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

NCT02876094 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2020-10-19

No results posted yet for this study

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.

Conditions

  • Spinal Muscular Atrophy (SMA)

Interventions

DRUG

celecoxib

dose-response

Sponsors & Collaborators

  • Families of Spinal Muscular Atrophy

    collaborator OTHER

  • Gwendolyn Strong Foundation

    collaborator OTHER

  • Hugh McMillan

    lead OTHER

Principal Investigators

  • Hugh McMillan, MD · Children's Hospital of Eastern Ontario Research Institute

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
80 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-01-29
Primary Completion
2020-08-06
Completion
2020-08-06

Countries

  • Canada

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02876094 on ClinicalTrials.gov