GM1 Ganglioside Effects on Parkinson's Disease
Condition: Parkinson Disease
MeSH terms: Parkinson Disease
Intervention: GM1 ganglioside; Placebo
Intervention description: 100 mg twice per day by subcutaneous injection; Twice per day subcutaneous injection, equal volume as active drug
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: Double (Participant, Investigator)
Enrollment: 94.0
Sex: All
Minimum age: 39 Years
Maximum age: 85 Years
Healthy volunteers: No
Keywords: Parkinson's disease; PD; GM1 Ganglioside; Sygen

Summary:

      The purpose of this trial is to examine the short term effects (24 Weeks) of GM1 on
      Parkinson's disease (PD) symptoms, as well as the effects of long-term treatment (120 Weeks)
      with GM1 on disease progression, and to examine the extent to which GM1 treatment influences
      the underlying disease process in PD.
    

Eligibility criteria:

        Inclusion criteria:

          -  Btwn ages of 39-85 yrs old.

          -  Females: at least 2 years post-menopausal; surgically sterile; or negative pregnancy
             test by quantitative serum ßHCG, & follow a reliable method of birth control for at
             least 2 months prior to entry, agree both to follow a reliable method of birth
             control, & to desist from breast feeding during, & for 1 month following, the study
             drug administration.

          -  Dx of idiopathic PD 6 months prior to screening. The diagnosis requires: the presence
             of at least 2 of the 4 cardinal clinical manifestations of the disease, tremor,
             rigidity, bradykinesia, and disturbances of posture or gait, & at least 1 must be
             rigidity or bradykinesia.

          -  Modified Hoehn and Yahr Staging between 1 and 3 as rated during an "off" period of at
             least 12 hours.

          -  Unified Parkinson's Disease Rating Scale motor component score between 10 and 40 as
             rated during an "off" period of at least 12 hours and a score of 6 or greater during
             "on" period.

          -  Antiparkinsonian treatments: stable treatment of l-dopa/carbidopa and/or dopamine
             agonist for at least 3 months prior to Screening.

          -  Mini Mental State Exam score > 25.

          -  Beck Depression Inventory score < 10.

          -  Signed informed consent.

        Exclusion criteria:

          -  Abrupt onset of Parkinsonism.

          -  Failure of Parkinsonian symptoms to have responded to l-dopa.

          -  Motor symptoms (such as peak dose dyskinesias (UPDRS score > 3), & random on-off
             phenomenon, other than end-of-dose wearing-off, persistently fluctuating over a 6
             month or longer period, in response to l-dopa.

          -  Hx of findings of any movement disorder other than idiopathic PD.

          -  A tremor score on the UPDRS motor scale of >5. Tremor score greater than 3 in an
             individual limb.

          -  High-dose vitamin E therapy (more than 1000 I.U./day) any time during the period
             starting 3 months prior baseline.

          -  Transient ischemic attack any time during the period starting 6 months prior baseline.

          -  Hx of 2 or more strokes. Hx of any stroke that resulted in motor deficit, movement
             disorder, ataxia, cognitive impairment, or a hemi-inattention syndrome. Any stroke
             with residua at the time of, or within 6 months preceding, study entry.

          -  Previous cerebral infarction, including lacunar infarction, in any area subserving
             motor function.

          -  Binswanger's disease or hx of hypertensive encephalopathy.

          -  Hx of encephalitis.

          -  Hx of extended exposure to any known neurotoxin that may cause parkinsonism, or
             chronic or sufficient use or consumption of any non-medicinal substance that could
             cause risk of developing a movement disorder or disturbance of posture or gait.

          -  Use of the following drugs within 6 months prior to screening: neuroleptics,
             metoclopramide, clozapine, flunarizine, alpha-methyldopa.

          -  Patients actively taking a medicine that is known to compete with the imaging agent
             for binding sites on the dopamine terminals.

          -  Hx of medication or drug use that may have caused atypical parkinsonism or history of
             Substance Use Disorder.

          -  Hx of a metabolic disorder that resulted or could have resulted in movement disorder
             or disturbance of posture or gait.

          -  Any disease or condition that resulted or could have resulted in a movement disorder
             or disturbance of posture or gait.

          -  Hx of intracranial hemorrhage, intracranial neoplasm, significant head trauma with
             loss of consciousness >24 hrs or other structural brain disease.

          -  Hx or clinical findings suggestive of progressive supranuclear palsy or multiple
             system atrophy.

          -  Acquired cognitive impairment reasonably possibly due to any cause other than
             idiopathic PD.

          -  Hx of a hereditary disorder associated with a movement disorder.

          -  Normal or low pressure hydrocephalus.

          -  Any ill-defined neuropathic disease, myelopathy, myopathy or other medical disorder or
             physical condition by history or clinical examination that could be expected to
             interfere with the diagnosis, treatment or assessment of PD, or with any of the study
             assessments.

          -  Hx of Guillain-Barré syndrome, chronic idiopathic polyneuropathy, or relapsing
             polyneuropathy.

          -  Hx of Axis I or Axis II major psychiatric disorder.

          -  Significant cardiac, pulmonary, hepatic, gastrointestinal, or renal disease.

          -  Any condition that could alter the distribution, accumulation, metabolism, or
             excretion of the study medication; or result in a life expectancy of less than 2
             years.

          -  Any primarily non-neurologic medical condition with a significant risk of secondarily
             causing neurologic dysfunction.

          -  Myocardial infarction within 6 months prior to screening.

          -  Presence of any medical condition or laboratory test abnormality or use of any licit
             or illicit substance which could cause unwarranted risk from participation in the
             study or result in worsening in the patient's medical condition during participation
             in study. Presence of any of the following laboratory test abnormalities are
             unwarranted risk: serum transaminase greater than twice the upper limit of
             normal;serum creatinine greater than 2.0 mg/dl in a man or greater than 1.7 mg/dl in a
             woman.

          -  Hx of a life-threatening allergic or immune-mediated reaction.

          -  Previous use of any ganglioside preparation.

          -  Use of any experimental drug in the period starting 60 days before Baseline.

          -  Hx of stereotaxic brain surgery for PD.