Study to assess the Clinical Efficacy and Safety of ISIS 396443 in SMA
Research type
Research Study
Full title
A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy
IRAS ID
170955
Contact name
Volker Straub
Contact email
Sponsor organisation
Isis Pharmaceuticals, Inc.
Eudract number
2014-001947-18
Duration of Study in the UK
2 years, 2 months, 1 days
Research summary
Spinal Muscular Atrophy is a neuromuscular disease resulting in wasting of the voluntary muscles of the limbs and trunk, occurs when SMN protein is missing in the spinal cord. The natural history of SMA includes four major types that are recognized dependent on age of onset and achieved motor abilities. The most severe form, Type I SMA (equivalent to infantile onset SMA), has a disease onset within the first few months of life; these children are never able to sit or walk and usually die from respiratory failure by the age of 2 years. Type II SMA patients are able to sit but never walk unaided, with symptoms presenting between 6-18 months of age. Type III SMA patients are able to sit and walk but individuals with this form may become severely and increasingly disabled. Type IV or adult onset SMA patients have an age of onset over 18 years of age and have normal life expectancies. ISIS is conducting this Clinical Trial in patients with spinal muscular atrophy. In animal studies, the ISIS 396443 drug has shown to increase the amount of SMN protein in the spinal cord, and there were positive effects in their movement and how long they lived. The purpose of this study is to see whether ISIS 396443 (the study drug) has any effects (good or bad) on patients with Later-onset Spinal Muscular. The study will be conducted at multiple centres worldwide and approximately 117 participantswill receive a scaled equivalent dose of ISIS 396443 or a sham procedure control on a 2:1 ratio.The study will consist of screening, treatment and posttreatment follow up periods.The total duration of participation in the study is approximately 15 months or when the study is stopped or patient withdrawsfrom the study.
REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
15/NE/0130
Date of REC Opinion
17 Jul 2015
REC opinion
Further Information Favourable Opinion