Trials / Terminated
TerminatedNCT02876094
Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy
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
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 1 (actual)
- Sponsor
- Hugh McMillan · Academic / Other
- Sex
- All
- Age
- 2 Years – 80 Years
- Healthy volunteers
- Not accepted
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | celecoxib | dose-response |
Timeline
- Start date
- 2019-01-29
- Primary completion
- 2020-08-06
- Completion
- 2020-08-06
- First posted
- 2016-08-23
- Last updated
- 2020-10-19
Locations
1 site across 1 country: Canada
Source: ClinicalTrials.gov record NCT02876094. Inclusion in this directory is not an endorsement.