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Pompe disease drug trial halted after just 3 patients

NCT ID NCT00688597

Disease control Sponsor: Amicus Therapeutics Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This study tested an oral drug called duvoglustat in adults with Pompe disease, a rare genetic disorder that causes muscle weakness. Only 3 people took part, and the study was stopped early. Researchers looked at safety and whether the drug could improve walking distance and other functions.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
duvoglustat (AT2220)
What this could lead to
If successful, duvoglustat could offer a new oral treatment option for Pompe disease, potentially improving muscle function and daily activity.
What could go wrong
This was a very small, early-phase study with only 3 participants, and it was terminated early. The results may not apply to a broader population, and the drug's safety and effectiveness are not yet proven.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

3 people

The number who actually took part.

Started

Dec 2008

Finished

Dec 2009

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 74 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Male or female, 18 to 74 years of age inclusive * Diagnosis of Pompe disease based on clinical assessment, enzyme assay, and/or genotyping. Confirmatory genotyping will be performed on all participants who are screened for the study * Naïve to enzyme replacement therapy (ERT) or has not received ERT in the 3 months prior to screening * Willing not to initiate ERT or other prohibited treatment during study participation * Functional grade for arms and/or legs ≥2 OR sitting forced vital capacity ≥30% and \<80% of predicted value, reproducible between screening and baseline (±15%) * Participants of reproductive potential agree to use reliable methods of contraception during the study * Participant or legal representative is willing and able to provide written informed consent Exclusion Criteria: * Any intercurrent condition that may preclude accurate interpretation of study data * Obstructive pulmonary disease * Invasive ventilatory support * Use of noninvasive ventilatory support \>8 hours/day while awake * History of QTc prolongation \>450 milliseconds (msec) for males and \>470 msec for females * History of allergy or sensitivity to the study drug, including any prior serious adverse reaction to iminosugars (such as miglustat or miglitol) * Pregnancy or breast-feeding * Current or recent drug or alcohol abuse * Treatment with another investigational drug within 30 days of study start * Use of prohibited medications ≤3 months prior to screening * Otherwise unsuitable for the study in the opinion of the Investigator

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Conditions

The condition(s) this trial relates to.

glycogen storage disease II Glycogen Storage Disease Type II

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Pompe disease

Contacts and locations

Locations

  • Study site

    Decatur, Georgia, 30033, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.