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

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

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

Study Overview

• Status: Completed
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Drug: SB-509

Detailed 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.

• Study Type: Interventional
• Enrollment (Actual): 45
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • La Jolla, California, United States, 92037
      • Coordinated Clinical Research
    • Orange, California, United States, 92868
      • University of California, Irvine; MDA ALS and Neuromuscular Center,
    • San Francisco, California, United States, 94115
      • California Pacific Medical Center (CPMC), The Forbes Norris MDA/ALS Research Center
  • Kansas
    • Kansas City, Kansas, United States, 66160
      • The University of Kansas Medical Center (KU)
  • Maryland
    • Baltimore, Maryland, United States, 21287
      • Johns Hopkins University
  • Texas
    • Houston, Texas, United States, 77030
      • Nerve And Muscle Center Of Texas

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 85 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Cohort 1; SB-509 drug administration via IM injection of neck, arms, and legs	Drug: SB-509; Intramuscular injection of 60 mg of SB-509. Two doses on Day 0 and Day 90.
Active Comparator: Cohort 2; SB-509 drug administration via IM injection of legs	Drug: SB-509; Intramuscular injection of 60 mg of SB-509. Two doses on Day 0 and Day 90.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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

Secondary Outcome Measures

Outcome Measure	Time Frame
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

Collaborators and Investigators

• Sponsor: Sangamo Therapeutics
• Investigators: Study Director: Ely Benaim, M.D., Sangamo Therapeutics

Study record dates

Study Major Dates

• Study Start: September 1, 2008
• Primary Completion (Actual): June 1, 2010
• Study Completion (Actual): June 1, 2010

Study Registration Dates

• First Submitted: September 4, 2008
• First Submitted That Met QC Criteria: September 5, 2008
• First Posted (Estimate): September 8, 2008

Study Record Updates

• Last Update Posted (Estimate): November 1, 2012
• Last Update Submitted That Met QC Criteria: October 30, 2012
• Last Verified: October 1, 2012

More Information

Terms related to this study

• Keywords: Lou Gehrig's Disease; Amyotrophic Lateral Sclerosis,; ALS,
• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: SB-509-0801