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

Primary Purpose

Amyotrophic Lateral Sclerosis

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
SB-509
Sponsored by
Sangamo Therapeutics
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis focused on measuring Amyotrophic Lateral Sclerosis,, ALS,, Lou Gehrig's Disease

Eligibility Criteria

18 Years - 85 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Coordinated Clinical Research
  • University of California, Irvine; MDA ALS and Neuromuscular Center,
  • California Pacific Medical Center (CPMC), The Forbes Norris MDA/ALS Research Center
  • The University of Kansas Medical Center (KU)
  • Johns Hopkins University
  • Nerve and Muscle Center of Texas

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Active Comparator

Arm Label

Cohort 1

Cohort 2

Arm Description

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

SB-509 drug administration via IM injection of legs

Outcomes

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).

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.

Full Information

First Posted
September 4, 2008
Last Updated
October 30, 2012
Sponsor
Sangamo Therapeutics
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1. Study Identification

Unique Protocol Identification Number
NCT00748501
Brief Title
Clinical Trial of SB-509 in Subjects With Amyotrophic Lateral Sclerosis (ALS)
Acronym
ALS
Official Title
A Phase 2 Repeat-Dosing Clinical Trial of SB-509 in Subjects With Amyotrophic Lateral Sclerosis
Study Type
Interventional

2. Study Status

Record Verification Date
October 2012
Overall Recruitment Status
Completed
Study Start Date
September 2008 (undefined)
Primary Completion Date
June 2010 (Actual)
Study Completion Date
June 2010 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Sangamo Therapeutics

4. Oversight

Data Monitoring Committee
No

5. Study Description

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
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis
Keywords
Amyotrophic Lateral Sclerosis,, ALS,, Lou Gehrig's Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
45 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Cohort 1
Arm Type
Active Comparator
Arm Description
SB-509 drug administration via IM injection of neck, arms, and legs
Arm Title
Cohort 2
Arm Type
Active Comparator
Arm Description
SB-509 drug administration via IM injection of legs
Intervention Type
Drug
Intervention Name(s)
SB-509
Intervention Description
Intramuscular injection of 60 mg of SB-509. Two doses on Day 0 and Day 90.
Primary Outcome Measure Information:
Title
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 Measure Information:
Title
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
85 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ely Benaim, M.D.
Organizational Affiliation
Sangamo Therapeutics
Official's Role
Study Director
Facility Information:
Facility Name
Coordinated Clinical Research
City
La Jolla
State/Province
California
ZIP/Postal Code
92037
Country
United States
Facility Name
University of California, Irvine; MDA ALS and Neuromuscular Center,
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
California Pacific Medical Center (CPMC), The Forbes Norris MDA/ALS Research Center
City
San Francisco
State/Province
California
ZIP/Postal Code
94115
Country
United States
Facility Name
The University of Kansas Medical Center (KU)
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Johns Hopkins University
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Nerve and Muscle Center of Texas
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States

12. IPD Sharing Statement

Learn more about this trial

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

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