Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies
Public ClinicalTrials.gov record NCT01451879. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Effects of Immunomodulation Therapy on Anti-rhGAA Immune Response in Subjects With Pompe Disease Receiving rhGAA Enzyme Replacement Therapy
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
Hypothesis: the effectiveness of treatment of Pompe Disease with rhGAA enzyme replacement therapy (ERT) is limited at least in part because patients develop antibodies against the provided rhGAA enzyme. Treatment with immunomodulatory drugs may dampen or eliminate the anti-rhGAA immune response in patients receiving ERT, thereby allowing for greater ERT efficacy. Studying the immune response to rhGAA may provide valuable insight into the role of the immune system in the effectiveness of ERT for Pompe Disease.
Study identification
- NCT ID
- NCT01451879
- Recruitment status
- Completed
- Study type
- Observational
- Phase
- Not listed
- Enrollment
- 11 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- Up to 65 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Sep 30, 2008
- Primary completion
- Sep 30, 2017
- Completion
- Sep 30, 2017
- Last update posted
- Dec 7, 2017
2008 – 2017
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Florida | Gainesville | Florida | 32610 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Recently updated Pompe Disease trials
Other public records listing Pompe Disease. Sorted by last update posted; newest first. Not a recommendation.
- NCT07123155: Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD) Recruiting
- NCT00231400: Pompe Disease Registry Protocol Recruiting
- NCT01665326: Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease Recruiting
- NCT06121011: A Global Prospective Observational Registry of Patients With Pompe Disease Recruiting
- NCT04639336: Cognitive and Neurological Pathologies in Pompe Disease Recruiting
- NCT05734521: Avalglucosidase Alfa Pregnancy Study Recruiting
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT01451879, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Dec 7, 2017 · Synced Sep 8, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT01451879 live on ClinicalTrials.gov.