Conditions: Parkinson Disease
Keywords: Parkinson's disease PD GM1 Ganglioside Sygen
Allocation: RANDOMIZED | Intervention model: CROSSOVER
Enrollment: 94
Healthy volunteers: no

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.

Detailed description:
The study is designed to further examine the extent to which GM1 ganglioside can improve symptoms, delay disease progression, and, perhaps, partially restore damaged brain cells in PD patients. GM1 ganglioside is a chemical that is normally found in the brain and is a normal part of the outer covering or membrane of nerve cells. This study will compare the effectiveness of GM1 to standard PD treatment. In addition to studying clinical measures of motor and cognitive functioning, the investigators will use PET (positron emission tomography) scanning to image the brain and the dopamine nerve endings in a subgroup of patients. Patients with mild to moderate idiopathic PD will be divided into 2 groups. One group will receive GM1 for 24 weeks and the other will receive placebo. At the end of this 24 week period, all patients will enter into a 96 week treatment period in which all patients will receive GM1.

In parallel, a group of standard-of-care patients with mild to moderate PD will be monitored over a 1 to 2 year period to assess the natural progression of PD. These patients will receive the same clinical evaluations as the treatment group subjects but they will not receive the experimental medication.

Arms:
arm 1: Subjects were randomized to receive GM1 ganglioside for 24 weeks. arm 2: Subjects were randomized to receive placebo for 24 weeks. arm 3: 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: intervention 1: GM1 ganglioside intervention 2: Placebo
Intervention descriptions: intervention 1: 100 mg twice per day by subcutaneous injection intervention 2: Twice per day subcutaneous injection, equal volume as active drug

Protocol document: