Biogen announce new clinical trial for SCA3
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. To find out more about how clinical trials work, please click here.
BIIB132 is antisense oligonucleotide (ASO), which means it can block the ability of a certain gene to make a protein. This is important as SCA3 is caused by faulty ATXN3 protein. BIIB132 has been tested in animal models and this will be the first in human study testing this therapy.
In this trial, there will be a 6 -week screening period, and then participants will be given either BIIB132 or a placebo every 4 weeks for 12 weeks. The treatment will be given an intrathecal injection by lumbar puncture so that it can reach the brain. Participants will be split into five different groups, where each group will be given a different 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. They also plan to look to see if there are changes in clinical assessments such as 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. Ataxia UK will also keep our members updated on this via our e-newsletter and social media.
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
