Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy
Status:
Terminated
Trial end date:
2020-08-06
Target enrollment:
Participant gender:
Summary
Several factors make the use of celecoxib in human SMA patients appealing including: 1)
low-dosing required for potential therapeutic effect (the corresponding dose in humans is
much lower than that commonly used in adults and children with; 2) favourable side effect
profile of this drug (particularly at the dosing required); 3) the fact that celecoxib
crosses the blood brain barrier and 4) demonstration of efficacy in a genetically and
pathophysiologically faithful animal mode. The investigators therefore believe that celecoxib
is a promising disease modifying therapy for SMA.
Phase:
Phase 2
Details
Lead Sponsor:
Hugh McMillan
Collaborators:
Families of SMA Canada Families of Spinal Muscular Atrophy Gwendolyn Strong Gwendolyn Strong Foundation