The goal of this study is to assess the impact of CoQ10 and GPI 1485 on the progression of
Parkinson's disease, in order to determine whether it is reasonable to proceed with further
study of either of these agents.
Parkinson's disease (PD) affects nearly a million Americans, a number that will increase
over the coming decades as the population ages. While available medical therapies are
usually effective for controlling symptoms in the initial years following diagnosis, higher
doses of multiple agents are required over time, with increasing side effects and incomplete
control of symptoms. Although these treatments can dramatically improve the lives of
patients with PD initially, they do not address the underlying causes of the disease or the
inevitable disease progression.
This multi-center, randomized, double-blind trial will involve 42 trial centers in the
United States and Canada, and enroll 195 people with PD. The primary objective of this
neuroprotection trial is to identify agents capable of slowing the progression of PD. In the
trial, investigators will assess the impact of CoQ10, an antioxidant, and GPI 1485, a novel
immunophilin compound, on the progression of PD and determine if it is futile or non-futile
to proceed with further study of these agents.
In this study, subjects with early, untreated PD will be equally randomized into one of the
three study arms: 1.) the group that receives active CoQ10 and placebo instead of GPI 1485;
2.) the group that receives active GPI 1485 and placebo instead of CoQ10; or 3.) the group
that receives placebo instead of CoQ10 and GPI 1485. Subjects will remain on the blinded
study drug for 12 months.
- Willing and able to give informed consent.
- Men and women with idiopathic PD of less than 5 years duration from diagnosis.
- Diagnosis must be confirmed by bradykinesia plus one of the other cardinal signs
(resting tremor, rigidity) being present, without any other known or suspected cause
of parkinsonism. The clinical signs must be asymmetric.
- Subjects must not require any therapy (including levodopa, dopamine agonists,
anticholinergics, amantadine, or selegiline) to treat symptoms of PD at the time of
enrollment. Subjects may have had prior exposure to any one of these agents, but
exposure with any agent may not have been longer than 60 days in duration and
subjects must not have been on any of these agents within 90 days prior to baseline.
Once a subject needs dopaminergic treatment, these medications may be added as part
of best medical management. The subject will still remain in the study.
- Age > 30 years.
- Willingness and ability to comply with study requirements.
- Women who are not postmenopausal or surgically sterile must use a medically accepted
contraceptive regimen for at least 60 days before the baseline visit, and agree to
continue such use throughout the duration of the study and for 30 days after the
final dose of study drug. Reliable forms of contraception include oral, implanted,
or injected contraceptives; intrauterine devices in place for at least 3 months; or
adequate barrier methods in conjunction with spermicide (abstinence is considered an
acceptable contraceptive regimen). Women must have a pregnancy test unless they are
at least 2 years postmenopausal or surgically sterile.
- Use of any of the following drugs within 180 days prior to baseline: neuroleptics,
metoclopramide, alpha-methyldopa, clozapine, olanzepine, and flunarizine.
- Use of any of the following drugs within 90 days prior to baseline: methylphenidate,
cinnarizine, reserpine, amphetamine, or MAO-A inhibitors (pargyline, phenelzine, and
- Presence of atypical Parkinson's syndromes due to drugs (e.g., metoclopramide,
flunarazine), metabolic identified neurogenetic disorders (e.g., Wilson's disease),
encephalitis, or other degenerative diseases (e.g., progressive supranuclear palsy).
- Use of CoQ10 or GPI 1485 90 days prior to baseline.
- Use of minocycline or creatine 90 days prior to baseline.
- Receipt of other investigational drugs within 90 days prior to baseline.
- Presence of freezing.
- Impairment of postural reflexes (pull test score > 0).
- Any clinically significant medical condition (e.g., active GI illness, angina, active
neoplasm) or laboratory abnormality, which would in the judgment of the investigator
interfere with the subjects ability to participate in the study or to be followed.
- History of stereotaxic brain surgery for PD (e.g., pallidotomy, deep brain
stimulation, fetal tissue implantation).
- Clinically significant structural brain disease that the investigator believes would
interfere with study evaluations.
- Significant psychiatric disorders that may interfere with complying with the
- History of known hypersensitivity or intolerability to CoQ10 or GPI 1485.