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NCT01898364 · ClinicalTrials.gov registry record · Phase 1
Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.
A Phase 1 study of Pompe Disease and Glycogen Storage Disease Type II (GSD-II), sponsored by Genzyme, a Sanofi Company.
- Completed
- Registry status
- Phase 1
- Development phase
- 24
- Enrollment target
- 17
- Study locations
NCT01898364: Completed Phase 1 study of Pompe Disease and Glycogen Storage Disease Type II (GSD-II), sponsored by Genzyme, a Sanofi Company.
NCT01898364 is a Phase 1 study of Pompe Disease and Glycogen Storage Disease Type II (GSD-II) that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 24 participants, below the 231-participant average among 14 other Pompe Disease trials with a reported enrollment target (90% lower). The trial reports 17 study locations across 8 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01898364, a Phase 1 study of Pompe Disease and Glycogen Storage Disease Type II (GSD-II), has completed, sponsored by Genzyme, a Sanofi Company.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 24 participants
- Enrollment target
- 17
- Study locations
Study Summary
Primary Objective: To evaluate the safety and tolerability of neoGAA in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients. Secondary Objective: To evaluate the pharmacokinetics, pharmacodynamics of neoGAA in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients. To evaluate the effect of neoGAA on exploratory efficacy endpoints in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients.
Conditions Studied
Interventions
- DRUG GZ402666
Study Locations (17)
Other
- Investigational Site Number 056001 - Leuven
- Investigational Site Number 208001 - København Ø
- Investigational Site Number 250001 - Marseille
- Investigational Site Number 250003 - Nice
- Investigational Site Number 250002 - Paris
- Investigational Site Number 276003 - Mainz
- Investigational Site Number 276001 - München
- Investigational Site Number 276002 - Münster
- Investigational Site Number 528001 - Rotterdam
- Investigational Site Number 826003 - Newcastle upon Tyne
Arizona
- Investigational Site Number 840006 - Phoenix
Florida
- Investigational Site Number 840010 - Jacksonville
Kansas
- Investigational Site Number 840001 - Kansas City
Missouri
- Investigational Site Number 840008 - St Louis
North Carolina
- Investigational Site Number 840002 - Durham
Texas
- Investigational Site Number 840009 - Dallas
Virginia
- Investigational Site Number 840003 - Fairfax
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 24 participants |
| Start Date | 2013-08-19 |
| Est. Completion | 2015-02-25 |
| Phase | Phase 1 |
What the finished NCT01898364 record still lists
NCT01898364 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 24 participants, a relatively small participant target, below the 231-participant average among 14 other Pompe Disease trials with a reported enrollment target (90% lower).
The record links to 3 conditions, with Pompe Disease appearing as the primary indexed condition, and to 1 intervention - of which GZ402666 is the first listed.
NCT01898364 lists 17 locations in 8 states (Other, Arizona, Florida).
Frequently Asked Questions
What is clinical trial NCT01898364 about?
NCT01898364 is a clinical study titled "Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.". Primary Objective: To evaluate the safety and tolerability of neoGAA in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients. Secondary Objective: To evaluate the pharmacokinetics, pharmacodynamics of neoGAA in treatment naïve and alglucosidase alfa treated late-onset ...
What is the current status of trial NCT01898364?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 24 participants. The study started on 2013-08-19. Estimated completion is 2015-02-25.
What conditions does trial NCT01898364 study?
This clinical trial studies the following conditions: Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency.
What interventions are being tested in trial NCT01898364?
The interventions under investigation include: GZ402666 (DRUG).
Who is sponsoring clinical trial NCT01898364?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01898364 being conducted?
This trial has 17 study locations across Arizona, Florida, Kansas, Missouri, North Carolina. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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