Summary:
The VALOR Study – A Clinical Trial for People Living with SOD1-ALS
Biogen is conducting a clinical trial to evaluate the safety and potential efficacy of an investigational drug for people living with Amyotrophic Lateral Sclerosis (ALS) caused by a SOD1 gene mutation.
STUDY OVERVIEW
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Motor neurons reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. The progressive degeneration – or loss of function – of the motor neurons in ALS eventually leads to their death. When the motor neurons die, the ability of the brain to start and control muscle movement is lost.
Sporadic ALS:
This is the most common type of ALS, and it affects approximately 90 to 95 percent of all people living with ALS. It is called “sporadic” because it can affect anyone, and there is no clear cause.
SOD-1 ALS:
This type of the disease is usually genetic, and it is inherited from a family member. SOD1-ALS affects approximately 2 percent of all people living with ALS. It is caused by a mutation, or change, to a gene called superoxide dismutase 1, or SOD1.
The VALOR Study is evaluating a potential new treatment for people living with ALS caused by a SOD1 gene mutation.
Study Duration: 32 weeks, including:
- 4-week screening
- 24-week study treatment period
- 4-week follow-up period
INVESTIGATIONAL DRUG
- BIIB067, an antisense oligonucleotide (ASO)
- Designed to reduce the amount of SOD1 protein in the body
- Given intrathecally, through a lumbar puncture
KEY ELIGIBILITY CRITERIA
- 18 years of age or older
- Have weakness caused by ALS
- Agree to genetic testing for the SOD1 mutation
ALL ELIGIBLE PARTICIPANTS WILL RECEIVE AT NO COST
- Comprehensive study-related health evaluations and assessments, including genetic testing
- Investigational drug or placebo
- All study-related visits and care
Assistance with travel and accommodations, and reimbursement for study-related expenses may also be available.