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A Pilot Study of Pyridostigmine in Pompe Disease

NCT02357225 · Status: TERMINATED · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 2

Last updated 2018-05-15

No results posted yet for this study

Summary

Pyridostigmine is an acetylcholinesterase inhibitor, which degrades acetylcholine at the neuromuscular junction. Based on recent studies, pyridostigmine may be an effective adjuvant treatment for people with Pompe disease, as it increases the functional impact of this neurotransmitter.

Hypothesis: the use of pyridostigmine in Pompe disease will improve transmission of acetylcholine across the neuromuscular junction, skeletal muscle function, respiratory function, and quality of life.

Conditions

  • Pompe Disease

Interventions

DRUG

Pyridostigmine Bromide

Pyridostigmine is an acetylcholinesterase inhibitor, which increases the amount of acetylcholine at the neuromuscular junction. It will be taken orally, either as a tablet or as a syrup.

Sponsors & Collaborators

  • University of Florida

    lead OTHER

Principal Investigators

  • Barry J Byrne, MD, PhD · University of Florida

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
8 Years
Max Age
60 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-08-31
Primary Completion
2018-01-01
Completion
2018-01-01

Countries

  • United States

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 NCT02357225 on ClinicalTrials.gov