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High Dose or High Dose Frequency Study of Alglucosidase Alfa

NCT00483379 · Status: COMPLETED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 13

Last updated 2014-03-07

Study results available
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Summary

Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. The objective of this exploratory study is to evaluate the safety and efficacy of alternative dosing regimens of alglucosidase alfa in patients with Pompe disease who have not demonstrated an optimal response to the standard dosing regimen of 20 mg/kg every other week after a minimum of 6 months treatment immediately prior to study entry.

Conditions

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

Interventions

BIOLOGICAL

alglucosidase alfa

intravenous infusion

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Medical Monitor · Genzyme, a Sanofi Company

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
6 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2007-05-31
Primary Completion
2009-12-31
Completion
2010-07-31

Countries

  • United States
  • Australia
  • Canada

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 NCT00483379 on ClinicalTrials.gov