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Completed Phase 1 Interventional

Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.

ClinicalTrials.gov ID: NCT01898364

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.

ClinicalTrials.gov public records Last synced Jul 21, 2026, 11:55 PM EDT

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

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 21, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT01898364 live on ClinicalTrials.gov.

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