A Pilot Study of Pyridostigmine in Pompe Disease

Stopped early · Early Phase 1

Conditions studied: Pompe Disease

In brief

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.

Key facts

Study ID

NCT02357225

Run by

University of Florida

People needed

2

Starts

2015-08-01

Expected to finish

2018-01-01

Last updated by the study team

2018-05-15

Who can join

Age: 8 and older, up to 60. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Males or females between 8 and 60 years of age;
• Diagnosis of Pompe disease (protein assay, genotyping, and positive clinical signs)
• No contraindication to pyridostigmine

You may not qualify if…

• Already receive pyridostigmine as part of their normal clinical care at screening
• Are pregnant - participants will receive a urine pregnancy test at screening
• Have received acute administration of antibiotic, corticosteroid, or neuromuscular blockade medications within 30 days prior to screening
• Any other concurrent medical condition which, in the opinion of the study team, would make the subject inappropriate to participate in the assessments

Where it is running

• University of Florida Clinical Research Center — Gainesville, Florida, United States

Full record on ClinicalTrials.gov

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