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

This study has been completed.
Information provided by (Responsible Party):
Weill Medical College of Cornell University Identifier:
First received: January 9, 2012
Last updated: February 28, 2017
Last verified: February 2017

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.

Condition Intervention Phase
Progressive Supranuclear Palsy
Drug: alpha-lipoic acid and L-acetyl carnitine
Phase 1
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Treatment
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

Resource links provided by NLM:

Further study details as provided by Weill Medical College of Cornell University:

Primary Outcome Measures:
  • Adverse Events [ Time Frame: at 25 weeks ]
    change of incidence and severity of adverse events

Secondary Outcome Measures:
  • 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

Enrollment: 11
Actual Study Start Date: September 14, 2010
Study Completion Date: April 7, 2015
Primary Completion Date: September 24, 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Juvenon Drug: alpha-lipoic acid and L-acetyl carnitine
alpha-lipoic acid and L-acetyl carnitine capsules, 600mg/1.5g daily for 6 months
Other Name: Juvenon

Detailed Description:

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 Alzheimer's disease. Importantly, it appeared that the effects of alpha-lipoic acid and L-acetyl carnitine when administered together were significantly augmented (100-1000 times), as opposed to when administered separately. This therefore provided a strong rationale to test the two in combination.

In addition to monitoring clinical features, we had also chosen to test physiologic effects of alpha-lipoic acid/L-acetyl carnitine in our PSP subjects using two biomarkers that provide measures of mitochondrial function and oxidative stress. This was particularly important, since both supplements may act by multiple mechanisms. 1H MRSI is a technique that provides insight into the metabolism of several endogenous brain compounds, most notably N-acetyl-L-aspartate (NAA), choline-containing compounds (Cho), and creatine and phosphocreatine (Cr). A number of studies of mitochondrial function had firmly established the utility of 1H MRSI in probing potential mitochondrial energy metabolism dysfunction. 31P MRSI provided complementary information to probe in vivo mitochondrial energy metabolism and tissue energetics. In addition, we proposed using markers of oxidative damage (including 8-hydroxydeoxyguanosine) as well as metabolomic analysis to test a composite panel of quantitative measures in plasma. We used an established metabolomic platform that has proven to identify specific combinations of metabolites differing between neurodegenerative disease states (including Parkinson's disease, Huntington's disease) and healthy controls. Our overall aim was to generate an "oxidative biomarker" and "metabolomic read-out" of the peripheral biochemical effects of alpha-lipoic acid/L-acetyl carnitine in PSP.


Ages Eligible for Study:   40 Years to 75 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Diagnosis of probable PSP by NINDS/PSP workshop criteria (see patient folder)
  • Age 40-75 years
  • Able to undergo MRI
  • Absence of significant medical, psychiatric, and other neurological disease
  • Stable intake of supplements and medication

Exclusion Criteria:

  • Failure to meet probable PSP diagnosis by NINDS/PSP workshop criteria
  • unable to comply with informed consent process
  • unable to undergo MRI
  • presence of significant medical, psychiatric (incl MDD) or other neurological (incl epilepsy, brain tumor, stroke) disease
  • possibility of pregnancy (negative test required in women of childbearing age)
  Contacts and Locations
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Please refer to this study by its identifier: NCT01537549

United States, New York
Weill Cornell Medical College
New York, New York, United States, 10021
Sponsors and Collaborators
Weill Medical College of Cornell University
Principal Investigator: Claire Henchcliffe, MD DPhil Weill Medical College of Cornell University
  More Information

Responsible Party: Weill Medical College of Cornell University Identifier: NCT01537549     History of Changes
Other Study ID Numbers: IRB1006011088
Study First Received: January 9, 2012
Results First Received: February 28, 2017
Last Updated: February 28, 2017

Keywords provided by Weill Medical College of Cornell University:
Progressive supranuclear palsy
alpha-lipoic acid
L-acetyl carnitine
magnetic resonance spectroscopy

Additional relevant MeSH terms:
Supranuclear Palsy, Progressive
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Movement Disorders
Ocular Motility Disorders
Cranial Nerve Diseases
Neurodegenerative Diseases
Eye Diseases
Thioctic Acid
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Vitamin B Complex
Growth Substances
Nootropic Agents processed this record on April 27, 2017