Investigating a vitamin metabolite for treating PKAN
A phase 2 study of a vitamin metabolite for PKAN
This study is testing a new vitamin treatment for people of all ages with pantothenate kinase-associated neurodegeneration (PKAN) to see if it can help improve their symptoms and quality of life.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Oregon Health & Science University NIH-funded |
| Lab location | 1 site (Portland, United States) |
| Project ID | NIH-10683049 on NIH RePORTER |
What this research studies
This research focuses on developing a new treatment for pantothenate kinase-associated neurodegeneration (PKAN), a rare genetic disorder caused by a defect in vitamin B5 metabolism. The study will evaluate the safety and effectiveness of a vitamin metabolite in both children and adults with PKAN through a phase 2 clinical trial. Participants will be randomly assigned to receive either the treatment or a placebo in a double-blind manner, followed by an open-label phase where all participants will receive the treatment. The goal is to assess how well this treatment can improve symptoms and quality of life for those affected by this debilitating condition.
Who could benefit from this research
Good fit: Ideal candidates for this research are children and adults diagnosed with pantothenate kinase-associated neurodegeneration (PKAN).
Not a fit: Patients with other neurological disorders or those not diagnosed with PKAN may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a new therapy that significantly improves the lives of patients suffering from PKAN.
How similar studies have performed: Previous research has shown promise in using similar metabolic approaches for treating other neurological conditions, indicating potential for success in this novel application.
Where this research is happening
Portland, United States
- Oregon Health & Science University — Portland, United States (Active)
Researchers
- Principal investigator: Hogarth, Penelope — Oregon Health & Science University
- Study coordinator: Hogarth, Penelope
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.