A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)
Public ClinicalTrials.gov record NCT04848779. 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
A Prospective Observational Study to Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
Primary Objective: To describe the effect of routine practice with alglucosidase alfa in patients with IOPD ≤6 months of age, on invasive ventilation-free survival after 52 weeks of treatment. Secondary Objectives: * To describe the effect of routine practice with alglucosidase alfa on invasive ventilation-free survival and survival at 12 and 18 months of age, as well as on change in left ventricular mass (LVM) Z score, Alberta Infant Motor Scale (AIMS) score, body weight, body length, and head circumference Z scores, and urinary glucose tetrasaccharide (Hex4), at Week 52 of treatment. * To describe the safety, tolerability, and immunogenicity of alglucosidase alfa in the routine practice of IOPD treatment.
Study identification
- NCT ID
- NCT04848779
- Recruitment status
- Active, not recruiting
- Study type
- Observational
- Phase
- Not listed
- Enrollment
- 16 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 0 Days to 6 Months
- 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
- Jun 9, 2021
- Primary completion
- Oct 27, 2026
- Completion
- Oct 27, 2026
- Last update posted
- Jun 23, 2026
2021 – 2026
United States locations
- U.S. sites
- 5
- U.S. states
- 5
- U.S. cities
- 5
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Advanced Medical Genetics- Site Number : 8400002 | Hawthorne | New York | 10532 | — |
| Duke University Medical Center- Site Number : 8400004 | Durham | North Carolina | 27710 | — |
| Cincinnati Children's Hospital Medical Center- Site Number : 8400001 | Cincinnati | Ohio | 45229 | — |
| Le Bonheur Children's Hospital- Site Number : 8400005 | Memphis | Tennessee | 38103 | — |
| Seattle Children's Hospital- Site Number : 8400003 | Seattle | Washington | 98105 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 10 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04848779, 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 Jun 23, 2026 · Synced Sep 5, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04848779 live on ClinicalTrials.gov.