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

• Status: Unknown status
• Trial end date: 2017-07-01
• Target enrollment: 0
• Participant gender: All

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).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Centre Hospitalier Régional de la Citadelle
• Treatments: Bromides; Pyridostigmine Bromide

Criteria

Inclusion Criteria:

• Spinal muscular atrophy type 3, genetically confirmed

• Age higher than 6 years old

• Ambulatory patient

• Informed consent signed

• More than 100 meters of walking at 6-minute walk test at screening

• Value at screening and baseline in a range of 20% of the highest value at 6-minute walk test

Exclusion Criteria:

• Patient who had surgical intervention or suffer from a recent traumatism (less than 6 months)

• Associated pathology such as endocrinopathy, infectious disease, allergy, myopathy, chronic or acute inflammatory pathology, during 3 weeks preceding the inclusion.

• Other therapeutics than food supplements or those frequently prescribed in spinal muscular atrophy or its complications

• Non tolerance of electromyography

• Limited collaboration due to trouble in information comprehension

• Pathology inducing contra-indication for pyridostigmine treatment (allergy at molecule, asthma, Parkinson disease, mechanic obstruction of urinary or digestive tracts)