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

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ClinicalTrials.gov Identifier: NCT00748501
Recruitment Status : Completed
First Posted : September 8, 2008
Last Update Posted : November 1, 2012
Sponsor:
Information provided by (Responsible Party):
Sangamo Therapeutics

Study Description
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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

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: SB-509 Phase 2

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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 45 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2 Repeat-Dosing Clinical Trial of SB-509 in Subjects With Amyotrophic Lateral Sclerosis
Study Start Date : September 2008
Actual Primary Completion Date : June 2010
Actual Study Completion Date : June 2010

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arms and Interventions
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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.


Outcome Measures
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Primary Outcome Measures :
  1. 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 ]

Secondary Outcome Measures :
  1. 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 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 85 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
Contacts and Locations
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00748501


Locations
United States, California
Coordinated Clinical Research
La Jolla, California, United States, 92037
University of California, Irvine; MDA ALS and Neuromuscular Center,
Orange, California, United States, 92868
California Pacific Medical Center (CPMC), The Forbes Norris MDA/ALS Research Center
San Francisco, California, United States, 94115
United States, Kansas
The University of Kansas Medical Center (KU)
Kansas City, Kansas, United States, 66160
United States, Maryland
Johns Hopkins University
Baltimore, Maryland, United States, 21287
United States, Texas
Nerve and Muscle Center of Texas
Houston, Texas, United States, 77030
Sponsors and Collaborators
Sangamo Therapeutics
Investigators
Study Director: Ely Benaim, M.D. Sangamo Therapeutics
More Information
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Responsible Party: Sangamo Therapeutics
ClinicalTrials.gov Identifier: NCT00748501     History of Changes
Other Study ID Numbers: SB-509-0801
First Posted: September 8, 2008    Key Record Dates
Last Update Posted: November 1, 2012
Last Verified: October 2012

Keywords provided by Sangamo Therapeutics:
Amyotrophic Lateral Sclerosis,
ALS,
Lou Gehrig's Disease

Additional relevant MeSH terms:
Sclerosis
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
 Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases