Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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: Completed Phase 4 study, sponsored by Genzyme, a Sanofi Company.
NCT01288027 is a Phase 4 study that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 16 participants, below the 518-participant average among 9,235 other Phase 4 trials with a reported enrollment target (97% lower). According to ClinicalTrials.gov, the official US trial registry.
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).
Primary Outcome
Tissue glycogen content was measured by quadriceps biopsies as 'percent area of tissue occupied by glycogen'.
Interventions
- BIOLOGICAL Alglucosidase Alfa
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 16 participants |
| Start Date | 2011-06 |
| Est. Completion | 2013-12 |
| Phase | Phase 4 |
What the finished NCT01288027 record still lists
NCT01288027 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 16 participants, a relatively small participant target, below the 518-participant average among 9,235 other Phase 4 trials with a reported enrollment target (97% lower).
The record links to 0 conditions, and to 1 intervention - of which Alglucosidase Alfa is the first listed.
NCT01288027 does not publish any study locations in the registry export this page uses.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01288027's enrollment target sits among peer trials
16 1869th of 2000 higher than 121 of 2,000 other Phase 4 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 4 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT02422641 · 16 participants · Phase 2
Prospective Evaluation Of High-Dose Systemic Methotrexate In Patients With Breast Cancer And Leptomeningeal Metastasis
- NCT02851758 · 16 participants · NA
Transplantation of Autologously Derived Mitochondria Following Ischemia
- NCT03291028 · 16 participants
Analysis of Primary and Metastatic Tumors in Patients With Renal Cell Carcinoma and Urothelial Carcinoma
- NCT03425526 · 16 participants · Phase 1
Donor T Cell Therapy in Treating Immunocompromised Patients With Adenovirus-Related Disease
Similar registry start date
- NCT05988463 · started 2027-01-01 · Phase 1
Dose-Escalation Study of Artesunate Patients With IPF
- NCT07219706 · started 2026-12-10 · NA
Dietary Fiber and Time Restricted Eating
- NCT02831335 · started 2026-12-01
Technical Development of Multi-Parametric Renocerebral MRI
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia