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Pompe disease study aims to clear path for gene therapy

NCT ID NCT06150820

Knowledge-focused Sponsor: Astellas Gene Therapies 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 This study
Running, but no longer taking on new participants.
Completed
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 18, 2026 · Updated 4 times

Summary

This study looks at people with late-onset Pompe disease to measure antibodies against a virus used in gene therapy and against the standard enzyme replacement therapy. It also checks for biomarkers in blood and urine. About 119 teenagers and adults will give samples over 2 years, but no new treatment is given. The goal is to gather information that could help design better gene therapies in the future.

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

Not a phased trial

Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.

Participants

119 people

The number who actually took part.

Started

Feb 2024

Expected to finish

Jun 2027

An estimate. End dates often move.

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

16 to 69 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: * Participant has a documented clinical diagnosis of LOPD. * Participant is enzyme replacement therapy ERT-naïve (ERT-N) or if the participant is currently taking an approved ERT treatment or is participating in an ERT-interventional study, the ERT must have been received for at least 6 months or more (ERT-experienced \[ERT-E\]). * Participant is willing and able to comply with study visits and procedures. * Participant agrees to not start participating in any other clinical study involving an investigational study treatment, including ERT, while participating in this study. Exclusion Criteria: * Participant previously received an AAV-related product (any serotype). * Participant is currently participating in a Pompe-related interventional study (other than ERT-interventional studies) or has received gene or cell therapy. * Participant requires any invasive or noninvasive ventilation support while awake and upright (non-invasive support while sleeping with either continuous positive airway pressure (CPAP) or bilevel positive airway pressure (BiPAP) is acceptable for eligibility). * Participant is unable to ambulate (assistive devices \[e.g., cane or walker\] are acceptable for eligibility). * Participants who have received any ERT for less than 6 months as of the Baseline visit are not eligible.

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Conditions

The condition(s) this trial relates to.

glycogen storage disease due to acid maltase deficiency, late-onset 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 (late-onset)

Contacts and locations

Locations

  • AU61001

    Herston, Australia

  • AU61003

    Adelaide, Australia

  • BR55001

    São Paulo, Brazil

  • BR55002

    Porto Alegre, Brazil

  • BR55003

    Flamengo, Brazil

  • CA15001

    Montreal, Canada

  • CN15003

    Edmonton, Canada

  • Children's Hospitals and Clinics of Minnesota

    Minneapolis, Minnesota, 55404, United States

  • DT49003

    Höchheim, Germany

  • DT49004

    Essen, Germany

  • DT49005

    Bonn, Germany

  • DT49006

    Münster, Germany

  • ES34001

    Madrid, Spain

  • ES34002

    Valencia, Spain

  • ES34003

    Albacete, Spain

  • ES34004

    Barcelona, Spain

  • ES34005

    Madrid, Spain

  • ES34007

    L'Hospitalet de Llobregat, Spain

  • ES34009

    Donostia / San Sebastian, Spain

  • Emory Clinic

    Atlanta, Georgia, 30322, United States

  • FR33001

    Strasbourg, France

  • FR33002

    Marseille, France

  • FR33003

    Nantes, France

  • FR33004

    Nice, France

  • FR33005

    Lille, France

  • FR33006

    Angers, France

  • FR33007

    Limoges, France

  • FR33009

    Garches, France

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601, United States

  • IT39002

    Florence, Italy

  • IT39003

    Udine, Italy

  • IT39004

    Roma, Italy

  • IT39005

    Gussago, Italy

  • IT39006

    Pisa, Italy

  • IT39008

    Pavia, Italy

  • IT39009

    Milan, Italy

  • IT39011

    Milan, Italy

  • IT39012

    Messina, Italy

  • Lysosomal and Rare Diseases Research and Treatment Center, Inc.

    Fairfax, Virginia, 22030, United States

  • National Center of Neurology and Psychiatry

    Kodaira-Shi, Japan

  • TW88601

    Taipei, Taiwan

  • TW88602

    Taipei, Taiwan

  • TW88603

    Taoyuan City, Taiwan

  • Tokyo Women's Medical University Hospital

    Shinjuku-Ku, Japan

  • UK44001

    Newcastle upon Tyne, United Kingdom

  • UK44003

    Cambridge, United Kingdom

  • UK44004

    Salford, United Kingdom

  • University of Cincinnati

    Cincinnati, Ohio, 45221, United States

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160, United States

  • University of Michigan

    Ann Arbor, Michigan, 48109, United States

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

  • University of Pittsburgh Medical Center

    Pittsburgh, Pennsylvania, 15213, United States

  • University of UTAH - PPDS

    Salt Lake City, Utah, 84132, United States

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