Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Oregon Health and Science University
With
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Washington State University
Oregon State University
Spoonbill Foundation
Spoonbill
Age
0–89
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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.
You may not be if
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.
Clareo Health | CoA-Z in Pantothenate Kinase-associated Neurodegeneration (PKAN)
