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

Purpose

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	Intervention	Phase
Amyotrophic Lateral Sclerosis	Drug: SB-509	Phase 2


Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	A Phase 2 Repeat-Dosing Clinical Trial of SB-509 in Subjects With Amyotrophic Lateral Sclerosis

Resource links provided by NLM:

Genetics Home Reference related topics: amyotrophic lateral sclerosis

MedlinePlus related topics: Amyotrophic Lateral Sclerosis

Genetic and Rare Diseases Information Center resources: Amyotrophic Lateral Sclerosis

U.S. FDA Resources

Further study details as provided by Sangamo Biosciences:

Primary Outcome Measures:

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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

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 ] [ Designated as safety issue: Yes ]


Enrollment:	45
Study Start Date:	September 2008
Study Completion Date:	June 2010
Primary Completion Date:	June 2010 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
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.

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.

Eligibility


Ages Eligible for Study:	18 Years to 85 Years (Adult, Senior)
Genders Eligible for Study:	Both
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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: 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 Biosciences

Investigators


Study Director:	Ely Benaim, M.D.	Sangamo Biosciences

More Information


Responsible Party:	Sangamo Biosciences
ClinicalTrials.gov Identifier:	NCT00748501 History of Changes
Other Study ID Numbers:	SB-509-0801
Study First Received:	September 4, 2008
Last Updated:	October 30, 2012
Health Authority:	United States: Food and Drug Administration

Keywords provided by Sangamo Biosciences:


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

ClinicalTrials.gov processed this record on September 28, 2016

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