drugset / Trial / NCT02876094
Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy
Phase 2
Terminated
1 enrolled
Hugh McMillan
Families of Spinal Muscular Atrophy · collabGwendolyn Strong Foundation · collab
NaSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2019-01-29
- Primary completion
- 2020-08-06
- Completion
- 2020-08-06
Outcome
Outcome not reported
Stopped: “Terminated: Study halted prematurely and will not resume; participants are no longer being examined or receiving intervention”
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Celecoxib | Other / unclassified | 40 ug/kg | Oral |
| Subject | Celecoxib | Other / unclassified | 80 ug/kg | Oral |
| Subject | Celecoxib | Other / unclassified | 160 ug/kg | Oral |