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Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

NCT01288027 · Status: COMPLETED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2014-12-19

Study results available
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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).

Conditions

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

Interventions

BIOLOGICAL

Alglucosidase Alfa

Alglucosidase alfa intravenous infusion 20 milligram per kilogram (mg/kg) every other week for 24 weeks.

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Medical Monitor · Genzyme, a Sanofi Company

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-06-30
Primary Completion
2013-12-31
Completion
2013-12-31

Countries

  • United States
  • Germany
  • Netherlands
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01288027 on ClinicalTrials.gov