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Participants with Late-Onset Pompe Disease 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

Study 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.Check my eligibility

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.See study design

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 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 2 years

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Occurrence of neutralizing antibodies to AAV8

Occurrence of total antibodies to AAV8

Secondary outcome measures

Creatine kinase [CK] levels

Occurrence of anti-GAA antibodies in participants on ERT

Seroconversion of antibodies to AAV8 over time

+1 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: Participants with Late-Onset Pompe DiseaseExperimental Treatment1 Intervention

Adolescent or adult participants with LOPD.

0 / 1Eligibility criteria met

Find a Location

Who is running the clinical trial?

Astellas Gene TherapiesLead Sponsor

4 Previous Clinical Trials

233 Total Patients Enrolled

Medical DirectorStudy DirectorAstellas Gene Therapies

2,777 Previous Clinical Trials

8,064,119 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is the primary objective of conducting this clinical trial?

"As per the trial sponsor, Astellas Gene Therapies, the main objective of this study is to assess the presence of neutralizing antibodies to AAV8 within a timeframe of up to 2 years. Secondary objectives include evaluating anti-GAA antibodies in participants receiving ERT through analysis of serum blood samples, monitoring urine glucose tetrasaccharide [Glc4]/hexose tetrasaccharide [Hex4] levels over time via collected urine samples, and measuring creatine kinase [CK] levels from obtained blood plasma samples."

Answered by AI

Are there any available slots for patients to participate in this clinical trial?

"Indeed, as indicated by clinicaltrials.gov, this research endeavor is actively seeking eligible participants. The trial was publicly announced on November 30th, 2023 and underwent its most recent update on January 4th, 2024."

Answered by AI

What is the current number of participants being recruited for this medical research study?

"Indeed, the details on clinicaltrials.gov indicate that this trial is presently in the recruitment phase. The initial posting date was November 30th, 2023, and it has been updated most recently on January 4th, 2024. The study aims to enroll a total of 100 participants at one designated site."

Answered by AI

Does this clinical trial include individuals who are aged 80 years or older as potential participants?

"To participate in this study, eligible individuals must fall within the age range of 16 to 69 years. There are a total of 9 trials specifically designated for patients below the age of 18 and another 10 trials tailored for patients above the age of 65."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease

2024. "A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06150820.

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