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A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease
NCT06150820 · View on ClinicalTrials.gov ↗
Study Summary
Pompe disease is a genetic condition which causes muscle weakness over time. People with Pompe disease have a faulty gene that makes an enzyme called acid alpha-glucosidase (or GAA). This enzyme breaks down a type of sugar called glycogen. Without this enzyme, there is a build-up of glycogen in the cells of the body. This causes muscle weakness and other symptoms. Pompe disease can happen at any age, but in late-onset Pompe disease, symptoms generally start from 12 months old onwards. The standard treatment for people with Pompe disease is to receive regular infusions of the GAA enzyme. This is known as enzyme replacement therapy. However, people can build up antibodies against the GAA enzyme over time. Gene therapy is used to treat conditions caused by a faulty gene. It works by replacing the faulty gene with a working gene inside the cells of the body. The working gene is delivered into the cells using certain viruses as carriers (vectors). Viruses are often used as carriers as they can easily get inside cells. The genetic material of the original virus is replaced with the working gene, so only the working gene gets inside the cells. A common virus used as a carrier in gene therapy is the adeno-associated virus (or AAV). This is like an adenovirus, which causes the common cold. The original type of AAV does not cause any harm to humans. However, people that have previously been infected with the original type of AAV may have built up antibodies against AAV. These antibodies may stop the AAV carrier with the working gene getting inside the cells. Researchers want to learn more about antibody levels against AAV and the GAA enzyme in people with late-onset Pompe disease. They also want to learn about other substances in the blood that provide more information about late-onset Pompe disease. These are known as biomarkers. In this study, older teenagers and adults with late-onset Pompe disease will take part. They will not have had gene therapy using AAV. There w
Conditions Studied
Interventions
- OTHER No Intervention
Study Locations (20)
Other
- AU61003 — Adelaide
- AU61001 — Herston
- BR55003 — Flamengo
- BR55002 — Porto Alegre
- BR55001 — São Paulo
- CN15003 — Edmonton
- CA15001 — Montreal
- FR33006 — Angers
- FR33009 — Garches
- FR33005 — Lille
Pennsylvania
- University of Pennsylvania — Philadelphia
- University of Pittsburgh Medical Center — Pittsburgh
Georgia
- Emory Clinic — Atlanta
Kansas
- University of Kansas Medical Center — Kansas City
Michigan
- University of Michigan — Ann Arbor
Minnesota
- Children's Hospitals and Clinics of Minnesota — Minneapolis
New Jersey
- Hackensack University Medical Center — Hackensack
Ohio
- University of Cincinnati — Cincinnati
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 119 participants |
| Start Date | 2024-02-01 |
| Est. Completion | 2027-06-30 |
| Phase | NA |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06150820
The ClinicalTrials.gov registry entry for NCT06150820 describes a study currently listed as active not recruiting. It is categorized as NA, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 119 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Astellas Gene Therapies, which has 36 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Pompe Disease (Late-onset) appearing as the primary indexed condition, and to 1 intervention — of which No Intervention is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT06150820 reports 20 study locations spanning 10 distinct geographic areas — top geographies include Other, Pennsylvania, Georgia. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT06150820 about?
NCT06150820 is a clinical study titled "A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease". Pompe disease is a genetic condition which causes muscle weakness over time. People with Pompe disease have a faulty gene that makes an enzyme called acid alpha-glucosidase (or GAA). This enzyme breaks down a type of sugar called glycogen. Without this enzyme, there is a build-up of glycogen in the ...
What is the current status of trial NCT06150820?
This trial is currently active not recruiting. It is a NA study. The enrollment target is 119 participants. The study started on 2024-02-01. Estimated completion is 2027-06-30.
What conditions does trial NCT06150820 study?
This clinical trial studies the following conditions: Pompe Disease (Late-onset). These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT06150820?
The interventions under investigation include: No Intervention (OTHER). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT06150820?
This trial is sponsored by Astellas Gene Therapies, which has 36 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT06150820 being conducted?
This trial has 20 study locations across Georgia, Kansas, Michigan, Minnesota, New Jersey. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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