Status:
COMPLETED
Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3
Lead Sponsor:
Centre Hospitalier Régional de la Citadelle
Conditions:
Spinal Muscular Atrophy Type 3
Eligibility:
All Genders
6+ years
Phase:
PHASE2
Brief 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, ...
Eligibility Criteria
Inclusion
- 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
- 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)
Key Trial Info
Start Date :
July 1 2014
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 1 2017
Estimated Enrollment :
4 Patients enrolled
Trial Details
Trial ID
NCT02227823
Start Date
July 1 2014
End Date
July 1 2017
Last Update
October 11 2023
Active Locations (1)
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1
Centre de référence des maladies neuromusculaire, Centre Hospitalier Régional de la Citadelle
Liège, Belgium, 4000