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New drug combo shows promise for rare pompe disease

NCT ID NCT02675465

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 This study
The trial has finished. Results may not be published yet.
Stopped early
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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested whether a new combination of two drugs (ATB200 and AT2221) is safe for adults with Pompe disease, a rare genetic disorder that causes muscle weakness. 29 participants received the drugs through an IV and by mouth. The main goal was to check for side effects and see how the body processes the medication.

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 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

29 people

The number who actually took part.

Started

Apr 2016

Finished

Aug 2024

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 75 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.

Adults with Diagnosis of Pompe disease Cohort 1: Enzyme Replacement Therapy (ERT)-experienced subject (ambulatory): * Male and female subjects between 18 and 65 years of age, inclusive * Received ERT with alglucosidase alfa (Myozyme/Lumizyme) for the previous 2-6 years, inclusive * Was receiving alglucosidase alfa at a frequency of once every other week * Must have been able to walk 200-500 meters on the 6-Minute Walk Test (6MWT) * Had upright Forced Vial Capacity (FVC) 30-80% of predicted normal value Cohort 2: ERT-experienced subjects (non-ambulatory): * Male and female subjects between 18 and 65 years of age, inclusive * Had been receiving ERT with alglucosidase alfa for ≥2 years at a regular or set frequency * Was wheelchair-bound Cohort 3: ERT-naïve subjects (ambulatory): * Male and female subjects between 18 and 65 years of age, inclusive * Must have been able to walk 200-500 meters on the 6MWT * Had upright FVC 30-80% of predicted normal value Cohort 4: ERT-experienced subject (ambulatory): * Male and female subjects between 18 and 75 years of age, inclusive * Had been receiving ERT with alglucosidase alfa for ≥7 years, inclusive * Was receiving alglucosidase alfa at a frequency of once every other week * Must have been able to walk 75-600 meters on the 6MWT * Had upright FVC 30-85% of predicted normal value Exclusion Criteria: * Received treatment with prohibited medications within 30 days of Baseline Visit * Subject, if female, was pregnant or breastfeeding at screening * Subject, whether male or female, planned to conceive a child during the study * Had a medical or any other extenuating condition or circumstance that may, in opinion of investigator, pose an undue safety risk to the subject or compromise his/her ability to comply with protocol requirements * Had a history of allergy or sensitivity to alglucosidase alfa, miglustat or other iminosugars (Cohorts 1, 2, and 4) * Required invasive ventilatory support, or used noninvasive ventilatory support ≥ 6 hours a day while awake (Cohorts 1, 3, and 4) * Had active systemic autoimmune disease such as lupus, scleroderma, or rheumatoid arthritis; subjects with autoimmune disease must have been discussed with the Amicus Medical Monitor * Had active bronchial asthma; subjects with bronchial asthma must have been discussed with the Amicus Medical Monitor

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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

  • Abramson Cancer Center Chester County Hospital

    West Chester, Pennsylvania, 19380, United States

  • Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • Emory University Division of Medical Genetics

    Decatur, Georgia, 30033, United States

  • Erasmus Medical Center

    Rotterdam, Netherlands

  • Friedrich-Baur-Institure, Dep of Neurology - University Munich

    Munich, 80336, Germany

  • Great Falls Clinic, LLP

    Great Falls, Montana, 59405, United States

  • Infusion Associates

    Grand Rapids, Michigan, 49525, United States

  • Lysosomal & Rare Disorders Research & Treatment Center (LDRTC)

    Fairfax, Virginia, 22030, United States

  • Neuromuscular Research Centre

    Phoenix, Arizona, 85028, United States

  • Perelman Center for Advanced Medicine

    Philadelphia, Pennsylvania, 19104, United States

  • Rutgers New Jersey Medical School

    Newark, New Jersey, 08103, United States

  • Salford Royal NHS Foundation Trust

    Salford, M6 8HD, United Kingdom

  • School of Medicine, University of Auckland

    Auckland, 01051, New Zealand

  • University Children's Hospital Department of Neuropediatrics and Inborn Metabolic Disorders, St. Josefs-Hospital

    Bochum, 44791, Germany

  • University Hospital Birmingham NHS Foundation Trust, Queen Elizabeth Medical Center

    Birmingham, B15 2TH, United Kingdom

  • University of California Irvine

    Orange, California, 92868, United States

  • University of Florida

    Gainesville, Florida, 32610, United States

  • University of Pittsburgh

    Pittsburgh, Pennsylvania, 15213, United States

  • Womens & Childrens Hospital, Adelaide

    North Adelaide, South Australia, 05006, Australia

More trials for these conditions

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