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Antibody Levels and Biomarkers for Pompe Disease

N/A
Recruiting
Research Sponsored by Astellas Gene Therapies
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Participant has a documented clinical diagnosis of LOPD.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 2 years
Awards & highlights
No Placebo-Only Group

Summary

This trial is studying Pompe disease, a genetic condition that causes muscle weakness. People with Pompe disease have a faulty gene that produces an enzyme called GAA. Without this enzyme, there is a build-up

Who is the study for?
This study is for older teens and adults with late-onset Pompe disease, a condition causing muscle weakness. Participants either have never had enzyme replacement therapy or have been on it for at least 6 months. They must be able to attend study visits and agree not to join other clinical trials during this one.
What is being tested?
The study isn't testing a new treatment but is looking at antibody levels against AAV8 (a harmless virus used in gene therapy) and the GAA enzyme involved in standard Pompe disease treatment. It also measures biomarkers related to the disease through regular blood and urine tests over two years.
What are the potential side effects?
Since there's no intervention being tested, such as a new drug or therapy, participants are not expected to experience side effects from the study itself. The focus is on monitoring existing conditions rather than introducing potential causes of side effects.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have been diagnosed with Late-Onset Pompe Disease (LOPD).

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 2 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 2 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Occurrence of neutralizing antibodies to AAV8
Occurrence of total antibodies to AAV8
Secondary study objectives
Creatine kinase [CK] levels
Occurrence of anti-GAA antibodies in participants on ERT
Seroconversion of antibodies to AAV8 over time
+1 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Participants with Late-Onset Pompe DiseaseExperimental Treatment1 Intervention
Adolescent or adult participants with LOPD.

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Who is running the clinical trial?

Astellas Gene TherapiesLead Sponsor
6 Previous Clinical Trials
304 Total Patients Enrolled
Medical DirectorStudy DirectorAstellas Gene Therapies
2,889 Previous Clinical Trials
8,089,093 Total Patients Enrolled
~67 spots leftby May 2027
Unbiased ResultsWe believe in providing patients with all the options.
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