Amyotrophic Lateral SclerosisPhase 2ALSNCT00748501
Clinical Trial of SB-509 in Subjects With Amyotrophic Lateral Sclerosis (ALS)
A Phase 2 study testing SB-509 in people with Amyotrophic Lateral Sclerosis, run by Sangamo Therapeutics at 6 sites in United States.
Trial 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 Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- SB-509
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 45 participants
- Where
- 6 sitesUnited States
- Study dates
- Sep 2008to Jun 2010
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
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- 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.
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Johns Hopkins University
Baltimore, Maryland, 21287
Nerve and Muscle Center of Texas
Houston, Texas, 77030
The University of Kansas Medical Center (KU)
Kansas City, Kansas, 66160
Coordinated Clinical Research
La Jolla, California, 92037
University of California, Irvine; MDA ALS and Neuromuscular Center,
Orange, California, 92868
California Pacific Medical Center (CPMC), The Forbes Norris MDA/ALS Research Center
San Francisco, California, 94115
About this study
Official title: 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
Read the 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.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- SB-509
- Keywords
- Amyotrophic Lateral Sclerosis,, ALS,, Lou Gehrig's Disease
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SB-509Drug
Intramuscular injection of 60 mg of SB-509. Two doses on Day 0 and Day 90.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Active comparator | SB-509 drug administration via IM injection of neck, arms, and legs Receives: Drug: SB-509 |
| Cohort 2 | Active comparator | SB-509 drug administration via IM injection of legs Receives: Drug: SB-509 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 11 months
Secondary outcomes (1)
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.
Measured: 11 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Sep 2008 (estimated)
- Study startSep 2008
- Primary completionJun 2010
- Study completionJun 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 45 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sangamo Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ely Benaim, M.D., Study director, Sangamo Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00748501
- Sponsor's protocol ID
- SB-509-0801
- Acronym
- ALS
- Status last verified
- Oct 2012
- First submitted
- 4 Sep 2008
- Last updated
- 1 Nov 2012
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Nov 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Repeat-Dosing Clinical Trial of SB-509 in Subjects With Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT00748501
- Acronym / sponsor ID
- ALS / SB-509-0801
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- Amyotrophic Lateral Sclerosis,, ALS,, Lou Gehrig's Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 45 participants (actual)
- Lead sponsor
- Sangamo TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Sep 2008
- Primary completion
- Jun 2010 (actual)
- Study completion
- Jun 2010 (actual)
- First posted
- 8 Sep 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Active comparator | Drug: SB-509SB-509 drug administration via IM injection of neck, arms, and legs |
| Cohort 2 | Active comparator | Drug: SB-509SB-509 drug administration via IM injection of legs |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SB-509Intramuscular injection of 60 mg of SB-509. Two doses on Day 0 and Day 90. | Drug | Cohort 1, Cohort 2 |
Primary outcome
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
1 secondary outcome
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Nov 2012.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ely Benaim, M.D. study director
Sangamo Therapeutics
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing SB-509 in people with Amyotrophic Lateral Sclerosis, run by Sangamo Therapeutics at 6 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Nov 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the 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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Nov 2012.