N/A
N=65
CoA-Z in Pantothenate Kinase-associated Neurodegeneration (PKAN)
Pantothenate Kinase-Associated Neurodegeneration
Bottom Line
View on ClinicalTrials.gov: NCT04182763 ↗Enrolled (actual)
65
Serious AEs
35.5%
Results posted
Oct 2025
Primary outcome: Primary: Number of Treatment-emergent Adverse Events Assessed Using CTCAE v4.0 — 1.15; 1.38; 1.14; 0.86 Events per person — p=0.50
Study Design & Population
- Study type
- Interventional
- Phase
- N/A
- Interventions
- CoA-Z (Other); Placebo (Other)
- Age
- Pediatric, Adult, Older Adult · 0+ yrs
- Sex
- All
- Sponsor
- Oregon Health and Science University
- Primary completion
- Sep 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Treatment-emergent Adverse Events Assessed Using CTCAE v4.0 |
1.15; 1.38; 1.14; 0.86 | 0.50 |
| PRIMARY Number of Treatment-emergent Clinically Significant Laboratory Abnormalities on Complete Blood Count. |
0; 1; 0; 0 | — |
| PRIMARY Number of Treatment-emergent Clinically Significant Laboratory Abnormalities on Comprehensive Metabolic Profile. |
1; 1; 0; 0 | — |
| PRIMARY Number of Participants Retained in Each Arm. |
15; 16; 17; 15 | — |
| PRIMARY Mean Percent of Study Product Consumed. |
98.98; 99.25; 98.66; 96.94 | — |
| SECONDARY CoASY mRNA Expression |
1.31; 1.13; 1.04; 1.07; 1.07; 0.97 | 0.105 |
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 disease in PKAN.
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.
Data sourced from ClinicalTrials.gov (NCT04182763). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.