Isis initates phase I spinal muscular atrophy study

Monday, December 19, 2011 11:34 AM

Isis Pharmaceuticals has initiated a phase I study of ISIS-SMNRx in patients with spinal muscular atrophy (SMA).  SMA is a severe motor-neuron disease that is the leading genetic cause of infant mortality. Isis is developing ISIS-SMNRx as a potential treatment for all types of SMA.

The phase I study of ISIS-SMNRx is a single-dose, dose-escalation study designed to assess the safety, tolerability and pharmacokinetic profile of the drug in children with SMA between the ages of 2-14 who are medically stable.  In this study, ISIS-SMNRx will be administered intrathecally as a single injection directly into the spinal fluid. Intrathecal administration of an antisense drug, ISIS-SOD1Rx, has been shown to be safe and well tolerated in an ongoing phase I study in patients with amyotrophic lateral sclerosis.

Share:          
CLINICAL TRIAL RESOURCES

Search:

NEWS ONLINE ARCHIVE

Browse by:

CWWeekly

July 28

WCG acquires Alliance Biosciences to expand its biosafety oversight capabilities, ensure compliance

Facilitated review meetings for protocol design catching on, decreasing amendments, complexity

Already a subscriber?
Log in to your digital subscription.

Subscribe to CWWeekly.

The CenterWatch Monthly

July

IRB market consolidating rapidly
Private equity driving a new commercial ethical review landscape

Lessons learned from medical device trials
Smaller, faster, more flexible trials hold important insights for pharmaceutical innovation

Already a subscriber?
Log in to your digital subscription.

Purchase the July issue.

Subscribe to
The CenterWatch Monthly.

The CenterWatch Monthly

June

CRO market poised for growth and consolidation
Sponsors expanding pipelines, shrinking infrastructure fuel strategic outsourcing

Strategies for success evolving in phase I space
Sites see increasing complexity, more patients, greater focus on efficacy

Already a subscriber?
Log in to your digital subscription.

Purchase the June issue.

Subscribe to
The CenterWatch Monthly.

JobWatch centerwatch.com/jobwatch

Featured Jobs