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

Primary Purpose

Spinal Muscular Atrophy Type 3

Status
Completed
Phase
Phase 2
Locations
Belgium
Study Type
Interventional
Intervention
Pyridostigmine Bromide
Sponsored by
Centre Hospitalier Régional de la Citadelle
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Spinal Muscular Atrophy Type 3 focused on measuring spinal muscular atrophy type 3, electromyography, fatigability, decrement

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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)

Sites / Locations

  • Centre de référence des maladies neuromusculaire, Centre Hospitalier Régional de la Citadelle

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

No Intervention

Arm Label

significant decrement

no decrement

Arm Description

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

Patient without significant decrement will not receive any treatment and will be the control group

Outcomes

Primary Outcome Measures

Change from Baseline in the distance walked at 6-minute walk test at 6 months

Secondary Outcome Measures

Change from baseline of decrement at 6 months
Change from baseline of MFM-D1
Comparison of treated and control group values will be made
Change from baseline of Moviplate values at 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
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.

Full Information

First Posted
August 20, 2014
Last Updated
October 9, 2023
Sponsor
Centre Hospitalier Régional de la Citadelle
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1. Study Identification

Unique Protocol Identification Number
NCT02227823
Brief Title
Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3
Acronym
EMOTAS
Official Title
Safety and Efficacy Study of Anti-cholinesterase Therapy on the Motor Functions in Patients With Spinal Muscular Atrophy Type 3.
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Completed
Study Start Date
July 2014 (undefined)
Primary Completion Date
July 2017 (Actual)
Study Completion Date
July 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Centre Hospitalier Régional de la Citadelle

4. Oversight

Data Monitoring Committee
No

5. Study Description

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, 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Spinal Muscular Atrophy Type 3
Keywords
spinal muscular atrophy type 3, electromyography, fatigability, decrement

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Factorial Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
4 (Actual)

8. Arms, Groups, and Interventions

Arm Title
significant decrement
Arm Type
Experimental
Arm Description
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
Arm Title
no decrement
Arm Type
No Intervention
Arm Description
Patient without significant decrement will not receive any treatment and will be the control group
Intervention Type
Drug
Intervention Name(s)
Pyridostigmine Bromide
Other Intervention Name(s)
Mestinon
Primary Outcome Measure Information:
Title
Change from Baseline in the distance walked at 6-minute walk test at 6 months
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Change from baseline of decrement at 6 months
Time Frame
6 months
Title
Change from baseline of MFM-D1
Description
Comparison of treated and control group values will be made
Time Frame
6 months
Title
Change from baseline of Moviplate values at 6 months
Description
Comparison between treated and control group value will be made
Time Frame
6 months
Title
Change from baseline of the ratio at 6 minutes walk test at 6 months
Description
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.
Time Frame
6 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility 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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Stephanie Delstanche
Organizational Affiliation
Centre de référence des maladies neuromusculaire de Liège
Official's Role
Principal Investigator
Facility Information:
Facility Name
Centre de référence des maladies neuromusculaire, Centre Hospitalier Régional de la Citadelle
City
Liège
ZIP/Postal Code
4000
Country
Belgium

12. IPD Sharing Statement

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

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