Clinical Trial: Clinical Trial of SB-509 in Subjects With Amyotrophic Lateral Sclerosis (ALS)

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Phase 2 Repeat-Dosing Clinical Trial of SB-509 in Subjects With Amyotrophic Lateral Sclerosis

Brief Summary: The purpose of the study is to evaluate the effects of the investigational drug, SB-509 on progression of the disease in subjects with ALS

Detailed Summary: SB-509 contains the gene (DNA—a kind of biological "blueprint") for a protein. When a study doctor injects SB-509 into the muscles of your neck, arms and/or legs, the drug enters the muscle and nerve cells around the injection sites and causes these cells to make a protein. This protein causes your cells to increase production of one of your own protein called vascular endothelial growth factor(VEGF-A), which may improve the structure and function of nerves and muscles. In addition, there are changes in the levels of 28 additional proteins in your cells. These proteins function to promote the growth of cells, are structures in cells, help synthesize products, and affect immune cells, and some have unknown functions. This increase in your own VEGF proteins may protect and repair the damaged nerves and muscles caused by ALS.
Sponsor: Sangamo Therapeutics

Current Primary Outcome: To evaluate the effect of SB-509 on progression of the disease in subjects with ALS, as measured by the ALS Functional Rating Scale -Revised (ALSFRS-R). [ Time Frame: 11 months ]

Original Primary Outcome: Same as current

Current Secondary Outcome: To evaluate a) the effect of SB-509 on Forced Vital Capacity, Neurophysiologic Index, Manual Muscle Test, and survival. b) safety and tolerability of SB-509 in ALS. c) stem cell mobilization in subjects with ALS receiving SB-509. [ Time Frame: 11 months ]

Original Secondary Outcome: Same as current

Information By: Sangamo Therapeutics

Dates:
Date Received: September 4, 2008
Date Started: September 2008
Date Completion:
Last Updated: October 30, 2012
Last Verified: October 2012