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drugset / Trial / NCT02227823

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

NCT02227823 ↗

Phase 2 Completed 4 enrolled Centre Hospitalier Régional de la Citadelle
Non-randomizedFactorialOpen-labelTreatment

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).

Timeline

Start
2014-07
Primary completion
2017-07
Completion
2017-07

Drugs

EvaluationDrugModalityDoseRoute
Subject Pyridostigmine Small molecule 1.5 mg/kg —
Subject Pyridostigmine Small molecule 60 mg —

Indications

spinal muscular atrophy, type III