233AS102 Extension Study in ALS
Research type
Research Study
Full title
An Extension Study to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB067 Administered to Previously Treated Adults with Amyotrophic Lateral Sclerosis Caused by Superoxide Dismutase 1 Mutation
IRAS ID
231203
Contact name
Pamela Shaw
Contact email
Sponsor organisation
Biogen Idec Research Limited
Eudract number
2016-003225-41
Duration of Study in the UK
2 years, 3 months, 3 days
Research summary
Amyotrophic lateral sclerosis (ALS), is a rapidly progressive, invariably fatal neurological disease that attacks the nerve cells (neurons) responsible for controlling voluntary muscles (muscle action we are able to control, such as those in the arms, legs, and face).
Although the majority of patients suffer from sporadic ALS (without family history), approximately 2%, have an inherited, or familial, form of ALS caused by a variety of genetic mutations known as superoxide dismutase 1 mutations (SOD1-ALS).BIIB067 is an investigational drug which reduces the levels of the toxic protein that causes the genetic mutation. The purpose of this extension study is to evaluate the long-term safety, tolerability and pharmacokinetics (PK) of BIIB067 (how the body processes the drug), in adults with SOD1-ALS who have completed Part B of the 233AS101 first in human study. The study will also characterise the effects of BIIB067 on disease progression by looking at the changes over time in clinical, electrical activity of certain muscles and quality of life assessments in patients. The extension study will allow for the collection of PK and pharmacodynamic (PD) data (how the drug affects the body) during dose interruption and resumption, providing information on the changing behaviour of the central nervous system.
The study is open-label, which means that both the researchers and participants will know which treatment is being administered. All patients will have a 4 week screening period, a 1 month run-in/loading dose period in which Subjects will receive 3 loading doses of BIIB067 by intrathecal bolus injection (via an injection into the spinal canal) approximately 2 weeks apart (Day 1, Day 14, & Day 28) followed by a 10 month dosing period and finally a 3 month follow-up period. The total study duration for each subject will be approximately 16 months. Up to 72 adults will be enrolled in this study (based on the sample size of the SOD1-ALS population in Study 233AS101).
Approximately 17 sites are planned in the United States, Canada, and Western Europe.
This study is sponsored by Biogen Idec Research Limited.REC name
North West - Liverpool Central Research Ethics Committee
REC reference
17/NW/0471
Date of REC Opinion
31 Aug 2017
REC opinion
Favourable Opinion