Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy — SMA

Spinal Muscular Atrophy (SMA) Clinical Trial

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

Clinical Trial 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.

Clinical Trial 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 Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT02876094
Study type	Interventional
Source	Children's Hospital of Eastern Ontario
Contact
Status	Terminated
Phase	Phase 2
Start date	January 29, 2019
Completion date	August 6, 2020

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See also
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