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

NCT ID: NCT00748501

Last Updated: 2012-11-01

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

45 participants

Study Classification

INTERVENTIONAL

Study Start Date

2008-09-30

Study Completion Date

2010-06-30

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.

Conditions

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Amyotrophic Lateral Sclerosis

Keywords

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Amyotrophic Lateral Sclerosis, ALS, Lou Gehrig's Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1

SB-509 drug administration via IM injection of neck, arms, and legs

Group Type ACTIVE_COMPARATOR

SB-509

Intervention Type DRUG

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

Cohort 2

SB-509 drug administration via IM injection of legs

Group Type ACTIVE_COMPARATOR

SB-509

Intervention Type DRUG

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

Interventions

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SB-509

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

Intervention Type DRUG

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.
Minimum Eligible Age

18 Years

Maximum Eligible Age

85 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Sangamo Therapeutics

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Ely Benaim, M.D.

Role: STUDY_DIRECTOR

Sangamo Therapeutics

Locations

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Coordinated Clinical Research

La Jolla, California, United States

Site Status

University of California, Irvine; MDA ALS and Neuromuscular Center,

Orange, California, United States

Site Status

California Pacific Medical Center (CPMC), The Forbes Norris MDA/ALS Research Center

San Francisco, California, United States

Site Status

The University of Kansas Medical Center (KU)

Kansas City, Kansas, United States

Site Status

Johns Hopkins University

Baltimore, Maryland, United States

Site Status

Nerve and Muscle Center of Texas

Houston, Texas, United States

Site Status

Countries

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United States

Other Identifiers

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SB-509-0801

Identifier Type: -

Identifier Source: org_study_id