Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 3 of 7 for:    Neurodegeneration with Brain Iron Accumulation (NBIA)

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04182763
Recruitment Status : Recruiting
First Posted : December 2, 2019
Last Update Posted : January 6, 2020
Sponsor:
Collaborators:
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Washington State University
Oregon State University
Spoonbill Foundation
Information provided by (Responsible Party):
Penelope Hogarth, Oregon Health and Science University

Brief 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 PKAN disease.

Condition or disease Intervention/treatment Phase
Pantothenate Kinase-Associated Neurodegeneration Other: CoA-Z Other: Placebo Not Applicable

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 51 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description: An initial 6-month, dose-ranging, parallel-group, randomized, double-blind, placebo-controlled phase will be followed by an 18-month, single-dose, open-label phase.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Other
Official Title: A Phase 2 Study of a Vitamin Metabolite for PKAN
Actual Study Start Date : December 4, 2019
Estimated Primary Completion Date : January 2024
Estimated Study Completion Date : January 2024


Arm Intervention/treatment
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
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.

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
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.

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
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.

Placebo Comparator: Placebo
6 months of placebo, followed by 18 months of CoA-Z at dose 2
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.




Primary Outcome Measures :
  1. 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

  2. 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

  3. 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.

  4. 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.

  5. 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.

  6. 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.

  7. 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.

  8. 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 Outcome Measures :
  1. 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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   3 Months to 89 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04182763


Contacts
Layout table for location contacts
Contact: Allison Gregory, M.S. 503-494-4344 CoAZinPKAN@ohsu.edu

Locations
Layout table for location information
United States, Oregon
Oregon Health & Science University Recruiting
Portland, Oregon, United States, 97239
Contact: Allison Gregory, MS    503-494-4344    CoAZinPKAN@ohsu.edu   
Sponsors and Collaborators
Oregon Health and Science University
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Washington State University
Oregon State University
Spoonbill Foundation
Investigators
Layout table for investigator information
Principal Investigator: Penelope Hogarth, M.D. Oregon Health and Science University
Additional Information:
Layout table for additonal information
Responsible Party: Penelope Hogarth, Professor, Oregon Health and Science University
ClinicalTrials.gov Identifier: NCT04182763    
Other Study ID Numbers: IRB00018782
R01HD097328 ( U.S. NIH Grant/Contract )
First Posted: December 2, 2019    Key Record Dates
Last Update Posted: January 6, 2020
Last Verified: January 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: IPD will be entered in a stand-alone repository that will allow for future sharing with other researchers. At this time we have not yet determined what IPD are to be shared.
Time Frame: Data will not be available until after September, 2024

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Keywords provided by Penelope Hogarth, Oregon Health and Science University:
Neurodegeneration with Brain Iron Accumulation
NBIA
PKAN
Additional relevant MeSH terms:
Layout table for MeSH terms
Pantothenate Kinase-Associated Neurodegeneration
Nerve Degeneration
Brain Diseases
Pathologic Processes
Basal Ganglia Diseases
Central Nervous System Diseases
Nervous System Diseases
Neuroaxonal Dystrophies
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn