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NCT02357225 · ClinicalTrials.gov registry record · Early Phase 1

A Pilot Study of Pyridostigmine in Pompe Disease

A Early Phase 1 study, sponsored by University of Florida.

Terminated
Registry status
Early Phase 1
Development phase
2
Enrollment target

NCT02357225: Clinical Trial Early Phase 1 study, sponsored by University of Florida.

NCT02357225 is a Early Phase 1 study that was terminated before completion, run by University of Florida. The registered enrollment target is 2 participants, below the 53-participant average among 2,764 other Early Phase 1 trials with a reported enrollment target (96% lower). According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT02357225, a Early Phase 1 study, was terminated before completion, sponsored by University of Florida.

TERMINATED
Registry status
Early Phase 1
Development phase
2 participants
Enrollment target

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.

Primary Outcome

Quantitative muscle testing and the 6 Minute Walk Test will be used to evaluate skeletal muscle function.

Interventions

  • DRUG Pyridostigmine Bromide

Trial Details

FieldValue
Enrollment Target 2 participants
Start Date 2015-08
Est. Completion 2018-01-01
Phase Early Phase 1
University of Florida

1,106 total trials

Why NCT02357225 stopped before completion

NCT02357225 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 2 participants, a relatively small participant target, below the 53-participant average among 2,764 other Early Phase 1 trials with a reported enrollment target (96% lower).

The record links to 0 conditions, and to 1 intervention - of which Pyridostigmine Bromide is the first listed.

NCT02357225 does not publish any study locations in the registry export this page uses.

Frequently Asked Questions

What is clinical trial NCT02357225 about?

NCT02357225 is a clinical study titled "A Pilot Study of Pyridostigmine in Pompe Disease". 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: ...

What is the current status of trial NCT02357225?

This trial is currently terminated. It is a Early Phase 1 study. The enrollment target is 2 participants. The study started on 2015-08. Estimated completion is 2018-01-01.

What interventions are being tested in trial NCT02357225?

The interventions under investigation include: Pyridostigmine Bromide (DRUG).

Who is sponsoring clinical trial NCT02357225?

This trial is sponsored by University of Florida, which has 1,106 total clinical trials registered on ClinicalTrials.gov.

How this trial's enrollment target compares

Where NCT02357225's enrollment target sits among peer trials

2 1973rd of 2000 higher than 16 of 2,000 other Early Phase 1 trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Early Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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Source: ClinicalTrials.gov NCT02357225, the US trial registry maintained by the National Library of Medicine. NCT02357225 (small enrollment · none site footprint · terminated) retrieved and formatted by PlainTrial, see methodology.