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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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
Sponsor:
Information provided by (Responsible Party):
Dr. Stéphanie Delstanche, Centre Hospitalier Régional de la Citadelle

Study Description
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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).

Condition or disease Intervention/treatment Phase
Spinal Muscular Atrophy Type 3 Drug: Pyridostigmine Bromide Phase 2

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.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: Non-Randomized
Intervention Model: Factorial Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
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


Outcome Measures
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Primary Outcome Measures :
  1. Change from Baseline in the distance walked at 6-minute walk test at 6 months [ Time Frame: 6 months ]

Secondary Outcome Measures :
  1. Change from baseline of decrement at 6 months [ Time Frame: 6 months ]
  2. Change from baseline of MFM-D1 [ Time Frame: 6 months ]
    Comparison of treated and control group values will be made

  3. Change from baseline of Moviplate values at 6 months [ Time Frame: 6 months ]
    Comparison between treated and control group value will be made

  4. 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.


Eligibility Criteria
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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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)
Contacts and Locations
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Information from the National Library of Medicine

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


Locations
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Belgium
Centre de référence des maladies neuromusculaire, Centre Hospitalier Régional de la Citadelle Recruiting
Liège, Belgium, 4000
Contact: Stephanie Delstanche       stephanie.delstanche@chrcitadelle.be   
Contact: Severine Denis       severine.denis@chrcitadelle.be   
Principal Investigator: Stephanie Delstanche         
Sponsors and Collaborators
Centre Hospitalier Régional de la Citadelle
Investigators
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Principal Investigator: Stephanie Delstanche Centre de référence des maladies neuromusculaire de Liège
More Information
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Responsible Party: Dr. Stéphanie Delstanche, Neurologist, Centre Hospitalier Régional de la Citadelle
ClinicalTrials.gov Identifier: NCT02227823    
Other Study ID Numbers: 1376
First Posted: August 28, 2014    Key Record Dates
Last Update Posted: August 28, 2014
Last Verified: August 2014
Keywords provided by Dr. Stéphanie Delstanche, Centre Hospitalier Régional de la Citadelle:
spinal muscular atrophy type 3
electromyography
fatigability
decrement
Additional relevant MeSH terms:
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Muscular Atrophy
Muscular Atrophy, Spinal
Spinal Muscular Atrophies of Childhood
Atrophy
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
 Neuromuscular Diseases
Heredodegenerative Disorders, Nervous System
Genetic Diseases, Inborn
Bromides
Pyridostigmine Bromide
Anticonvulsants
Cholinesterase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Cholinergic Agents
Neurotransmitter Agents
Physiological Effects of Drugs