second VOUS in PANK2, or (c) be a symptomatic sibling of a proband subject meeting a or b.
  • Be between 12 months old and 89 years old.
  • Be able to take study by mouth or gastronomy 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 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.

Gender Eligibility: All

Minimum Age: 1 Year

Maximum Age: 89 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Oregon Health and Science University
  • Collaborator
    • Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
  • Provider of Information About this Clinical Study
    • Principal Investigator: Penelope Hogarth, Professor – Oregon Health and Science University
  • Overall Official(s)
    • Penelope Hogarth, M.D., Principal Investigator, Oregon Health and Science University
  • Overall Contact(s)
    • Allison Gregory, M.S., 503-494-4344, gregorya@ohsu.edu

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