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

NCT02227823 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 4

Last updated 2023-10-11

No results posted yet for this study

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

Interventions

DRUG

Pyridostigmine Bromide

Sponsors & Collaborators

  • Centre Hospitalier Régional de la Citadelle

    lead OTHER

Principal Investigators

  • Stephanie Delstanche · Centre de référence des maladies neuromusculaire de Liège

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
FACTORIAL

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-07-31
Primary Completion
2017-07-31
Completion
2017-07-31

Countries

  • Belgium

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02227823 on ClinicalTrials.gov