Approximately 2% of people living with ALS have a mutation in the superoxide dismutase 1 (SOD1) gene. The mutation of the SOD1 gene leads to the production of an abnormal SOD1 protein that is likely to be toxic to cells and could possibly lead to the nerve cell death seen in people with ALS. The VALOR study is evaluating a potential new treatment for people living with ALS caused by a SOD1 gene mutation (SOD1-ALS). Tofersen (BIIB067) is an antisense oligonucleotide (ASO), which is designed to reduce levels of SOD1 protein in people with SOD1-ALS.
You will be assigned to a study drug group at random (by chance) to receive either the investigational drug or placebo. A placebo is a substance that looks like the investigational drug but contains no actual active drug. They help us to make sure that any changes seen during the study are due to the investigational drug alone and not another reason. Study participants will have a 2-in-3 chance of being assigned to the investigational drug group, and a 1-in-3 chance of being assigned to the placebo group. Neither the participant nor the study team will be told which group they have been placed into until after the study has finished.
The investigational drug or placebo is delivered intrathecally. Intrathecally means that the study drug is given to you by a procedure called a lumbar puncture. All participants will receive 8 lumbar punctions throughout the study duration. The study duration is 32-36 weeks, including:
- 4-week screening
- 24-week study treatment period
- 4 to 8-week follow-up period
Key eligibility criteria
To be able to enrol in the VALOR study, participants need to be:
- 18 years of age or older
- Have weakness caused by ALS
- Agree to genetic testing for the SOD1 mutation
If you are eligible and choose to participate, you will receive the following 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 be available.