Biogen announces new clinical trial for SCA3 

Biogen - Michaelmantz ataxia blogs2
Marth’s website: and Post Published: April 29, 2022

Biogen have announced that they are starting a Phase I clinical trial for their Spinocerebellar Ataxia Type 3 (SCA3) treatment BIIB132. A Phase I trial tests a treatment in a small group of people to look at its safety and gain information about dosage levels.

BIIB132 is an antisense oligonucleotide (ASO),

Which means it can block the ability of a certain gene to make a protein. This holds significance as faulty ATXN3 protein is the cause of SCA3. BIIB132 has undergone testing in animal models, and this study will mark the first human trial for evaluating this therapy.

In this trial, there will be a 6-week screening period. Subsequently, participants will receive either BIIB132 or a placebo every 4 weeks for a duration of 12 weeks. The administration of the treatment will involve an intrathecal injection through a lumbar puncture, facilitating its targeted delivery to the brain. The participants will divide into five distinct groups, and each group will receive a varying dosage level of BIIB132. After this, there will be a follow-up period of 26 weeks. 

The researchers will look at the safety of the treatment and its pharmacokinetics, which is how the treatment moves into, through, and out of the body. They will collect cerebral spinal fluid to evaluate ATXN3 protein and safety markers. Furthermore, they plan to assess whether any changes are observable in clinical evaluations like the Scale for Assessment and Rating of Ataxia (SARA) and patient-reported outcomes.

To find out more information on the inclusion and exclusion criteria for participants see here.  

There are 21 study sites for this trial, including Oxford and London. This is a small trial, with a total of 48 participants across the 21 sites. The UK sites are not currently recruiting, but when study recruitment begins it will be updated here. Furthermore, Ataxia UK will ensure our members are well-informed by providing updates through our e-newsletter and various social media platforms.

To find out more about the trial you can watch the National Ataxia Foundation’s PrepRARE Webinar with Dr Rosalind Chuang (Biogen) here.  

Date posted: 29/04/2022 

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