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NCT01288027 · ClinicalTrials.gov registry record · Phase 4
Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa
A Phase 4 study, sponsored by Genzyme, a Sanofi Company.
- Completed
- Registry status
- Phase 4
- Development phase
- 16
- Enrollment target
NCT01288027 is a Phase 4 study that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 16 participants.
The verdict
NCT01288027, a Phase 4 study, has completed, sponsored by Genzyme, a Sanofi Company.
- COMPLETED
- Registry status
- Phase 4
- Development phase
- 16 participants
- Enrollment target
Study Summary
This is an open-label, multicenter study of participants with late-onset Pompe disease naive to treatment with enzyme replacement therapy (ERT). The primary objective of this study is to evaluate glycogen clearance in muscle tissue samples collected pre and post alglucosidase alfa treatment in participants with Late-Onset Pompe disease. The secondary objectives are to characterize the disease burden in participants with late-onset Pompe disease and explore imaging, histologic, and functional assessments in these participants and to explore potential plasma or urine biomarkers relative to late-onset Pompe disease and participant's response to treatment with alglucosidase alfa (Myozyme®/Lumizyme®/GZ419829).
Interventions
- BIOLOGICAL Alglucosidase Alfa
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 16 participants |
| Start Date | 2011-06 |
| Est. Completion | 2013-12 |
| Phase | Phase 4 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01288027
The ClinicalTrials.gov registry entry for NCT01288027 describes a study currently listed as completed, categorized as Phase 4. The registered enrollment target is 16 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Genzyme, a Sanofi Company, which has 196 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which Alglucosidase Alfa is the first listed.
NCT01288027 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT01288027 about?
NCT01288027 is a clinical study titled "Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa". This is an open-label, multicenter study of participants with late-onset Pompe disease naive to treatment with enzyme replacement therapy (ERT). The primary objective of this study is to evaluate glycogen clearance in muscle tissue samples collected pre and post alglucosidase alfa treatment in parti...
What is the current status of trial NCT01288027?
This trial is currently completed. It is a Phase 4 study. The enrollment target is 16 participants. The study started on 2011-06. Estimated completion is 2013-12.
What interventions are being tested in trial NCT01288027?
The interventions under investigation include: Alglucosidase Alfa (BIOLOGICAL).
Who is sponsoring clinical trial NCT01288027?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
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