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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Sangamo Biosciences
ClinicalTrials.gov Identifier:
NCT00748501
First received: September 4, 2008
Last updated: October 30, 2012
Last verified: October 2012

September 4, 2008
October 30, 2012
September 2008
June 2010   (final data collection date for primary outcome measure)
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 ]
Same as current
Complete list of historical versions of study NCT00748501 on ClinicalTrials.gov Archive Site
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 ]
Same as current
Not Provided
Not Provided
 
Clinical Trial of SB-509 in Subjects With Amyotrophic Lateral Sclerosis (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

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.

Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Amyotrophic Lateral Sclerosis
Drug: SB-509
Intramuscular injection of 60 mg of SB-509. Two doses on Day 0 and Day 90.
  • Active Comparator: Cohort 1
    SB-509 drug administration via IM injection of neck, arms, and legs
    Intervention: Drug: SB-509
  • Active Comparator: Cohort 2
    SB-509 drug administration via IM injection of legs
    Intervention: Drug: SB-509
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
45
June 2010
June 2010   (final data collection date for primary outcome measure)

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.
Both
18 Years to 85 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00748501
SB-509-0801
No
Sangamo Biosciences
Sangamo Biosciences
Not Provided
Study Director: Ely Benaim, M.D. Sangamo Biosciences
Sangamo Biosciences
October 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP