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

Spinal Muscular Atrophy Type 3 Clinical Trial

— EMOTAS  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02227823
• Other study ID #: 1376
• Status: Completed
• Phase: Phase 2
• Start date: July 2014
• Est. completion date: July 2017

Study information

• Verified date: October 2023
• Source: Centre Hospitalier Régional de la Citadelle
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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, 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 4
• Est. completion date: July 2017
• Est. primary completion date: July 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years and older

Eligibility

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)

Related Conditions & MeSH terms

• Atrophy
• Muscular Atrophy
• Muscular Atrophy, Spinal
• Spinal Muscular Atrophy Type 3

Intervention

Drug:
• Pyridostigmine Bromide

Locations

Country	Name	City	State
Belgium	Centre de référence des maladies neuromusculaire, Centre Hospitalier Régional de la Citadelle	Liège

Sponsors (1)

Lead Sponsor	Collaborator
Centre Hospitalier Régional de la Citadelle

Country where clinical trial is conducted

Belgium, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from Baseline in the distance walked at 6-minute walk test at 6 months		6 months
Secondary	Change from baseline of decrement at 6 months		6 months
Secondary	Change from baseline of MFM-D1	Comparison of treated and control group values will be made	6 months
Secondary	Change from baseline of Moviplate values at 6 months	Comparison between treated and control group value will be made	6 months
Secondary	Change from baseline of the ratio at 6 minutes walk test at 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.	6 months