Spinocerebellar Ataxia Saffron Study

About the Saffron Spinocerebellar Ataxia 2 (SC2) Study:

Current therapies for SCA2 are supportive and aimed at helping ease symptoms. However, there are no available treatments that delay, slow, or reverse the progress of the condition. The Saffron Study will look at a potential new medication (study drug) in adults with SCA2. This study drug may lower the amount of a protein called ATXN2 in the body, which may stop or slow SCA2 disease progression.

The study will look at:

• How safe the study drug is
• What side effects it may cause
• What is the most suitable dose for further research
• The study drug will be compared with a placebo, which looks like the study drug but contains no actual medicine.

What will this study involve?

• Participants will receive either the study drug at 1 of 4 dose levels or a placebo. Participants are 2 times more likely to receive the study drug than the placebo.
• Participants will receive the study medication (study drug or placebo) once, as an injection in the fluid around the spine.
• Participants will have 5 lumbar punctures (a procedure to collect spinal fluid) throughout the study.
• An overnight stay at the study centre for 1 night.
• 12 study centre 'visits' for study assessments.

The study is made up of 3 parts lasting 10 months:

1) Screening period (up to 2 months)

• You will have at least 1 study centre visit to see if the study is suitable for you and whether you want to take part.

2) Study treatment period (about 24 hours, overnight)

• You will receive the study medication and have study assessments.
• This will include 2 of the 12 study centre visits – 1 'visit' before and 1 'visit' after the study medication dose.

3) Follow-up period (about 8 months)

• You will have 9 study centre visits for study assessments.
• At the end of 8 months, follow-up may be extended with further study centre visits every 2 month