Brief Title
A Pilot Clinical Trial of Pyruvate, Creatine, and Niacinamide in Progressive Supranuclear Palsy.
Official Title
Energy Metabolism in Neurodegenerative Diseases: A Randomized, Double Blind, Placebo-Controlled Clinical Pilot Trial of Pyruvate, Creatine, and Niacinamide in Progressive Supranuclear Palsy.
Brief Summary
This study intends to study the safety and tolerance of the combination of pyruvate,
creatine, and niacinamide over 6 months in patients with PSP.
Detailed Description
There are no effective symptomatic or biologic treatments for progressive supranuclear palsy
(PSP), a relatively rare neurodegenerative disease that presents late in life with relentless
progressive postural balance disturbances, non-levodopa responsive parkinsonism, supranuclear
vertical gaze palsy, pseudobulbar palsy, and frontal behavioral and dysexecutive symptoms. In
light of currently proposed etiopathogenic mechanisms in PSP and based on successful
experiments inhibiting cellular neurotoxicity, it is hypothesized that preservation of brain
energy homeostasis may allow endogenous neuroprotective mechanisms to reverse or impede free
radical injury or other neurotoxic events leading to neurodegeneration in this disease. An
emerging literature has described the neuroprotective effects of pyruvate, (as a neuronal
energy fuel and free radical scavenger); niacinamide, (which boosts cofactor NAD), and
creatine, (which buffers and selectively parcels cellular energy utilization) in various
animal models of brain injury or degeneration.
Ajay Verma et al. have further demonstrated a synergistic neuroprotective effect of these
three nutrients in various neural injury models. We thus propose using these nutrients as a
novel and safe neuroprotective approach for treating PSP patients. This randomized,
double-blind, placebo, control pilot study will test the safety and tolerance of this
nutrient combination over 6 months in patients with PSP, and will measure their transport
across the blood brain barrier. In addition to clinical and neuropsychological outcome
measures, brain creatine will also be evaluated using magnetic resonance spectroscopy before
and after therapy
Study Type
Interventional
Primary Outcome
Clinical features of PSP, including motor function, neuropsychological function, and blood chemistry
Secondary Outcome
CSF metabolite concentrations
Condition
Progressive Supranuclear Palsy
Intervention
Pyruvate, creatine, niacinamide
Study Arms / Comparison Groups
Pyruvate, creatine, niacinamide
Description: Pyruvate, creatine, niacinamide administered
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Dietary Supplement
Estimated Enrollment
20
Start Date
April 2004
Completion Date
December 2009
Primary Completion Date
December 2009
Eligibility Criteria
Inclusion Criteria:
- All subjects must meet the clinically definite or probable NINDS-SPSP PSP diagnostic
research criteria that includes the presence of postural instability at disease onset,
as well as supranuclear vertical ophthalmoparesis.
- All subjects must be able to tolerate oral feedings and be ambulatory
- All subjects or their caregivers must be able to read and understand the consent
Exclusion Criteria:
- Any contraindications to the use of pyruvate, creatine, and niacinamide
- the presence of a medical condition that can reasonably be expected to subject the
patient to unwarranted risk or require frequent changes in medication.
- Pregnancy, nursing, or lack of effective contraception, if still at child-bearing age.
- History of prior sever traumatic brain injury or other severe neurologic or
psychiatric condition, such as psychosis, stroke, multiple sclerosis, or spinal cord
injury, which will interfere with outcome evaluation, in the opinion of the local
principal investigator
- Subject unable to discontinue prohibited medication, which includes antiparkinsonian
medications with potential neuroprotective effects such as amantadine, deprenyl, and
vitamin E supplements > 400 IU per day.
Gender
All
Ages
N/A - N/A
Accepts Healthy Volunteers
No
Contacts
Irene Litvan, MD, ,
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT00605930
Organization ID
083.03
Secondary IDs
420
Responsible Party
Sponsor
Study Sponsor
University of Louisville
Study Sponsor
Irene Litvan, MD, Principal Investigator, University of Louisville, Division of Movement Disorders
Verification Date
April 2017