Extension Study for SMA patients who participated in ISIS396443 study
Research type
Research Study
Full title
An Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443
IRAS ID
192217
Contact name
Francesco Muntoni
Contact email
Sponsor organisation
Ionis Pharmaceuticals, Inc.
Eudract number
2015-001870-16
ISRCTN Number
ISRCTN00000000
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
000, 000
Duration of Study in the UK
3 years, 5 months, 15 days
Research summary
The purpose of this study is to see whether Nusinersen (the study drug) has any effects (good or bad), when it is given over a longer period of time to children with Spinal Muscular Atrophy (SMA) who previously completed a research study testing Nusinersen (parent study).
It also provides an option for these children to continue receiving Nusinersen after they completed the parent study (or to start using Nusinersen in case they received a sham procedure in the parent study).
Spinal muscular atrophy is a disease that causes severe muscle weakness in infants and children. No drug has been approved for its treatment. The infantile form of the disease is the most severe and even with maximal supportive treatment many infants with the disease die before the age of 2.
Spinal muscular atrophy is caused by a lack of a protein called “survival motor neuron”. Nusinersen has the potential to increase the amount of this protein, i.e. to address the root cause of the disease.
Results of smaller studies have been encouraging, but not conclusive. Larger studies with control groups will determine if Nusinersen benefits infants and children with spinal muscular atrophy. Should Nusinersen be approved, it would have to be given to children and infants with spinal muscular atrophy for long time periods, potentially even lifelong. Therefore, it is important to understand the effects of Nusinersen (good and bad) when it is given for a long time period. The current study addresses this question.
REC name
East of England - Cambridge Central Research Ethics Committee
REC reference
16/EE/0367
Date of REC Opinion
4 Oct 2016
REC opinion
Further Information Favourable Opinion