NCT02227823

Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3

Safety and Efficacy Study of Anti-cholinesterase Therapy on the Motor Functions in Patients With Spinal Muscular Atrophy Type 3.

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: Centre Hospitalier Régional de la Citadelle · Academic / Other
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate safety and efficacy of anti-cholinesterase therapy on the motor function in SMA type 3 patients with impaired neuromuscular junction (NMJ).

Detailed description

Spinal muscular atrophy (SMA) is the second neuromuscular disease meet in children. SMA is a genetically transmitted disease inducing muscular weakness predominating on shoulders and hips. Currently, there is no effective therapy to slow the progression of the disease. SMA is due to a neuron motor attempt of the spinal cord and recently it has been demonstrated a neuromuscular junction (NMJ) involvement, according to recent studies. EMOTAS study aim to understand if NMJ abnormalities could have an impact on motor performance and fatigue in SMA type 3 ambulatory patients by electromyogram and to improve by non-invasive therapy quality of life of patients.

Conditions

• Spinal Muscular Atrophy Type 3

Interventions

Type	Name	Description
DRUG	Pyridostigmine Bromide

Timeline

• Start date: 2014-07-01
• Primary completion: 2017-07-01
• Completion: 2017-07-01
• First posted: 2014-08-28
• Last updated: 2023-10-11

Locations

1 site across 1 country: Belgium