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

NCT02227823 | Completed Phase 2

• 1 other identifier: 1376
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 1

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).

Conditions

Spinal Muscular Atrophy Type 3

Keywords

spinal muscular atrophy type 3; electromyography; fatigability; decrement

Outcome Measures

Primary Outcomes (1)

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

  6 months

Secondary Outcomes (4)

• Change from baseline of decrement at 6 months

  6 months

• Change from baseline of MFM-D1

  6 months

• Change from baseline of Moviplate values at 6 months

  6 months

• Change from baseline of the ratio at 6 minutes walk test at 6 months

  6 months

Study Arms (2)

significant decrement

EXPERIMENTAL

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

no decrement

NO INTERVENTION

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

Interventions

Pyridostigmine Bromide DRUG

Also known as: Mestinon

significant decrement

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• Patient who had surgical intervention or suffer from a recent traumatism (less than 6 months)
• 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)

Sponsors & Collaborators

• Centre Hospitalier Régional de la Citadelle: lead

Study Sites (1)

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

Liège, 4000, Belgium

Location

MeSH Terms

Conditions

Spinal Muscular Atrophies of Childhood

Interventions

Pyridostigmine Bromide

Condition Hierarchy (Ancestors)

Muscular Atrophy, Spinal; Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Motor Neuron Disease; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Pyridinium Compounds; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Stephanie Delstanche

  Centre de référence des maladies neuromusculaire de Liège

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: FACTORIAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Neurologist

Study Record Dates

• First Submitted: August 20, 2014
• First Posted: August 28, 2014
• Study Start: July 1, 2014
• Primary Completion: July 1, 2017
• Study Completion: July 1, 2017
• Last Updated: October 11, 2023

Record last verified: 2023-10

Locations

BE (1)