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

Study Overview
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Brief Summary
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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 Description
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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.

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

Conditions
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Amyotrophic Lateral Sclerosis

Intervention / Treatment
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* Drug: SB-509


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 85 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

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


Arms and Interventions

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

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | 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 | Measure Description | 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Amyotrophic Lateral Sclerosis,, ALS,, Lou Gehrig's Disease