Improving treatment for disorders that affect fat metabolism
Optimizing medium-chain lipids for the treatment of long-chain fatty acid oxidation disorders
['FUNDING_R01'] · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · NIH-11041006
This study is looking at ways to make treatments with medium-chain fatty acids better for people with long-chain fatty acid oxidation disorders, which make it hard for their bodies to use certain fats for energy, with the goal of helping reduce symptoms like heart problems and muscle weakness.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF PITTSBURGH AT PITTSBURGH (nih funded) |
| Locations | 1 site (PITTSBURGH, UNITED STATES) |
| Trial ID | NIH-11041006 on ClinicalTrials.gov |
What this research studies
This research focuses on long-chain fatty acid oxidation disorders (LC-FAODs), which hinder the body's ability to break down certain fats for energy. The study aims to enhance the effectiveness of medium-chain fatty acids (MCFAs) in treating these disorders by exploring new lipid species and delivery methods. It seeks to understand how MCFAs are metabolized in the liver, heart, and muscle, and to address the limitations of current MCFA therapies that have not significantly improved patient outcomes. By optimizing these treatments, the research hopes to alleviate symptoms like cardiomyopathy and muscle weakness in affected patients.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with long-chain fatty acid oxidation disorders, particularly those experiencing muscle symptoms or cardiomyopathy.
Not a fit: Patients with other metabolic disorders unrelated to fatty acid oxidation may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatments for patients with long-chain fatty acid oxidation disorders, improving their quality of life and reducing symptoms.
How similar studies have performed: While there have been studies on medium-chain fatty acids, this research aims to explore novel approaches that have not been extensively tested in human patients.
Where this research is happening
PITTSBURGH, UNITED STATES
- UNIVERSITY OF PITTSBURGH AT PITTSBURGH — PITTSBURGH, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: GOETZMAN, ERIC S — UNIVERSITY OF PITTSBURGH AT PITTSBURGH
- Study coordinator: GOETZMAN, ERIC S
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.