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

Brief Title

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

Official Title

A Phase 2 Study of a Vitamin Metabolite for PKAN

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.
    



Study Type

Interventional


Primary Outcome

Number of participants experiencing adverse events assessed by CTCAE v4.0

Secondary Outcome

 Ratio of CoASY mRNA expression level to that of 18s, an internal control

Condition

Pantothenate Kinase-Associated Neurodegeneration

Intervention

CoA-Z

Study Arms / Comparison Groups

 CoA-Z dose 1
Description:  6 months of CoA-Z at the highest assigned dose followed by 18 months of CoA-Z at dose 2

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Other

Estimated Enrollment

51

Start Date

December 4, 2019

Completion Date

January 2024

Primary Completion Date

January 2024

Eligibility 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.
      

Gender

All

Ages

3 Months - 89 Years

Accepts Healthy Volunteers

No

Contacts

Penelope Hogarth, M.D., 503-494-4344, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT04182763

Organization ID

IRB00018782

Secondary IDs

R01HD097328

Responsible Party

Principal Investigator

Study Sponsor

Oregon Health and Science University

Collaborators

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

Study Sponsor

Penelope Hogarth, M.D., Principal Investigator, Oregon Health and Science University


Verification Date

January 2020