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Growth and Development Study of Alglucosidase Alfa

NCT00486889 · Status: COMPLETED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2022-08-26

Study results available
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Summary

Pompe disease (also known as glycogen storage disease Type II) is a rare autosomal recessive metabolic muscle disease caused by the deficiency of acid α glucosidase (GAA), an enzyme that degrades lysosomal glycogen. As opposed to the exclusively cytoplasmic accumulation of glycogen that occurs in other glycogen storage disorders, Pompe disease is characterized by organelle bound (lysosomal) and extra-lysosomal accumulation of glycogen in many body tissues, ultimately leading to multisystemic pathology. The overall objective of this study was to evaluate the long-term growth and development of participants with infantile-onset Pompe disease with alglucosidase alfa before 1 year of age. Participants were to be followed for a 10-year period.

Conditions

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

Interventions

BIOLOGICAL

alglucosidase alfa

Dose: 20 mg/kg every 2 weeks; Route of administration: Intravenous infusion

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Medical Monitor · Genzyme, a Sanofi Company

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
24 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2008-08-26
Primary Completion
2021-11-23
Completion
2021-11-23
FDA Drug
Yes

Countries

  • United States

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 NCT00486889 on ClinicalTrials.gov