CoA-Z in Pantothenate Kinase-associated Neurodegeneration (PKAN)
Status
Unknown
Conditions
Pantothenate Kinase-Associated Neurodegeneration
Treatments
Other: CoA-Z
Other: Placebo
Study type
Interventional
Funder types
Other
NIH
Identifiers
Details and patient eligibility
About
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.
Enrollment
77 patients
Sex
All
Ages
3 months to 89 years old
Volunteers
No Healthy Volunteers
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.
Trial design
Primary purpose
Other
Allocation
Randomized
Interventional model
Single Group Assignment
Masking
Quadruple Blind
77 participants in 5 patient groups, including a placebo group
CoA-Z dose 1
Experimental group
Description:
6 months of CoA-Z at the highest assigned dose followed by 18 months of CoA-Z at dose 2
Treatment:
Other: CoA-Z
CoA-Z dose 2
Experimental group
Description:
6 months of CoA-Z at the medium assigned dose followed by 18 months of CoA-Z at dose 2
Treatment:
Other: CoA-Z
CoA-Z dose 3
Experimental group
Description:
6 months of CoA-Z at the lowest assigned dose followed by 18 months of CoA-Z at dose 2
Treatment:
Other: CoA-Z
Placebo
Placebo Comparator group
Description:
6 months of placebo, followed by 18 months of CoA-Z at dose 2 (medium dose)
Treatment:
Other: CoA-Z
Other: Placebo
Open-label arm
Experimental group
Description:
Up to 24 months of CoA-Z at dose 2
Treatment:
Other: CoA-Z
Trial contacts and locations
1
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Data sourced from clinicaltrials.gov
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