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
Clinicaltrials.gov Identifier
Duration of Study in the UK
3 years, 10 months, 10 days
Research summary
The purpose of this study is to see whether ISIS 396443 (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 an ISIS 396443 research study.
ISIS 396443 is not approved for marketing or use. It is approved for clinical research, and thus its safety or efficacy has not been determined yet.
The symptoms of Spinal Muscular Atrophy (SMA) occur when Survival motor neuron (SMN) protein is missing in the nerves of the spinal cord and the brain. In animal studies, the drug ISIS 396443 was shown to increase the amount of Survival motor neuron (SMN) protein in the spinal cord and brain, and there were positive effects in the animals’ movements and how long they lived.
ISIS 396443 has been studied in animals and it has been tested in 10 clinical studies that are either completed or are currently still ongoing. As of October 2015 it has been given to 190 patients with SMA as single and multiple doses. At this time, the possible effects of the drug, good and bad are unknown.
This study will be performed at research sites around the whole world. Up to 274 persons around the world may take part in this study.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
16/LO/0987
Date of REC Opinion
27 May 2016
REC opinion
Unfavourable Opinion