232SM202 - Spinal Muscular Atrophy
Research type
Research Study
Full title
A phase 2, randomized, double-blind, sham-procedure controlled study to assess the safety and tolerability and explore the efficacy of ISIS 396443 (BIIB058) administered intrathecally in subjects with spinal muscular atrophy who are not eligible to participate in the clinical studies ISIS 396443-CS3B or ISIS 396443-CS4
IRAS ID
180876
Contact name
Imelda Hughes
Contact email
Sponsor organisation
Biogne Idec
Eudract number
2014-003657-33
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 9 months, 6 days
Research summary
ISIS 396443 is an investigational product that is being developed by Biogen as a potential treatment for Spinal Muscular Atrophy (SMA). SMA is a genetic disorder that affects the control of muscle movement. The main purpose of this study is to find out if ISIS 396443 has any effects (good or bad) on infants or children with Spinal (SMA).
To learn if ISIS 396443 is safe and effective, this study will include a fake or ‘sham control’ procedure. The participant will be randomly, i.e. by chance, assigned to their procedure group.
Up to 21 subjects will be randomized in a ratio of 2:1 to receive ISIS 396443 or a sham-procedure control. This means that only 2 out of every 3 subjects will receive the study drug and 1 out of every 3 subjects will not. Randomization will be stratified based on age at onset of clinical signs and symptoms consistent with SMA: >6 months versus <6 months.
The study will also be “double blind”, which means that the only people who know if the participant receives the study drug are a small team of people who prepare and give either the study drug or the fake procedure (sham control). Those who do not know if the participant received drug or not are considered ‘blinded’, and this includes the parent/guardian, the Principal Investigator (doctor in charge at the study site) and their staff, the sponsors, and their agents who manage the study.
For those receiving study drug, the drug will be administered “intrathecally”. Intrathecally means that the study drug is injected by lumbar puncture using a thin needle into fluid filled space below the end of the spinal cord through the lower back. For those not receiving study drug, a fake procedure (‘sham procedure’) will be done by making a small needle prick on the skin of the lower back to make it look like the injection was given.Participants will be in the study for about 15 months. The study consists of a screening period, which can take up to 28 days, a 303 day treatment period, and a follow-up evaluation approximately 4 months after the last dose of study drug.
Around 21 subjects in about 8 study centres in 3 countries will take part in this study.
REC name
Yorkshire & The Humber - Leeds East Research Ethics Committee
REC reference
15/YH/0368
Date of REC Opinion
17 Nov 2015
REC opinion
Further Information Favourable Opinion