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NCT01942590 · ClinicalTrials.gov registry record · Phase 1

Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy

A Phase 1 study, sponsored by Dwight Koeberl, M.D., Ph.D..

Completed
Registry status
Phase 1
Development phase
17
Enrollment target

NCT01942590: Completed Phase 1 study, sponsored by Dwight Koeberl, M.D., Ph.D..

NCT01942590 is a Phase 1 study that has completed, run by Dwight Koeberl, M.D., Ph.D.. The registered enrollment target is 17 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (72% lower). According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT01942590, a Phase 1 study, has completed, sponsored by Dwight Koeberl, M.D., Ph.D..

COMPLETED
Registry status
Phase 1
Development phase
17 participants
Enrollment target

Study Summary

Funding Source- FDA OOPD The purpose of this study is to investigate the safety and efficacy of clenbuterol on motor function in individuals with late-onset Pompe disease (LOPD) who are treated with enzyme replacement therapy (ERT).

Primary Outcome

Worsening muscle involvement, as defined by \>3x increase in CK from baseline that is \>2x the upper limit of normal

Interventions

  • DRUG Placebo
  • DRUG Clenbuterol

Trial Details

FieldValue
Enrollment Target 17 participants
Start Date 2013-09
Est. Completion 2016-09-02
Phase Phase 1
Dwight Koeberl, M.D., Ph.D.

2 total trials

What the finished NCT01942590 record still lists

NCT01942590 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 17 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (72% lower).

The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.

NCT01942590 does not publish any study locations in the registry export this page uses.

Frequently Asked Questions

What is clinical trial NCT01942590 about?

NCT01942590 is a clinical study titled "Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy". Funding Source- FDA OOPD The purpose of this study is to investigate the safety and efficacy of clenbuterol on motor function in individuals with late-onset Pompe disease (LOPD) who are treated with enzyme replacement therapy (ERT).

What is the current status of trial NCT01942590?

This trial is currently completed. It is a Phase 1 study. The enrollment target is 17 participants. The study started on 2013-09. Estimated completion is 2016-09-02.

What interventions are being tested in trial NCT01942590?

The interventions under investigation include: Placebo (DRUG), Clenbuterol (DRUG).

Who is sponsoring clinical trial NCT01942590?

This trial is sponsored by Dwight Koeberl, M.D., Ph.D., which has 2 total clinical trials registered on ClinicalTrials.gov.

How this trial's enrollment target compares

Where NCT01942590's enrollment target sits among peer trials

17 1785th of 2000 higher than 215 of 2,000 other Phase 1 trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other 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 NCT01942590, the US trial registry maintained by the National Library of Medicine. NCT01942590 (small enrollment · none site footprint · completed) retrieved and formatted by PlainTrial, see methodology.