Clinical Trial: Alpha-lipoic Acid/L-acetyl Carnitine for Progressive Supranuclear Palsy

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: An Open-label Trial of Alpha-lipoic Acid/L-acetyl Carnitine for Progressive Supranuclear Palsy (PSP): Effect Upon Oxidative Damage and Mitochondrial Biomarkers

Brief Summary:

Studies have shown that alpha-lipoic acid and L-acetyl carnitine may have some neuroprotective activities and it is hoped that they could be helpful for people with neurodegenerative illnesses such as progressive supranuclear palsy (PSP).

The purpose of this study is to find out whether the nutritional supplement alpha-lipoic acid/L-acetyl carnitine is safe and well-tolerated in individuals with PSP when given daily, and whether it affects their well-being, brain scan measurements and blood tests that measure the energy metabolism in cells.


Detailed Summary:

Multiple lines of evidence support mitochondrial dysfunction and oxidative stress playing a role in the pathogenesis of atypical Parkinsonism, including PSP. Such dysfunction may well contribute to the tau pathology that is well-recognized in PSP, thus providing a link between the two processes. This pathway therefore represents an excellent potential target for novel therapeutic intervention in neurodegenerative disorders, and a number of well-tolerated and safe nutritional supplements have been identified that appear to augment mitochondrial function, and improve oxidative stress.

Alpha-lipoic acid and L-acetyl carnitine are two nutritional supplements that have received increasing attention as potential neuroprotective interventions in neurodegenerative and other disease states. Alpha-lipoic acid/L-acetyl carnitine had been demonstrated to improve learning in aged beagles over 2 months of administration, and showed a trend to improve cognitive function in a mouse model of Alzheimer's disease (human apoE4 transgene). Moreover, alpha-lipoic acid/L-acetyl carnitine was neuroprotective in a mouse model of Parkinson's disease (rotenone-induced parkinsonism), with effects including decreased oxidative stress, and increased mitochondrial biogenesis. In fibroblasts derived from individuals with Alzheimer's disease, alpha-lipoic acid/L-acetyl carnitine reduced increased levels of oxidative stress. In healthy men exposed to intensive exercise, alpha-lipoic acid provided antioxidant effects systemically (decreased peroxidation). L-acetyl carnitine improved neuroimaging correlates of cerebral blood flow in 30 subjects with dementia. These nutritional supplements have been safe and well-tolerated, and they have been tested in age groups including children, up to the elderly. Alpha-lipoic acid had been successfully administered over an extended period in an open-label trial in Alzhe
Sponsor: Weill Medical College of Cornell University

Current Primary Outcome: Adverse Events [ Time Frame: at 25 weeks ]

change of incidence and severity of adverse events


Original Primary Outcome: Adverse Events [ Time Frame: at baseline, 4, 12 and 24 weeks ]

change of incidence and severity of adverse events


Current Secondary Outcome: Cerebral Oxidative Stress Markers [ Time Frame: at baseline and at week 5 ]

changes of cerebral lactate and glutathione levels as determined by magnetic resonance spectroscopy


Original Secondary Outcome:

  • Clinical assessments [ Time Frame: at baseline, at weeks 5, 13, 25 ]
    changes of scores for UPDRS, Golbe PSP Rating Scale, Hoehn and Yahr Scale and neuropsychological tests
  • Quality of life [ Time Frame: at baseline, at weeks 5, 13, 25 ]
    changes of scores for PSP quality of life questionnaire (PSP QOL)
  • Routine safety laboratory parameters [ Time Frame: at baseline, at weeks 5, 13, 25 ]

    Changes in

    • Electrolyte panel (CPT 80051)
    • Liver function panel (CPT 80076)
    • CBC (CPT 85027)
  • Cerebral Oxidative Stress Markers [ Time Frame: at baseline and at week 5 ]
    changes of cerebral lactate and glutathione levels as determined by magnetic resonance spectroscopy


Information By: Weill Medical College of Cornell University

Dates:
Date Received: January 9, 2012
Date Started: September 14, 2010
Date Completion:
Last Updated: February 28, 2017
Last Verified: February 2017