Research and Clinical Trials

A Phase 1, Double-Blind, Placebo-Controlled, Dose-Escalation Study of the Safety, Tolerability, and Pharmacokinetics of ISIS 333611 Administered Intrathecally to Patients with Familial Amyotrophic Lateral Sclerosis Due to Superoxide Dismutase 1 Gene
Brief Description  
Carolinas Neuroscience and Spine Institute is conducting a study on patients with Familial Amyotrophic Lateral Sclerosis (FALS) , also known as Lou Gehrig’s disease, carrying the SOD1 gene using a pharmaceutical company called ISIS. This is a phase 1 study and will require admission into Carolinas Medical Center for 2-3 days. The study involves a 12 hour infusion of ISIS 333611 through a line placed in the back. Preliminary studies suggest that 333611 stops the SOD1 gene from duplicating and the
Who may be Eligible  
Patients diagnosed with Familial Amyotrophic Lateral Sclerosis (FALS) with the SOD1 gene mutation. Patients must be at least 18 years of age. Patients may not have taken other study drugs in the 30 days before they are screened for the study. Patients will not be eligible if they have a condition which may impact intrathecal infusion, such as history of spinal disease (see coordinator for more details), or if the study doctor decides they have a medical condition that would make it unsafe for them to take part in the study.
ALS - Amyotrophic Lateral Sclerosis
IRB Number  
Principal Investigator  
Brooks, Benjamin

For More Information, Contact  Jaime  , Shue
Phone:  (704) 446-6253  Fax:  (704) 446-1915  
Address:1010 Edgehill Road North Charlotte, NC 28207