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

1376

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).

2014-07-01

2017-07-01

2017-07-01

Completed

Early phase I

4

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)

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'FACTORIAL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 4, 'type': 'ACTUAL'}}

2023-10-11