« Return to News | Clinical Intelligence

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

May 20

IRB consolidation trend continues, as private equity firm Audax buys majority stake in Chesapeake

Quintiles, Merck Serono ink exclusive partnership

Already a subscriber?
Log in to your digital subscription.

Subscribe to CWWeekly.

The CenterWatch Monthly

May

INC Research, PPD, inVentiv named top CROs
Yet most CROs slipping in ability to manage site relationships

R&D cuts by sponsors spur more collaboration, innovation
New strategies share pipeline activity costs

Already a subscriber?
Log in to your digital subscription.

Purchase the May issue.

Subscribe to
The CenterWatch Monthly.

The CenterWatch Monthly

April

20 innovators changing the face of the clinical trials industry
The CenterWatch Monthly honors individuals, teams and companies for their forward-thinking ideas and their ability to implement them.

Already a subscriber?
Log in to your digital subscription.

Purchase the April issue.

Subscribe to
The CenterWatch Monthly.

JobWatch centerwatch.com/jobwatch

Featured Jobs

More Jobs

 

Copyright © 1995-2013 CenterWatch.