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
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
45
Intramuscular injection of 60 mg of SB-509. Two doses on Day 0 and Day 90.
Coordinated Clinical Research
La Jolla, California, United States
University of California, Irvine; MDA ALS and Neuromuscular Center,
Orange, California, United States
California Pacific Medical Center (CPMC), The Forbes Norris MDA/ALS Research Center
San Francisco, California, United States
The University of Kansas Medical Center (KU)
Kansas City, Kansas, United States
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
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
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Johns Hopkins University
Baltimore, Maryland, United States
Nerve and Muscle Center of Texas
Houston, Texas, United States