GM1 Ganglioside Effects on Parkinson's Disease

NCT00037830 | Completed Phase 2 Results

• 1 other identifier: R01NS038681
• Study Type: interventional
• Target: 94
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Parkinson Disease

Keywords

Parkinson's disease; PD; GM1 Ganglioside; Sygen

Outcome Measures

Primary Outcomes (2)

• Change in Unified Parkinson's Disease Rating Scale (UPDRS) (Part III) Motor Score From Baseline to Week 24 Assessed Off Medication.

  The Unified Parkinson's Disease Rating Scale (UPDRS) (Part III) motor scores assess 14 symptoms some of which separately assess symptoms in different body parts (e.g. right arm, left arm, right leg, left leg) and each symptom is rated on a scale from 0 (normal) to 4 (severe). The minimum total score possible is 0 and the maximum total score possible is 108. Each subject was independently rated by two observers at each study visit and a mean score was calculated for analysis.

  Baseline to Week 24

• Change in Unified Parkinson's Disease Rating Scale (UPDRS) (Part III) Motor Scores From Baseline to Week 120 Assessed Off Medication.

  The Unified Parkinson's Disease Rating Scale (UPDRS) (Part III) motor scores assess 14 symptoms some of which separately assess symptoms in different body parts (e.g. right arm, left arm, right leg, left leg) and each symptom is rated on a scale from 0 (normal) to 4 (severe). The minimum total score possible is 0 and the maximum total score possible is 108. Each subject was independently rated by two observers at each study visit and a mean score was calculated for analysis.

  Baseline to Week 120

Secondary Outcomes (6)

• Change From Baseline to Week 24 in Total Unified Parkinson's Disease Rating Scale (UPDRS)Score Assessed Off Medication

  Baseline to Week 24

• Change in Total UPDRS Score From Baseline to Week 120 Assessed Off Medication

  Baseline to Week 120

• Change in Unified Parkinson's Disease Rating Scale (UPDRS) (Part III) Motor Score From Baseline to Week 24 Assessed Off Medication.

  Baseline to Week 24

• Change in Unified Parkinson's Disease Rating Scale (UPDRS) (Part III) Motor Score From Baseline to Week 48 Assessed Off Medication.

  Baseline to Week 48

• Change in Unified Parkinson's Disease Rating Scale (UPDRS) (Part III) Motor Score From Baseline to Week 72 Assessed Off Medication.

  Baseline to Week 72

Study Arms (3)

Early-Start Group

ACTIVE COMPARATOR

Subjects were randomized to receive GM1 ganglioside for 24 weeks.

Drug: GM1 ganglioside

Delayed-Start Group

PLACEBO COMPARATOR

Subjects were randomized to receive placebo for 24 weeks.

Drug: Placebo

Comparison Group

NO INTERVENTION

A separate group of Parkinson's disease patients who received standard of care were followed for one to two years to provide comparative information about natural disease progression. This comparison group was not compared statistically to the treatment groups since they were not randomized.

Interventions

GM1 ganglioside DRUG

100 mg twice per day by subcutaneous injection

Also known as: Sygen

Early-Start Group

Placebo DRUG

Twice per day subcutaneous injection, equal volume as active drug

Delayed-Start Group

Eligibility Criteria

• Age: 39 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Thomas Jefferson University: lead
• National Institute of Neurological Disorders and Stroke (NINDS): collaborator

Study Sites (1)

Parkinson's Disease Research Unit, Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States

Location

MeSH Terms

Conditions

Parkinson Disease

Interventions

G(M1) Ganglioside

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Intervention Hierarchy (Ancestors)

Gangliosides; Acidic Glycosphingolipids; Glycosphingolipids; Glycolipids; Glycoconjugates; Carbohydrates; Lipids; Sphingolipids; Membrane Lipids

Results Point of Contact

• Title: Dr. Jay Schneider
• Organization: Thomas Jefferson University

jay.schneider@jefferson.edu

215-503-0370

Study Officials

• Jay S. Schneider, Ph.D.

  Parkinson's Disease Research Unit, Thomas Jefferson University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 22, 2002
• First Posted: May 23, 2002
• Study Start: November 1, 1999
• Primary Completion: June 1, 2010
• Study Completion: June 1, 2010
• Last Updated: December 21, 2012
• Results First Posted: August 9, 2012

Record last verified: 2012-12

Locations

US (1)