A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
Public ClinicalTrials.gov record NCT01597596. 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 Phase 3/4, Prospective, Multinational, Open-label, Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
A study to demonstrate comparable safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa manufactured at the 160 litre (L) and 4000 L scales in participants who had been diagnosed with infantile-onset Pompe disease. Participants were treated with alglucosidase alfa 160 L scale product in the United States (US) and 4000 L scale product in the regions outside the US.
Study identification
- NCT ID
- NCT01597596
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 4
- Enrollment
- 4 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- Up to 12 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
- Aug 2012 (Type not available)
- Primary completion
- Dec 2014 (Actual)
- Completion
- Dec 2014 (Actual)
- Last update posted
- Jan 18, 2016
2012 – 2014
United States locations
- U.S. sites
- 14
- U.S. states
- 14
- U.S. cities
- 14
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Not listed | Little Rock | Arkansas | — | — |
| Not listed | Oakland | California | — | — |
| Not listed | Gainsville | Florida | — | — |
| Not listed | Decatur | Georgia | — | — |
| Not listed | Chicago | Illinois | — | — |
| Not listed | Cambridge | Massachusetts | — | — |
| Not listed | Detroit | Michigan | — | — |
| Not listed | Las Vegas | Nevada | — | — |
| Not listed | New Brunswick | New Jersey | — | — |
| Not listed | New York | New York | — | — |
| Not listed | Durham | North Carolina | — | — |
| Not listed | Cincinnati | Ohio | — | — |
| Not listed | Fort Worth | Texas | — | — |
| Not listed | Seattle | Washington | — | — |
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 3 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT01597596, 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 Jan 18, 2016 · Synced Oct 24, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT01597596 live on ClinicalTrials.gov.