Ignite Creation Date:
2025-12-25 @ 1:33 AM
Ignite Modification Date:
2026-03-11 @ 5:02 PM
Study NCT ID:
NCT02876094
Status:
TERMINATED
Last Update Posted:
2020-10-19
First Post:
2016-08-09
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy
Sponsor:
Hugh McMillan
Organization:
Study Overview
Official Title:
A Pilot, Open-Label, Dose Response Study Investigating the Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy (SMA)
Status:
TERMINATED
Status Verified Date:
2020-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Terminated: Study halted prematurely and will not resume; participants are no longer being examined or receiving intervention
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
SMA
Brief 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.
Detailed Description:
This is a pilot, open-label, dose-response study in patients with SMA type II or III. All patients will be treated at each dose of once daily celecoxib (40, 80 and 160 mcg/kg) for a period of two weeks, for a total of 6 weeks (42 days) of treatment.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: