Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.
Public ClinicalTrials.gov record NCT01898364. 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
An Open-label, Multicenter, Multinational, Ascending Dose Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Repeated Biweekly Infusions of neoGAA in naïve and Alglucosidase Alfa Treated Late-onset Pompe Disease Patients.
Study identification
- NCT ID
- NCT01898364
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 1
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Enrollment
- 24 participants
Conditions and interventions
Interventions
- GZ402666 Drug
Drug
Eligibility (public fields only)
- Age range
- 18 Years and older
- 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 18, 2013
- Primary completion
- Feb 24, 2015
- Completion
- Feb 24, 2015
- Last update posted
- Mar 22, 2023
2013 – 2015
United States locations
- U.S. sites
- 7
- U.S. states
- 7
- U.S. cities
- 7
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Investigational Site Number 840006 | Phoenix | Arizona | 85013 | — |
| Investigational Site Number 840010 | Jacksonville | Florida | 32209 | — |
| Investigational Site Number 840001 | Kansas City | Kansas | 66160-7321 | — |
| Investigational Site Number 840008 | St Louis | Missouri | 63110 | — |
| Investigational Site Number 840002 | Durham | North Carolina | 27710 | — |
| Investigational Site Number 840009 | Dallas | Texas | 75390 | — |
| Investigational Site Number 840003 | Fairfax | Virginia | 22030 | — |
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 NCT01898364, 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 Mar 22, 2023 · Synced Jul 22, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT01898364 live on ClinicalTrials.gov.