Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3 (EMOTAS)
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|ClinicalTrials.gov Identifier: NCT02227823|
Recruitment Status : Unknown
Verified August 2014 by Dr. Stéphanie Delstanche, Centre Hospitalier Régional de la Citadelle.
Recruitment status was: Recruiting
First Posted : August 28, 2014
Last Update Posted : August 28, 2014
|Condition or disease||Intervention/treatment||Phase|
|Spinal Muscular Atrophy Type 3||Drug: Pyridostigmine Bromide||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Factorial Assignment|
|Masking:||None (Open Label)|
|Official Title:||Safety and Efficacy Study of Anti-cholinesterase Therapy on the Motor Functions in Patients With Spinal Muscular Atrophy Type 3.|
|Study Start Date :||July 2014|
|Estimated Primary Completion Date :||July 2017|
|Estimated Study Completion Date :||July 2017|
Experimental: significant decrement
Patients with significant decrement at electromyogram will be treated by pyridostigmine bromide 60mg 3 times a day for patients older than 18 and 1.5mg/kg 3 times a day for children less than 40kg
Drug: Pyridostigmine Bromide
Other Name: Mestinon
No Intervention: no decrement
Patient without significant decrement will not receive any treatment and will be the control group
- Change from Baseline in the distance walked at 6-minute walk test at 6 months [ Time Frame: 6 months ]
- Change from baseline of decrement at 6 months [ Time Frame: 6 months ]
- Change from baseline of MFM-D1 [ Time Frame: 6 months ]Comparison of treated and control group values will be made
- Change from baseline of Moviplate values at 6 months [ Time Frame: 6 months ]Comparison between treated and control group value will be made
- Change from baseline of the ratio at 6 minutes walk test at 6 months [ Time Frame: 6 months ]It's the ratio between the number of meters during the last minute of the 6-minute walk test and the first minute of the 6-minute walk test.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02227823
|Centre de référence des maladies neuromusculaire, Centre Hospitalier Régional de la Citadelle||Recruiting|
|Liège, Belgium, 4000|
|Contact: Stephanie Delstanche firstname.lastname@example.org|
|Contact: Severine Denis email@example.com|
|Principal Investigator: Stephanie Delstanche|
|Principal Investigator:||Stephanie Delstanche||Centre de référence des maladies neuromusculaire de Liège|