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

Amyotrophic Lateral Sclerosis Clinical Trial

— ALS  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00748501
• Other study ID #: SB-509-0801
• Status: Completed
• Phase: Phase 2
• First received: September 4, 2008
• Last updated: October 30, 2012
• Start date: September 2008
• Est. completion date: June 2010

Study information

• Verified date: October 2012
• Source: Sangamo Biosciences
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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

Description:

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 45
• Est. completion date: June 2010
• Est. primary completion date: June 2010
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 85 Years

Eligibility

Inclusion Criteria:

• Male or female between the ages of 18 and 85 with clinical diagnosis of ALS

• Forced Vital Capacity (FVC) > 60% of predicted

• Less than 3 years of ALS since the onset of the first symptom with clinical evidence of limb muscle atrophy and weakness.

• Subjects taking Riluzole must have been at a stable dose for at least 30 days with no evidence of toxicity

• Female of childbearing potential and male of child-creating potential must agree to use a medically acceptable physical barrier (condom, diaphragm, and cervical cap) through the treatment phase and for at least 30 days after the last study treatment.

Exclusion Criteria:

• Women who are pregnant or currently breast-feeding

• Dependent upon invasive or non-invasive artificial ventilation

• Patients with bulbar onset ALS or with other active neuromuscular/ neurodegenerative diseases.

• Type 1 or Type 2 diabetes.

• Evidence of chronic or active heart, liver, kidney, or lung diseases, or Age-related macular degeneration.

• Current or history of known immune or immunodeficiency disorders

• Patients with cognitive impairment with significant decision making incapacity, or major depression, or schizophrenia, or dementia (e.g. Alzheimer's disease).

• Malignancy or history of malignancy, except it has been in complete remission for at least 5 years

• Pre-cancerous conditions (e.g. Barrett's Esophagus, dysplasias) or benign tumors which have the potential for significant growth due to VEGF stimulation.

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• SB-509
Intramuscular injection of 60 mg of SB-509. Two doses on Day 0 and Day 90.

Locations

Country	Name	City	State
United States	Johns Hopkins University	Baltimore	Maryland
United States	Nerve and Muscle Center of Texas	Houston	Texas
United States	The University of Kansas Medical Center (KU)	Kansas City	Kansas
United States	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

Sponsors (1)

Lead Sponsor	Collaborator
Sangamo Biosciences

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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).		11 months	No
Secondary	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.		11 months	Yes