Who is sponsoring NCT01288027?

NCT01288027 is sponsored by Genzyme, a Sanofi Company.

What condition does NCT01288027 study?

NCT01288027 studies Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenesis 2 Acid Maltase Deficiency.

What drugs are being tested in NCT01288027?

Interventions: Alglucosidase Alfa.

What is the status of NCT01288027?

NCT01288027 is currently COMPLETED.

Last reviewed 2014-12-04 · How we verify

A Phase 4 Prospective Exploratory Muscle Biopsy, Biomarker, and Imaging Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

NCT01288027 Phase 4 COMPLETED Results posted

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).

Details

Lead sponsor	Genzyme, a Sanofi Company
Phase	Phase 4
Status	COMPLETED
Enrolment	16
Start date	2011-06
Completion	2013-12

Conditions

Pompe Disease (Late-Onset)
Glycogen Storage Disease Type II (GSD II)
Glycogenesis 2 Acid Maltase Deficiency

Interventions

Alglucosidase Alfa

Primary outcomes

Change From Baseline in Tissue Glycogen Content in Quadriceps Muscle Biopsy Samples at Week 26 — Baseline, Week 26
Tissue glycogen content was measured by quadriceps biopsies as 'percent area of tissue occupied by glycogen'.

Countries

United States, Germany, Netherlands, United Kingdom